Title |
Treatment of hip dysplasia in patients with mucopolysaccharidosis type I after hematopoietic stem cell transplantation: results of an international consensus procedure
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Published in |
Orphanet Journal of Rare Diseases, October 2013
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DOI | 10.1186/1750-1172-8-155 |
Pubmed ID | |
Authors |
Eveline J Langereis, Andrea Borgo, Ellen Crushell, Paul R Harmatz, Peter M van Hasselt, Simon A Jones, Paula M Kelly, Christina Lampe, Johanna H van der Lee, Thierry Odent, Ralph Sakkers, Maurizio Scarpa, Matthias U Schafroth, Peter A Struijs, Vassili Valayannopoulos, Klane K White, Frits A Wijburg |
Abstract |
Mucopolysaccharidosis type I (MPS-I) is a lysosomal storage disorder characterized by progressive multi-organ disease. The standard of care for patients with the severe phenotype (Hurler syndrome, MPS I-H) is early hematopoietic stem cell transplantation (HSCT). However, skeletal disease, including hip dysplasia, is almost invariably present in MPS I-H, and appears to be particularly unresponsive to HSCT. Hip dysplasia may lead to pain and loss of ambulation, at least in a subset of patients, if left untreated. However, there is a lack of evidence to guide the development of clinical guidelines for the follow-up and treatment of hip dysplasia in patients with MPS I-H. Therefore, an international Delphi consensus procedure was initiated to construct consensus-based clinical practice guidelines in the absence of available evidence. |
X Demographics
Geographical breakdown
Country | Count | As % |
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Spain | 2 | 33% |
Mexico | 1 | 17% |
Unknown | 3 | 50% |
Demographic breakdown
Type | Count | As % |
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Members of the public | 5 | 83% |
Scientists | 1 | 17% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
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Spain | 1 | 2% |
Unknown | 58 | 98% |
Demographic breakdown
Readers by professional status | Count | As % |
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Student > Ph. D. Student | 8 | 14% |
Researcher | 8 | 14% |
Student > Master | 8 | 14% |
Student > Bachelor | 6 | 10% |
Student > Doctoral Student | 4 | 7% |
Other | 8 | 14% |
Unknown | 17 | 29% |
Readers by discipline | Count | As % |
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Medicine and Dentistry | 23 | 39% |
Social Sciences | 3 | 5% |
Nursing and Health Professions | 3 | 5% |
Pharmacology, Toxicology and Pharmaceutical Science | 2 | 3% |
Agricultural and Biological Sciences | 2 | 3% |
Other | 8 | 14% |
Unknown | 18 | 31% |