ReviewPediatric nephrology (Berlin, Germany)2013
Primary hyperoxaluria type 1: practical and ethical issues.
Review in Pediatric nephrology (Berlin, Germany), 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
19 citing papers in PubMed.
- The efficacy and safety of RNA interference for the treatment of primary hyperoxaluria: a systematic review and meta-analysis.Clinical kidney journal · 2025Review
- Application of four pricing models for orphan medicines: a case study for lumasiran.Orphanet journal of rare diseases · 2024Article
- Diagnosis and management of primary hyperoxalurias: best practices.Pediatric nephrology (Berlin, Germany) · 2024Review
- Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 30-month analysis of the phase 3 ILLUMINATE-B trial.Frontiers in pediatrics · 2024Article
- Qualitative assessment of the patient experience of primary hyperoxaluria type 1: an observational study.BMC nephrology · 2023Observational
- Clinical and molecular characterization of a large primary hyperoxaluria cohort from Saudi Arabia: a retrospective study.Pediatric nephrology (Berlin, Germany) · 2023Article
- Effect of liver transplantation with primary hyperoxaluria type 1: Five case reports and review of literature.World journal of clinical cases · 2023Article
- Case Report: effect of lumasiran treatment in a late preterm baby with antenatal diagnosis of primary hyperoxaluria type 1.Frontiers in pediatrics · 2023Article
- Primary hyperoxaluria type 1 in developing countries: novel challenges in a new therapeutic era.Clinical kidney journal · 2022Review
- Primary hyperoxaluria: the pediatric nephrologist's point of view.Clinical kidney journal · 2022Review
- Clinical and economic impact of primary hyperoxaluria: a retrospective claims analysis.Journal of managed care & specialty pharmacy · 2022Article
- DeleteriousGenes · 2020Article
- Importance of Assessing Compliance with Conservative Treatment of Primary Hyperoxaluria Type 1: A Case Report of a Patient with I244T/c.969-3C>G Mutation.The Permanente journal · 2020Article
- Molecular basis of primary hyperoxaluria: clues to innovative treatments.Urolithiasis · 2019Review
- Skin microvascular dysfunction as an early cardiovascular marker in primary hyperoxaluria type I.Pediatric nephrology (Berlin, Germany) · 2019Article
- Successful long-term outcome of pediatric liver-kidney transplantation: a single-center study.Pediatric nephrology (Berlin, Germany) · 2018Article
- Clinical spectrum of primary hyperoxaluria type 1: Experience of a tertiary center.Nephrologie & therapeutique · 2017Article
- Primary hyperoxaluria type 1 in 18 children: genotyping and outcome.International journal of nephrology · 2015Article
- Mutational analysis of AGXT in two Chinese families with primary hyperoxaluria type 1.BMC nephrology · 2014Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Primary hyperoxaluria type 1 (PH1) is a rare inborn error of glyoxylate metabolism of autosomal recessive inheritance, leading to progressive systemic oxalate storage (named 'oxalosis') with a high rate of morbidity and mortality, as well as an unacceptable quality of life for most patients. The adverse outcome, however, is partly due to issues that can be overcome. First, the diagnosis of PH is often delayed due to a general lack of knowledge of the disease among physicians. This accounts specifically for patients with pyridoxine sensitive PH, a group that is paradoxically most easy to treat. Second, lack of adherence to a strict conduction of conservative treatment and optimal urological management may enhance an adverse outcome of the disease. Third, specific techniques to establish PH1 and specific therapies are currently often not available in several low-resources countries with a high prevalence of PH. The management of patients with advanced disease is extremely difficult and warrants a tailor-made approach in most cases. Comprehensive programs for education of local physicians, installation of national centers of expertise, European support of low-resources countries for the management of PH patients and intensified international collaboration on the management of current patients, as well as on conduction of clinical studies, may further improve outcome of PH.
Indexed as
Identifiers
23494551What OpenQuestion holds
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.