ArticleJIMD reports2013
In vivo bone architecture in pompe disease using high-resolution peripheral computed tomography.
Article in JIMD reports, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
8 citing papers in PubMed, 14 citations in OpenAlex.
- The clinical application of high-resolution peripheral computed tomography (HR-pQCT) in adults: state of the art and future directions.Osteoporosis international : a journal established as result of cooperation between the European Foundation for Osteoporosis and the National Osteoporosis Foundation of the USA · 2021Review
- Spectrum of microarchitectural bone disease in inborn errors of metabolism: a cross-sectional, observational study.Orphanet journal of rare diseases · 2020Observational
- Multisystem late onset Pompe disease (LOPD): an update on clinical aspects.Annals of translational medicine · 2019Review
- Nutrition and exercise in Pompe disease.Annals of translational medicine · 2019Review
- Neuromuscular Diseases and Bone.Frontiers in endocrinology · 2019Review
- Improvement of bone mineral density after enzyme replacement therapy in Chinese late-onset Pompe disease patients.BMC research notes · 2017Article
- Improvement in Bone Mineral Density and Architecture in a Patient with Gaucher Disease Using Teriparatide.JIMD reports · 2015Article
- Non-muscle involvement in late-onset glycogenosis II.Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology · 2013Review
Corrections and comments
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Authors and funding
7 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Pompe disease (lysosomal acid alpha-glucosidase deficiency) in adolescents and adults presents primarily with muscle weakness. Bone weakness is an under-recognized finding in patients with Pompe disease, but there is emerging evidence that loss of muscle function and mobility can lead to loss of mineral content and a higher risk of fracture. In addition to the mineral content, architecture is also important in determining the overall strength of the bone. We present the results of the longest longitudinal duration study to date using a novel application of high-resolution peripheral quantitative computed tomography (HR-pQCT) in four patients with Pompe disease over 4 years of observation during the normal course of their disease management. The subjects varied in treatment status with recombinant human alpha-glucosidase (rhGAA), use of anti-resorptive therapy (such as bisphosphonates), mobility and weight-bearing status, and the use of side-alternating vibration therapy. Our observations were that HR-pQCT can measure trends in mineral density and architecture over a long period of observation and may be an early indicator of the response to interventional therapies. In addition, a combination of decreased loading forces due to decreased mobility likely contributes to the compromise of bone integrity in Pompe disease. These trends can be reversed by applying increased loading forces such as vibration therapy and maintaining weight-bearing and mobility. We conclude that HR-pQCT can serve as a valuable tool to monitor bone health in patients with Pompe disease.
Identifiers
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.