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Philadelphia chromosome-negative myeloproliferative neoplasms: clinical aspects and treatment options

Akihiko Gotoh1

  • 1Department of Hematology, Tokyo Medical University, 6-7-1, Nishishinjuku, Shinjuku-ku, Tokyo, 160-0023, Japan. akihikog@tokyo-med.ac.jp.

International Journal of Hematology
|April 10, 2022
PubMed

Insights

Discoveries in JAK2, MPL, and calreticulin mutations advance myeloproliferative neoplasms (MPN) treatment. While JAK inhibitors improve outcomes, hematopoietic stem cell transplantation remains the only cure for MPN.

Area of Science:

  • Hematology
  • Oncology
  • Molecular Biology

Background:

  • Philadelphia chromosome-negative myeloproliferative neoplasms (MPN) are driven by mutations in JAK2, MPL, and calreticulin.
  • Genetic factors significantly influence MPN pathogenesis and progression, leading to the development of prognostic systems.
  • JAK2 activation is a common consequence of these driver mutations.

Purpose of the Study:

  • To review the current understanding of genetic drivers in MPN.
  • To evaluate the efficacy of JAK inhibitors and other treatments.
  • To discuss the role of hematopoietic stem cell transplantation (HSCT) as a curative option.

Main Methods:

  • Review of clinical studies on MPN.
  • Analysis of genetic mutations (JAK2, MPL, calreticulin) and their impact.
  • Assessment of treatment outcomes for JAK inhibitors and HSCT.

Main Results:

  • JAK inhibitors have improved outcomes for primary myelofibrosis and polycythemia vera by targeting JAK2 activation.
  • Current JAK inhibitors show limited efficacy in inhibiting clonogenic growth compared to ABL inhibitors in CML.
  • Hematopoietic stem cell transplantation (HSCT) remains the sole curative treatment for MPN despite its high mortality rate.

Conclusions:

  • Further research is needed to enhance MPN treatments beyond current JAK inhibitors.
  • Refining HSCT methods and exploring combination therapies (JAK inhibitors with other agents) are crucial.
  • Investigating the safety and clonogenic inhibitory effects of interferon-alfa may offer new therapeutic avenues.
Keywords:
Hematopoietic stem cell transplantationInterferon-αJAK inhibitorMyeloproliferative neoplasms

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