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Myelofibrosis Treatment Options: A Guide for Pharmacists
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Treatment options may include treating the anemia with blood transfusions, androgen therapy, thalidomide, and other medications.
Myelofibrosis is a type of cancer that affects the bone marrow and disrupts the normal production of red blood cells. It causes scarring in the bone marrow, which in turn leads to severe anemia, resulting in weakness and fatigue. It also affects the blood-clotting cells including platelets, which may cause the patient to be more prone to risk of bleeding.1
Bone marrow illustration, myelofibrosis | Image credit: catalin | stock.adobe.com
There are 2 types of myelofibrosis: primary and secondary.¹ Primary myelofibrosis is a rare chronic condition impacting about 1 person per 100,000 population. It can happen at any age, but usually these patients are diagnosed later in life, between the ages of 60 and 70.2 Secondary myelofibrosis develops from another bone marrow disorder.1
Some patients affected by myelofibrosis have no symptoms and may not need acute treatment. On the other hand, depending on the patient, the disease may be severe and require aggressive treatments right away. This disease develops slowly, so some patients may not experience the signs and symptoms until later in the course of disease. Patients may feel tired, weakened, and short of breath due to the anemia. They may also experience pain or fullness below the ribs due to the enlarged spleen. Some of these patients experience easy bruising, easy bleeding, excessive sweating at night, bone pain, and fever.¹
Many patients with myelofibrosis have an overabundance of white blood cells and different levels of platelets. The most common genetic mutations causing myelofibrosis include Janus Kinase 2 (JAK2) gene mutations, CALR, and MPL mutations. The diagnosis, prognosis, and treatment options depend on the type of mutation.¹
Diagnosis of myelofibrosis includes physical exam by the provider or the specialist; various blood tests, including white blood cell, red blood cells, and platelet counts; and imaging tests. Bone marrow examinations and biopsy are also performed many times to analyze the liquid portion of the bone marrow and the cells found in it. Lastly, cells for gene mutations are analyzed from blood or bone marrow cells to check for JAK2, CALR and MPL mutations.¹
Treatment options may include treating the anemia with blood transfusions, androgen therapy, thalidomide (Thalomid; Bristol Myers Squibb), and other medications. Treatment for the enlarged spleen may include targeted drug therapies, targeting JAK2 gene mutations, chemotherapy, and surgical removal of the spleen. Radiation therapy may also be an option for patients.¹
Some patients may benefit from bone marrow transplantation, also called an allogeneic stem cell transplant, for those that qualify for it. This is a treatment option to replace the diseased bone marrow using healthy blood stem cells.¹ Targeted therapy options for myelofibrosis may include JAK inhibitors such as ruxolitinib (Jakafi; Incyte), fedratinib (Inrebic; Bristol Myers Squibb) and momelotinib (Ojjaara; GSK).3
Pharmacists play a vital role in the successful management of myelofibrosis. By providing comprehensive medication therapy management, including thorough medication reviews, adherence counseling, and proactive monitoring for adverse reactions, pharmacists contribute significantly to optimizing treatment outcomes.
Perhaps most importantly, pharmacists serve as invaluable patient educators. By clearly explaining complex treatment regimens, addressing potential adverse effects, and empowering patients with knowledge about their disease and available therapies, pharmacists foster a strong patient-centered approach to care. This collaborative effort between physicians, pharmacists, and patients is crucial to navigating the challenges of myelofibrosis and improving the quality of life for patients.
