{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,7]],"date-time":"2026-03-07T19:50:58Z","timestamp":1772913058436,"version":"3.50.1"},"reference-count":21,"publisher":"Wiley","issue":"8","license":[{"start":{"date-parts":[[2011,11,23]],"date-time":"2011-11-23T00:00:00Z","timestamp":1322006400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Pediatric Transplantation"],"published-print":{"date-parts":[[2011,12]]},"abstract":"<jats:p>Grigull L, Sykora K\u2010W, Tenger A, Bertram H, Meyer\u2010Marcotty M, Hartmann H, B\u00fcltmann E, Beilken A, Zivicnjak M, Mynarek M, Osthaus AW, Schilke R, Kollewe K, L\u00fccke T. Variable disease progression after successful stem cell transplantation: Prospective follow\u2010up investigations in eight patients with Hurler syndrome. \u2028Pediatr Transplantation 2011: 15: 861\u2013869. \u00a9 2011 John Wiley &amp; Sons A\/S.<\/jats:p><jats:p><jats:bold>Abstract:\u2002<\/jats:bold> We report the results of a prospective, standardized follow\u2010up programme of eight children (median age at SCT 1.2\u2003yr) with mucopolysaccharidosis (MPS1H, M. Hurler) transplanted using a fludarabine\u2010based SCT. SCT resulted in stable engraftment without transplant\u2010related mortality. All patients are alive, engrafted and in ambulatory care. During follow\u2010up (median five\u2003yr, 1.9\u20138\u2003yr), six of eight showed developmental delay (two severe, two mild\/no), all eight had spinal deformities and one received hip surgery for acetabular dysplasia. Hand surgery for carpal tunnel release and trigger digits was required in five of the patients. The cranio\u2010cervical junction was narrowed in four patients, one child having already received surgery. CC was present in all patients prior to SCT. It remained unchanged in seven and regressed in one child. Severe cardiac dysfunction was present in two of the eight children before SCT. Cardiac pump function was normal in six patients and ameliorated in two, while valve abnormalities could be detected in six patients. Currently, transplantation seems no longer the major obstacle for MPS1H patients, but the variable musculoskeletal disease progression after successful SCT remains a challenge. Patients with Hurler syndrome need specialized follow\u2010up care because of their manifold health problems. The standardized follow\u2010up presented here is a step to optimize care for MPS children and their families after SCT.<\/jats:p>","DOI":"10.1111\/j.1399-3046.2011.01595.x","type":"journal-article","created":{"date-parts":[[2011,11,24]],"date-time":"2011-11-24T05:44:45Z","timestamp":1322113485000},"page":"861-869","source":"Crossref","is-referenced-by-count":20,"title":["Variable disease progression after successful stem cell transplantation: Prospective follow\u2010up investigations in eight patients with Hurler 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