Hemophilia Symptoms in Children: A Guide for Parents

Early recognition of hemophilia symptoms in children and prompt treatment are essential to prevent lifelong disabilities. Symptoms can range from minor cuts to potentially life-threatening internal bleeding. 

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What Is Hemophilia?

Hemophilia is a bleeding disorder that prevents blood from clotting as usual. Children born with this condition have little or no clotting factor in their blood. A clotting factor (coagulation factor) is a protein in the blood that helps blood clot. Consequently, children are more likely to have severe bleeding and other complications. 

The two main types are:

  • Hemophilia A: Children either lack or have too little clotting factor VIII. 
  • Hemophilia B: Children either lack or have too little clotting factor IX. 

Depending on the clotting factor levels, hemophilia may be [1]:

  • Mild: 5% to 40% of clotting factor levels
  • Moderate: 1% to 4% of clotting factor levels
  • Severe: Less than 1% of clotting factor levels

Though incurable, hemophilia is treatable. With appropriate treatments, children with hemophilia can lead healthy lives. 

Recognizing Hemophilia Symptoms in Children

Hemophilia symptoms in children can vary depending on the severity of the condition. 

Many parents first notice hemophilia symptoms soon after their baby starts crawling or walking. Symptoms at this stage can include large bruises, swollen and warm joints, and avoiding using the affected arm or leg. Likewise, some parents may notice prolonged bleeding during vaccination, circumcision, or routine dental care. 

In mild cases, obvious symptoms may not appear unless severe bleeding occurs following surgery or injury. 

Hemophilia can cause external and internal bleeding. The signs of external bleeding can include:

  • Bleeding in the child’s mouth from a cut, bite, or loss of a baby tooth
  • Bleeding from a cut that stops for a short period, then continues
  • Unusual bleeding from a minor cut
  • Nosebleeds without any obvious cause
  • Heavy periods in girls 

Internal bleeding means bleeding in the organs and muscles. When bleeding occurs in the bladder or kidneys, it can cause blood in urine (may be red or brown). Likewise, bleeding in the digestive tract can cause black, tarry stool or stool mixed with bright red blood. Large bruises may indicate bleeding into large muscles. 

Bleeding Into a Joint

Joint bleeding (hemarthrosis) is common in children with hemophilia. The most commonly affected joints are knees, ankles, and elbows. Joint bleeding may occur without a major injury. If a child with joint bleeding does not get immediate treatment, the affected joint may be permanently damaged. 

A baby with joint bleeding may be cranky for no obvious reason. Older children may describe having an “aura” or tingling in the joint before the external signs. Other signs and symptoms can include:

  • Stiff, painful, swollen, and warm joints
  • Inability to move or straighten an arm or leg

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Bleeding in the Brain

Bleeding in the brain is the most serious complication of hemophilia. It is more common in children younger than two years. Notably, it is the major cause of death and disability in children with hemophilia. 

The signs of bleeding in the brain include:

  • Seizures
  • Constant vomiting
  • Double vision
  • Neck pain or stiffness
  • Long-lasting headaches
  • Sleepiness 
  • Behavior changes
  • Sudden weakness of arms or legs 
  • Problems with balance and coordination

If your child shows any of these signs, particularly after a bump on the head, seek emergency medical care. 

Hemophilia Symptoms in Children: When To Call a Healthcare Provider

Seek immediate medical care if your child:

  • Has a major injury 
  • Passes out
  • Vomits after a bump on the head
  • Has seizures

Does Hemophilia Affect the Vaccine Schedule in Children With Hemophilia?

Children with hemophilia should receive all necessary vaccines according to the schedule, regardless of the severity of hemophilia. Your child’s doctor may administer a vaccine by injecting it under the skin (subcutaneous) instead of injecting it into the muscle tissue (intramuscular).

How To Deal With Ulcerative Colitis Flare-Ups

Together with medications, diet and lifestyle changes may help reduce the severity of ulcerative colitis symptoms during flare-ups.  

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Ulcerative colitis (UC) is a long-term condition. It causes inflammation and ulcers (sores) on the inner lining of the large intestine. While the exact cause is unclear, health experts believe a combination of genes, an abnormal immune response, and gut microbiome may play a role. 

UC symptoms typically worsen over time. However, in some people, the symptoms may improve or disappear temporarily. This period, when you experience minimal or no symptoms, is known as remission. 

What Are Ulcerative Colitis Flare-Ups?

An ulcerative colitis flare-up is a period when the symptoms reappear or worsen after remission. Flare-ups can cause mild or severe symptoms, which may last for hours, a few days, or months. Likewise, the gap between flares might be weeks or years. 

What Causes Ulcerative Colitis Flare-Ups?

Researchers have yet to find the exact cause of ulcerative colitis flare-ups. What they know is that diet and stress may trigger flare-ups.

You may be at a higher risk of developing recurrent UC flare-ups if you have [1]:

  • Bowel incontinence (failure to control bowel movements)
  • A family history of ulcerative colitis

Symptoms of Flare-Ups

Patient leaning over and holding stomach, experiencing abdominal pain

Symptoms vary widely and may include:

  • Diarrhea six or more times a day
  • Blood or mucus in the stool
  • Severe abdominal pain and cramps
  • Urgency to pass stool
  • Fatigue 
  • Fever 
  • Weight loss
  • Anemia (low hemoglobin level or fewer red blood cells in the blood)
  • Shortness of breath 
  • Irregular heartbeat 

Some people may have joint pain, mouth ulcers, and irritated eyes. 

How To Manage Ulcerative Colitis Flare-Ups

Because UC is a lifelong condition, you will need to visit your provider regularly. Regular visits are necessary even when you are in remission. When you first experience a flare-up, inform your provider. They will help you determine the cause and adjust medication if needed. 

To reduce irritation during a flare-up, replace your toilet paper with wipes. You may also take acetaminophen to relieve pain. 

Along with your prescribed medications, use the following tips to help manage symptoms. 

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Identify the Triggers

Triggers can vary from one person to another. Common triggers include:

  • Pain medications called nonsteroidal anti-inflammatory drugs (NSAIDs like ibuprofen, naproxen, aspirin)
  • Antibiotics (they can disrupt microbial balance in your gut)
  • Foods, such as dairy products, high-fiber foods, greasy foods, caffeine, spices, sodas, and alcohol
  • Stopping your UC medications without talking to your provider 
  • Hormonal changes during periods and pregnancy
  • Abnormal electrolyte levels 

Manage Stress

Stress does not cause ulcerative colitis, but it can trigger a flare-up or worsen symptoms. Try meditation, yoga, mindfulness, or exercise to reduce stress. 

Regular exercise may help boost your feeling of well-being and diminish some UC symptoms [2].

Watch What You Eat

Certain foods may trigger flare-ups. Avoid them if you know which foods are the culprit. Eat mindfully whenever possible. Instead of large meals, eat four or five small meals every day. 

According to the Crohn’s & Colitis Foundation, the following foods may help reduce discomfort during a flare-up [3]:

  • Fruits like bananas and raspberries
  • Cooked green vegetables 
  • Healthy fats (fatty fish, walnut butter, flaxseed oil)
  • Cooked or reheated starches (rice, potatoes, sweet potatoes, oatmeal)
  • High-protein foods, such as chicken, tofu, fish, turkey, eggs, yogurt, beans, and chia seeds

Drink plenty of fluids every day to maintain electrolyte balance and prevent dehydration. Apart from water, which is the best for rehydration, you may take broth, smoothies, or oral rehydration solution. 

Keep a food journal to determine potential triggers. 

Further Reading: Ulcerative Colitis Diet Plan: What To Eat & Avoid

Stick to Your Treatment Regimen

Take your medications exactly as prescribed by your healthcare provider. Never discontinue your medication without talking to your provider. Abruptly discontinuing your medication may trigger a flare-up. 

When To Seek Medical Help

Talk to your healthcare provider immediately if you:

  • Notice blood clots in your stool
  • Have heavy and persistent diarrhea
  • Have a high fever
  • Have severe vomiting
  • Have continuous pain

Probiotics for Ulcerative Colitis Flare-Ups: Do They Work?

Probiotics may be effective in preventing flare-ups and reducing symptom severity. However, more studies are needed to confirm these benefits, determine the beneficial bacterial species, and the dosage. 

Moreover, there’s insufficient evidence to recommend probiotics alone as the primary treatment for UC. 

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Frequently Asked Questions

What are the new prescription treatments for ulcerative colitis?

The new prescription treatments for ulcerative colitis are:

  • Interleukin-23 selective inhibitors (risankizumab-rzaa and mirikizumab-mrkz)
  • Sphingosine 1-phosphate modulators (ozanimod and etrasimod)
  • Janus kinase inhibitors (tofacitinib and upadacitinib)

What are the apps that track ulcerative colitis flare-ups and symptoms?

Examples of apps for tracking and managing UC are: mySymptoms Food Diary, Aila Health, LyfeMD, We Can’t Wait, and Trellus Health. 

How can I control ulcerative colitis?

No cure for ulcerative colitis is available. However, medications and lifestyle changes can help manage symptoms, reduce relapse, and prevent complications. Always take your medications as prescribed and never stop medical treatment without talking to your provider. 

Can coffee trigger ulcerative colitis flare-ups?

Coffee may trigger a flare-up in some people. The exact mechanism is unclear, but health experts think it might involve stimulation of colon contractions. Avoid or try small servings if you think coffee worsens your condition. 

What foods should I avoid during a flare-up?

Foods to avoid during a UC flare-up include:

  • Alcohol
  • Sugary foods and drinks
  • Caffeine
  • Dairy products (milk, cheese, cream)
  • Greasy food
  • Foods that cause gas, such as beans and carbonated beverages
  • Heavily seasoned foods
  • Foods rich in insoluble fiber (raw vegetables, some raw fruits)
  • Red meat
  • Spicy food

How can I prevent UC flare-ups?

The following may help lower your risk of flare-ups:

  • Avoiding triggers
  • Engaging in regular physical activity
  • Managing stress 
  • Seeing your healthcare provider regularly 
  • Sticking to the treatment regimen 
  • Avoiding pain medications called nonsteroidal anti-inflammatory drugs (NSAIDs)

IVIG Mechanism of Action: A Detailed Look

IVIG works through multiple pathways rather than a single mechanism. These mechanisms include suppression of specialized white blood cells, neutralization of autoantibodies, and regulation of immune responses. 

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What Is IVIG?

Intravenous immunoglobulin (IVIG) is a blood product administered through an intravenous (IV) infusion. It contains purified antibodies obtained from the pooled plasma of healthy donors. 

When a foreign substance enters your body, your immune system produces protective proteins called antibodies or immunoglobulins (Ig). These antibodies attach to the foreign invader and eliminate it from the body. 

There are five types of antibodies: IgA, IgD, IgE, IgG, and IgM. IVIG products mainly contain IgG antibodies (over 90% of the total). 

Your healthcare provider may prescribe IVIG to boost the immune system, treat autoimmune conditions, and prevent infections in individuals with weakened immune systems. 

IVIG Mechanism of Action

IVIG is not a single-target therapy; its effects vary depending on dose, disease, and patient factors.

For example, IVIG acts as a replacement therapy in individuals with antibody deficiencies. Also, it acts as an immunomodulator and an anti-inflammatory in many autoimmune and inflammatory disorders. 

Moreover, IVIG can be both pro-inflammatory and anti-inflammatory, depending on the dose. 

IVIG Mechanisms of Action in Immunodeficiencies

As a replacement therapy, IVIG primarily acts by replacing the deficient IgG. This helps neutralize pathogens and reduce recurrent infections.

For this purpose, the typical replacement dose is 200 mg/kg to 400 mg/kg body weight, administered every 3 weeks [1].

At low doses, IVIG interacts with the foreign invaders to help activate the first line of immune defense. This part of the immune system (also known as the complement system):

  • Targets the foreign substance, such as bacteria
  • Triggers inflammation to prevent infection
  • Eliminates the foreign substance from the body

So, low-dose IVIG usually promotes inflammation to protect your body from foreign organisms [2].

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IVIG Mechanism of Action in Autoimmune and Inflammatory Diseases

IVIG nurse standing next to a pole  set up with an IVIG bottle and a pump

IVIG can simultaneously interact with different components of the immune system. Thus, the immunomodulatory actions of IVIG involve multiple mechanisms and vary widely among diseases. 

High doses, usually 2 g/kg/month, are used in autoimmune and inflammatory diseases. Unlike replacement IVIG, high-dose IVIG is more immunosuppressive and anti-inflammatory [3]. 

The immunomodulatory effects of IVIG are thought to occur as a result of the following interactions [4,5,6,7,8]:

Interaction With Specific Blood Cells

Monocytes and macrophages are white blood cells that identify and remove pathogens from the body. They do so by releasing inflammatory substances and engulfing pathogens. In a healthy individual, the lifespan of these cells is tightly regulated to prevent excessive inflammation.  

However, in chronic inflammatory diseases, these cells survive for longer than usual. Consequently, their accumulation further increases inflammation. IVIG binds to these cells and helps reduce inflammation. 

IVIG may help reduce inflammation by suppressing T cell multiplication. T cells are white blood cells that fight off disease-causing microorganisms. 

According to a 2024 study, T cells that migrate from the periphery to the site of tissue injury are involved in the development of many chronic inflammatory disorders, including [9]:

IVIG can enhance or block the activity of natural killer cells (NK cells). NK cells are white blood cells that kill virally infected cells and cancer cells. IVIG blocks NK cell activity in women experiencing recurrent spontaneous abortions, leading to favorable pregnancy outcomes. 

Contrarily, IVIG enhances NK activity in Kawasaki disease and seizure disorders. 

Interaction With Autoantibodies

Autoantibodies are proteins made by the immune system. They attack the body’s healthy tissues instead of foreign substances. In some individuals, they can lead to the development of autoimmune disorders. 

IVIG neutralizes the pathogenic autoantibodies. By doing this, it prevents the immune system from attacking the body’s healthy tissues and organs. IVIG reduces the production of autoantibodies by blocking the expansion of B cells. 

IVIG is used in many autoimmune disorders, such as: 

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Frequently Asked Questions

What is the mechanism of action of IVIG?

IVIG mechanisms of action vary widely depending on the dose, disease being treated, and patient factors. As a replacement therapy, IVIG works by replacing the missing antibodies in individuals with immunodeficiencies. As an immunomodulator, IVIG affects various immune cells and steps involved in immune regulation. 

What are IgM, IgG, IgA, IgD, and IgE?

They are five major types of immunoglobulins (antibodies). The subclasses of IgG and IgA are IgG1, IgG2, IgG3, IgG4, and IgA1 and IgA2, respectively. 

How does IVIG work to increase platelets?

Though the exact mechanism is unclear, IVIG may:

  • Reduce platelet destruction
  • Enhance signals for platelet production

How does IVIG work for Guillain-Barre syndrome?

Researchers have yet to determine the exact mechanism of action. However, they think IVIG can work for Guillain-Barre syndrome (GBS) by decreasing the pathogenic antibodies that attack the nerve cells. 

How does IVIG work in autoimmune disease? 

IVIG can help prevent tissue/organ damage in autoimmune disease by neutralizing the harmful antibodies and reducing the production of antibodies by B cells. 

How does IVIG help with myasthenia gravis?

The exact mechanism is not entirely understood. However, IVIG can help improve myasthenia gravis (MG) symptoms and muscle strength by neutralizing autoantibodies that impair communication between muscles and nerves.

Sevenfact: A New Treatment Option for Hemophilia With Inhibitors

Sevenfact is an FDA-approved medication used to treat or control bleeding in patients with hemophilia A or B with inhibitors. In this article, you will learn about its mechanism of action, dosage, side effects, cost, and more.

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What Is Sevenfact?

Sevenfact is a brand-name prescription medication. The active ingredient in this product is coagulation factor VIIa (recombinant)-jncw, also called eptacog beta. 

Sevenfact belongs to a class of medications known as hemostatics.

The term “recombinant” means this medication is made in a lab using recombinant DNA technology. The four lowercase letters “jncw” are used to distinguish Sevenfact from other similar products. 

The U.S. FDA approved Sevenfact in April 2020 to treat and control bleeding in individuals 12 years and older with hemophilia A or B with inhibitors. 

This product should not be used to treat individuals with congenital Factor VII deficiency.

What Is Sevenfact Used For?

Sevenfact is used to treat and control bleeding episodes in patients with hemophilia A or B who have developed inhibitors to standard clotting factor treatments.

Inhibitors are antibodies that develop in some patients and block standard clotting factor treatments, making bleeding more difficult to control.

Sevenfact Mechanism of Action

Recombinant coagulation Factor VIIa (rFVIIa) in Sevenfact helps the blood to clot by activating another clotting factor called Factor X. 

Normally, activation of Factor X depends on Factors VIII and IX. Sevenfact bypasses this pathway, allowing clot formation even when these factors are absent. For this reason, it is known as a “bypassing agent.”

Sevenfact Dosing

This medication comes as a white to off-white powder for solution (reconstitution) for intravenous (IV) injection in single-dose vials. 

The following strengths are available in the U.S.:

  • 1 mg of coagulation factor VIIa (recombinant)-jncw
  • 2 mg of coagulation factor VIIa (recombinant)-jncw
  • 5 mg of coagulation factor VIIa (recombinant)-jncw

The liquid to prepare the solution comes in single-dose prefilled glass syringes. Each syringe contains 1.1 ml, 2.2 ml, or 5.2 ml of sterile water for injection. 

The dose and duration of treatment depend on:

  • Location and severity of bleeding
  • Need for urgent bleeding control
  • Frequency of administration
  • Response to bypassing agents during earlier bleeding episodes

Recommended Sevenfact Dosing:

Mild and moderate bleeding: 75 mcg/kg repeated every 3 hours until bleeding stops. Alternatively, a 225 mcg/kg initial dose may be used. If bleeding doesn’t stop within 9 hours, an extra dose of 75 mcg/kg every 3 hours may be given as needed to stop bleeding.

If bleeding doesn’t stop within 24 hours after the first dose, your healthcare provider will likely switch you to an alternative treatment. 

Severe bleeding: 225 mcg/kg initial dose. If needed after 6 hours, give 75 mcg/kg every 2 hours until bleeding stops.

The duration of treatment depends on the site and severity of bleeding. 

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Sevenfact Side Effects

Common Side Effects

Patient with fever, a side effect of taking Sevenfact
  • Headache
  • Dizziness
  • Pain, redness, or swelling at the infusion site
  • Fever
  • Joint pain
  • Mild itching 
  • Nausea 
  • Vomiting 

Serious Side Effects

Call your healthcare provider immediately if you experience any of the following signs:

Stroke

  • Sudden numbness or weakness
  • Severe headache
  • Slurred speech
  • Difficulty with vision or balance

Blood clot in the lung

  • Chest pain
  • Sudden cough or breathing difficulty
  • Dizziness
  • Coughing up blood

Blood clot deep in the body

  • Pain, swelling, or warmth in one of your legs

Allergic Reactions

This medication may cause allergic reactions, which can be fatal. Seek emergency medical care if you experience:

  • Hives
  • Breathing difficulty
  • Swelling in the face or throat
  • Chest tightness
  • Fast heartbeat

Warnings and Precautions

This medication can cause severe or life-threatening reactions. Call your healthcare provider immediately if you experience:

  • Hives, itching, or rash
  • Chest pain or tightness
  • Shortness of breath
  • Wheezing
  • Dizziness
  • Low blood pressure
  • Pain, swelling, and warmth in one leg
  • Sudden numbness or weakness (particularly affecting one side of the body)
  • Severe headache
  • Slurred speech
  • Problems with vision or balance

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Before Using Sevenfact

You should not use this medication if you:

  • Have a history of allergic reactions to rabbits or rabbit proteins
  • Are allergic to this product or any product components

Before you receive your first dose of Sevenfact, inform your healthcare provider if you have or have ever had:

  • Coronary artery disease (narrowing of the arteries that supply oxygen to the heart)
  • Heart attack or stroke
  • Severe injury or infection
  • Allergic reactions to mouse, hamster, or cow proteins
  • Liver disease

Also, inform your healthcare provider if you are:

  • Pregnant or planning to get pregnant
  • Breastfeeding or planning to breastfeed

Sevenfact vs. NovoSeven

Sevenfact and NovoSeven are coagulation factors. The differences are listed below:

FeaturesSevenfactNovoSeven
Approved Use Hemophilia A or B with inhibitors in adults and adolescents 12 years and older Adults and children with hemophilia A or B with inhibitors, congenital Factor VII deficiencyAdults with acquired hemophilia 
FDA approval date20201999
Active ingredient eptacog beta eptacog alfa
Available strengths 1 mg, 2 mg, and 5 mg  of coagulation factor VIIa (recombinant)-jncw 1 mg, 2 mg, 5 mg, or 8 mg recombinant coagulation factor VIIa (FVIIa)
Manufacturer HEMA Biologics Novo Nordisk Limited
ContraindicationsKnown allergy to rabbits or rabbit proteinsSevere allergy to this product or any product componentsNone

Sevenfact Cost

Cost can vary depending on your insurance plan, location, and pharmacy. Contact your insurance provider to find out if your plan covers this medication or if you need prior authorization.

The manufacturer of Sevenfact, HEMA Biologics, offers several support services through its “HEMA Biologics Cares” program for eligible patients. These include:

Sevenfact Co-pay Savings Program

Eligible patients may receive up to $12,000 in savings per calendar year through HEMA Biologics Cares. 

Patient Assistance Program (PAP) 

The Patient Assistance Program (PAP) provides this medication at no cost if patients meet specific income requirements. 

Quick Start Program

This program is for new users who are having an insurance coverage-related delay in getting their treatment. 

Bridge Program

This program is for current users who are having a delay in getting their treatment due to changes in their insurance coverage. 

Contact us if you are interested in exploring financial assistance opportunities or copay assistance for Sevenfact. 

IVIG for Long COVID: Early Findings Suggest Potential Relief for Patients

Use of IVIG for long COVID is experimental, with some small studies showing IVIG may improve some symptoms. Large trials are currently underway. IVIG therapy may be considered in selected cases under specific circumstances.

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What Is Long COVID?

Synonyms: Post COVID-19 Condition, Infection-associated chronic condition, long-haul COVID, post-COVID-19 syndrome, post-acute sequelae of SARS-CoV-2 (PASC)

Long COVID is a chronic (long-term) condition. It occurs when an individual affected by the SARS-CoV-2 infection continues to have or develops new symptoms 3 months after initial infection. These symptoms usually last for at least 2 months. 

According to a 2022 WHO report, long COVID may affect 10% to 20% of individuals infected by the virus that causes COVID-19 [1].

Long COVID Symptoms

Man with headache suffering from symptoms of long COVID

Research shows long COVID may have over 200 symptoms. Common symptoms can include:

  • Excessive tiredness, typically after activity
  • Brain fog (problems with memory)
  • Dizziness
  • Shortness of breath
  • Changes in smell and taste
  • Sleep problems
  • Cough
  • Headache
  • Rapid or irregular heartbeat
  • Digestive issues, such as loose stools or constipation 

Scientists are studying how COVID-19 causes these symptoms. COVID-19 can affect almost every system in the body. 

While the exact mechanism is unclear, long-term illness may result due to:

  • Changes in immune system communication, causing the immune cells to attack the body’s healthy cells and tissues (autoimmunity)
  • Reactivation of viruses that were previously dormant
  • Unfavorable changes in the gut, blood vessels, and certain areas of the brain

IVIG for Long COVID: What Does the Latest Research Say?

Limited evidence suggests IVIG for long COVID may be an option. Below are the findings of the most recent studies. 

2021 

In a 2021 case report, a 12-year-old girl who developed burning pain, limb weakness, slurred speech, and impaired cognition following COVID-19 received IVIG 2 g/kg/month for 5 months. After IVIG therapy, she achieved overall initial improvement [2].

2023

In a 2023 study, researchers from the University of Kentucky administered IVIG to six participants with long COVID. The most common symptoms in the participants were brain fog, fatigue, chest pain, and shortness of breath. 

IVIG treatment was initiated 101 days to 547 days after initial COVID infection. The IVIG dosage used in this study was 0.5 g/kg every 2 weeks for up to one year. At the end of the study, the researchers concluded that IVIG may bring meaningful benefits to patients with long COVID, such as improvements in neurological problems, chest pain, and fatigue [3]. 

Interestingly, nearly half of the participants in this study reported getting back to “almost normal.”

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2024

According to a 2024 study, IVIG might be a candidate treatment for improving neurological symptoms, fatigue, and disability risk in individuals with long COVID [4]. This study involved 21 participants who received 3 monthly courses of 0.5 g/kg IVIG, along with supportive therapy. 

In the study, the control group (29 participants) received only supportive therapy. 

Ongoing Research: IVIG for Long COVID 

The National Institutes of Health (NIH) is currently conducting a large study to evaluate IVIG for long COVID. Known as Researching COVID to Enhance Recovery (RECOVER), it is one of the world’s largest studies dedicated to understanding, diagnosing, and treating long COVID. 

This trial is evaluating three potential treatments:

  • Gamunex-C (an IVIG preparation)
  • Ivabradine (an oral medicine to lower heart rate)
  • Coordinator-guided, non-pharmaceutical care 

The result of this trial is scheduled to be presented at the American College of Cardiology (ACC) Annual Conference. 

IVIG for Long COVID: What Are the Potential Advantages?

IVIG therapy has been found to be effective in over 100 hematologic, inflammatory, autoimmune, and infectious diseases. Moreover, many studies have established that IVIG can effectively regulate the immune system. 

Also, a growing body of evidence suggests that autoimmunity might be one of the drivers of long COVID [5]. Given the demonstrated benefits of IVIG for autoimmune disorders, there is a potential for IVIG for long COVID as well. 

IVIG for Long COVID: What Are the Limitations?

First and foremost, the efficacy of IVIG for long COVID has not been established in large trials. Available information about potential benefits is largely based on small studies. Besides, other factors like cost, accessibility, and a lack of standard dose/timing all seem to affect the routine use of IVIG for long COVID. 

Current Treatments for Long COVID

Currently, there is no definitive treatment for long COVID. Treatment can vary depending on the bodily systems involved and an individual’s unique signs and symptoms.

Symptomatic treatment aims to relieve specific symptoms in an individual. For example, if they have a fever, a healthcare provider may give them acetaminophen. Likewise, vitamin C may be given to relieve fatigue. 

Supportive treatment can include appropriate counseling and talk therapy, such as cognitive-behavioral therapy (CBT). 

Other options can include rehabilitation for chronic cough, breathing exercises, and self-monitoring. 

Key Takeaways

Because long COVID affects multiple bodily systems and has a wide range of symptoms, health experts agree that IVIG may not work as a standalone therapy. However, IVIG can be a valuable addition to a multidisciplinary treatment approach. 

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IVIG for Long COVID: Frequently Asked Questions

What is the best treatment for long COVID symptoms?

There is no single best treatment for long COVID. Treatment can be symptomatic, supportive, and rehabilitative, depending on the specific situation. Long COVID treatment requires a multidisciplinary and individualized approach. 

Is IVIG used for long COVID?

Studies evaluating IVIG for long COVID are underway. Some small studies show IVIG may improve certain symptoms of long COVID. 

What are the top-rated IVIG brands for long COVID?

So far, studies have used only two IVIG brands for long COVID: Privigen and Gamunex-C. 

Does insurance cover IVIG for long COVID?

Because the use of IVIG for long COVID is still experimental, it is unlikely that your insurance will cover it. 

What medication is used for long COVID fatigue? 

Some emerging therapies, such as low-dose naltrexone, are being explored for managing long COVID fatigue.

Corifact (Factor XIII Concentrate): FDA-Approved Treatment for Congenital Factor XIII Deficiency

Corifact (also known by its generic name, factor XIII concentrate) is an injectable prescription medicine used to prevent excessive bleeding in people with congenital factor XIII deficiency, which is a rare inherited bleeding disorder. This condition affects around 1 in every 2 to 3 million people, with an incidence in the U.S. of approximately 150 people.

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Corifact belongs to the drug class of coagulation factors and is also classified as a hemostatic agent that helps prevent and control bleeding by supporting proper blood clot formation. 

This drug was approved by the U.S. Food and Drug Administration (FDA) in 2011 and is available in 12 different countries around the world under the brand name Fibrogammin®- P. 

Read on to learn when it is recommended, how it works, its dosage, and important precautionary measures. 

Approved Uses

Corifact is approved only for the treatment of congenital factor XIII deficiency in both adults and pediatric patients. It is prescribed in the following situations:

  • To help prevent unexpected bleeding before it happens, even if there is no injury.
  • To prevent and treat bleeding during or after minor or major surgical procedures.
  • To prevent uncontrolled bleeding after minor injuries.

How Corifact Works 

To better understand how Corifact works, it is important to first understand congenital factor XIII deficiency. 

This extremely rare bleeding disorder occurs when a person is born with too little or no factor XIII protein. The factor XIII protein usually helps blood clots become strong and stable. 

Normally, when you get a cut or injury, your body forms a clot to stop bleeding. Factor XIII protein strengthens the clot so it stays in place while healing occurs. 

However, when you have factor XIII deficiency, blood clots do form, but they are weak and unstable. Without enough Factor XIII, the clot breaks down too quickly, leading to prolonged or unexpected bleeding, even after minor injuries. 

Corifact Working Mechanism

Corifact contains a purified form of human factor XIII protein extracted from pooled human plasma donated by healthy individuals. It works like the body’s natural clotting protein and replaces the missing factor XIII in the bloodstream. Once infused, it:

  • Increases the level of Factor XIII in the blood
  • Strengthens blood clots
  • Helps clots stay in place long enough for proper healing
  • Reduces the risk of spontaneous or uncontrolled bleeding
  • Helps prevent spontaneous bleeding episodes

By maintaining adequate Factor XIII levels, Corifact helps the body form stronger, more stable clots, lowering the risk of serious bleeding episodes.

Drug Form and Strengths

Corifact comes as a white, lyophilized (freeze-dried) powder form that must be mixed with a sterile diluent before use. It is available in two vial strengths:

  • 250 IU, which is mixed with 4 ml of Sterile Water for Injection
  • 1,250 IU, which is mixed with 20 ml of Sterile Water for Injection

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Dosage for Adults and Children

The dose of Corifact is based on body weight, not age. This means adults and children generally receive the same weight-based dosing schedule.

Routine Preventive Treatment (Prophylaxis)

Both adults and children with congenital Factor XIII deficiency receive a dose of 40 IU/kg body weight intravenously, once every 4 weeks (every 28 days). 

The doctor may adjust the dose based on the factor XIII blood levels, bleeding history, or any upcoming surgeries or procedures. 

For Perioperative Management of Surgical Bleeding

If you need surgery or experience a bleeding episode, your healthcare provider may adjust the dose based on your Factor XIII levels, the type of surgery, and your clinical response.

Corifact: Administration 

Corifact is given as an intravenous (IV) infusion, administered directly into a vein. Before infusing, Corifact is dissolved with sterile water provided in the package.

Possible Side Effects

Man experiencing join inflammation, one of the side effects of Corifact

Corifact is generally a well-tolerated drug. But you may experience some common side effects such as:

  • Joint inflammation
  • Joint pain (arthralgia)
  • Mild rash or itching
  • Headache
  • Pain at the injection site
  • Mild fever
  • Elevated thrombin-antithrombin levels
  • Increased blood lactate dehydrogenase

Serious Side Effects (Rare)

Seek immediate medical attention if you experience:

  • Hypersensitivity or allergic reactions (rash, itching, swelling)
  • Acute ischemia

Corifact: Contraindications and Warnings

You should not use Corifact if you have experienced hypersensitivity reactions, including anaphylactic or severe systemic reactions to human plasma-derived products or to any ingredient in Corifact.  

In rare cases, the body may develop antibodies (inhibitors) against Factor XIII, which can reduce how well Corifact works. Your doctor may monitor your blood levels if treatment seems less effective.

Moreover, because Corifact is made from human plasma, there is a very small risk of viral transmission, including viruses and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent.

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Precautionary Measures You Should Take

Before using Corifact, it is important to take some safety measures to help ensure the medicine works effectively and safely. Always consult your healthcare provider before taking Corifact if you:

  • Are pregnant or plan to become pregnant, as it is not known whether Corifact can harm your unborn baby.
  • Are breastfeeding or plan to breastfeed, because it is not known if Corifact passes into breast milk.
  • Have experienced allergic reactions to this drug, its ingredients, or components of the container. 
  • Are taking any over-the-counter medications or supplements, or herbal products.
  • Have or have had any liver disease. 
  • Have been told that you have inhibitors (neutralizing antibodies) against factor XIII, as Corifact may not work effectively in this case. 

Corifact: Estimated Cost

The cost of Corifact can vary widely depending on your healthcare coverage and how much medication you need. Because Corifact is a specialized clotting factor product, it is typically very expensive, often costing several thousand dollars per vial depending on dosage and insurance coverage.

Contact us if you are interested in exploring financial assistance or copay assistance for Corifact. 

Natural Treatments for Myasthenia Gravis: Do They Really Work?

Natural treatments for myasthenia gravis and lifestyle habits may help control symptoms and improve quality of life. These can include acupuncture, speech/swallowing therapy, a balanced diet, and supervised exercise. However, they should not replace standard medical therapy. 

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Myasthenia gravis (MG) is a long-term autoimmune disorder. MG causes weakness of muscles you can control, especially those in the eye, face, throat, neck, and limbs. It occurs when the immune system disrupts the communication between the nerves and muscles. 

Standard treatments for myasthenia gravis include medications, surgery, plasma exchange, and intravenous immunoglobulin (IVIG).

Natural Treatments for Myasthenia Gravis: A Closer Look at the Evidence

Natural treatments may help some people with myasthenia gravis improve their quality of life. Nonetheless, they should not replace conventional MG treatments. 

Acupuncture

Acupuncture is a form of traditional Chinese medicine. During an acupuncture session, a practitioner inserts fine needles through the skin at specific points on the body. According to the practitioners, doing so will help rebalance the flow of “life force” in the body. 

Acupuncture is mainly used to treat:

  • Dental pain 
  • Neck pain
  • Low back pain 
  • Labor pain
  • Menstrual cramps 
  • Joint pain

Acupuncture may help reduce symptom severity and improve quality of life in some MG patients by stimulating local nerves. 

For example, in a 2024 review, researchers found that acupuncture (combined with conventional treatments) may benefit MG patients as a supportive therapy. In this study, acupuncture was associated with improved quality of life and reduced symptom severity [1].

Likewise, participants in a small 2025 trial reported improvements in MG symptoms after twice-weekly acupuncture for 12 weeks. According to the researchers, acupuncture is safe and feasible for MG patients [2].

However, this is an ongoing area of research. Large high-quality studies are necessary to establish acupuncture as an effective MG treatment. 

Speech and Swallowing Therapy

People with MG often have difficulty speaking and swallowing. These issues can occur because MG significantly weakens the muscles in the head, neck, and throat. A 2025 study found that people with MG have a significantly higher risk of speech and swallowing difficulties compared with the general population [3]. 

Common speech problems include vocal fatigue, monotonous or hypernasal voice, and periods of voice loss. 

Speech therapy can help people with MG improve their speech clarity. Moreover, they can learn compensatory strategies to increase understandability, such as [4]:

  • Using short phrases
  • Taking regular breaks to avoid vocal fatigue
  • Developing alternative modes of communication 
  • Using gestures or facial expressions for effective communication 
  • Avoiding speaking in loud environments 

Common swallowing problems include coughing or choking during or after eating, slow eating, and shortness of breath when eating or drinking. 

Swallowing therapy is an evidence-based approach. This rehabilitation program may help reduce the risk of choking, aid efficient eating, and strengthen swallowing-related muscles.

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Respiratory Muscle Training 

Sometimes, MG can weaken muscles involved in breathing, such as the diaphragm and the intercostal muscles. If this happens, a person may experience shortness of breath. 

Respiratory muscle training (RMT) can help increase respiratory muscle endurance, improve functional outcomes, and reduce fatigue in MG patients [5,6].

A respiratory therapist may recommend breathing exercises, such as:

  • Belly breathing 
  • Inspiratory muscle training
  • Pursed-lip breathing 

Lifestyle Measures for Myasthenia Gravis

Find Ways To Save Your Energy

Fatigue is a common, often debilitating feature of myasthenia gravis. To conserve your energy, try the following:

  • Schedule important or physically demanding tasks appropriately. 
  • Sit more often than you stand.
  • Rest when you need to. 
  • Ask for help when you need to. 
  • Try to eat when you feel the strongest. 
  • Use tools to save your energy. 

Manage Potential Triggers

Stress, infection, heat, and overexertion may worsen myasthenia gravis symptoms. Take steps to avoid these triggers. These can include:

  • Avoiding extreme heat when possible. 
  • Avoiding exposure to people with signs of an infection. 
  • Saving energy to reduce fatigue. 
  • Consulting a healthcare provider before using a new medicine or getting a vaccine.

Symptoms may also worsen during pregnancy. Inform your provider if you are pregnant or plan to get pregnant. 

Exercise 

A woman walking in the park for exercise

Because exercise can increase fatigue, many people with MG tend to avoid physical activity. However, exercise is still crucial for overall health, mood, balance, and strength. 

If you have mild to moderate MG, consider an appropriate activity to meet the weekly goal of 150 minutes of exercise. Always consult your provider before starting any new activity. Also, make sure you don’t overexert yourself. Low-impact exercises such as swimming, walking, or yoga are effective and less rigorous on the body.

A Healthy Diet 

A healthy diet is the foundation of health. While there’s no MG-specific dietary guideline, the fundamentals of healthy eating still apply. 

We have an entire article dedicated to healthy eating when you have myasthenia gravis. Read more about the Best Diet for Myasthenia Gravis. 

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Myasthenia Gravis Natural Treatments: Frequently Asked Questions 

Can myasthenia gravis be completely cured?

No. Currently available treatments help manage symptoms and prevent complications, but don’t cure myasthenia gravis. 

Are there natural supplements for myasthenia gravis?

Limited evidence suggests certain Chinese herbal formulas and vitamin supplementation may offer additional benefits. However, there is no evidence that these remedies significantly affect disease progression. Always talk to your healthcare provider before trying supplements. 

What medications can trigger myasthenia gravis?

Certain antibiotics, blood pressure medications, and anti-seizure medications may worsen MG symptoms. 

What foods should I avoid if I have myasthenia gravis?

Foods to avoid with myasthenia gravis include:

  • Difficult-to-swallow foods, such as dry/crunchy/crumbly foods
  • Dry bread products such as bagels, muffins, and sandwiches 
  • Tough meats that are hard to chew and swallow
  • Ultraprocessed foods, which are high in added salt or sugar
  • Foods high in caffeine, such as coffee and soda
  • Hot, spicy foods

Does vitamin B12 benefit people with myasthenia gravis?

There is no evidence to suggest that vitamin B12 affects MG disease activity or that vitamin B12 supplementation can improve symptoms. 

AlphaNine SD Guide: How It Works, Dosing, Risks, and Benefits

AlphaNine SD injection is a human plasma-derived factor IX preparation. It is used to prevent or control bleeding in patients with Factor IX deficiency caused by hemophilia B. In this article, you will learn about its mechanism of action, dosage, side effects, cost, and more.

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AlphaNine SD Introduction and Uses

AlphaNine SD is a brand-name injectable medication used to prevent bleeding in people aged 16 years and older with hemophilia B. It contains the active ingredient “factor IX or Christmas factor”, which is derived from human plasma. This medication belongs to the drug class known as coagulation factors or hemostatics. 

“SD” in the brand name stands for “solvent detergent” treated, which is a virus-inactivation technology. 

AlphaNine SD should not be used to treat patients with other bleeding problems, such as:

  • Factor II, VII, or X deficiencies
  • Severe bleeding caused by certain blood thinners (anticoagulants)
  • Hemophilia A patients with antibodies to Factor VIII

AlphaNine SD Mechanism of Action

AlphaNine SD temporarily replaces deficient factor IX in patients with hemophilia B with working clotting factors, which helps to stop or prevent bleeds. 

AlphaNine SD Dosing

This medication comes as a sterile powder for solution (reconstitution) for intravenous (IV) injection in single-dose vials. It is supplied with a 10-ml vial of “sterile water for injection” and a filter transfer set. 

The following strengths are available in the U.S.:

  • 500 IU factor IX (FIX)/10 ml single-dose vial
  • 1,000 IU FIX/10 ml single dose vial
  • 1,500 IU FIX/10 ml single dose vial

The amount of AlphaNine SD required to stop or prevent bleeds depends on a patient’s specific needs. Before administering this medication, your healthcare provider will use a specific formula to determine the dose. They will individualize dosing based on:

  • Severity of factor IX deficiency
  • Extent and location of bleeding
  • Your clinical status

Then they will prepare the medication according to the instructions in the package insert.

Recommended AlphaNine SD Dosing

Minor bleeding: 20-30 IU per kilogram twice daily until bleeding stops and healing has been achieved. Treatment typically lasts 1 to 2 days. 

Moderate bleeding: 25-50 IU per kilogram twice daily until healing has been achieved. Treatment typically lasts 2 to 7 days. 

Major bleeding: 30-50 IU per kilogram twice daily for 3 to 5 days. Then, 20 IU per kilogram twice daily until healing has been achieved. Treatment may last for up to 10 days. 

AlphaNine SD Side Effects

Common Side Effects

  • Nausea
  • Headache
  • Dizziness
  • Altered sense of taste
  • Mild skin rash
  • Pain, redness, or itching at the injection site

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Serious Side Effects

  • Weight gain, swelling in the waist, hands, or lower legs
  • Loss of appetite
  • Fever or chills
  • Continued bleeding after treatment
  • New or worsened bleeding
  • Signs of excessive blood clotting, such as sudden numbness, slurred speech, problems with vision or balance, and coughing up blood

AlphaNine SD Proper Use and Storage

Doctor consults with a boy and his father about AlphaNine SD and hemophilia
  • Use this medication exactly as directed by your prescriber. 
  • You will likely receive your first dose in a hospital or clinic setting. Then, your healthcare provider will teach you how to prepare and self-administer this medication at home, if deemed appropriate. 
  • Ask your prescriber or pharmacist if you have problems understanding any part of the “Instruction for Use” that comes with the medication.
  • After mixing your dose with the liquid, use the prepared medication within 3 hours. Never use expired medication. Contact your prescriber or pharmacy if you need a refill.
  • Don’t use the mixture if it looks cloudy or contains particles. 
  • Store between 2 and 8°C (36°F to 46°F). You may store the medication at room temperature (not exceeding 30°C or 86°F) for up to 30 days. 

Before Using AlphaNine SD

You should not use this medication if you:

  • Have a history of allergic reactions to clotting factor medicines
  • Have signs of excessive blood clotting
  • Are allergic to hamster proteins

Before you receive your first dose of AlphaNine SD for hemophilia B, inform your healthcare provider if you’ve ever had:

  • Kidney or liver disease
  • Coronary artery disease (narrowing of the arteries that supply oxygen to the heart)
  • A heart attack or stroke

Also, inform your healthcare provider if you:

  • Are pregnant or planning to get pregnant
  • Are breastfeeding

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Warnings and Precautions

Disease Transmission Risk

Because this medication is derived from donated human plasma, it may contain viruses or other disease-causing agents. While donated plasma is tested and treated to lower the risk of disease transmission, a small possibility of transmission cannot be ruled out. Ask your healthcare provider about any potential risk.

Allergic Reactions

This medication may cause allergic reactions, which can be fatal. Seek emergency medical care if you experience:

  • Hives
  • Breathing difficulty
  • Swelling in the face or throat
  • Chest tightness
  • Fast heartbeat

In previously untreated patients, severe allergic reactions are most likely to occur after 11 days of exposure to this medication. 

Inhibitor (Antibody) Formation

In some cases, your body can produce proteins called “factor IX inhibitors”, which can prevent this medication from working as intended.

AlphaNine SD vs. Alprolix

Both products are used to treat people with hemophilia B. AlphaNine SD is a human plasma-derived preparation with a standard half-life. On the other hand, Alprolix is a recombinant product with a long half-life, allowing for less frequent administration.

AlphaNine SD Cost

Cost can vary depending on your insurance plan, location, and pharmacy. Contact your insurance provider to find out whether your plan covers this medication or requires prior authorization.

The manufacturer of AlphaNine SD, Grifols, offers several assistance programs for eligible patients. These include:

  • The $0 Copay Assistance Program may cover out-of-pocket expenses not covered or partially covered by insurance. 
  • The Free Trial Program for those who are new to AlphaNine SD.
  • Benefits investigation and support services that help patients coordinate with their provider. 
  • The Patient Assistance Program for patients with no coverage or lapsed coverage.
  • Care Coordination to help patients gain access to and remain on AlphaNine SD.

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Plasmapheresis (Therapeutic Plasma Exchange) for Myasthenia Gravis

Plasmapheresis (or plasma exchange) is a type of intravenous therapy used in severe cases of myasthenia gravis (MG), especially when there is a high risk of respiratory failure. MG is a rare, long-term neurological condition that causes profound muscle weakness and extreme fatigue. 

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In the United States, around 30,000 to 60,000 people are living with myasthenia gravis. With modern treatments and improved critical care, most people with MG can live a normal or near-normal lifespan. However, severe complications such as myasthenic crisis — which can affect breathing — require urgent medical attention.

There are several treatment options available for MG, but plasmapheresis is one of the fastest-acting and most effective rescue therapies for severe MG symptoms, such as difficulty breathing or swallowing. 

If your doctor has recommended plasmapheresis, this article will help you understand what plasmapheresis is, why it is recommended, how the treatment works, what you can expect during the procedure, and its possible benefits and limitations. 

Myasthenia Gravis: Brief Overview

Myasthenia gravis, or MG, is an antibody-mediated autoimmune neurological disorder that affects the voluntary muscles, especially the muscles of the eyes, face, throat, neck, arms, and legs. This condition occurs when the immune system produces autoantibodies that attack the connection between nerves and muscles (known as the neuromuscular junction). 

This immune attack makes it harder for muscles to receive signals from the nerves, so they become weak and tire easily. Common symptoms of MG include:

  • Drooping eyelids
  • Double or blurred vision
  • Trouble speaking or swallowing
  • General muscle weakness

In severe cases, the weakness can involve the respiratory muscles. This medical emergency, known as a myasthenic crisis, is life-threatening and requires urgent medical care. During a crisis, a person may struggle to breathe, have difficulty speaking clearly, or be unable to swallow food or liquids safely.

While there is no cure for MG, certain treatments, such as oral medications (e.g., corticosteroids, immunosuppressants, cholinesterase inhibitors), surgery (e.g., thymectomy), and intravenous therapy (e.g., IVIG and plasmapheresis), can help manage symptoms and reduce immune activity in patients. 

Plasmapheresis: A Form of Treatment for Myasthenia Gravis

Plasmapheresis, or therapeutic plasma exchange (TPE), is a medical procedure in which the liquid part of the blood (plasma) is removed and replaced. 

Your blood is made up of four main components: red blood cells, white blood cells, platelets, and plasma. Plasma is the liquid portion that generally contains proteins, antibodies, hormones, and other substances. 

In patients with myasthenia gravis (MG), harmful antibodies that attack the neuromuscular junction are present in the plasma. By removing these harmful antibodies, plasmapheresis helps alleviate symptoms, provides rapid symptom relief, and improves muscle strength and motor performance in MG patients.

When It Is Recommended

Doctors recommend plasmapheresis to MG patients under the following circumstances: 

  • Myasthenic crisis, when breathing or swallowing muscles are severely weakened
  • Severe symptom flare-ups, even if a full crisis hasn’t developed
  • Before surgery (including thymectomy) to reduce the risk of complications
  • When medications are not working well enough or take too long to show benefit

Many MG patients experience rapid symptom improvement within a few days of receiving plasmapheresis.

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Plasmapheresis and Myasthenia Gravis: Key Research Insights

Arm of an individual giving a plasma donation for plasmapheresis

Research shows that plasmapheresis can be an effective treatment for people with moderate to severe myasthenia gravis, especially during sudden worsening of symptoms or a myasthenic crisis. 

In fact, a real-world study found that plasmapheresis produced rapid clinical improvement in approximately two-thirds (66.7%) of patients with acetylcholine receptor antibody (AChR-Ab)–positive generalized myasthenia gravis. The most notable benefit was improvement in respiratory and limb muscle weakness during disease exacerbations or myasthenic crisis. This study also suggests that plasmapheresis treatment was most effective in patients with higher antibody levels and those with thymus involvement.

Similarly, another study found that people with moderate-to-severe myasthenia gravis showed clear improvement within 2 weeks of plasmapheresis. Patients who had more severe symptoms at baseline, tested positive for acetylcholine receptor antibodies, and had thymic abnormalities or a history of thymectomy were more likely to experience significant benefit.

These findings suggest that certain clinical features can help predict who will respond best to plasmapheresis.

What To Expect During Plasmapheresis

The plasmapheresis procedure is only performed in a hospital or specialized treatment centers. It usually takes 2 to 3 hours, depending on your height, weight, and blood health. 

During the treatment:

  • You will sit in a reclining chair or lie on a bed. 
  • A healthcare provider will insert a needle or catheter into your vein (usually in the arm) to draw out the blood.  
  • The needle or catheter is connected to tubing that carries your blood to the plasmapheresis machine.
  • The blood will then slowly pass through a machine to separate the plasma from the blood cells (red cells, white cells, and platelets).
  • The plasma, which may contain harmful antibodies, is removed and discarded. 
  • The remaining blood cells are mixed with a replacement fluid, such as a protein-albumin solution or plasma collected from healthy donors, before they are returned to the body.
  • This cycle is repeated until the required volume of plasma is exchanged (usually 1–1.5 times your total plasma volume per session).

You may receive multiple sessions (often four to six sessions) over 1 to 2 weeks, depending on your symptom severity and how well you respond.

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Benefits of Plasmapheresis 

Plasmapheresis offers several benefits to MG patients, which include:

1. Rapid Symptom Relief

Unlike many MG medications that take time to resolve symptoms, plasmapheresis removes harmful antibodies directly and helps reduce symptoms quickly, often within days. Patients with severe weakness or breathing problems (myasthenic crisis) benefit the most. 

2. Short-Term Treatment

For patients who cannot tolerate high doses of steroids or other immunosuppressive drugs, or who experience other complications, plasmapheresis can be a useful short-term treatment, as it does not rely on immune suppression over the long term.

3. Improved Daily Function and Quality of Life

Plasmapheresis strengthens muscles and improves motor performance. This makes everyday activities like breathing, eating, speaking, and performing routine tasks safer and easier for MG patients.

Side Effects and Limitations of Plasmapheresis 

Generally, plasmapheresis is a very safe procedure, but it also has some side effects and limitations. 

Possible Side Effects

Serious side effects are rare, but you may feel cold (hypothermic), dizzy, or nauseous during or after the treatment. Hypotension (low blood pressure) and hypocalcemia or hypomagnesemia (low calcium) are also common. 

Tell your healthcare provider or medical staff immediately if you experience any of the above side effects.

Limitations

A major limitation is that plasmapheresis provides temporary relief from severe MG symptoms. Because the body continues to produce antibodies, symptoms may gradually return weeks after treatment. For ongoing disease control, doctors rely on medications like immunosuppressants and acetylcholinesterase inhibitors. 

Hence, plasmapheresis cannot be used as a long-term standalone treatment for myasthenia gravis. Moreover, if it is used repeatedly, it may increase the risk of serious infections such as catheter-related infections. 

Key Takeaway

Plasmapheresis is an effective and widely recommended treatment for providing rapid symptom relief during a myasthenic crisis. However, it does not replace your regular MG medications. Even after plasmapheresis, you will usually need to continue your regular MG medications, such as immunosuppressants or acetylcholinesterase inhibitors, to help control the disease and prevent future flare-ups.

Pazopanib (Votrient): Uses, How it Works, Dosage, Side Effects, and More

Pazopanib, sold under the brand name Votrient, is a targeted cancer medication frequently prescribed to treat specific types of cancers. It belongs to the class of potent kinase inhibitors that work to slow down tumor growth and spread to other parts of the body. 

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Pazopanib was initially approved by the FDA in 2009 for the treatment of kidney cancer. Later, its approval was expanded to include the treatment of sarcoma as well. 

This cancer drug comes in the form of a capsule-shaped tablet that is taken orally, as prescribed. It is available only with a prescription from a physician. Read on to learn how Pazopanib works, how it is used, possible side effects, and what to expect during treatment. 

Pazopanib (Votrient): Uses

Pazopanib is an FDA-approved drug and is typically prescribed to adult patients with the following types of cancers:

  • Kidney cancer, also known as advanced renal cell carcinoma (RCC), is a type of cancer that has advanced or has spread to other organs.
  • Soft-tissue sarcoma (a cancer that occurs in bone and connective tissues, such as fat, muscle, and blood vessels). 

Pazopanib may be prescribed for other purposes as determined by your healthcare provider. 

Important Safety Information (Boxed Warning)

Pazopanib may cause severe or life-threatening liver damage (hepatotoxicity). Therefore, it is recommended that liver function be monitored during treatment with this drug. Call your doctor immediately if you experience any of the following symptoms: yellowing of the skin or eyes, dark urine, extreme tiredness, nausea, vomiting, loss of appetite, pain in the upper right part of the stomach, or unusual bleeding or bruising. 

Pazopanib: Working Mechanism

Pazopanib is a type of targeted cancer medicine that attacks cancer in a more specific way than traditional chemotherapy. Typically, cancer cells need oxygen and nutrients to grow and spread. To achieve this, they release specific proteins (tyrosine kinases) that signal the body to form new blood vessels (a process called angiogenesis). 

Pazopanib, which is a potent tyrosine kinase inhibitor (TKI), blocks these proteins (tyrosine kinases) and helps:

  • Stop the growth of new blood vessels that feed the tumor
  • Reduce the supply of oxygen and nutrients to cancer cells
  • Slow down tumor growth and spread

In short, it starves the cancer cells by cutting off their blood supply and growth signals that tumors need to grow.

Pazopanib: Drug Form

Pazopanib is available as a capsule-shaped oral tablet in strengths of 200 mg and 400 mg. 

Recommended Dosage and Administration

The recommended dose of pazopanib for patients with advanced kidney cancer and soft-tissue sarcoma is 800 mg once a day. 

Pazopanib tablets are taken on an empty stomach with a glass of water, at least 1 hour before or 2 hours after a meal. You should swallow the tablet whole; do not crush, chew, or break it. 

Also, do not eat grapefruit or drink grapefruit juice during treatment with pazopanib. Grapefruit products may increase the amount of pazopanib in your body.

If you miss a dose, take it as soon as you remember. Do not take it if it is close (within 12 hours) to your next dose. Just take the next dose at your regular time. 

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Possible Side Effects

Patient suffering from side effects of pazopanib

Pazopanib may cause some side effects. You might have one or more of them. The commonly reported side effects in individuals with kidney cancers after taking pazopanib are:

  • Diarrhea
  • Hypertension
  • Hair color changes (depigmentation)
  • Nausea
  • Anorexia
  • Vomiting

People with soft-tissue sarcoma may experience the following side effects after taking Pazopanib:

  • Fatigue
  • Diarrhea
  • Nausea
  • Decreased weight
  • Hypertension
  • Decreased appetite
  • Vomiting
  • Tumor pain
  • Hair color changes
  • Musculoskeletal pain
  • Headache
  • Dysgeusia (taste changes)
  • Dyspnea (shortness of breath)
  • Skin hypopigmentation

Adverse Side Effects

Pazopanib may cause serious side effects, including: 

  • Liver problems (yellowing of skin or eyes, dark urine, severe tiredness)
  • Severe high blood pressure (hypertension)
  • Irregular or fast heartbeat or fainting
  • Heart failure
  • Heart attack or stroke
  • Bleeding problems (unusual bruising, coughing up blood, black stools)
  • Blood clots (sudden leg pain or swelling, trouble breathing)
  • Severe stomach or bowel problems (tear in your stomach or intestinal wall [perforation] or an abnormal connection between two parts of your gastrointestinal tract [fistula])
  • Lung problems (inflammation in the lungs)
  • Reversible Posterior Leukoencephalopathy Syndrome (a serious brain condition in which blood vessels in your brain swell)
  • Hypothyroidism (low thyroid hormones)
  • Proteinuria (high amount of protein in the urine)
  • Serious infections

Although less common, these require immediate medical attention. Always tell your doctor if you notice new or worsening symptoms.

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Pazopanib: Precautionary Measures 

Before taking Pazopanib, tell your doctor if you:

  • Have or had liver problems
  • Have high blood pressure 
  • Have heart problems or an irregular heartbeat, including QT prolongation 
  • Have a history of a stroke 
  • Have headaches, seizures, or vision problems
  • Have coughed up blood in the last 6 months
  • Had bleeding in your stomach or intestines in the last 6 months
  • Have a history of a tear (perforation) in your stomach or intestine, or an abnormal connection between two parts of your gastrointestinal tract (fistula) 
  • Have had blood clots in a vein or in the lung 
  • Have thyroid problems 
  • Had recent surgery (within the last 7 days) or are going to have surgery 
  • Have any other medical conditions 
  • Are allergic to pazopanib or any of the other ingredients of this medicine
  • Are pregnant or plan to become pregnant. Pazopanib can harm your unborn baby. 
  • Are breastfeeding or plan to breastfeed. It is not known if pazopanib passes into your breast milk. You and your healthcare provider should decide if you will take Pazopanib or breastfeed. You should not do both. 
  • Are taking any prescription or non-prescription medicines, vitamins, and herbal supplements. Pazopanib may affect the way other medicines work, and other medicines may affect how pazopanib works.

Medicines To Avoid 

You should avoid taking the following medications, as they may alter the blood level of pazopanib, may affect how Votrient works, or make it more likely that you’ll have side effects. These include:

  • A proton pump inhibitor (PPI), such as esomeprazole (Nexium)
  • Any medicine that can cause QT prolongation, which is a potentially dangerous heart rhythm problem
  • Dextromethorphan (a medicine used to treat a cough caused by the common cold or other illnesses)
  • Ketoconazole (a medicine used for fungal infections)
  • Lapatinib (Tykerb) (a medicine used to treat certain types of breast cancer)
  • Midazolam (a medicine used to relieve anxiety before surgery)
  • Medicines that contain simvastatin to treat high cholesterol levels
  • Herbal supplements containing St. John’s wort

Pazopanib Cost

Pazopanib (Votrient) can be very expensive without insurance because it is a branded medication. The cost may reach several thousand dollars per month, depending on the tablet strength and how many tablets are prescribed. 

For example, a 120-tablet supply of 200 mg pazopanib may cost between $9,495 and $22,701, whereas a 30-tablet supply of 400 mg may cost approximately $3,749 or more before discounts, insurance, or assistance programs.

Many patients do not pay the full price because insurance coverage, copay assistance, or pharmacy discount cards can help reduce the cost. Contact us if you are interested in exploring financial assistance or copay assistance for Pazopanib (Votrient).