SynthesisJournal of inherited metabolic disease2011
Orthopaedic management of Hurler's disease after hematopoietic stem cell transplantation: a systematic review.
Synthesis in Journal of inherited metabolic disease, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 32 papers.
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Who cites it
32 citing papers in PubMed, 67 citations in OpenAlex.
- Adult disease burden in patients with mucopolysaccharidosis type I H (Hurler syndrome): A comprehensive literature review with patient case analysis.Molecular genetics and metabolism reports · 2026Review
- Diagnostic utility of ultra-microangiography and shear wave elastography in pediatric carpal tunnel syndrome associated with mucopolysaccharidosis.Pediatric radiology · 2026Article
- Proteomics identifies novel biomarkers of synovial joint disease in a canine model of mucopolysaccharidosis I.Molecular genetics and metabolism · 2023Article
- Effects of lithium administration on vertebral bone disease in mucopolysaccharidosis I dogs.Bone · 2022Article
- Long term disease burden post-transplantation: three decades of observations in 25 Hurler patients successfully treated with hematopoietic stem cell transplantation (HSCT).Orphanet journal of rare diseases · 2021Article
- Hurdles in treating Hurler disease: potential routes to achieve a "real" cure.Blood advances · 2020Review
- Screening for Carpal Tunnel Syndrome in Patients With Mucopolysaccharidosis.Journal of child neurology · 2020Article
- Surgical Management of Spinal Disorders in People with Mucopolysaccharidoses.International journal of molecular sciences · 2020Review
- Tarsal tunnel syndrome in the mucopolysaccharidoses: A case series and literature review.JIMD reports · 2019Article
- Treatment of thoracolumbar kyphosis in patients with mucopolysaccharidosis type I: results of an international consensus procedure.Orphanet journal of rare diseases · 2019Review
- Spine challenges in mucopolysaccharidosis.International orthopaedics · 2019Review
- The new frame for Mucopolysaccharidoses.Italian journal of pediatrics · 2018Article
- Pain in Mucopolysaccharidoses: Analysis of the Problem and Possible Treatments.International journal of molecular sciences · 2018Review
- Bone health in patients with inborn errors of metabolism.Reviews in endocrine & metabolic disorders · 2018Review
- Does orthopaedic surgery improve quality of life and function in patients with mucopolysaccharidoses?Journal of children's orthopaedics · 2017Article
- Open issues in Mucopolysaccharidosis type I-Hurler.Orphanet journal of rare diseases · 2017Review
- Pathogenesis and treatment of spine disease in the mucopolysaccharidoses.Molecular genetics and metabolism · 2016Review
- Musculoskeletal manifestations in mucopolysaccharidosis type I (Hurler syndrome) following hematopoietic stem cell transplantation.Orphanet journal of rare diseases · 2016Article
- Characterization of the MPS I-H knock-in mouse reveals increased femoral biomechanical integrity with compromised material strength and altered bone geometry.Molecular genetics and metabolism reports · 2015Article
- Characterization of knee alignment in children with mucopolysaccharidosis types I and II and outcome of treatment with guided growth.Journal of children's orthopaedics · 2015Article
Corrections and comments
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Authors and funding
6 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectiveThe introduction of hematopoietic stem cell transplantation (HSCT) has significantly improved the life-span of Hurler patients (mucopolysaccharidosis type I-H, MPS I-H). Yet, the musculoskeletal manifestations seem largely unresponsive to HSCT. In order to facilitate evidence based management, the aim of the current study was to give a systematic overview of the orthopaedic complications and motor functioning of Hurler's patients after HSCT.
methodsA systematic review was conducted of the medical literature published from January 1981 to June 2010. Two reviewers independently assessed all eligible citations, as identified from the Pubmed and Embase databases. A pre-developed data extraction form was used to systematically collect information on the prevalence of radiological and clinical signs, and on the orthopaedic treatments and outcomes.
resultsA total of 32 studies, including 399 patient reports were identified. The most frequent musculoskeletal abnormalities were odontoid hypoplasia (72%), thoracolumbar kyphosis (81%), genu valgum (70%), hip dysplasia (90%) and carpal tunnel syndrome (63%), which were often treated surgically during the first decade of life. The overall complication rate of surgical interventions was 13.5%. Motor functioning was further hampered due to reduced joint mobility, hand dexterity, motor development and longitudinal growth.
conclusionStem cell transplantation does not halt the progression of a large range of disabling musculoskeletal abnormalities in Hurler's disease. Although prospective data on the quantification, progression and treatment of these deformities were very limited, early surgical intervention is often advocated. Prospective data collection will be mandatory to achieve better evidence on the effect of treatment strategies.
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