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

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

Health Sciences

Research output: Contribution to journal › Article › peer-review

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
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.

Original language	English
Number of pages	11
Journal	Cancer Medicine
Early online date	19 Dec 2022
DOIs	https://doi.org/10.1002/cam4.5487
Publication status	E-pub ahead of print - 19 Dec 2022

Bibliographical note

© 2022 The Authors.

Presented in part at the 63rd Annual Meeting of the American Society of Hematology (ASH). December 11–14, 2021. Atlanta (GA), USA.

Keywords

myelodysplasia
myelodysplastic syndromes
patient-reported outcomes
quality of life
questionnaire
symptom burden

Access to Document

10.1002/cam4.5487Licence: CC BY

Cancer Medicine - 2022 - Efficace - Raising the standards of patient‐centered outcomes research in myelodysplastic
© 2022 The Authors.
Final published version, 550 KBLicence: CC BY

Cite this

Efficace, F., Koinig, K., Cottone, F., Bowen, D., Mittelman, M., Sommer, K., Langemeijer, S., Culligan, D., Filanovsky, K., Storck, M., Smith, A., van Marrewijk, C., Dugas, M., Stojkov, I., Siebert, U., de Witte, T., & Stauder, R. (2022). 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 Medicine. Advance online publication. https://doi.org/10.1002/cam4.5487

@article{3a108282acd44688bbdc4e4d5ecc3a56,

title = "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",

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 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",

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

note = "{\textcopyright} 2022 The Authors. Presented in part at the 63rd Annual Meeting of the American Society of Hematology (ASH). December 11–14, 2021. Atlanta (GA), USA.",

year = "2022",

month = dec,

day = "19",

doi = "10.1002/cam4.5487",

language = "English",

journal = "Cancer Medicine",

issn = "2045-7634",

publisher = "Wiley-Blackwell",

}

Efficace, F, Koinig, K, Cottone, F, Bowen, D, Mittelman, M, Sommer, K, Langemeijer, S, Culligan, D, Filanovsky, K, Storck, M, Smith, A, van Marrewijk, C, Dugas, M, Stojkov, I, Siebert, U, de Witte, T & Stauder, R 2022, '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 Medicine. https://doi.org/10.1002/cam4.5487

TY - JOUR

T1 - Raising the standards of patient-centered outcomes research in myelodysplastic syndromes

T2 - Clinical utility and validation of the subscales of the QUALMS from the MDS-RIGHT project

AU - Efficace, Fabio

AU - Koinig, Karin

AU - Cottone, Francesco

AU - Bowen, David

AU - Mittelman, Moshe

AU - Sommer, Kathrin

AU - Langemeijer, Saskia

AU - Culligan, Dominic

AU - Filanovsky, Kalman

AU - Storck, Michael

AU - Smith, Alexandra

AU - van Marrewijk, Corine

AU - Dugas, Martin

AU - Stojkov, Igor

AU - Siebert, Uwe

AU - de Witte, Theo

AU - Stauder, Reinhard

PY - 2022/12/19

Y1 - 2022/12/19

N2 - 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 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.

AB - 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 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.

KW - myelodysplasia

KW - myelodysplastic syndromes

KW - patient-reported outcomes

KW - quality of life

KW - questionnaire

KW - symptom burden

U2 - 10.1002/cam4.5487

DO - 10.1002/cam4.5487

M3 - Article

SN - 2045-7634

JO - Cancer Medicine

JF - Cancer Medicine

ER -