ArticleJournal of the Endocrine Society2019
Sirolimus: Efficacy and Complications in Children With Hyperinsulinemic Hypoglycemia: A 5-Year Follow-Up Study.
Article in Journal of the Endocrine Society, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 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.
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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
18 citing papers in PubMed, 1 synthesis or guideline pooled it, 31 citations in OpenAlex.
- International Guidelines for the Diagnosis and Management of Hyperinsulinism.Hormone research in paediatrics · 2024Guideline
- Single-center experience description of surgical management of diffuse congenital hyperinsulinism in a pediatric non-current cohort.Frontiers in endocrinology · 2026Article
- Advances in Pharmacotherapy for Congenital Hyperinsulinism.Paediatric drugs · 2026Review
- Case Report: Pasireotide treatment in neonatal congenital hyperinsulinism due to a homozygousFrontiers in endocrinology · 2026Article
- Prenatal Diagnosis and Management of Tuberous Sclerosis Complex with Cardiac Rhabdomyoma: A Case Report Highlighting the Role of Sirolimus and Postnatal Complications.Diagnostics (Basel, Switzerland) · 2025Article
- A Focal Form of Diazoxide-resistant Congenital Hyperinsulinism with Good Response to Long-acting Somatostatin.Journal of the ASEAN Federation of Endocrine Societies · 2024Article
- Non-surgical Treatment May be Appropriate for Most Chinese Children With Monogenic Congenital Hyperinsulinism Based on a Retrospective Study of 121 Patients.Pediatric diabetes · 2024Article
- Effect of sirolimus on insulin dynamics in horses.Journal of veterinary internal medicine · 2023Article
- Safety of Sirolimus in Patients with Tuberous Sclerosis Complex under Two Years of Age-A Bicenter Retrospective Study.Journal of clinical medicine · 2023Article
- Standardised practices in the networked management of congenital hyperinsulinism: a UK national collaborative consensus.Frontiers in endocrinology · 2023Review
- Response to sirolimus in a case of diffuse congenital hyperinsulinaemic hypoglycaemia due to homozygousBMJ case reports · 2022Article
- Congenital Hyperinsulinaemic Hypoglycaemia-A Review and Case Presentation.Journal of clinical medicine · 2022Review
- Congenital hyperinsulinism in infancy and childhood: challenges, unmet needs and the perspective of patients and families.Orphanet journal of rare diseases · 2022Review
- Molecular Characterization and Management of Congenital Hyperinsulinism: A Tertiary Centre Experience.Indian pediatrics · 2022Article
- Sirolimus in infants with congenital hyperinsulinism (CHI) - a single-centre experience.European journal of pediatrics · 2022Article
- Stem Cell Based Models in Congenital Hyperinsulinism - Perspective on Practicalities and Possibilities.Frontiers in endocrinology · 2022Review
- Hyperinsulinemic hypoglycemia in children and adolescents: Recent advances in understanding of pathophysiology and management.Reviews in endocrine & metabolic disorders · 2020Review
- Possible New Strategies for the Treatment of Congenital Hyperinsulinism.Frontiers in endocrinology · 2020Article
Corrections and comments
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Authors and funding
8 authors at 4 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
introductionSirolimus, a mammalian target of rapamycin inhibitor, has been used in congenital hyperinsulinism (CHI) unresponsive to diazoxide and octreotide. Reported response to sirolimus is variable, with high incidence of adverse effects. To the best of our knowledge, we report the largest group of CHI patients treated with sirolimus followed for the longest period to date.
methodsRetrospective study of CHI patients treated with sirolimus in a tertiary service and review of the 15 publications reporting CHI patients treated with mammalian target of rapamycin inhibitors. Comparison was made between the findings of this study with those previously published.
resultsTwenty-two CHI patients treated with sirolimus were included in this study. Twenty showed partial response, one showed complete response, and one was unresponsive. Five of the partially/fully responsive patients had compound heterozygous
conclusionSirolimus candidates must be carefully selected given its frequent and potentially life-threatening side effects. Its use as a short-term, last-resort therapy until normoglycemia is achieved with other agents such as lanreotide could avoid pancreatectomy. Further studies evaluating the use of sirolimus in patients with CHI are required.
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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.