Educational content on VJNeurology is intended for healthcare professionals only. By visiting this website and accessing this information you confirm that you are a healthcare professional.

Share this video  

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

Christine Duncan • 24 Apr 2022

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.

Choose your cookie settings

VJNeurology uses necessary cookies to make the site work. With your permission we'd also like to use analytics, preference and marketing cookies to understand how the site is used and improve it.

Choose an option to continue. You can change it at any time on our Cookie Policy page, linked at the bottom of every page.

Cookie Policy (opens in a new tab)
The content of VJNeurology is intended for healthcare professionals