Is there a role for pomalidomide in the treatment of myelofibrosis?

ABSTRACT

Introduction: Myelofibrosis is a complex heterogeneous disease which continues to challenge the treating clinician. Ruxolitinib, the first JAK inhibitor approved, delivers a reduction in splenomegaly and disease-related symptoms, and likely prolongs survival. JAK inhibitors are being trialled in combination and appear to alleviate anaemia, thrombocytopenia or deepen response. However, a number of patients may not gain benefit, lose their response or are not able to tolerate ruxolitinib.

Areas covered: There remains a need for alternative agents in myelofibrosis patients in whom JAK inhibitors have failed or are not tolerable. We discuss the different therapeutic options currently available and explore pomalidomide as a therapeutic option in anaemic, and/or thrombocytopenic patients with or without splenomegaly.

Expert opinion: The responses documented in clinical trials suggest pomalidomide efficacy might be variable with a limited benefit in improving constitutional symptom burden or reversing the disease process. However, a number of phase-II trials have demonstrated pomalidomide is a safe and efficacious option in managing splenomegaly, reducing transfusion requirements and in a minority improving platelet number. As we discuss, pomalidomide, which is currently approved for use in multiple myeloma, is a potential alternative in the challenging myelofibrosis patient where thrombocytopenia and anaemia exist.

KEYWORDS:

• Myelofibrosis
• pomalidomide
• immunomodulatory drugs
• anaemia
• thrombocytopenia

Article highlights

• POM is a potential alternative therapeutic agent for patients with MF who are intolerant to or have failed to respond to JAK inhibitors.

• MF subjects with thrombocytopenia and/or anemia my benefit from a trial of POM either in combination with PRED or as monotherapy.

• POM may have a role for management of complex MF subjects in combination with other agents such as JAK inhibitors or azacitidine.

• To date, there remains no strong evidence for the use of POM as first-line therapy in patients with MF.

• There are on-going studies evaluating the role of POM in MF in combination with other agents.

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Declaration of interest

C Harrison has received a research grant from Novartis and speaker fees from Novartis and Shire. 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. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.