Defibrotide Treatment: Uses, Dosage & Side Effects Explained

Summary of Key Points

Defibrotide is a medication primarily used to treat complications that can arise after a bone marrow transplant, such as veno-occlusive disease (VOD) and hepatic sinusoidal obstruction syndrome (SOS). It works by protecting blood vessels and improving blood flow. Defibrotide is typically administered in a hospital setting through an intravenous (IV) infusion. Always consult your healthcare provider before starting or making any changes to your treatment plan.

Names of the Medication

US Brand Name: Defitelio (Defibrotide)

The generic name of a medication refers to its active ingredient, while the brand name is the name given by the company that markets the drug. In this case, Defibrotide is the generic name, and Defitelio is the brand name.

Pharmacologic Category

1. Fibrinolytic Agent
2. Endothelial Protective Agent

Defibrotide Dosage Guidelines

Defibrotide is used to manage various conditions, primarily complications following bone marrow transplants. Below are the dosing guidelines for each condition. It is essential to consult your doctor before adjusting your dose or starting a new treatment. For personalized advice, you can also schedule an appointment with one of our telemedicine providers.

Veno-Occlusive Disease (VOD) / Hepatic Sinusoidal Obstruction Syndrome (SOS)

The recommended dose is 6.25 mg/kg every six hours, administered as an intravenous infusion over two hours. Treatment typically lasts for at least 21 days but may continue until symptoms improve or resolve.

Bone Marrow Transplant Complications

The dosage for preventing or treating complications such as VOD is the same as for VOD: 6.25 mg/kg every six hours via IV infusion. The duration of treatment depends on the patient’s clinical response.

Acute Myeloid Leukemia (AML)

Defibrotide is not commonly used as a primary treatment for AML but may be prescribed to manage complications like VOD. The dosing remains 6.25 mg/kg every six hours via IV infusion.

Acute Lymphoblastic Leukemia (ALL)

Similar to AML, Defibrotide is used to address complications such as VOD, with the same dosing of 6.25 mg/kg every six hours.

Non-Hodgkin Lymphoma

Defibrotide may be prescribed to treat VOD in patients undergoing stem cell transplants. The recommended dose is 6.25 mg/kg every six hours via IV infusion.

Multiple Myeloma

Defibrotide is used to manage VOD in patients undergoing stem cell transplants, with the same dosing of 6.25 mg/kg every six hours.

Sickle Cell Disease

Defibrotide is not typically used to treat sickle cell disease directly but may be utilized to manage transplant-related complications such as VOD. The dosing remains 6.25 mg/kg every six hours.

Thrombotic Microangiopathy (TMA)

Defibrotide may be prescribed for cases of transplant-related thrombotic microangiopathy. The dose is 6.25 mg/kg every six hours via IV infusion.

Graft-Versus-Host Disease (GVHD)

Defibrotide is not a primary treatment for GVHD but may be used to manage VOD in patients with GVHD. The dosing is 6.25 mg/kg every six hours.

Dosage Forms and Strengths

Injection: 200 mg/2.5 mL (80 mg/mL) solution in a single-use vial

Administration Instructions

Defibrotide is administered as an intravenous (IV) infusion, meaning it is delivered directly into a vein. Each infusion typically lasts two hours and is repeated every six hours. It is crucial to follow your healthcare provider’s instructions carefully and attend all scheduled treatments. If you miss a dose, contact your healthcare provider immediately for guidance.

Defibrotide Side Effects and Adverse Reactions

Like all medications, Defibrotide can cause side effects. It is important to be aware of these and to contact your healthcare provider if you experience any concerning symptoms. Below are some potential side effects:

Common Side Effects

1. Bleeding: Defibrotide can increase the risk of bleeding, particularly in patients with other risk factors. This may include nosebleeds, gum bleeding, or, in rare cases, more serious internal bleeding.
2. Low Blood Pressure (Hypotension): Some patients may experience a drop in blood pressure, which can lead to dizziness or fainting.

Less Common Side Effects

1. Allergic Reactions: Although rare, some patients may develop an allergic reaction, which could include symptoms such as rash, itching, or difficulty breathing.
2. Kidney Problems: Defibrotide may affect kidney function in certain patients, potentially causing changes in urine output or swelling in the legs.

Contraindications for Defibrotide Treatment

A contraindication refers to a specific condition or circumstance in which a medication, such as Defibrotide, should not be used because it may cause harm. For Defibrotide, certain situations make its use inadvisable due to an increased risk of serious side effects or complications.

Active bleeding: Defibrotide can heighten the risk of bleeding. If you are experiencing active bleeding, using this medication could exacerbate the condition and should be avoided.

Allergy to Defibrotide: If you have previously had an allergic reaction to Defibrotide, taking it again could provoke a severe allergic response. In such cases, its use is contraindicated.

Concurrent use of anticoagulants or fibrinolytics: Defibrotide influences blood clotting. Combining it with anticoagulants like heparin or warfarin, or fibrinolytics (clot-dissolving medications), significantly increases the risk of bleeding complications.

Severe liver disease: Because Defibrotide is processed by the liver, severe liver impairment can hinder its metabolism, potentially leading to toxicity or heightened side effects.

Drug-to-Drug Interactions with Defibrotide

Defibrotide may interact with other medications, increasing the likelihood of side effects or reducing the effectiveness of either Defibrotide or the other drugs. It is essential to inform your healthcare provider about all medications you are taking, including over-the-counter drugs and supplements, before starting Defibrotide treatment.

1. Heparin: Both Defibrotide and heparin affect blood clotting. Using them together can substantially increase the risk of severe bleeding.
2. Warfarin (Coumadin): Combining Warfarin, a blood thinner, with Defibrotide further elevates the risk of bleeding complications.
3. Aspirin (Bayer, Bufferin): Aspirin, an antiplatelet medication, can amplify the risk of bleeding when taken alongside Defibrotide.
4. Clopidogrel (Plavix): Clopidogrel, another antiplatelet drug, also increases the likelihood of bleeding when used in combination with Defibrotide.
5. Thrombolytics (e.g., Alteplase, Streptokinase): These clot-dissolving medications, when taken with Defibrotide, can significantly heighten the risk of severe bleeding.

Defibrotide Use During Pregnancy

Is it safe to take Defibrotide when pregnant?

There is limited information regarding the safety of Defibrotide during pregnancy. While animal studies suggest potential risks to the fetus, there are no well-controlled studies in humans. Consequently, Defibrotide should only be used during pregnancy if the potential benefits outweigh the risks. If you are pregnant or planning to become pregnant, it is crucial to discuss this with your healthcare provider. Our telemedicine providers are available to guide you in making an informed decision about Defibrotide treatment.

Defibrotide and Breastfeeding

Is it safe to take Defibrotide while breastfeeding?

It is currently unknown whether Defibrotide passes into breast milk. Due to this uncertainty, breastfeeding is generally not recommended during Defibrotide treatment. You may need to explore alternative feeding options after consulting your healthcare provider. If you are breastfeeding or planning to breastfeed, our telemedicine providers can help you determine the safest course of action for both you and your baby.

Estimated Cost of Defibrotide Medication

The estimated cost of a 30-day supply of Defibrotide without insurance, when using a GoodRX coupon, ranges from $25,000 to $30,000. Prices may vary depending on the pharmacy and location. It is advisable to check with your pharmacy and explore insurance coverage options to help manage the cost of Defibrotide treatment.

Possible Alternatives to Defibrotide Treatment

Depending on your condition, alternative treatments or lifestyle adjustments may be effective. It is important to discuss these options with your healthcare provider before making any changes to your treatment plan. Our telemedicine providers are available to assist you in exploring these alternatives.

1. Veno-occlusive disease (VOD): Supportive care, such as managing fluid levels and avoiding liver-toxic medications, may be beneficial. In severe cases, liver transplantation might be considered.
2. Hepatic sinusoidal obstruction syndrome (SOS): Similar to VOD, supportive care and avoiding hepatotoxic drugs are critical. Your provider may also recommend medications like ursodiol.
3. Bone marrow transplant complications: Treatment options may include immunosuppressive therapy, antibiotics, or other supportive measures, depending on the specific complication.
4. Acute myeloid leukemia (AML): Common treatments include chemotherapy, targeted therapies, and stem cell transplants. Your healthcare provider can help determine the most appropriate approach for your condition.
5. Acute lymphoblastic leukemia (ALL): Treatment options often include chemotherapy, radiation therapy, and targeted therapies. Your provider will work with you to identify the best course of action.
6. Non-Hodgkin lymphoma: Treatment options include chemotherapy, radiation therapy, and immunotherapy. Additionally, maintaining a healthy diet and regular exercise may support overall well-being during treatment.
7. Multiple myeloma: Treatments may involve chemotherapy, immunotherapy, and stem cell transplants. Discuss with your provider whether these options are suitable for your condition.
8. Sickle cell disease: Common treatments include hydroxyurea, blood transfusions, and bone marrow transplants. Lifestyle changes, such as staying well-hydrated and avoiding extreme temperatures, can also help manage symptoms.
9. Thrombotic microangiopathy (TMA): Treatment may involve plasma exchange, immunosuppressive therapy, and supportive care. Consult your provider to determine the most effective approach for your condition.
10. Graft-versus-host disease (GVHD): Immunosuppressive medications and supportive care are commonly used. Your provider can help you decide if these treatments are appropriate for your situation.

Recent Updates on Defibrotide Research

Recent studies have continued to explore the effectiveness of Defibrotide treatment in managing veno-occlusive disease (VOD) and related conditions. A 2022 study published in the journal Bone Marrow Transplantation demonstrated that Defibrotide significantly improved survival rates in patients with severe VOD following stem cell transplantation. These findings highlight the increasing importance of Defibrotide in critical care settings, particularly for patients facing life-threatening complications.

In addition to its established role in treating VOD, ongoing research is investigating the potential use of Defibrotide in other thrombotic conditions, such as thrombotic microangiopathy (TMA) and graft-versus-host disease (GVHD). These studies aim to deepen our understanding of how Defibrotide works in addressing these complex and challenging conditions. While early results are encouraging, further research is essential to fully assess the long-term benefits, potential risks, and any associated Defibrotide side effects in these emerging areas of treatment.

If you have questions about how these advancements might impact your care, our telemedicine providers are available to assist you. They can help you understand the latest research and guide you in making well-informed decisions about Defibrotide treatment and its potential role in your healthcare plan.

James Kingsley

Learn More