Treosulfan-Based Conditioning in Allogeneic Stem Cell Transplantation for Myelofibrosis: A Systematic Review

Area of Science:

• Hematology
• Oncology
• Transplantation Medicine

Background:

• Allogeneic hematopoietic stem cell transplantation (allo-HCT) is a curative option for myelofibrosis (MF).
• High transplant-related morbidity and mortality limit allo-HCT use, particularly in older or comorbid patients.
• Treosulfan presents a potentially less toxic conditioning regimen compared to busulfan.

Purpose of the Study:

• To evaluate the efficacy and safety of treosulfan-based conditioning regimens in patients with myelofibrosis undergoing allo-HCT.
• To synthesize data on engraftment, graft-versus-host disease (GVHD), and survival outcomes.

Main Methods:

• Comprehensive literature search across multiple databases (PubMed, Scopus/EMBASE, Cochrane Library, Web of Science, grey literature) for studies from 2000-2025.
• Synthesis of data from eight studies involving over 800 patients.
• Analysis of patient characteristics, conditioning regimens, engraftment, GVHD, and survival outcomes.

Main Results:

• Treosulfan, often combined with fludarabine, demonstrated high engraftment rates (94-100%).
• Low non-relapse mortality (NRM) and favorable progression-free survival (PFS) were observed.
• A registry study showed superior survival and lower NRM compared to busulfan-based regimens, with benefits in older patients and those undergoing second transplants.

Conclusions:

• Treosulfan-based conditioning is an effective and well-tolerated option for myelofibrosis allo-HCT.
• The regimen shows promise for improving outcomes in older patients, those with alternative donors, and for second transplants.
• Further prospective trials are warranted to optimize treosulfan dosing and patient selection.