Treatment of anemia in myelofibrosis: focusing on novel therapeutic options

ABSTRACT

Introduction

Myelofibrosis is a clonal myeloproliferative neoplasm associated with the proliferation of hematopoietic stem cells, increased bone marrow fibrosis, extramedullary hematopoiesis, hepatosplenomegaly, abnormal cytokine production, and constitutional symptoms. These and many other factors contribute to the development of anemia in myelofibrosis patients.

Areas covered

This review summarizes novel and promising treatments for anemia in myelofibrosis including transforming growth factor-β inhibitors luspatercept and KER-050, JAK inhibitors momelotinib, pacritinib, and jaktinib, BET inhibitors pelabresib and ABBV-744, antifibrotic PRM-151, BCL2/BCL-XL inhibitor navitoclax, and telomerase inhibitor imetelstat.

Expert opinion

Standard approaches to treat myelofibrosis-related anemia have limited efficacy and are associated with toxicity. New drugs have shown positive results in myelofibrosis-associated anemia when used alone or in combination.

KEYWORDS:

• Anemia
• imetelstat
• luspatercept
• momelotinib
• MPN
• myeloproliferative neoplasm
• navitoclax
• pelabresib

Article highlights

• Luspatercept has shown promising results in patients treated with or without ruxolitinib by increasing the rate of transfusion independence.

• New generation of JAK inhibitors momelotinib, pacritinib, and jaktinib are effective drugs and have fewer side effects, especially in terms of anemia.

• A BET inhibitor pelabresib has been shown to increase transfusion independence alone or in combination. A novel BET inhibitor ABBV-744 is under investigation.

• An antifibrotic PRM-151, BCL2/BCL-XL inhibitor navitoclax, and telomerase inhibitor imetelstat offer new treatment options for myelofibrosis-related anemia.

Declaration of interest

AE Eşkazan has received advisory board and speaker bureau honoraria from Novartis and Bristol-Myers Squibb. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This paper was not funded.