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NEJM Group

Sep 10, 2026, 15:21

Pathogenesis and Emerging Targeted Therapies in Myeloproliferative Neoplasms – NEJM Group

NEJM Group shared a post on LinkedIn about a recent article by Isabelle Plo and William Vainchenker, adding:

“A new review summarizes the pathogenesis and treatment of myeloproliferative neoplasms, highlighting the role of driver mutations, inflammation, and emerging targeted therapies that may achieve durable disease modification.

Classic myeloproliferative neoplasms, including essential thrombocythemia, polycythemia vera, and primary myelofibrosis, are chronic, clonal hematopoietic stem-cell disorders.

These disorders are driven by gain-of-function mutations in the genes Janus kinase 2 (JAK2), calreticulin (CALR), or the thrombopoietin receptor (MPL) that activate cytokine signaling.

These mutations arise decades before clinical disease develops and confer a clonal advantage that is further shaped by comutations in epigenetic, splicing, or signaling genes.

Inflammation enhances clonal dominance, favoring the development of myelofibrosis and thrombotic complications.

Disease evolution may culminate in secondary acute myeloid leukemia, which has a poor prognosis.

Current therapies primarily aim to control symptoms, thrombosis, and splenomegaly, but they have limited disease-modifying effects, except for pegylated interferon alfa and JAK2 inhibitors in some patients.

Emerging therapies that selectively target mutant CALR and JAK2 V617F using immunotherapy and selective inhibitors could be a breakthrough in the treatment of persons with myeloproliferative neoplasms, with the expectation of achieving durable disease modification and potentially clonal eradication.”

Title: Myeloproliferative Neoplasms

Authors: Isabelle Plo, William Vainchenker

NEJM Group