Neurodegenerative disease after hematopoietic stem cell transplantation in metachromatic leukodystrophy.
Murtadha L Al-SaadyShanice BeerepootBonnie C PlugMarjolein BreurHristina GalabovaPetra J W PouwelsJaap-Jan BoelensCaroline LindemansPeter M van HasseltUlrich MatznerAdeline VanderverMarianna BugianiMarjo S van der KnaapNicole I WolfPublished in: Annals of clinical and translational neurology (2023)
Neurological deterioration may occur after hematopoietic stem cell transplantation in metachromatic leukodystrophy despite successfully treated leukodystrophy. MRI shows gray matter atrophy, and histological data demonstrate absence of donor cells in gray matter structures. These findings point to a clinically relevant gray matter component of metachromatic leukodystrophy, which does not seem sufficiently affected by transplantation.