ArticleOrphanet journal of rare diseases2017
Extreme caution on the use of sirolimus for the congenital hyperinsulinism in infancy patient.
Article in Orphanet journal of rare diseases, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers, 1 of them a synthesis that pooled it.
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.
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, 1 synthesis or guideline pooled it, 35 citations in OpenAlex.
- International Guidelines for the Diagnosis and Management of Hyperinsulinism.Hormone research in paediatrics · 2024Guideline
- Advances in Pharmacotherapy for Congenital Hyperinsulinism.Paediatric drugs · 2026Review
- Case Report: Pasireotide treatment in neonatal congenital hyperinsulinism due to a homozygousFrontiers in endocrinology · 2026Article
- Genotype-histotype-phenotype correlations in hyperinsulinemic hypoglycemia.Histology and histopathology · 2024Review
- Standardised practices in the networked management of congenital hyperinsulinism: a UK national collaborative consensus.Frontiers in endocrinology · 2023Review
- Congenital hyperinsulinism in infancy and childhood: challenges, unmet needs and the perspective of patients and families.Orphanet journal of rare diseases · 2022Review
- Congenital hyperinsulinism in clinical practice: From biochemical pathophysiology to new monitoring techniques.Frontiers in pediatrics · 2022Review
- Hyperinsulinemic hypoglycemia in children and adolescents: Recent advances in understanding of pathophysiology and management.Reviews in endocrine & metabolic disorders · 2020Review
- Update of variants identified in the pancreatic β-cell KHuman mutation · 2020Review
- Clinical Efficacy Evaluation of Sirolimus in Congenital Hyperinsulinism.International journal of endocrinology · 2020Article
- Possible New Strategies for the Treatment of Congenital Hyperinsulinism.Frontiers in endocrinology · 2020Article
- Clinical characteristics and phenotype-genotype review of 25 Omani children with congenital hyperinsulinism in infancy. A one-decade single-center experience.Saudi medical journal · 2019Article
- Current and Emerging Agents for the Treatment of Hypoglycemia in Patients with Congenital Hyperinsulinism.Paediatric drugs · 2019Review
- Therapies and outcomes of congenital hyperinsulinism-induced hypoglycaemia.Diabetic medicine : a journal of the British Diabetic Association · 2019Review
- Characterization of diabetes following pancreatic surgery in patients with congenital hyperinsulinism.Orphanet journal of rare diseases · 2018Article
- Genetic characteristics of patients with congenital hyperinsulinism.Current opinion in pediatrics · 2018Review
- The burden of congenital hyperinsulinism in the United Kingdom: a cost of illness study.Orphanet journal of rare diseases · 2018Article
- Morphoproteomics and biomedical analytics coincide with clinical outcomes in supporting a constant but variable role for the mTOR pathway in the biology of congenital hyperinsulinism of infancy.Orphanet journal of rare diseases · 2017Article
- Hyperinsulinemic hypoglycemia: clinical, molecular and therapeutical novelties.Journal of inherited metabolic disease · 2017Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 3 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
We have recently published on the limited effectiveness of sirolimus as a treatment option for hypoglycaemia as a consequence of hyperinsulinism. Our data oppose the view that mTOR inhibitors provide new opportunities for the treatment of patients with hyperinsulinism. We are not convinced by the argument that any benefit for some patients outweighs the potential and later long-term problems that accompany mTOR inhibition in the neonate. We also express the opinion that caution must be taken when repurposing/repositioning therapies in the field of rare disease.
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What 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.