Inventory of current practices regarding hematopoietic stem cell transplantation in metachromatic leukodystrophy in Europe and neighboring countries.

ABSTRACT:

Background

For decades, early allogeneic stem cell transplantation (HSCT) has been used to slow neurological decline in metachromatic leukodystrophy (MLD). There is lack of consensus regarding who may benefit, and guidelines are lacking. Clinical practice relies on limited literature and expert opinions. The European Reference Network for Rare Neurological Diseases (ERN-RND) and the MLD initiative facilitate expert panels for treatment advice, but some countries are underrepresented. This study explores organizational and clinical HSCT practices for MLD in Europe and neighboring countries to enhance optimization and harmonization of cross-border MLD care.

Methods

A web-based EUSurvey was distributed through the ERN-RND and the European Society for Blood and Marrow Transplant

SUBMITTER: Schoenmakers DH 

PROVIDER: S-EPMC10848395 | biostudies-literature | 2024 Feb

REPOSITORIES: biostudies-literature