{"doi":"10.1002/oby.23928","title":"Diazoxide choline extended‐release tablet in people with <scp>Prader‐Willi</scp> syndrome: results from long‐term open‐label study","abstract":"OBJECTIVE: This study assessed the effect of 1-year administration of diazoxide choline extended-release tablet (DCCR) on hyperphagia and other complications of Prader-Willi syndrome (PWS). METHODS: The authors studied 125 participants with PWS, age ≥ 4 years, who were enrolled in the DESTINY PWS Phase 3 study and who received DCCR for up to 52 weeks in DESTINY PWS and/or its open-label extension. The primary efficacy endpoint was Hyperphagia Questionnaire for Clinical Trials (HQ-CT) score. Other endpoints included behavioral assessments, body composition, hormonal measures, and safety. RESULTS: DCCR administration resulted in significant improvements in HQ-CT (mean [SE] -9.9 [0.77], p < 0.0001) and greater improvements in those with more severe baseline hyperphagia (HQ-CT > 22). Improvements were seen in aggression, anxiety, and compulsivity (all p < 0.0001). There were reductions in leptin, insulin, and insulin resistance, as well as a significant increase in adiponectin (all p < 0.004). Lean body mass was increased (p < 0.0001). Disease severity was reduced as assessed by clinician and caregiver (both p < 0.0001). Common treatment-emergent adverse events included hypertrichosis, peripheral edema, and hyperglycemia. Adverse events infrequently resulted in discontinuation (7.2%). CONCLUSIONS: DCCR administration to people with PWS was well tolerated and associated with broad-ranging improvements in the syndrome. Sustained administration of DCCR has the potential to reduce disease severity and the burden of care for families.","journal":"Obesity","year":2023,"id":328469,"datarank":0.6056027585107467,"base_score":3.1780538303479458,"endowment":3.1780538303479458,"self_citation_contribution":0.47670807455219194,"citation_network_contribution":0.12889468395855483,"self_endowment_contribution":0.47670807455219194,"citer_contribution":0.12889468395855483,"corpus_percentile":null,"corpus_rank":null,"citation_count":23,"citer_count":19,"citers_with_citation_signal":9,"citers_with_endowment":9,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9569,"is_data_producer":true,"deposit_databanks":{"ClinicalTrials.gov":["NCT03440814","NCT03714373"]},"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2023-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":1030630,"name":"Evelien Gevers","orcid":"0000-0002-4397-4126","position":1,"is_corresponding":false},{"id":1030631,"name":"Nicola Bridges","orcid":"0000-0002-2915-4877","position":2,"is_corresponding":false},{"id":402285,"name":"Jack A. Yanovski","orcid":"0000-0001-8542-1637","position":3,"is_corresponding":false},{"id":1030632,"name":"Parisa Salehi","orcid":"0000-0003-1586-6336","position":4,"is_corresponding":false},{"id":1030633,"name":"Kathryn Obrynba","orcid":"0000-0003-1443-577X","position":5,"is_corresponding":false},{"id":231482,"name":"Eric I. Felner","orcid":"0000-0002-6567-0123","position":6,"is_corresponding":false},{"id":249069,"name":"Lynne M. Bird","orcid":"0000-0003-4833-3747","position":7,"is_corresponding":false},{"id":701643,"name":"Ashley H. Shoemaker","orcid":"0000-0003-1628-3677","position":8,"is_corresponding":false},{"id":1031395,"name":"Moris Angulo","orcid":null,"position":9,"is_corresponding":false},{"id":326229,"name":"Merlin G. Butler","orcid":"0000-0002-2911-0524","position":10,"is_corresponding":false},{"id":633176,"name":"David A. Stevenson","orcid":"0000-0001-8601-0020","position":11,"is_corresponding":false},{"id":876161,"name":"Anthony P. Goldstone","orcid":"0000-0001-8179-7071","position":12,"is_corresponding":false},{"id":1030639,"name":"John Wilding","orcid":"0000-0003-2839-8404","position":13,"is_corresponding":false},{"id":1030634,"name":"Melissa Lah","orcid":"0000-0002-2406-8961","position":14,"is_corresponding":false},{"id":1049898,"name":"M Guftar Shaikh","orcid":"0000-0002-2316-3334","position":15,"is_corresponding":false},{"id":1031397,"name":"Elizabeth Littlejohn","orcid":null,"position":16,"is_corresponding":false},{"id":702658,"name":"M. Jennifer Abuzzahab","orcid":null,"position":17,"is_corresponding":false},{"id":1030638,"name":"Amy Fleischman","orcid":"0000-0002-5497-4367","position":18,"is_corresponding":false},{"id":1050402,"name":"Patricia Hirano","orcid":null,"position":19,"is_corresponding":false},{"id":1050403,"name":"Kristen Yen","orcid":null,"position":20,"is_corresponding":false},{"id":1030635,"name":"Neil Cowen","orcid":"0000-0003-4189-1009","position":21,"is_corresponding":false},{"id":1030636,"name":"Anish Bhatnagar","orcid":"0000-0001-7078-6959","position":22,"is_corresponding":false},{"id":1050404,"name":"the C601/C602 Investigators","orcid":null,"position":23,"is_corresponding":false},{"id":1030629,"name":"Jennifer Miller","orcid":"0000-0003-4370-3438","position":0,"is_corresponding":true}],"reference_count":18,"raw_metadata":null,"created_at":"2026-07-19T01:08:52.196069Z","pmid":"37919617","pmcid":null,"fwci":null,"citation_percentile":null,"influential_citations":0,"oa_status":null,"license":null,"views":0,"total_file_size_bytes":0,"version_count":0,"fair_f":null,"fair_a":null,"fair_i":null,"fair_r":null,"fair_zscore":null,"fair_rationale":null,"fair_model":null,"fair_agent_version":null,"fair_fulltext_source":null,"fair_has_llm":null,"fair_computed_at":null,"clinical_trials":[],"software_tools":[],"db_accessions":[],"linked_datasets":[],"topics":[]}