{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,15]],"date-time":"2026-07-15T16:12:18Z","timestamp":1784131938568,"version":"3.55.0"},"reference-count":41,"publisher":"S. Karger AG","license":[{"start":{"date-parts":[[2026,5,18]],"date-time":"2026-05-18T00:00:00Z","timestamp":1779062400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/karger.com\/pages\/terms-and-conditions"},{"start":{"date-parts":[[2026,5,18]],"date-time":"2026-05-18T00:00:00Z","timestamp":1779062400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/karger.com\/pages\/terms-and-conditions"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Horm Res Paediatr"],"abstract":"<jats:p>Introduction: Duchenne muscular dystrophy (DMD) is a progressive neuromuscular disorder associated with obesity due to low energy expenditure and high-dose corticosteroid treatment. Obesity significantly impacts quality of life in individuals with DMD and is consistently identified as a top family concern. Despite FDA approval of weight-loss medications for adolescents, including phentermine\/topiramate (PHEN\/TPM), there is limited evidence for their use in children with DMD, particularly regarding potential cardiac complications in a population already at elevated risk. Method: Individuals with a genetic diagnosis of DMD, who were prescribed PHEN\/TPM from January 1, 2015, to April 25, 2025, were identified from the electronic medical record system of Children\u2019s Hospital Colorado (CHCO). A final cohort of six individuals treated for at least 6 months had clinic-obtained weights documented and were included in the analysis. Results: All patients were aged 9.9\u201316.9 years, with baseline weight 45\u2013108.2 kg and BMI 29.5\u201339.7 kg\/m2. Treatment duration ranged from 0.6 to 4.2 years. In the first year of therapy, median BMI decreased by 3.75 kg\/m2 (interquartile ranges [IQRs]: \u22125.5 to \u22122.6), BMI standard deviation (SD) by 0.57 (IQR: \u22120.99 to \u22120.31), and weight SD by 0.73 (IQR: \u22121.17 to \u22120.59). Over the full treatment course (0.6\u20134.2 years), median annualized changes were BMI \u22121.73 kg\/m2 (IQR: \u22122.93 to 0.17), BMI SD \u22120.31 (IQR: \u22120.44 to \u22120.15), and weight SD \u22120.30 (IQR: \u22120.70 to \u22120.11). Longitudinal analysis revealed significant nonlinear trends for BMI and BMI SD, with nadir effect by year two. Cardiac parameters showed initial transient heart rate increase (HR, median +13.5 bpm at 0.5 years) but no significant changes in longitudinal follow-up of HR, systolic blood pressure, diastolic blood pressure, or left ventricular ejection fraction. Two individuals developed kidney stones; other adverse events were mild to moderate. Conclusion: Treatment with PHEN\/TPM in a small group of children with DMD and obesity was associated with clinically relevant reductions in measures of obesity and was generally well-tolerated with no significant adverse cardiac effects observed during extended follow-up. These findings support the potential use of PHEN\/TPM as a weight management option in carefully selected individuals with DMD, though larger prospective studies are warranted.<\/jats:p>","DOI":"10.1159\/000552576","type":"journal-article","created":{"date-parts":[[2026,5,18]],"date-time":"2026-05-18T16:01:02Z","timestamp":1779120062000},"page":"1-11","source":"Crossref","is-referenced-by-count":0,"title":["Outcomes of Phentermine-Topiramate in Children Living with Duchenne Muscular Dystrophy"],"prefix":"10.1159","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-0981-7757","authenticated-orcid":false,"given":"Li-Min","family":"Chen","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Po-Chih","family":"Shen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Philip","family":"Zeitler","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"127","published-online":{"date-parts":[[2026,5,18]]},"reference":[{"key":"ref1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(09)70271-6"},{"key":"ref2","doi-asserted-by":"publisher","DOI":"10.1038\/ejcn.2014.93"},{"key":"ref3","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpeds.2016.02.067"},{"key":"ref4","doi-asserted-by":"publisher","DOI":"10.1002\/ajmg.a.40517"},{"key":"ref5","doi-asserted-by":"publisher","DOI":"10.1002\/mus.28065"},{"key":"ref6","doi-asserted-by":"publisher","DOI":"10.3390\/nu14163304"},{"key":"ref7","doi-asserted-by":"publisher","DOI":"10.1002\/mus.28309"},{"key":"ref8","doi-asserted-by":"publisher","DOI":"10.1542\/peds.2018-0333F"},{"key":"ref9","doi-asserted-by":"publisher","DOI":"10.1002\/mus.28062"},{"key":"ref10","doi-asserted-by":"publisher","DOI":"10.1089\/chi.2024.0297"},{"key":"ref11","doi-asserted-by":"publisher","DOI":"10.1111\/ijpo.13113"},{"key":"ref12","doi-asserted-by":"publisher","DOI":"10.1038\/s41366-025-01790-w"},{"key":"ref13","doi-asserted-by":"publisher","DOI":"10.1007\/s40272-022-00532-z"},{"key":"ref14","doi-asserted-by":"publisher","DOI":"10.1001\/jamanetworkopen.2024.33326"},{"key":"ref15","doi-asserted-by":"publisher","DOI":"10.1001\/jama.2024.11809"},{"key":"ref16","doi-asserted-by":"publisher","DOI":"10.1002\/1098-2396(20010101)39:1%3c32::AID-SYN5%3e3.0.CO;2-3"},{"key":"ref17","doi-asserted-by":"publisher","DOI":"10.3390\/life13091845"},{"key":"ref18","doi-asserted-by":"publisher","DOI":"10.1001\/jamanetworkopen.2023.29178"},{"key":"ref19","doi-asserted-by":"publisher","DOI":"10.1016\/0167-5273(90)90082-g"},{"key":"ref20","doi-asserted-by":"publisher","DOI":"10.1161\/jaha.123.032960"},{"key":"ref21","doi-asserted-by":"publisher","DOI":"10.2147\/ahmt.s383454"},{"key":"ref22","doi-asserted-by":"publisher","DOI":"10.1016\/j.jbi.2008.08.010"},{"key":"ref23","doi-asserted-by":"publisher","DOI":"10.1016\/j.jbi.2019.103208"},{"key":"ref24","doi-asserted-by":"publisher","DOI":"10.3390\/nu9060594"},{"key":"ref25","doi-asserted-by":"publisher","DOI":"10.1542\/peds.2022-060640"},{"key":"ref26","doi-asserted-by":"publisher","DOI":"10.3389\/fendo.2022.1043650"},{"key":"ref27","doi-asserted-by":"publisher","DOI":"10.1089\/chi.2022.0062"},{"key":"ref28","doi-asserted-by":"publisher","DOI":"10.1161\/CIR.0000000000000526"},{"key":"ref29","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(18)30025-5"},{"key":"ref30","doi-asserted-by":"publisher","DOI":"10.3233\/JND-180340"},{"key":"ref31","doi-asserted-by":"publisher","DOI":"10.1016\/s0140-6736(11)60205-5"},{"key":"ref32","doi-asserted-by":"publisher","DOI":"10.1038\/oby.2011.94"},{"key":"ref33","doi-asserted-by":"publisher","DOI":"10.3945\/ajcn.111.024927"},{"key":"ref34","doi-asserted-by":"publisher","DOI":"10.1001\/archinternmed.2010.334"},{"key":"ref35","doi-asserted-by":"publisher","DOI":"10.1016\/j.hrcr.2024.07.023"},{"key":"ref36","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2125.2009.03521.x"},{"key":"ref37","doi-asserted-by":"publisher","DOI":"10.1111\/bcp.12283"},{"key":"ref38","doi-asserted-by":"publisher","DOI":"10.1016\/j.urology.2010.06.048"},{"key":"ref39","doi-asserted-by":"publisher","DOI":"10.1111\/bcp.12751"},{"key":"ref40","doi-asserted-by":"publisher","DOI":"10.1002\/mus.21420"},{"key":"ref41","doi-asserted-by":"publisher","DOI":"10.1542\/peds.2018-0333i"}],"container-title":["Hormone Research in Paediatrics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/karger.com\/article-pdf\/doi\/10.1159\/000552576","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,7,15]],"date-time":"2026-07-15T16:00:52Z","timestamp":1784131252000},"score":1,"resource":{"primary":{"URL":"https:\/\/karger.com\/article\/doi\/10.1159\/000552576"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,5,18]]},"references-count":41,"URL":"https:\/\/doi.org\/10.1159\/000552576","archive":["Portico"],"relation":{"is-supplemented-by":[{"id-type":"doi","id":"10.6084\/m9.figshare.32314155","asserted-by":"subject"}]},"ISSN":["1663-2818","1663-2826"],"issn-type":[{"value":"1663-2818","type":"print"},{"value":"1663-2826","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,5,18]]}}}