کد مقاله کد نشریه سال انتشار مقاله انگلیسی نسخه تمام متن
5524068 1546236 2017 9 صفحه PDF دانلود رایگان
عنوان انگلیسی مقاله ISI
Hematopoietic Stem Cell Transplantation for Patients with Mucopolysaccharidosis II
موضوعات مرتبط
علوم زیستی و بیوفناوری بیوشیمی، ژنتیک و زیست شناسی مولکولی تحقیقات سرطان
پیش نمایش صفحه اول مقاله
Hematopoietic Stem Cell Transplantation for Patients with Mucopolysaccharidosis II
چکیده انگلیسی


- Hematopoietic stem cell transplantation reduces glycosaminoglycan levels and improves activity of daily living and magnetic resonance imaging findings in patients with mucopolysaccharidosis II
- Improvements in transplantation therapy have dramatically reduced mortality rates
- Outcomes for patients with mucopolysaccharidosis II treated with hematopoietic stem cell transplantation are better than those for enzyme replacement therapy

There is limited information regarding the long-term outcomes of hematopoietic stem cell transplantation (HSCT) for mucopolysaccharidosis II (MPS II). In this study, clinical, biochemical, and radiologic findings were assessed in patients who underwent HSCT and/or enzyme replacement therapy (ERT). 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. 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. Long-term magnetic resonance imaging (MRI) findings were investigated in 13 treated patients (6 ERT and 7 HSCT). 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. None of the 27 new patients died as a direct result of the HSCT procedure. Graft-versus-host disease occurred in 8 (9%) out of 85 published cases, and 9 (8%) patients died from transplantation-associated complications. Most HSCT patients showed greater improvement in somatic features, joint movements, and activity of daily living than the ERT patients. GAG levels in blood were significantly reduced by ERT and levels were even lower after HSCT. HSCT patients showed either improvement or no progression of abnormal findings in brain MRI while abnormal findings became more extensive after ERT. HSCT seems to be more effective than ERT for MPS II in a wide range of disease manifestations and could be considered as a treatment option for this condition.

ناشر
Database: Elsevier - ScienceDirect (ساینس دایرکت)
Journal: Biology of Blood and Marrow Transplantation - Volume 23, Issue 10, October 2017, Pages 1795-1803
نویسندگان
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