Article type
Year
Abstract
Background: The minimally important difference (MID) provides a measure of the smallest change in a patient reported outcome (PRO) instrument that patients perceive as important. An anchor-based approach is widely considered the most appropriate method for MID determination. No study or database currently exists that documents and appraises all anchor-based MIDs associated with PRO instruments in children.
Objectives: To complete a systematic survey of the literature to collect, characterize and appraise published anchor-based MIDs associated with PRO instruments used in evaluating the effects of interventions on chronic medical and psychiatric conditions in children.
Methods: We searched MEDLINE, EMBASE, and PsycINFO (1989 to 11 February 2015) to identify studies reporting empirical ascertainment of anchor-based MIDs in pediatric PROs. Teams of two reviewers screened titles and abstracts, reviewed full texts of citations identified as potentially eligible, extracted relevant data, and assessed the credibility of all studies.
Results: Of 3910 citations, 706 were reviewed in full text; 32 proved eligible. Anchor-based MID determinations were assessed in 23 (72%) disease-specific PROs and 10 (28%) generic PROs (one study reported both). Fourteen PROs and anchors were self-reported, 10 PROs and 13 anchors were proxy-reported (parent or guardian), eight PROs and three anchors were both self- and proxy-reported, and reporting of two anchors was unclear. Anchor-based approaches consisted of a standard approach (range of scores on an independent anchor that was classified as 'minimally important change') in 27 studies, three used Receiver Operating Characteristic curves, and two used an approach based on hypothetical scenarios. Detailed results and the credibility assessments of the anchor-based approaches will be presented at the Cochrane Colloquium.
Conclusions: We have compiled the first comprehensive synthesis of anchor-based MIDs for PROs in children. This compendium will help clinical trialists in child health improve sample size calculations, and will enhance the interpretability of treatment effects in systematic reviews and practice guidelines.
Objectives: To complete a systematic survey of the literature to collect, characterize and appraise published anchor-based MIDs associated with PRO instruments used in evaluating the effects of interventions on chronic medical and psychiatric conditions in children.
Methods: We searched MEDLINE, EMBASE, and PsycINFO (1989 to 11 February 2015) to identify studies reporting empirical ascertainment of anchor-based MIDs in pediatric PROs. Teams of two reviewers screened titles and abstracts, reviewed full texts of citations identified as potentially eligible, extracted relevant data, and assessed the credibility of all studies.
Results: Of 3910 citations, 706 were reviewed in full text; 32 proved eligible. Anchor-based MID determinations were assessed in 23 (72%) disease-specific PROs and 10 (28%) generic PROs (one study reported both). Fourteen PROs and anchors were self-reported, 10 PROs and 13 anchors were proxy-reported (parent or guardian), eight PROs and three anchors were both self- and proxy-reported, and reporting of two anchors was unclear. Anchor-based approaches consisted of a standard approach (range of scores on an independent anchor that was classified as 'minimally important change') in 27 studies, three used Receiver Operating Characteristic curves, and two used an approach based on hypothetical scenarios. Detailed results and the credibility assessments of the anchor-based approaches will be presented at the Cochrane Colloquium.
Conclusions: We have compiled the first comprehensive synthesis of anchor-based MIDs for PROs in children. This compendium will help clinical trialists in child health improve sample size calculations, and will enhance the interpretability of treatment effects in systematic reviews and practice guidelines.