Sunisha Arora: New EBMT Recommendations Transform the Approach to HSCT in Thalassemia
Sunisha Arora, Consultant Pediatric Hematologist-Oncologist at Fortis Memorial Research Institute – India, shared a post on LinkedIn about a recent article by Mohammed Essa et al, published in Bone Marrow Transplantation, adding:
“New EBMT Recommendations 2026: HSCT Conditioning in Transfusion-Dependent Thalassemia (TDT)
1. HSCT remains a curative standard for TDT, with DFS greater than 90 percent in favorable-risk children with matched sibling donors.
2. Earlier transplant matters: Optimal pediatric outcomes are seen when HSCT is performed at less than or equal to 14 years
3. Don’t delay HSCT just to reduce iron burden.
4. Pre-HSCT optimization: In suboptimally transfused children, maintain pre-transfusion Hb around 10.5–11 g/dL for greater than or equal to 2 months to suppress ineffective erythropoiesis.
5. Treosulfan is firmly established: Treo plus Fludarabine plus Thiotepa plus ATG provides outcomes comparable to busulfan-based conditioning.
6. Busulfan plus Fludarabine remains standard, usually combined with thiotepa or low-dose cyclophosphamide.
7. Avoid TBI-based conditioning in TDT because of long-term toxicity.
8. Routine prophylactic defibrotide is NOT recommended for VOD prevention.
9. Haploidentical HSCT is now considered a valid alternative for children without matched donor.
10. Both TCRαβ-depleted haplo-HSCT and PTCy platforms are acceptable.
11. Routine splenectomy should be avoided.”
Title: Conditioning regimen for hematopoietic cell transplant in transfusion dependent thalassemia: Recommendations from the EBMT practice harmonisation and guidelines committee
Authors: Mohammed Essa, Mattia Algeri, Mohsen Alzahrani, Francoise Bernaudin, Victoria Bordon, Sonali Choudhury, Josu de la Fuente, Fabio Giglio, Erfan Nur, Valeria Maria Pinto, Francesco Onida, Annalisa Ruggeri, Isabel Sánchez-Ortega, Akif Yesilipek, Ibrahim Yakoub-Agha, Emanuele Angelucci
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