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

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

SUBMITTER: Patni N 

PROVIDER: S-EPMC6456945 | biostudies-literature | 2018 Jun

REPOSITORIES: biostudies-literature