Defining Remission following Hematopoietic Cell Transplant for Myelofibrosis: An International Expert Panel Consensus

作者：Rachel B. Salit, E. Hexner, N. Gagelmann, N. Kröger, D. McLornan, Tania Jain, V. Gupta, Gabriela S. Hobbs, R. Tamari, M. Robin, Bart Scott, Wael Saber · 发表于：Leukemia · 年份：2025 · DOI：10.1038/s41375-025-02754-9 · 被引用次数：5 · 研究领域：Medicine

Background: Persistent marrow fibrosis, splenomegaly and cytopenias following hematopoietic cell transplant (HCT) in patients with myelofibrosis (MF) are common and make it difficult to define disease remission. Consequently, there has been inconsistency in reporting MF HCT outcomes. Our aim was to create an MF HCT remission definition that could be applied when evaluating patients, completing data forms, and interpreting clinical trial data. Methods: A modified Delphi technique was used to develop consensus criteria for remission following MF HCT. Twelve MF experts from the United States, Canada, and Europe contributed by completing multiple choice and open-ended surveys as well as serial discussions by videoconference. While initial surveys and discussions were not anonymous, the final criteria were voted on by anonymous questionnaire. Results: There was consensus that based on recent data, clearance of molecular mutations is central to determining remission after HCT. Patients in molecular remission could then be further categorized as: 1) complete remission, 2) remission with poor graft function, or 3) remission with incomplete marrow recovery. Conclusion: Using molecular remission to define remission status following HCT for MF represents a practical approach to harmonizing data reporting. Additional studies are required to determine whether molecular remission correlates with long-term disease-free survival.