ReviewTherapeutics and clinical risk management2016
Growth hormone therapy for Prader-willi syndrome: challenges and solutions.
Review in Therapeutics and clinical risk management, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 43 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
43 citing papers in PubMed, 73 citations in OpenAlex.
- The Efficacy, Safety, and Pharmacology of a Ghrelin O-Acyltransferase Inhibitor for the Treatment of Prader-Willi Syndrome.The Journal of clinical endocrinology and metabolism · 2022Trial
- Effects of recombinant human growth hormone treatment on growth, body composition, and safety in infants or toddlers with Prader-Willi syndrome: a randomized, active-controlled trial.Orphanet journal of rare diseases · 2019Trial
- Effectiveness and Safety of Hormonal Treatments in Children with Growth Disorders: A Systematic Review of Clinical Evidence.Clinics and practice · 2026Review
- Patient advocacy group perspectives on treatment priorities and clinical trials for the rare neurodevelopmental condition, Prader-Willi syndrome.Orphanet journal of rare diseases · 2026Article
- Long-term impact of growth hormone therapy on mortality and type 2 diabetes in Prader-Willi syndrome: a nationwide cohort study.Frontiers in endocrinology · 2025Article
- Novel Case of Prader-Willi Syndrome and Ebstein's Anomaly: Implications for Complex Care Management.Journal of pediatric genetics · 2024Article
- The Pivotal Role of Oxytocin's Mechanism of Thermoregulation in Prader-Willi Syndrome, Schaaf-Yang Syndrome, and Autism Spectrum Disorder.International journal of molecular sciences · 2024Review
- A bibliometric analysis of Prader-Willi syndrome from 2002 to 2022.Open medicine (Warsaw, Poland) · 2024Article
- Endocrine features of Prader-Willi syndrome: a narrative review focusing on genotype-phenotype correlation.Frontiers in endocrinology · 2024Review
- Growth hormone treatment in children with Prader-Willi syndrome: safety and effectiveness data from the PATRO Children study.Therapeutic advances in endocrinology and metabolism · 2024Article
- Hormonal Imbalances in Prader-Willi and Schaaf-Yang Syndromes Imply the Evolution of Specific Regulation of Hypothalamic Neuroendocrine Function in Mammals.International journal of molecular sciences · 2023Review
- Multicentric Italian case-control study on 25OH vitamin D levels in children and adolescents with Prader-Willi syndrome.Journal of endocrinological investigation · 2023Article
- Current Treatments for Patients with Genetic ObesityJournal of clinical research in pediatric endocrinology · 2023Review
- The Italian registry for patients with Prader-Willi syndrome.Orphanet journal of rare diseases · 2023Article
- Effects of early recombinant human growth hormone treatment in young Chinese children with Prader-Willi syndrome.Orphanet journal of rare diseases · 2023Article
- Long-term effects of GH therapy in adult patients with Prader-Willi syndrome: a longitudinal study.Frontiers in endocrinology · 2023Article
- Prader-Willi syndrome: Symptoms and topiramate response in light of genetics.Frontiers in neuroscience · 2023Article
- Hypothalamic AAV-BDNF gene therapy improves metabolic function and behavior in theMolecular therapy. Methods & clinical development · 2022Article
- Prader-Willi Syndrome in Adults: An Update On Nutritional Treatment and Pharmacological Approach.Current obesity reports · 2022Review
- Patients with Prader-Willi Syndrome (PWS) Underwent Bariatric Surgery Benefit more from High-Intensity Home Care.Obesity surgery · 2022Observational
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 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Prader-Willi syndrome (PWS) is characterized by a dysregulation of growth hormone (GH)/insulin-like growth factor I axis, as the consequence of a complex hypothalamic involvement. PWS' clinical picture seems to resemble the classic non-PWS GH deficiency (GHD), including short stature, excessive body fat, decreased muscle mass, and impaired quality of life. GH therapy is able to ameliorate the phenotypic appearance of the syndrome, as well as to improve body composition, physical strength, and cognitive level. In this regard, however, some pathophysiologic and clinical questions still remain, representing a challenge to give the most appropriate care to PWS patients. Data about the prevalence of GHD in PWS children are not unequivocal, ranging from 40% to 100%. In this context, to establish whether the presence (or not) of GHD may have a different effect on clinical course during GH therapy may be helpful. In addition, the comparison of GH effects in PWS children diagnosed as small for gestational age with those obtained in subjects born appropriate for gestational age is of potential interest for future trials. Emerging information seems to demonstrate the maintenance of beneficial effects of GH therapy in PWS subjects after adolescent years. Thus, GH retesting after achievement of final height should be taken into consideration for all PWS patients. However, it is noteworthy that GH administration exerts positive effects both in PWS adults with and without GHD. Another critical issue is to clarify whether the genotype-phenotype correlations may be relevant to specific outcome measures related to GH therapy. Moreover, progress of our understanding of the role of GH replacement and concomitant therapies on bone characteristics of PWS is required. Finally, a long-term surveillance of benefits and risks of GH therapy is strongly recommended for PWS population, since most of the current studies are uncontrolled and of short duration.
Indexed as
Identifiers
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.