State of the Art of Patient-reported Outcomes in Acromegaly or GH Deficiency: A Systematic Review and Meta-analysis

Merel van der Meulen; Amir H Zamanipoor Najafabadi; Leonie H A Broersen; Jan W Schoones; Alberto M Pereira; Wouter R van Furth; Kim M J A Claessen; Nienke R Biermasz

doi:10.1210/clinem/dgab874

State of the Art of Patient-reported Outcomes in Acromegaly or GH Deficiency: A Systematic Review and Meta-analysis

J Clin Endocrinol Metab. 2022 Apr 19;107(5):1225-1238. doi: 10.1210/clinem/dgab874.

Authors

Merel van der Meulen¹, Amir H Zamanipoor Najafabadi^{1

2}, Leonie H A Broersen¹, Jan W Schoones³, Alberto M Pereira¹, Wouter R van Furth², Kim M J A Claessen¹, Nienke R Biermasz¹

Affiliations

¹ Department of Medicine, Division of Endocrinology, Leiden University Medical Center, Leiden, the Netherlands.
² University Neurosurgical Center Holland, Leiden University Medical Center, Haaglanden Medical Centre and Haga Teaching Hospital, Leiden and The Hague, The Netherlands.
³ Directorate of Research Policy, Leiden University Medical Center, Leiden, The Netherlands.

Abstract

Context: Insight into the current landscape of patient-reported outcome (PRO) measures (PROM) and differences between PROs and conventional biochemical outcomes is pivotal for future implementation of PROs in research and clinical practice. Therefore, in studies among patients with acromegaly and growth hormone deficiency (GHD), we evaluated (1) used PROMs, (2) their validity, (3) quality of PRO reporting, (4) agreement between PROs and biochemical outcomes, and (5) determinants of discrepancies.

Evidence acquisition: We searched 8 electronic databases for prospective studies describing both PROs and biochemical outcomes in acromegaly and GHD patients. Quality of PRO reporting was assessed using the International Society for Quality of Life Research (ISOQOL) criteria. Logistic regression analysis was used to evaluate determinants.

Evidence synthesis: Ninety studies were included (acromegaly: n = 53; GHD: n = 37). Besides nonvalidated symptom lists (used in 37% of studies), 36 formal PROMs were used [predominantly Acromegaly Quality of Life Questionnaire in acromegaly (43%) and Quality of Life-Assessment of Growth Hormone Deficiency in Adults in GHD (43%)]. Reporting of PROs was poor, with a median of 37% to 47% of ISOQOL items being reported per study. Eighteen (34%) acromegaly studies and 12 (32%) GHD studies reported discrepancies between PROs and biochemical outcomes, most often improvement in biochemical outcomes without change in PROs.

Conclusions: Prospective studies among patients with acromegaly and GHD use a multitude of PROMs, often poorly reported. Since a substantial proportion of studies report discrepancies between PROs and biochemical outcomes, PROMs are pivotal in the evaluation of disease activity. Therefore, harmonization of PROs in clinical practice and research by development of core outcome sets is an important unmet need.

Keywords: acromegaly; growth hormone deficiency; patient-reported outcomes; prospective studies; quality of life; trials.

Publication types

Meta-Analysis
Systematic Review
Research Support, Non-U.S. Gov't

MeSH terms

Acromegaly* / diagnosis
Acromegaly* / therapy
Adult
Human Growth Hormone* / adverse effects
Humans
Patient Reported Outcome Measures
Prospective Studies
Quality of Life

Substances

Human Growth Hormone

Grants and funding

Chiasma, Inc