Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2022 Jul;22(7):e507-e520.
doi: 10.1016/j.clml.2022.01.016. Epub 2022 Feb 5.

Thrombocytopenia in Patients With Myelofibrosis: Pathogenesis, Prevalence, Prognostic Impact, and Treatment

Dahniel Sastow  1 , John Mascarenhas  2 , Douglas Tremblay  3
Affiliations
Review

Thrombocytopenia in Patients With Myelofibrosis: Pathogenesis, Prevalence, Prognostic Impact, and Treatment

Dahniel Sastow et al. Clin Lymphoma Myeloma Leuk. 2022 Jul.

Abstract

Myelofibrosis (MF) is a clonal hematopoietic stem cell neoplasm, characterized by pathologic myeloproliferation associated with inflammatory and pro-angiogenic cytokine release, that results in functional compromise of the bone marrow. Thrombocytopenia is a disease-related feature of MF, which portends a poor prognosis impacting overall survival (OS) and leukemia free survival. Thrombocytopenia in MF has multiple causes including ineffective hematopoiesis, splenic sequestration, and treatment-related effects. Presently, allogeneic hematopoietic stem cell transplantation (HSCT) remains the only curable treatment for MF, which, unfortunately, is only a viable option for a minority of patients. All other currently available therapies are either focused on improving cytopenias or the alleviating systemic symptoms and burdensome splenomegaly. While JAK2 inhibitors have moved to the forefront of MF therapy, available JAK inhibitors are advised against in patients with severe thrombocytopenia (platelets < 50 × 109/L). In this review, we describe the pathogenesis, prevalence, and prognostic significance of thrombocytopenia in MF. We also explore the value and limitations of treatments directed at addressing cytopenias, splenomegaly and symptom burden, and those with potential disease modification. We conclude by proposing a treatment algorithm for patients with MF and severe thrombocytopenia.

Keywords: Danazol; Immunomodulators; JAK inhibitors; Pacritinib; Ruxolitinib.

PubMed Disclaimer

Conflict of interest statement

Declaration of Competing Interest DS has no conlicts of interest; JM receives research funding paid to the institution from Incyte, Novartis, Roche, Merck, Geron, CTI Bio, BMS, Celgene, Kartos, PharmaEssentia and consulting fees from Incyte, CTI Bio, Constellation, Geron, Kartos, BMS, Celgene, Novartis, Karyopharm, Sierra Oncology, PharmaEssentia, and Galecto; DT receives contracted research funding paid to his institution from Astellas Pharma and consulting fees from AbbVie and CTI Biopharma.