Validation and reference values of the EORTC QLQ-CML24 questionnaire to assess health-related quality of life in patients with chronic myeloid leukemia

Abstract

Health-related quality of life (HRQOL) assessment is important to facilitate decisions in the current treatment landscape of chronic myeloid leukemia (CML). Therefore, the availability of a validated HRQOL questionnaire, specifically developed for CML patients treated with tyrosine kinase inhibitors (TKIs), may enhance quality of research in this area. We performed an international study including 782 CML patients to assess the validity of the EORTC QLQ-CML 24 questionnaire, and to generate HRQOL reference values to facilitate interpretation of results in future studies. Internal consistency, assessed with Cronbach’s alpha coefficients, ranged from 0.66 to 0.83. In the confirmatory factor analysis, all standardized factor loadings exceeded the threshold of 0.40 (range 0.49–0.97), confirming the hypothesized scale structure. Reference values stratified by age and sex were also generated. Our findings support the use of the EORTC QLQ-CML 24, in conjunction with the EORTC QLQ-C30, as a valuable measure to assess HRQOL in CML patients.

Keywords:

• Quality of life
• tyrosine kinase inhibitors
• symptoms
• chronic myeloid leukemia
• patient-reported outcomes

Acknowledgements

The authors thank Kathrin Sommer from the GIMEMA Data Center, Rome, Italy. The authors acknowledge the essential contribution of all patients who participated in this study.

Disclosure statement

AI: Speaker honoraria: Novartis, Pfizer, and Incyte, outside the submitted work. CE: advisory board Novartis e Pfizer, outside the submitted work. EA: consultancy for BMS, Incyte, Novartis, Pfizer, outside the submitted work. FCa: Consultancy: Novartis, Incyte, Pfizer, BMS; Honoraria: Novartis, Incyte, Pfizer, BMS, outside the submitted work. FE: Consultancy: BMS, Amgen, Orsenix, Takeda and research grant (to his Institution) from Amgen, outside the submitted work. FE, also declares a grant by the EORTC Quality of Life Group (to his Institution) for the conduct of this study. FSt: Honoraria: BMS, Incyte, Novartis, Pfizer, outside the submitted work. GR: speaker honoraria: Novartis, Pfizer, Incyte. Research grant (to his institution): Novartis, Pfizer, Incyte, outside the submitted work. MBa: Honararia: Novartis, Incyte, Takeda, Fusion Pharma; consultancy: Novartis, Incyte, Takeda, Fusion Pharma, outside the submitted work. MBo: Novartis (research support and honoraria), Pfizer (honoraria), Incyte (honoraria), Amgen (honoraria), outside the submitted work. MBr: Honoraria by Novartis, Pfizer, Incyte, Celgene, outside the submitted work. MV: personal fees from Jazz Healthcare Italy SRL, Amgen, Millennium Pharmaceuticals Inc., Celgene, Janssen, Novartis and Incyte, outside the submitted work. SS: Honoraria: BMS, Incyte, Novartis, Pfizer; Research support: BMS, Incyte, Novartis, outside the submitted work. FA: Honoraria by Incyte, Novartis, outside the submitted work; VK: honorarium for advisory board for Novartis and Pfizer, outside the submitted work.

Additional information

Funding

This study was partly supported by a research grant from the EORTC Quality of Life Group, grant number 004/2015.