EBMT Releases Updated Guidance on HCT, CAR-T, and Gene Therapies

PARIS, FRANCE – October 2, 2025 – The European Society for Blood and Marrow Transplantation (EBMT) has published its ninth special report, providing updated clinical guidance on indications for Hematopoietic Cell Transplantation (HCT) and Chimeric Antigen Receptor T-cell (CAR-T) therapy. The comprehensive report, which also addresses advances in gene therapy, is designed to support clinicians in optimizing treatment strategies for hematological diseases, solid tumors, and immune disorders. The full work is published in the journal Bone Marrow Transplantation.

Harmonizing Practice in an Evolving Landscape

The 2025 report underscores the significant influence of innovative therapies, such as CAR-T and advanced therapy medicinal products (ATMPs), on modern treatment paradigms. It emphasizes the critical need to harmonize practices across transplant centers to improve patient outcomes and facilitate robust data aggregation.  “Since the last edition, innovative cellular and gene therapies have entered clinical practice across several indications, significantly influencing decision-making,” said co-lead author Annalisa Ruggeri.

The document provides detailed recommendations for HCT and CAR-T indications, categorized by disease status, donor availability, and level of evidence for both adult and pediatric populations.

Focus on Quality, Data, and Multidisciplinary Care

The EBMT guidance covers several key areas:

• Gene Therapy: The report includes updates on the latest advances in gene therapy, reflecting its growing role in the treatment landscape.
• Quality Standards: It highlights the importance of quality standards, such as JACIE certification, for transplant centers.
• Real-World Evidence: The EBMT strongly encourages comprehensive reporting to the EBMT Registry to strengthen real-world evidence and evaluate long-term patient outcomes.

Ultimately, the report recommends a multidisciplinary approach to treatment decisions, emphasizing the need to carefully balance disease risk, procedural risks, and alternative strategies, with a core focus on maximizing both patient survival and quality of life.