Health-Related Quality of Life as Assessed by the EQ-5D-5L Predicts Outcomes of Patients Treated with Azacitidine-A Prospective Cohort Study by the AGMT

Cancers (Basel). 2023 Feb 22;15(5):1388. doi: 10.3390/cancers15051388.


In this prospective study (NCT01595295), 272 patients treated with azacitidine completed 1456 EuroQol 5-Dimension (EQ-5D) questionnaires. Linear mixed-effect modelling was used to incorporate longitudinal data. When compared with a matched reference population, myeloid patients reported more pronounced restrictions in usual activities (+28%, p < 0.0001), anxiety/depression (+21%, p < 0.0001), selfcare (+18%, p < 0.0001) and mobility (+15%, p < 0.0001), as well as lower mean EQ-5D-5L indices (0.81 vs. 0.88, p < 0.0001), and lower self-rated health on the EuroQol Visual Analogue Scale (EQ-VAS) (64 vs. 72%, p < 0.0001). After multivariate-adjustment, (i) the EQ-5D-5L index assessed at azacitidine start the predicted time with clinical benefit (TCB) (9.6 vs. 6.6 months; p = 0.0258; HR = 1.43), time to next treatment (TTNT) (12.8 vs. 9.8 months; p = 0.0332; HR = 1.42) and overall survival (OS) (17.9 vs. 12.9 months; p = 0.0143; HR = 1.52); (ii) Level Sum Score (LSS) predicted azacitidine response (p = 0.0160; OR = 0.451) and the EQ-5D-5L index showed a trend (p = 0.0627; OR = 0.522); (iii) up to 1432 longitudinally assessed EQ-5D-5L response/clinical parameter pairs revealed significant associations of EQ-5D-5L response parameters with haemoglobin level, transfusion dependence and hematologic improvement. Significant increases of the likelihood ratios were observed after addition of LSS, EQ-VAS or EQ-5D-5L-index to the International Prognostic Scoring System (IPSS) or the revised IPSS (R-IPSS), indicating that they provide added value to these scores.

Keywords: Austrian Registry of Hypomethylating Agents; EuroQol 5-Dimension 5-Level questionnaire (EQ-5D-5L); acute myeloid leukaemia; azacitidine; chronic myelomonocytic leukaemia; health-related quality of life (HRQoL); mixed-effects linear models; myelodysplastic syndromes; patient-reported outcome (PRO); prognosis.

Grants and funding

The Austrian Group for Medical Tumor Therapy (AGMT) is the sponsor for the Austrian Registry of Hypomethylating Agents and received funding from Celgene/BMS, AbbVie, and Janssen Cilag. The AGMT is a non-for-profit organisation and an academic study group. The group performed administrative and legal management, as well as funding acquisition. No pharmaceutical company and no other funding source were involved in any way and had no role in the study design, data collection, data analysis, data interpretation, or writing of the report. No medical writer or editor was involved.