Unvalidated patient-reported outcome measures limit acromegaly, GH studies

Jennifer E. Engen

January 05, 2022

2 min read

Chiasma funded this study but had no role in the study design, data collection, analysis or writing. The authors report no relevant financial disclosures.

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One-third of acromegaly and growth hormone deficiency studies report discrepancies between patient-reported outcomes and biochemical outcomes, with many using unvalidated and poorly reported outcome measures, data from a meta-analysis show.

In both acromegaly and GH deficiency, biochemical outcomes do not always correlate with health-related quality of life-reported symptoms, Merel van der Meulen, BSc, a doctoral student in the division of endocrinology at Leiden University Medical Center, the Netherlands, and colleagues wrote in the study background. To capture the patients’ perspective by measuring symptoms and health-related quality of life, researchers have advocated for the use of patient-reported outcome measures in clinical trials and practice, in addition to biochemical evaluation.

Patient-reported vs. biochemical outcomes in acromegaly and GH deficiency studies

Roughly one-third of acromegaly and GH deficiency studies have discrepancies between patient-reported outcomes and biochemical outcomes. Data were derived from van der Meulen M, et al. J Clin Endocrinol Metab. 2021;doi:10.1210clinem/dgab874.

“Patient-reported outcome measures can cover a wide range of unidimensional or multidimensional concepts of health-related quality of life, ranging from specific symptoms and bodily limitations to restrictions in social participation,” van der Meulen and colleagues wrote. “Moreover, incorporation of patient-reported outcome measures in trials and clinical practice safeguards comprehensive patient-centered outcome measurement. However, no clear criteria nor consensus exist for the use of patient-reported outcomes in clinical trials in patients with acromegaly or GH deficiency. As a result, a great variety of validated and unvalidated generic and disease-specific patient-reported outcomes are currently being used in patients with pituitary disease, limiting comparability between trials.”

Researchers analyzed data from 90 prospective studies describing patient-reported outcomes and biochemical outcomes in patients with acromegaly (n = 53 studies) and GH deficiency (n = 37 studies), with the quality of patient-reported outcomes reporting assessed using the International Society for Quality of Life (ISOQOL) research criteria. The primary outcome was the pooled mean difference before vs. after study for the total scores of the Acromegaly Quality of Life Questionnaire (AcroQoL) and Patient Assessed Acromegaly Symptom Questionnaire (PASQ) for studies in acromegaly, and the Quality of Life Assessment of Growth Hormone Deficiency in Adults (QoL-AGDHA) for studies in GH deficiency. Researchers used logistic regression analysis to evaluate determinants.

The findings were published in The Journal of Clinical Endocrinology & Metabolism.

Researchers found that nonvalidated symptom lists were used in 37% of studies, and 36 formal patient-reported outcome measures were used, predominantly the AcroQoL in acromegaly (43%) and QoL-AGHDA in GH deficiency (43%). Reporting of patient-reported outcomes was poor, with a median of 37% to 47% of ISOQOL items reported per study. Researchers also found that 34% of acromegaly studies and 32% of GH deficiency studies reported discrepancies between patient-reported outcomes and biochemical outcomes, most often for improvement in biochemical outcomes without change in patient-reported outcomes.

“Many different, often unvalidated, patient-reported outcome measures are used, limiting comparability between trials,” the researchers wrote. “Reporting of these patient-reported outcomes is not according to current standards, hampering proper interpretation, comparability and implementation of results in clinical practice. Interestingly, we found that in a third of studies, discrepancies exist between patient-reported outcomes and biochemical outcomes. In the studies with discrepant results, biochemical outcomes generally improved with treatment, while patients’ symptoms and health-related quality of life remained stable across most domains.”

The researchers wrote that the findings emphasize the need to standardize outcomes to improve the use and reporting of patient-reported outcomes next to conventional clinical outcomes in clinical trials with patients with acromegaly or GH deficiency.

“This may improve interpretability of patient-reported outcome results and consequently facilitate implementation of these outcomes in clinical practice,” the researchers wrote.


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