Raising the standards of patient-centered outcomes research in myelodysplastic syndromes: Clinical utility and validation of the subscales of the QUALMS from the MDS-RIGHT project

Fabio Efficace; Karin Koinig; Francesco Cottone; David Bowen; Moshe Mittelman; Kathrin Sommer; Saskia Langemeijer; Dominic Culligan; Kalman Filanovsky; Michael Storck; Alexandra Smith; Corine van Marrewijk; Martin Dugas; Igor Stojkov; Uwe Siebert; Theo de Witte; Reinhard Stauder

doi:10.1002/cam4.5487

Raising the standards of patient-centered outcomes research in myelodysplastic syndromes: Clinical utility and validation of the subscales of the QUALMS from the MDS-RIGHT project

Cancer Med. 2023 Mar;12(6):7529-7539. doi: 10.1002/cam4.5487. Epub 2022 Dec 19.

Authors

Fabio Efficace¹, Karin Koinig², Francesco Cottone¹, David Bowen³, Moshe Mittelman⁴, Kathrin Sommer¹, Saskia Langemeijer⁵, Dominic Culligan⁶, Kalman Filanovsky⁷, Michael Storck⁸, Alexandra Smith⁹, Corine van Marrewijk⁵, Martin Dugas¹⁰, Igor Stojkov¹¹, Uwe Siebert^{11

12

13}, Theo de Witte¹⁴, Reinhard Stauder²

Affiliations

¹ Italian Group for Adult Hematologic Diseases (GIMEMA), Data Center and Health Outcomes Research Unit, Rome, Italy.
² Department of Internal Medicine V (Hematology and Oncology), Medical University Innsbruck, Innsbruck, Austria.
³ St. James's Institute of Oncology, Leeds Teaching Hospitals, Leeds, UK.
⁴ Department of Hematology, Tel Aviv Sourasky (Ichilov) Medical Center, Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv-Yafo, Israel.
⁵ Department of Hematology, Radboud University Medical Center, Nijmegen, The Netherlands.
⁶ Department of Haematology, Aberdeen Royal Infirmary, Aberdeen, UK.
⁷ Department of Medicine, Kaplan Medical Center, Rehovot, Israel.
⁸ Institute of Medical Informatics, University of Münster, Münster, Germany.
⁹ Epidemiology and Cancer Statistics Group, Department of Health Sciences, University of York, York, UK.
¹⁰ Institute of Medical Informatics, Heidelberg University Hospital, Heidelberg, Germany.
¹¹ Department of Public Health, Health Services Research and Health Technology Assessment, Institute of Public Health, Medical Decision Making and Health Technology Assessment, UMIT - University for Health Sciences, Medical Informatics and Technology, Hall in Tirol, Austria.
¹² Departments of Epidemiology and Health Policy & Management, Center for Health Decision Science, Harvard Chan School of Public Health, Boston, Massachusetts, USA.
¹³ Institute for Technology Assessment and Department of Radiology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
¹⁴ Department of Tumor Immunology, Nijmegen Center for Molecular Life Sciences, Radboud University Medical Center, Nijmegen, The Netherlands.

Abstract

Background: Clinical decision-making for patients with myelodysplastic syndromes (MDS) is challenging, and both disease and treatment effects heavily impact health-related quality of life (HRQoL) of these patients. Therefore, disease-specific HRQoL measures can be critical to harness the patient voice in MDS research.

Methods: We report a prospective international validation study of the Quality of Life in Myelodysplasia Scale (QUALMS) with a main focus on providing information on the psychometric characteristics of its three subscales: physical burden (QUALMS-P), emotional burden (QUALMS-E), and benefit finding (QUALMS-BF). The analysis is based on patients enrolled from three European countries and Israel, participating to the MDS-RIGHT Project. The scale structure and psychometric properties of the QUALMS were assessed.

Results: Overall, 270 patients with a median age of 74 years were analyzed and the majority of them (60.3%) had a low MDS-Comorbidity Index score. Results of the confirmatory factor analysis supported the underlying scale structure of the QUALMS, which, in addition to a total score, includes three subscales: QUALMS-P, QUALMS-E, and the QUALMS-BF. The QUALMS-P exhibited the highest Cronbach's alpha coefficients. Discriminant validity analysis indicated good results with the QUALMS-P and QUALMS-E distinguishing between patients with different performance status, comorbidity, anemia, and transfusion dependency status. No floor and ceiling effects were observed. Responsiveness to change analysis supported the validity of the measure. Patients with a hemoglobin (Hb) level of <11 g/dL at study entry, who subsequently showed an improvement in their Hb levels, also reported a mean score change of 9 and 8 points (scales ranging between 0 and 100) in the expected direction of the QUALMS-E and QUALMS-P, respectively.

Conclusions: Our study provides additional validation data on the QUALMS from the international MDS-RIGHT Project. The use of this disease-specific HRQoL measure may contribute to raise quality standards of patient-centered outcomes research in MDS.

Keywords: myelodysplasia; myelodysplastic syndromes; patient-reported outcomes; quality of life; questionnaire; symptom burden.

Publication types

Research Support, Non-U.S. Gov't

MeSH terms

Aged
Anemia*
Humans
Myelodysplastic Syndromes* / drug therapy
Myelodysplastic Syndromes* / therapy
Patient Outcome Assessment
Prospective Studies
Quality of Life