The prognostic value of health-related quality of life for early molecular response in patients with chronic myeloid leukemia: analysis of the GIMEMA-SUSTRENIM trial

Fabio Efficace; Laura Cannella; Isabella Capodanno; Rosaria Sancetta; Veronica Porcu; Monia Lunghi; Annalisa Biagi; Fausto Castagnetti; Giovanni Caocci; Thomas Baldi; Roberto Marasca; Massimiliano Bonifacio; Antonella Gozzini; Bruno Martino; Federica Sora; Peter E Westerweel; Paola Fazi; Marco Vignetti; Fabrizio Pane; Massimo Breccia

doi:10.3389/fonc.2025.1645217

The prognostic value of health-related quality of life for early molecular response in patients with chronic myeloid leukemia: analysis of the GIMEMA-SUSTRENIM trial

Front Oncol. 2025 Aug 27:15:1645217. doi: 10.3389/fonc.2025.1645217. eCollection 2025.

Authors

Fabio Efficace¹, Laura Cannella¹, Isabella Capodanno², Rosaria Sancetta³, Veronica Porcu⁴, Monia Lunghi⁵, Annalisa Biagi⁶, Fausto Castagnetti⁷, Giovanni Caocci⁸, Thomas Baldi¹, Roberto Marasca⁹, Massimiliano Bonifacio^{10

11}, Antonella Gozzini¹², Bruno Martino¹³, Federica Sora¹⁴, Peter E Westerweel¹⁵, Paola Fazi¹, Marco Vignetti¹, Fabrizio Pane¹⁶, Massimo Breccia¹⁷

Affiliations

¹ Italian Group for Adult Hematologic Diseases (GIMEMA), Data Center and Health Outcomes Research Unit, Rome, Italy.
² Hematology Unit, Azienda Unità Sanitaria Locale-IRCCS, Reggio Emilia, Italy.
³ Hematology Unit, Dell'Angelo Hospital, Venezia-Mestre, Italy.
⁴ Hematology Unit, S. Eugenio Hospital, ASL Roma 2, Tor Vergata University, Rome, Italy.
⁵ Division of Hematology, Department of Translation Medicine, University of Eastern Piedmont, Novara, Italy.
⁶ Hematology Unit, Santa Maria Goretti Hospital, Latina, Italy.
⁷ Institute of Hematology "Seràgnoli" IRCCS Azienda Ospedaliero-Universitaria di Bologna, Department of Medical and Surgical Sciences University of Bologna, Bologna, Italy.
⁸ Hematology Unit, Businco Hospital, Department of Medical Sciences and Public Health, University of Cagliari, Cagliari, Italy.
⁹ Section of Hematology, Department of Medical and Surgical Sciences, University of Modena and Reggio Emilia, Azienda Ospedaliero-Universitaria di Modena, Modena, Italy.
¹⁰ Department of Medicine, Azienda Ospedaliera Universitaria Integrata di Verona, Verona, Italy.
¹¹ Department of Engineering for Innovation Biomedicine, Section of Innovation Biomedicine, Hematology Area, University of Verona, Verona, Italy.
¹² Hematology Unit, AOU Careggi, University of Florence, Florence, Italy.
¹³ Division of Hematology, Grande Ospedale Metropolitano 'Bianchi Melacrino Morelli', Reggio Calabria, Italy.
¹⁴ Dipartimento di Diagnostica per Immagini, Radioterapia Oncologica ed Ematologia, Fondazione Policlinico Universitario A. Gemelli IRCCS, Università Cattolica Sacro Cuore, Rome, Italy.
¹⁵ Department of Internal Medicine, Albert Schweitzer Hospital, Dordrecht, Netherlands.
¹⁶ Hematology Section, Department of Clinical Medicine and Surgery, University of Naples "Federico II", Naples, Italy.
¹⁷ Hematology, Department of Translational and Precision Medicine, Azienda Ospedaliera Policlinico Umberto I, Sapienza University of Rome, Rome, Italy.

Abstract

We aimed to explore the prognostic value of patient-reported health-related quality of life (HRQoL) data for the achievement of early molecular response (EMR) at 3 months in patients with chronic phase chronic myeloid leukemia (CP-CML). We analyzed HRQoL baseline data of 436 newly diagnosed patients with CML patients enrolled in the GIMEMA Sustrenim trial. HRQoL was assessed by the EORTC QLQ-30 and the QLQ-CML24 questionnaires. In the multivariate analysis, the following factors were found to be independently associated with achievement of EMR: Sokal risk (low vs intermediate risk p=0.046 and low vs high risk p<0.001), nilotinib treatment (p<0.001) and higher patient-reported role functioning (EORTC QLQ-C30) (p<0.001). Current findings suggest the importance of assessing HRQoL at diagnostic workup of patients with CML as it may provide valuable prognostic information.

Keywords: chronic myeloid leukemia (CML); early molecular response (EMR); functioning; patient reported outcome (PRO); quality of life.