Body Height of MPS I and II Patients after Hematopoietic Stem Cell Transplantation: The Impact of Dermatan Sulphate.

ABSTRACT:

Introduction

Hematopoietic stem cell transplantation (HSCT) comprises one of the two main treatment regimens for patients with mucopolysaccharidoses (MPS). There is a scarcity of literature concerning the process of growth in children with Mucopolysaccharidosis type I (MPS I) and Mucopolysaccharidosis type I (MPS II) after HSCT. The aim of this manuscript was to evaluate the therapeutic effect of HSCT on the heights of patients with MPS I and MPS II.

Material and methods

It was an observational, single-center study on patients with MPS I and II treated with HSCT.

Results

6 MPS patients, including 4 MPS I and 2 MPS II, underwent HSCT at a median age of 2 years. All patients are alive to date, with a median age of 7.7 years (range 5.5-12 years) at the last follow-up. In

SUBMITTER: Lipinski P 

PROVIDER: S-EPMC11394317 | biostudies-literature | 2024 Sep

REPOSITORIES: biostudies-literature