Orlistat Therapy for Children with Type 1 Hyperlipoproteinemia: A Randomized Clinical Trial

Nivedita Patni, Claudia Quittner, Abhimanyu Garg

Research output: Contribution to journalArticle

Abstract

Context 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. Objective To determine the efficacy of orlistat, a gastric and pancreatic lipase inhibitor, in reducing serum triglyceride levels in patients with T1HLP. Design and Setting We conducted a randomized, open-label, clinical trial with a four-period, two-sequence ("orlistat" and "off orlistat" for 3 months), crossover study design. Patients 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. Results 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. Conclusion 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.

Original languageEnglish (US)
Pages (from-to)2403-2407
Number of pages5
JournalJournal of Clinical Endocrinology and Metabolism
Volume103
Issue number6
DOIs
StatePublished - Jun 1 2018

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Randomized Controlled Trials
Triglycerides
Fats
Fat-Restricted Diet
Nutrition
Vitamins
Therapeutics
Serum
Cross-Over Studies
Inborn Genetic Diseases
Familial hyperchylomicronemia syndrome
orlistat
Lipase
Constipation
Labels
Pancreatitis
Gases
Fasting
Stomach
Clinical Trials

ASJC Scopus subject areas

  • Endocrinology, Diabetes and Metabolism
  • Biochemistry
  • Endocrinology
  • Clinical Biochemistry
  • Biochemistry, medical

Cite this

Orlistat Therapy for Children with Type 1 Hyperlipoproteinemia : A Randomized Clinical Trial. / Patni, Nivedita; Quittner, Claudia; Garg, Abhimanyu.

In: Journal of Clinical Endocrinology and Metabolism, Vol. 103, No. 6, 01.06.2018, p. 2403-2407.

Research output: Contribution to journalArticle

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abstract = "Context 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. Objective To determine the efficacy of orlistat, a gastric and pancreatic lipase inhibitor, in reducing serum triglyceride levels in patients with T1HLP. Design and Setting We conducted a randomized, open-label, clinical trial with a four-period, two-sequence ({"}orlistat{"} and {"}off orlistat{"} for 3 months), crossover study design. Patients 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. Results 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. Conclusion 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.",
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