Trial reportPediatric nephrology (Berlin, Germany)2018
Teenagers and young adults with nephropathic cystinosis display significant bone disease and cortical impairment.
Trial report in Pediatric nephrology (Berlin, Germany), 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers, 1 of them a synthesis that pooled it.
What it found
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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
15 citing papers in PubMed, 1 synthesis or guideline pooled it, 26 citations in OpenAlex.
- Adult complications of nephropathic cystinosis: a systematic review.Pediatric nephrology (Berlin, Germany) · 2021Pooled it
- Cystinosis-associated metabolic bone disease: pathogenesis and outcome.Pediatric nephrology (Berlin, Germany) · 2026Review
- Clinical practice recommendations for the diagnosis and management of nephropathic cystinosis.Nature reviews. Nephrology · 2026Review
- Long-term outcomes in nephropathic cystinosis: a review.Pediatric nephrology (Berlin, Germany) · 2026Review
- Adherence to delayed-release cysteamine in nephropathic cystinosis over time: data from the prospective CrYSTobs cohort study.Pediatric nephrology (Berlin, Germany) · 2025Article
- The assessment and treatment of the musculoskeletal manifestations of cystinosis.Frontiers in nephrology · 2025Review
- Changes in bone density and microarchitecture in adolescents undergoing a first kidney transplantation: a prospective study.European journal of pediatrics · 2024Article
- Addressing the psychosocial aspects of transition to adult care in patients with cystinosis.Pediatric nephrology (Berlin, Germany) · 2024Review
- Cortical impairment and reduced muscle mass in children and young adults with nephropathic cystinosis.Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research · 2024Article
- Review
- Patients With Infantile Nephropathic Cystinosis in Germany and Austria: A Retrospective Cohort Study.Frontiers in medicine · 2022Article
- Peripheral Blood Mononuclear Cells (PBMCs) to Dissect the Underlying Mechanisms of Bone Disease in Chronic Kidney Disease and Rare Renal Diseases.Current osteoporosis reports · 2021Review
- Article
- Bone Disease in Nephropathic Cystinosis: Beyond Renal Osteodystrophy.International journal of molecular sciences · 2020Review
- Skeletal Consequences of Nephropathic Cystinosis.Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research · 2018Article
Corrections and comments
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Authors and funding
11 authors at 8 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundBone impairment appears to be a novel complication of nephropathic cystinosis despite cysteamine therapy. Its exact underlying pathophysiology is nevertheless unclear. The objective of this study was to evaluate bone status among patients included in the French Crystobs study.
methodsIn addition to clinical data, bone status was evaluated using biomarkers (ALP, PTH, 25-D, 1-25D, FGF23), DXA (spine and total body), and high-resolution peripheral quantitative computed tomography (HR-pQCT) at the tibia and radius. Results were compared to age- and gender-matched healthy controls (1:2 basis) from the local reference cohorts.
resultsAt a median age of 22.5 (10.2-34.6) years, 10 patients with nephropathic cystinosis were included (2 receiving conservative therapies, 2 undergoing hemodialysis, 6 with a past of renal transplantation); 7 out of 10 patients complained of a bone symptom (past of fracture, bone deformations, and/or bone pain). Biochemicals and spine DXA did not show any significant abnormalities. Using HR-pQCT, significant decreases in cortical parameters (e.g., cortical thickness 850 (520-1100) versus 1225 (480-1680) μm; p < 0.05) and total volumetric bone mineral density (290 (233-360) versus 323 (232-406) mg/cm
conclusionsIn this pilot study, bone impairment (rather cortical than trabecular) is a significant clinical problem in nephropathic cystinosis; 70% of patients displayed significant bone symptoms, during teenage or young adulthood. This new complication should be known by physicians because of its potential dramatic impact on quality of life.
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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.