[Myeloproliferative neoplasms: recent progresses in therapy].

Area of Science:

• Hematology
• Oncology
• Pharmacology

Background:

• Polycythemia vera (PV) and essential thrombocythemia (ET) patients have prognoses similar to the general population, with treatment focused on preventing thrombohemorrhagic events.
• Current first-line treatments include phlebotomy, hydroxyurea (HU), and aspirin for PV, and HU or anagrelide for ET.
• Primary myelofibrosis (PMF) has a poorer prognosis, with allogeneic hematopoietic stem cell transplantation (HSCT) as the only curative option.

Purpose of the Study:

• To evaluate the efficacy and safety of ropeginterferon alfa-2b compared to hydroxyurea (HU) in polycythemia vera (PV) patients.
• To review current therapeutic strategies for PV, ET, and primary myelofibrosis (PMF).
• To explore the role of JAK inhibitors and other novel agents in managing myeloproliferative neoplasms.

Main Methods:

• A phase 3 randomized trial compared ropeginterferon alfa-2b with HU in PV patients, assessing hematological response and adverse events.
• Review of existing literature on treatments for PV, ET, and PMF.
• Analysis of data on JAK inhibitors, including ruxolitinib, and emerging therapies for anemia and bone marrow fibrosis.

Main Results:

• Ropeginterferon alfa-2b demonstrated superior hematological response and a lower incidence of adverse events compared to HU in PV patients.
• Ruxolitinib provides survival benefits and symptom amelioration for intermediate-2 or high-risk PMF patients.
• Several JAK inhibitors have been developed, though some faced discontinuation due to toxicity; newer agents show promise.

Conclusions:

• Ropeginterferon alfa-2b represents a promising therapeutic advancement for polycythemia vera.
• Management of primary myelofibrosis relies on HSCT or symptomatic treatment with agents like ruxolitinib.
• Ongoing research into JAK inhibitors and targeted therapies offers potential for improved outcomes in myeloproliferative neoplasms.