5 targeted therapies for myelofibrosis(MF)

Myelofibrosis is a rare bone marrow disorder that disrupts the body's normal production of blood cells. Treatment for myelofibrosis depends on the severity and symptoms of the disease. Some of the drugs used in the treatment of myelofibrosis include:

JAK inhibitors

These drugs target the Janus kinase (JAK) pathway, which is often overactive in myelofibrosis patients. The most common JAK inhibitor used to treat myelofibrosis is ruxolitinib (Jakafi). It helps to reduce spleen size and alleviate symptoms related to the disease.

Pacritinib^[2]

On February 28, the U.S. Food and Drug Administration (FDA) approved pacritinib (Vonjo) for the treatment of adults with intermediate- or high-risk primary or secondary (post–polycythemia vera or post–essential thrombocythemia) myelofibrosis with a platelet count below 50 × 10⁹/L.

Immunomodulatory drugs

Thalidomide (Thalomid) and its derivatives, such as lenalidomide (Revlimid) and pomalidomide (Pomalyst), are immunomodulatory drugs that can help manage the symptoms of myelofibrosis. These drugs may help to reduce spleen size, improve blood counts, and alleviate symptoms.

Thalidomide^[3]

In the 1950s and 1960s, thalidomide was used to treat morning sickness during pregnancy. But it was found to cause disabilities in the babies born to those taking the drug. Now, decades later, thalidomide (Thalomid) is being used to treat a skin condition and cancer.

Lenalidomide^[4]

In 2006, lenalidomide, an orally administered thalidomide analogue, received FDA approval for use with dexamethasone in patients with multiple myeloma who received at least one prior therapy. In 2015, the indication was expanded for use in combination with dexamethasone for the treatment of patients with multiple myeloma, to include newly diagnosed multiple myeloma patients who are not eligible for autologous stem cell transplant. Lenalidomide is also approved in myelodysplastic syndromes and mantle cell lymphoma.

Pomalidomide^[5]

On February 8, 2013, the FDA approved pomalidomide (Pomalyst; Celgene), the most recent IMiD, for the treatment of patients with MM who have received at least 2 previous therapies, including lenalidomide and bortezomib, and have demonstrated disease progression within 60 days of the completion of the last therapy.

Others

Hydroxyurea

Hydroxyurea (Hydrea) can help to control the excessive production of blood cells in myelofibrosis. This drug is often used in combination with other medications for better symptom management.

Interferon

Interferon therapy, such as pegylated interferon alpha-2a or alpha-2b (e.g., Pegasys, Intron A), may help to reduce the symptoms of myelofibrosis and slow down the progression of the disease. Interferon can stimulate the immune system and help control the production of blood cells.

Androgen therapy

Androgens such as danazol can stimulate the production of red blood cells and may be used in some cases of myelofibrosis, particularly for patients with anemia.

Blood thinners

If you are at risk of blood clots due to myelofibrosis, your doctor may prescribe blood thinners, such as aspirin or warfarin, to help reduce the risk.

Supportive care

In some cases, the focus of treatment may be on managing symptoms and improving quality of life. This can include blood transfusions to address anemia, medications to reduce inflammation and pain, and treatments to manage other complications related to myelofibrosis.