{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,4,21]],"date-time":"2026-04-21T02:32:56Z","timestamp":1776738776857,"version":"3.51.2"},"reference-count":21,"publisher":"The Endocrine Society","issue":"6","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2018,6,1]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:sec>\n                  <jats:title>Context<\/jats:title>\n                  <jats:p>Patients with type 1 hyperlipoproteinemia (T1HLP), a rare genetic disorder, have extreme chylomicronemia and recurrent episodes of acute pancreatitis. Currently, the only therapeutic option is to consume an extremely low-fat diet because the triglyceride-lowering medications are not efficacious.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Objective<\/jats:title>\n                  <jats:p>To determine the efficacy of orlistat, a gastric and pancreatic lipase inhibitor, in reducing serum triglyceride levels in patients with T1HLP.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Design and Setting<\/jats:title>\n                  <jats:p>We conducted a randomized, open-label, clinical trial with a four-period, two-sequence (\u201corlistat\u201d and \u201coff orlistat\u201d for 3 months), crossover study design.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Patients<\/jats:title>\n                  <jats:p>Two unrelated young Asian Indian males (11 and 9 years old) with T1HLP due to homozygous large GPIHBP1 deletions were enrolled at the UT Southwestern Medical Center. The patients were randomized to receive 3 months of orlistat or no therapy (off), then crossed over to the other arm, and this sequence was then repeated. Fasting serum triglyceride levels, fat-soluble vitamins, and gastrointestinal side effects were assessed.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Results<\/jats:title>\n                  <jats:p>Compared with the two off periods, orlistat therapy reduced serum triglycerides by 53.3% and 53.0% in patient 1 and 45.8% and 62.2% in patient 2. There was no deficiency of fat-soluble vitamin levels, and their growth continued. There were no serious adverse effects of orlistat; patient 1 had a mild increase in passage of gas and bloating, and patient 2 had constipation with mild stool leakage.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Conclusion<\/jats:title>\n                  <jats:p>Orlistat is safe and highly efficacious in lowering serum triglycerides in children with T1HLP and should be the first-line therapy in conjunction with an extremely low-fat diet.<\/jats:p>\n               <\/jats:sec>","DOI":"10.1210\/jc.2018-00369","type":"journal-article","created":{"date-parts":[[2018,4,6]],"date-time":"2018-04-06T20:21:15Z","timestamp":1523046075000},"page":"2403-2407","source":"Crossref","is-referenced-by-count":22,"title":["Orlistat Therapy for Children With Type 1 Hyperlipoproteinemia: A Randomized Clinical Trial"],"prefix":"10.1210","volume":"103","author":[{"given":"Nivedita","family":"Patni","sequence":"first","affiliation":[{"name":"Division of Pediatric Endocrinology, Department of Pediatrics and the Center for Human Nutrition, UT Southwestern Medical Center, Dallas, Texas"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Claudia","family":"Quittner","sequence":"additional","affiliation":[{"name":"Division of Nutrition and Metabolic Diseases, Department of Internal Medicine and the Center for Human Nutrition, UT Southwestern Medical Center, Dallas, Texas"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Abhimanyu","family":"Garg","sequence":"additional","affiliation":[{"name":"Division of Nutrition and Metabolic Diseases, Department of Internal Medicine and the Center for Human Nutrition, UT Southwestern Medical Center, Dallas, Texas"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"80","published-online":{"date-parts":[[2018,4,5]]},"reference":[{"issue":"2","key":"2020071621290951900_B1","doi-asserted-by":"crossref","first-page":"455","DOI":"10.1016\/S0025-7125(16)31430-4","article-title":"Chylomicronemia syndrome: interaction of genetic and acquired hypertriglyceridemia","volume":"66","author":"Brunzell","year":"1982","journal-title":"Med Clin North Am"},{"key":"2020071621290951900_B2","unstructured":"Burnett\n              JR\n            , HooperAJ, HegeleRA. 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