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^1*Laura Cannella¹Isabella Capodanno²Rosaria Sancetta³Veronica Porcu⁴Monia Lunghi⁵Annalisa Biagi⁶Fausto Castagnetti⁷Giovanni Caocci⁸Thomas Baldi¹Roberto Marasca⁹Massimiliano Bonifacio^10,11Antonella Gozzini¹²Bruno Martino¹³Federica Sora¹⁴Peter E. Westerweel¹⁵Paola Fazi¹Marco Vignetti¹Fabrizio Pane¹⁶Massimo Breccia¹⁷

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

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.