Most CVD outcome measurements reported by patients are inadequate

Only a minority of the tools used in clinical and research settings to assess patient-reported outcome measurements (PROMs) in CVD care adhere to quality standards set by the U.S. Food and Drug Administration or to consensus-based metrics, according to a review of 50 such tools.

Health status PROMs, which assess symptom burden, functional status, and health-related quality of life, give the patient a voice in their treatment, provide tools to measure treatment effects in research, and have proven to be more sensitive measures of health changes over time than in some cases a doctor’s assessment.

Harriette Van Spall, MD, MPH (McMaster University, Hamilton, Canada), senior author of the published review Annals of Internal Medicinesaid that while traditional instruments such as the NYHA grade and the Canadian Cardiovascular Society (CCS) scale for grading angina are the ones that many physicians are most familiar with, many disease-specific and generic PROMs are available, but are commonly used in CVD treatment are still underused.

Her group evaluated all existing health status PROMs that have been developed and validated in CVD populations.

“We found that few PROMs have reported validation of all FDA-recommended psychometric properties,” Van Spall told TCTMD. “Few had characteristics assessed as sufficient by the COSMIN standards, which are standards that assess the validation process and reporting of PROMs.” The review also concluded that some PROMs had minimally important Differences (MIDs) were identified, meaning that there is no known improvement threshold that is clinically relevant. Therefore, results derived from these PROMs can be difficult to interpret.

“Not all PROMs are the same. When I choose PROMs for my clinical trials, I often choose them based on what patients prefer,” she added. “I can now present you with a smaller group of PROMs based on how they were validated.”

A card for clinicians and researchers

Van Spall and colleagues, led by Derek S. Chew, MD, MSc (Libin Cardiovascular Institute, University of Calgary, Canada, and Duke Clinical Research Institute, Durham, NC), evaluated 50 health status PROMs described in 83 studies . Of these, 45 were disease-specific and five were generic. The disease-specific tools were developed for a range of cardiovascular diseases, including arrhythmia, CAD, heart failure and general cardiovascular diseases. The average number of items was 25 for each item and the average time it took patients to complete them on paper or tablet was 10 minutes.

Only 16% of PROMs reported validation of all FDA-recommended psychometric properties, including invariance across sex, sex, and culture. Also only 11 out of 50 had available MID data or included a description of how to interpret changes in scores over time. With reference to According to COSMIN standards, only two out of 50 PROMs rated all psychometric properties as qualitatively sufficient. In 64% of all PROMs, less than half of the psychometric properties were rated as sufficient.

None of the five generic PROMs and only one out of six of the disease-specific PROMs met all FDA requirements for product approval. Similarly, none of the generic PROMs and only two of the disease-specific PROMs rated all nine COSMIN-defined properties as sufficient.

The PROMs that most completely met FDA and COSMIN criteria: Atrial Fibrillation Effect on QualiTy-of-life (AFEQT), Kansas City Cardiomyopathy Questionnaire (KCCQ), MacNew Heart Disease Health-related Quality of Life Questionnaire, Minnesota Living with Heart Failure Questionnaire ( MLHFQ), Patient-Reported Outcomes Measurement Information System-Plus-Heart Failure (PROMIS-Plus-HF), Orthostatic Hypotension Questionnaire (OHQ) and HeartQOL.

The researchers note that while generic PROMs such as EuroQol-5D (EQ-5D) are widely used in clinical CVD trials, “there appears to be a discrepancy between the frequency of their use and the extent to which they meet FDA Recommendations meet or meet COSMIN criteria for good measurement performance in CVD populations.” Problems found with the generic PROMs included insufficient content validity and the fact that none of them were developed with patient input.

“Although generic PROMs quantify an individual’s overall well-being, they do not include symptoms or functional limitations specific to CVD and are less sensitive to clinical changes compared to disease-specific instruments,” the researchers write. “Notwithstanding these limitations, generic PROMs

remain useful as they can be broadly applied to different disease states to facilitate comparison.” They may also be useful to assess the health economics of drugs or devices, they add.

“The take-home message is that we now have a comprehensive map of the characteristics and performance of each of these health status PROMs, so researchers and clinicians have a guide that can help implement the right PROM for the right condition,” Van Spall said. “Our work underscores the need for careful adherence to recognized and standardized methodological criteria for PROM development and validation also clear reporting of the psychometric properties of PROMs.”