Myelofibrosis medical therapy
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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]Associate Editor(s)-in-Chief: Sabawoon Mirwais, M.B.B.S, M.D.[2]Sujit Routray, M.D. [3]
Overview
Red blood cell transfusion, danazol therapy, or thalidomide are recommended for patients who develop anemia. Ruxolitinib, an inhibitor of Janus kinase 1 (JAK1) and Janus kinase 2 (JAK2), can reduce the splenomegaly and the constitutional symptoms of weight loss, fatigue, and night sweats for patients with Janus kinase 2 (JAK2)-positive or Janus kinase 2 (JAK2)-negative primary myelofibrosis (PMF), post–essential thrombocythemia myelofibrosis, or post–polycythemia vera myelofibrosis. Hydroxyurea, chemotherapy, or radiotherapy are recommended for patients who develop splenomegaly.
Medical Therapy
Treatment for Anemia
Red blood cell transfusion
- The profound anemia that develops in this disease usually requires red blood cell transfusion.[1][2]
Glucocorticoids
- Glucocorticoids can improve the red blood cell (RBC) survival, which can be markedly decreased in some patients.[1][3]
Androgen therapy
- Danazol can be given to stimulate the production of red blood cells (RBCs).
- Preferred regimen: 600-800 mg/day till normalization of cell counts and then reduced to 200-400 mg/day
Erythropoietic growth factors
- Erythropoietin and darbepoetin can help if patients are not dependent on transfusion.
- Preferred regimen: SubQ: 150 to 300 mcg once weekly or 500 mcg once every 2 to 3 weeks
Thalidomide, lenalidomide, or pomalidomide
- These may help in improving the red blood cell counts, thus improving anemia and help in the reversal of splenomegaly. However, patients on these drugs require prophylaxis for avoiding thrombosis and careful monitoring for hematologic toxicity.[1][4]
- Preferred regimen: Oral: 10 mg once daily
Janus kinase (JAK) Inhibitor Therapy
- Ruxolitinib, an inhibitor of Janus kinase 1 (JAK1) and Janus kinase 2 (JAK2), can reduce the splenomegaly and the symptoms of weight loss, fatigue, and night sweats in patients with Janus kinase 2 (JAK2)-positive or Janus kinase 2 (JAK2)-negative primary myelofibrosis, post-essential thrombocythemia myelofibrosis, or post-polycythemia vera myelofibrosis.[1][5]
- Ruxolitinib can also be integrated as pretransplantation treatment.[6]
- Ruxolitinib preferred regimen: Oral: Initial dose (based on platelet count, titrate dose thereafter based on efficacy and safety):
- Platelets >200,000/mm3: 20 mg twice daily
- Platelets 100,000 to 200,000/mm3: 15 mg twice daily
- Platelets 50,000 to <100,000/mm3: 5 mg twice daily
- Momelotinib is a JAK inhibitor that is currently in clinical trials.
- Pacritinib is a JAK inhibitor that is currently in clinical trials.
Treatment for Splenomegaly
JAK2 inhibitor therapy
- Ruxolitinib can provide temporary relief.[1][7] This medication is FDA-approved for intermediate- and high-risk myelofibrosis.
Hydroxyurea
- Hydroxyurea can reduce the splenomegaly but may have a potential leukemogenic effect.
- Preferred regimen: Starting dose of 500 mg/day and then subsequently adjusted to the individual efficacy.
Chemotherapy
- Drugs like thalidomide, lenalidomide, or cladribine may also reduce the spleen size, and relieve the pain symptoms.
Radiation therapy
- Radiation may be used to reduce the size of the spleen, when splenectomy is contraindicated.
Splenectomy
- Splenectomy can also be performed.
Interferon therapy
- Peg-interferon alpha 2a and peg-interferon alpha 2b have been shown to reduce spleen size, improve constitutional symptoms, and improve blood counts.
References
- ↑ 1.0 1.1 1.2 1.3 1.4 Treatment overview of primary myelofibrosis. National cancer institute 2016. http://www.cancer.gov/types/myeloproliferative/hp/chronic-treatment-pdq#section/_9. Accessed on March 10, 2016
- ↑ Hoffman, Ronald (2018). Hematology : basic principles and practice. Philadelphia, PA: Elsevier. ISBN 9780323357623.
- ↑ Hoffman, Ronald (2018). Hematology : basic principles and practice. Philadelphia, PA: Elsevier. ISBN 9780323357623.
- ↑ Hoffman, Ronald (2018). Hematology : basic principles and practice. Philadelphia, PA: Elsevier. ISBN 9780323357623.
- ↑ Hoffman, Ronald (2018). Hematology : basic principles and practice. Philadelphia, PA: Elsevier. ISBN 9780323357623.
- ↑ Gupta V, Kosiorek HE, Mead A, Klisovic RB, Galvin JP, Berenzon D, Yacoub A, Viswabandya A, Mesa RA, Goldberg J, Price L, Salama ME, Weinberg RS, Rampal R, Farnoud N, Dueck AC, Mascarenhas JO, Hoffman R (September 2018). "Ruxolitinib Therapy Followed by Reduced-Intensity Conditioning for Hematopoietic Cell Transplantation for Myelofibrosis: Myeloproliferative Disorders Research Consortium 114 Study". Biol. Blood Marrow Transplant. doi:10.1016/j.bbmt.2018.09.001. PMID 30205231.
- ↑ Hoffman, Ronald (2018). Hematology : basic principles and practice. Philadelphia, PA: Elsevier. ISBN 9780323357623.