TCT 2022 | Eli-cel gene therapy vs alloHSCT in children with CALD

Christine Duncan, MD, Dana-Farber Cancer Institute, Boston, MA, talks on the results of a study comparing the outcomes of pediatric patients with cerebral adrenoleukodystrophy (CALD) receiving elivaldogene autotemcel (eli-cel) gene therapy in clinical trials versus allogeneic hematopoietic stem cell transplantation (alloHSCT). Overall, whilst the efficacy was similar in patients receiving gene therapy or alloHSCT, the study reported a significantly higher progression-free survival (PFS) for patients treated with eli-cel, and a distinct toxicity profile. Importantly, some patients treated with eli-cel developed treatment-related myelodysplasia. Overall, this data suggests eli-cel as a promising alternative to alloHSCT in this patient population. This interview took place at the Transplantation & Cellular Therapy (TCT) Meetings of ASTCT™ and CIBMTR® 2022 in Salt Lake City, Utah.