Scope and heterogeneity of outcomes reported in randomized trials in patients receiving peritoneal dialysis

Abstract

Background: Randomized trials can provide evidence to inform decision-making but this may be limited if the outcomes of importance to patients and clinicians are omitted or reported inconsistently. We aimed to assess the scope and heterogeneity of outcomes reported in trials in peritoneal dialysis (PD).

Methods: We searched the Cochrane Kidney and Transplant Specialized Register for randomized trials in PD. We extracted all reported outcome domains and measurements and analyzed their frequency and characteristics.

Results: From 128 reports of 120 included trials, 80 different outcome domains were reported. Overall, 39 (49%) domains were surrogate, 23 (29%) patient-reported and 18 (22%) clinical. The five most commonly reported domains were PD-related infection [59 (49%) trials], dialysis solute clearance [51 (42%)], kidney function [45 (38%)], protein metabolism [44 (37%)] and inflammatory markers/oxidative stress [42 (35%)]. Quality of life was reported infrequently (4% of trials). Only 14 (12%) trials included a patient-reported outcome as a primary outcome. The median number of outcome measures (defined as a different measurement, aggregation and metric) was 22 (interquartile range 13-37) per trial. PD-related infection was the most frequently reported clinical outcome as well as the most frequently stated primary outcome. A total of 383 different measures for infection were used, with 66 used more than once.

Conclusions: Trials in PD include important clinical outcomes such as infection, but these are measured and reported inconsistently. Patient-reported outcomes are infrequently reported and nearly half of the domains were surrogate. Standardized outcomes for PD trials are required to improve efficiency and relevance.

Keywords: outcomes; patient-reported outcomes; peritoneal dialysis; trials.

FIGURE 1:

Search results.

FIGURE 2:

Number of trials reporting each outcome domain (total 120 trials, 80 outcome domains).

FIGURE 3:

(A) Frequency of outcome measures (definitions and time points) of dialysis solute clearance reported in >4% of trials (17 of 84 outcomes measures shown). (B) Frequency of outcome measures (definitions and time points) of kidney function reported in >4% of trials (17 of 73 outcomes measures shown). (C) Frequency of outcome measures (definitions and time points) of PD-related infection reported in >5% of trials (36 of 383 outcomes measures shown). (D) Frequency of outcome measures (definitions and time points) among trials reporting mortality (37 trials, 65 outcomes measures). (E) Frequency of outcome measures (definitions and time points) among trials reporting gastrointestinal symptoms (17 trials, 32 outcomes measures). (F) Frequency of outcome measures (definitions and time points) among trials reporting pain (non-PD-specific) (14 trials and 18 outcomes measures).

FIGURE 3:

(A) Frequency of outcome measures (definitions and time points) of dialysis solute clearance reported in >4% of trials (17 of 84 outcomes measures shown). (B) Frequency of outcome measures (definitions and time points) of kidney function reported in >4% of trials (17 of 73 outcomes measures shown). (C) Frequency of outcome measures (definitions and time points) of PD-related infection reported in >5% of trials (36 of 383 outcomes measures shown). (D) Frequency of outcome measures (definitions and time points) among trials reporting mortality (37 trials, 65 outcomes measures). (E) Frequency of outcome measures (definitions and time points) among trials reporting gastrointestinal symptoms (17 trials, 32 outcomes measures). (F) Frequency of outcome measures (definitions and time points) among trials reporting pain (non-PD-specific) (14 trials and 18 outcomes measures).