title: Impact of Enzyme Replacement Therapy and Hematopoietic Stem Cell Therapy on Growth in Patients with Hunter Syndrome.
abstract: Patients with Hunter syndrome (mucopolysaccharidosis II) present with skeletal dysplasia including short stature as well as CNS and visceral organ involvement. A previous study on Hunter syndrome indicated an impact on brain and heart involvement after hematopoietic stem cell therapy (HSCT) at an early stage but little impact after enzyme replacement therapy (ERT) (Tanaka et al 2012). Meanwhile, impact on growth in patients with Hunter syndrome treated with ERT and HSCT has not been compared until now. We recently developed baseline growth charts for untreated patients with Hunter syndrome to evaluate the natural history of growth of these patients compared to unaffected controls (Patel et al, 2014). To assess impact of ERT and HSCT on growth, clinical data were obtained from 44 Japanese male patients with MPS II; 26 patients had been treated with ERT, 12 patients had been treated with HSCT, and 6 had been treated with both ERT and HSCT. Height and weight were compared to untreated patients and unaffected controls from the previous study. We demonstrated 1) that MPS II patients, who had been treated with either ERT or HSCT, had increased height and weight when compared to untreated patients, and 2) that HSCT and ERT were equally effective in restoring growth of MPS II patients. In conclusion, HSCT should be considered as one of the primary therapeutic options for early stage treatment of MPS II, as HSCT has also been reported to have a positive effect on brain and heart valve development (Tanaka et al 2012).
authors: Nemours/Alfred I. duPont Hospital for Children, Wilmington, DE.
authors: Pravin
authors: P
authors: Patel
authors: Medical Education Development Center, Gifu University.
authors: Yasuyuki
authors: Y
authors: Suzuki
authors: Department of Pediatrics, Osaka City University Graduate School of Medicine, Osaka, Japan.
authors: Akemi
authors: A
authors: Tanaka
authors: Department of Cell Transplantation, Tokai University School of Medicine, Isehara, Japan.
authors: Hiromasa
authors: H
authors: Yabe
authors: Department of Cell Transplantation, Tokai University School of Medicine, Isehara, Japan.
authors: Shunichi
authors: S
authors: Kato
authors: Nemours/Alfred I. duPont Hospital for Children, Wilmington, DE.
authors: Tsutomu
authors: T
authors: Shimada
authors: Nemours/Alfred I. duPont Hospital for Children, Wilmington, DE.
authors: Robert W
authors: RW
authors: Mason
authors: Division of Neonatal Intensive Care Unit, Gifu University Hospital.
authors: Kenji E
authors: KE
authors: Orii
authors: Department of Pediatrics, Gifu University, School of Medicine, Gifu, Japan.
authors: Toshiyuki
authors: T
authors: Fukao
authors: Department of Pediatrics, Gifu University, School of Medicine, Gifu, Japan.
authors: Tadao
authors: T
authors: Orii
authors: Nemours/Alfred I. duPont Hospital for Children, Wilmington, DE ; Department of Pediatrics, Gifu University, School of Medicine, Gifu, Japan.
authors: Shunji
authors: S
authors: Tomatsu
keywords: Hunter syndrome; enzyme replacement therapy; growth impact; height; hematopoietic stem cell therapy