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Updated: Nov 28, 2025

Use of Hematopoietic Stem Cell Transplantation to Assess the Origin of Myelodysplastic Syndrome

Use of Hematopoietic Stem Cell Transplantation to Assess the Origin of Myelodysplastic Syndrome

Published on: October 3, 2018

[Myelofibrosis: A review].

A Genthon¹, M Killian², P Mertz³

¹Service d'hématologie clinique et de thérapie cellulaire, hôpital Saint-Antoine, AP-HP, Paris, France; Médecine Sorbonne université, Paris, France.

La Revue De Medecine Interne

|November 27, 2020

Summary

This summary is machine-generated.

Myelofibrosis is a serious blood cancer. Current treatments are palliative, and while stem cell transplant offers a cure, novel strategies are needed to reverse disease progression and improve survival.

Keywords:

JAK2.Myelofibrosis Myeloproliferative neoplasms Myeloproliferative syndrome Myélofibrose Myélofibrose primitive Néoplasie myéloproliférative Primary myelofibrosis Syndrome myéloprolifératif

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Area of Science:

Hematology
Oncology
Molecular Biology

Background:

Myelofibrosis is a BCR-ABL1-negative chronic myeloproliferative neoplasm.
Characterized by clonal stem cell proliferation and somatic mutations (JAK2, CALR, MPL).
Commonly presents with splenomegaly, constitutional symptoms, and cytopenias.

Purpose of the Study:

To review the current understanding of myelofibrosis pathogenesis.
To highlight the limitations of existing treatments.
To emphasize the need for novel therapeutic strategies.

Main Methods:

Literature review of myelofibrosis pathogenesis and treatment.
Analysis of current therapeutic options including JAK inhibitors and stem cell transplantation.
Identification of unmet needs in myelofibrosis management.

Main Results:

Myelofibrosis pathogenesis is complex, involving driver and subclonal mutations.
Ruxolitinib (JAK inhibitor) provides symptomatic relief but does not eliminate the clone.
Allogeneic hematopoietic stem cell transplantation is the only curative option.

Conclusions:

Despite advances in understanding, myelofibrosis treatment remains largely palliative.
There is a critical need for novel therapies to slow or reverse disease progression.
Preventing transformation to blast-phase and improving long-term survival are key goals.