Recombinant interferon alfa in BCR/ABL-negative chronic myeloproliferative neoplasms

Area of Science:

• Hematology
• Oncology
• Pharmacology

Background:

• Myeloproliferative neoplasms (MPNs) are driven by JAK2, CALR, and MPL mutations.
• The treatment landscape for these conditions has evolved, with recent approvals of JAK inhibitors and interferon alfa-2 (IFN-α).
• IFN-α has been used off-label for over 30 years as a cytoreductive agent in MPNs.

Purpose of the Study:

• To review clinical research and advances leading to the regulatory approval of IFN-α in BCR/ABL-negative MPNs.
• To highlight the potential of IFN-α as a disease-modifying therapeutic agent.
• To discuss the efficacy and safety of IFN-α therapy in polycythemia vera (PV) and essential thrombocythemia.

Main Methods:

• Review of clinical trials and regulatory data.
• Analysis of long-term IFN-α therapy effects on clinical, hematologic, and molecular responses.
• Evaluation of pegylated IFN-α formulations, including ropeginterferon alfa-2b.

Main Results:

• IFN-α therapy demonstrates significant clinical, hematologic, and molecular responses in MPNs.
• Long-term IFN-α treatment can lead to a reduction in mutant JAK2 allele burden, potentially achieving a measurable residual disease state.
• Pegylated IFN-α, such as ropeginterferon alfa-2b, offers improved stability and tolerability.

Conclusions:

• Ropeginterferon alfa-2b has received regulatory approval for PV, marking a significant advance.
• IFN-α therapy holds promise as a disease-modifying agent for BCR/ABL-negative MPNs.
• Continued research into IFN-α mechanisms and applications is warranted.