Effect of plasmapheresis versus standard medical treatment in patients with hypertriglyceridemia-associated acute pancreatitis complicated by early organ failure (PERFORM-R): Study design and rationale of a multicenter, pragmatic, registry-based randomized controlled trial.

Abstract

Background: The prevalence of hypertriglyceridemia-associated acute pancreatitis (HTG-AP) is increasing. Studies have demonstrated the association between higher initial plasma triglyceride (TG) levels and worse clinical prognosis; therefore, lowering plasma TG has been the mainstay when managing HTG-AP. For TG-lowering therapy, plasmapheresis, which is costly and of potential complications, is currently widely used to clear TG from plasma, but whether it confers clinical benefits is unclear. In this trial, we aimed to evaluate the effect of plasmapheresis versus standard medical treatment on the duration of organ failure in HTG-AP patients with early organ failure.

Methods: This is a multicenter, pragmatic, registry-based, randomized controlled trial. Based on previous studies, up to 236 HTG-AP patients with early organ failure are projected to be randomly assigned to either the plasmapheresis group or the standard medical treatment group (insulin plus low molecular weight heparin therapy). The PERFORM registry will be used as the platform for patient enrollment. The primary outcome is organ failure-free days to 14 days of enrollment. Organ failure in this trial is defined as an individual sequential organ failure assessment (SOFA) score of two or more for the respiratory, cardiovascular, or renal system. Patients who died before day 14 will be assigned zero organ failure-free days.

Discussion: This trial will provide top-class evidence regarding the clinical impact of plasmapheresis in HTG-AP patients with early organ failure. The findings of this trial will have a direct influence on the current clinical practice concerning the management of HTG-AP.