KNEES-ACL has superior responsiveness compared to the most commonly used patient-reported outcome measures for anterior cruciate ligament injury

et al. 2021

Results by patient‐reported outcome measures (PROMs) from randomized controlled trials (RCTs) in musculoskeletal research often influence healthcare strategies. We aimed to evaluate to which extent these RCTs use adequate PROMs, and how this influences the results and conclusions. We identified RCTs of sports research relevance with PROMs as primary outcomes published in 13 preselected journals between January 1, 2008, and November 1, 2019; all journals regularly publish results from musculoskeletal research. Five journals have a high impact factor (>15), and eight with lower impact factors are widely read journals. It was assessed whether the RCTs had used PROMs with high content validity and whether the most adequate PROMs were used (ie, the most well developed and well validated for the patients enrolled in the study). We registered journal impact factor, year of publication, existence of a registered protocol, and whether the study showed significant difference between interventions. A total of 54 RCTs with 56 primary outcomes comprising 26 different PROMs were identified. For 13 RCTs (24%), a protocol was not published. In only 24 of RCTs (44%), the most appropriate PROM had been used as primary outcome, independent of a registered protocol, ranking of the journal, and year of publication. In seven cases, PROMs were used to evaluate a condition that they had not been developed for. RCTs that used the most adequate PROM showed significantly more often (46%) difference in outcomes in contrast to RCTs that used inadequate PROMs (22%) (P = 0.0483). In conclusion, in the majority of RCTs, the most adequate PROM had not been used. Studies, in which the most adequate PROM had been used as outcome, were significantly more likely to show significant difference between interventions. The extent to which protocols were not available was surprisingly high. Journals should request that adequate PROMs are used in RCTs, and if this is not the case that it is discussed how it might influence the results and conclusions. Likewise, it should be requested that a protocol is published or registered.

Section: Discussionmentioning

confidence: 99%

Are adequate PROMs used as outcomes in randomized controlled trials? an analysis of 54 trials

Hansen

et al. 2021

“…It has been shown that PROMs developed and validated for the patient group and intervention in question are more responsive than PROMs that do not possess these qualities. 12 However, it has not been studied how this influences the results and conclusions of randomized treatment studies. In other words, how often do data from inappropriate PROMs lead to false conclusions because of type 1 or type 2 errors?…”

Section: Discussionmentioning

confidence: 99%

Potential problems in the use of patient reported outcome measures (PROMs) and reporting of PROM data in sports science

Krogsgaard

et al. 2020

To evaluate the effect of a treatment for anterior cruciate ligament insufficiency (ACLD) in the knee using a patient reported outcome measure (PROM) that is specific for shoulder instability is obviously inappropriate. Likewise, it may be suboptimal to use PROMs for hip and knee osteoarthritis (like the Western Ontario and McMaster Universities Arthritis Index [WOMAC]) or meniscal injury (like the Western Ontario Meniscal Evaluation Tool [WOMET]) for

“…The responsiveness of various PROMs used in studies is in most cases not known and can therefore not be used as an argument for the choice of PROM. As high responsiveness is most importantly the result of the content and construct validity of the PROM, 34 a minimum requirement should be that PROMs are developed through feedback from the same patient group targeted in the planned study (ie, the PROM has high content validity for the actual study population) and are adequately validated with respect to its measurement properties (ie, has construct validity for the actual study population). 31,35 Cohen's ES relates the change in PROM score to the heterogeneity of the scores (ie, the variance of the PROM scores at baseline), thus assigning high responsiveness to PROMs that attain a narrow range of values.…”

Section: Discussionmentioning

confidence: 99%

Responsiveness, minimal important difference, minimal relevant difference, and optimal number of patients for a study

Comins

et al. 2020

Self Cite

Several terms are used to describe changes in PROM scores in relation to treatments.Whether the change is small, large, or relevant is defined in different ways, yet these change scores are used to recommend or oppose treatments. They are also used to calculate the necessary number of patients for a study. This article offers a theoretical explanation behind the terms responsiveness, minimal important difference (MID), minimal important change (MIC), minimal relevant difference (MIREDIF), and threshold of clinical importance. It also gives instructions on how these and the optimal number of patients for a study are calculated. Responses to two domains of the Knee Injury and Osteoarthritis Outcome Score (KOOS), before and 1 year after reconstruction of the anterior cruciate ligament of 164 patients, are used to illustrate the calculations. This paper presents the most common methods used to calculate and interpret MID. Results vary substantially across domains, patient location on the scale, and health conditions. The optimal number of patients depends on the minimal relevant difference (MIREDIF), the standard error of the measure (SEM), the desired statistical power for the measurement, and the responsiveness of the measurement instrument (the PROM). There is often uncertainty surrounding the calculation and interpretation of responsiveness, MID, and MIREDIF, as these concepts are complex.When MID is used to evaluate research results, authors should specify how the MID was calculated, and its relevance for the study population. These measures should only be used after thorough consideration to justify healthcare decisions.