![]() ![]() GAG levels in blood were significantly reduced by ERT and levels were even lower after HSCT. ![]() Most HSCT patients showed greater improvement in somatic features, joint movements, and activity of daily living than the ERT patients. Graft-versus-host disease occurred in 8 (9%) out of 85 published cases, and 9 (8%) patients died from transplantation-associated complications. None of the 27 new patients died as a direct result of the HSCT procedure. Mean age at HSCT was 5.5 years (range, 2 to 21.4 years) in new patients and 5.5 years (range, 10 months to 19.8 years) in published cases. Long-term magnetic resonance imaging (MRI) findings were investigated in 13 treated patients (6 ERT and 7 HSCT). Glycosaminoglycan (GAG) levels were analyzed by liquid chromatography tandem mass spectrometry in blood samples from HSCT, ERT, and untreated patients as well as age-matched controls. Demographic data for 146 HSCT patients were collected from 27 new cases and 119 published cases and were compared with 51 ERT and 15 untreated cases. In this study, clinical, biochemical, and radiologic findings were assessed in patients who underwent HSCT and/or enzyme replacement therapy (ERT). Electronic address: is limited information regarding the long-term outcomes of hematopoietic stem cell transplantation (HSCT) for mucopolysaccharidosis II (MPS II). duPont Hospital for Children, Wilmington, Delaware Department of Pediatrics, Shimane University, Shimane, Japan Department of Pediatrics, Graduate School of Medicine, Gifu University, Gifu, Japan Department of Pediatrics, Thomas Jefferson University, Philadelphia, Pennsylvania.
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