Journal Article

Growth and endocrine function in patients with Hurler syndrome after hematopoietic stem cell transplantation

Polgreen, L E, Himes, J H, Petryk, A, Miller, B S, Tolar, J, Plog, M, Orchard, P J, Whitley, C B

2008

Overview

Short stature is characteristic of Hurler syndrome, or mucopolysaccharidosis type IH (MPS IH). Hematopoietic stem cell transplantation (HSCT) is used to treat children with MPS IH. While HSCT corrects some of the metabolic features of MPS IH, its effects on growth are not well delineated. We investigated growth in patients with MPS IH after HSCT and described accompanying endocrine abnormalities. A cohort of 48 patients with MPS IH who had received HSCT between 1983 and 2005 were included. The prevalence of short stature (height <−2 s.d. score, SDS) before HSCT was 9%, and increased to 71% at last follow-up (6.9±5.1 years after HSCT). Short stature was positively associated with increased age at HSCT ( P =0.002) and TBI ( P =0.009). In total, 23% had growth hormone deficiency and/or low insulin-like growth factor-1, one female patient had premature adrenarche, one precocious puberty and 27% had clinical or subclinical hypothyroidism. Growth failure is highly prevalent in children with MPS IH after HSCT. Children who had no TBI exposure and were younger at the time of HSCT had a better height outcome.

• Publisher: Nature Publishing Group UK,Nature Publishing Group
• Subject: Abnormalities / Adolescent / Adolescent Development - radiation effects / Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy / Biological and medical sciences / Bone marrow / Bone marrow, stem cells transplantation. Graft versus host reaction / Carbohydrates (enzymatic deficiencies). Glycogenosis / Care and treatment / Causes of / Cell Biology / Child / Child Development - radiation effects / Child, Preschool / Children / Diagnosis / Endocrine gland diseases / Errors of metabolism / Female / Follow-Up Studies / Growth factors / Growth hormones / Hematology / Hematopoietic Stem Cell Transplantation / Hematopoietic stem cells / Humans / Hurler's syndrome / Hypothyroidism / Infant / Insulin / Insulin-like growth factor I / Internal Medicine / Male / Medical sciences / Medicine / Medicine & Public Health / Metabolic diseases / Mucopolysaccharidosis / Mucopolysaccharidosis I - therapy / original-article / Patient outcomes / Patients / Physical growth / Puberty / Public Health / Retrospective Studies / Stem cell transplantation / Stem Cells / Transfusions. Complications. Transfusion reactions. Cell and gene therapy / Transplantation / Transplantation Conditioning - adverse effects / Transplantation Conditioning - methods / Whole-Body Irradiation - adverse effects