Allogeneic haematopoietic cell transplantation for advanced systemic mastocytosis: Best practice recommendations on...

Area of Science:

• Hematology
• Oncology
• Stem Cell Transplantation

Background:

• Systemic Mastocytosis (SM) is a heterogeneous disease, with Advanced SM (AdvSM) encompassing aggressive SM (ASM), mast cell leukemia (MCL), and SM with an associated hematologic neoplasm (SM-AHN).
• KIT D816V mutations are prevalent in AdvSM, leading to targeted therapies like midostaurin and avapritinib.
• While KIT inhibitors improve outcomes, they are not curative, and allogeneic hematopoietic cell transplantation (allo-HCT) is recommended for high-risk patients, yet guidance is lacking.

Purpose of the Study:

• To establish best practice recommendations for allogeneic hematopoietic cell transplantation (allo-HCT) in Advanced Systemic Mastocytosis (AdvSM).
• To provide a practical framework for clinicians regarding patient selection and the integration of KIT inhibitors within the allo-HCT algorithm for AdvSM.

Main Methods:

• Development of best practice recommendations by an expert panel specializing in allo-HCT and mastocytosis.
• Adherence to European Society of Blood and Marrow Transplantation (EBMT) Practice Harmonisation guidelines and European Competence Network on Mastocytosis (ECNM) methodology.

Main Results:

• The study generated comprehensive guidelines for allo-HCT in AdvSM.
• The recommendations address the optimal use of KIT inhibitors pre- and post-transplant to enhance patient outcomes.

Conclusions:

• These guidelines provide a crucial, up-to-date framework for managing AdvSM with allo-HCT in 2024 and beyond.
• The recommendations aim to optimize treatment strategies by integrating novel targeted therapies with established transplant procedures for AdvSM patients.