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STOCKHOLM — The prognosis of older adults with acute myeloid leukemia (AML) can be gleaned partly from what patients themselves report about their health-related quality of life (HRQoL) before treatment. Findings from the international EORTC AML21 phase 3 trial, which will be presented at the European Hematology Association (EHA) 2026 Congress, show that a pretreatment assessment of HRQoL can add valuable information to that derived from well-established clinical and genetic data on overall survival (OS).

“To my knowledge, this is one of the biggest studies with data on HRQoL in the AML setting to investigate this research question,” Fabio Efficace, PhD, who will present the results, told Medscape News Europe. Efficace is head of the Health Outcomes Research Unit and chair of the Working Party of Quality of Life at the Italian Group for Adult Hematologic Disease.

Assessing a patient’s pretreatment fitness is crucial to informing the therapeutic choice in AML, Efficace explained. Several options are now available, ranging from intensive chemotherapy to nonchemotherapy regimens or supportive care. “Although pretreatment fitness assessment is standard practice in AML and plays an important role in guiding treatment decisions, it typically does not include patient-reported outcomes (PROs),” he added, noting that the analysis evaluated the role of patient-reported QoL measures in defining the prognosis of older adults with the disease.

The Numbers Speak Loudly 

In the phase 3 EORTC AML21 trial, investigators randomly assigned 606 older adults with AML (33.7% aged ≥ 70 years) to receive 10-day decitabine or intensive chemotherapy. The EORTC QLQ-C30 and QLQ-ELD14 questionnaires were used to assess baseline (ie, pretreatment) HRQoL alongside other fitness indicators, with the researchers focusing specifically on physical functioning (PF), role functioning (RF), global health status/QoL, fatigue, and burden of illness. Prognostic factor analyses were performed for OS and complete remission (CR)/CR with incomplete hematological recovery probability.

The main multivariable analyses were adjusted for age, sex, white blood cell count at diagnosis, and ELN 2022 risk category, while sensitivity analyses were further adjusted for frailty indicators. PF, RF, and global health status/QoL remained independently associated with OS, with hazard ratios (HRs) of 0.949 (P = .0433), 0.962 (P = .0296), and 0.940 (P = .0096), respectively. This finding translates to as much as a 6% decrease in the instantaneous rate of death for every clinically significant increase (ie, 10 points on a 100-point scale) in the PF, RF, and global health status/QoL scales of the EORTC QLQ-C30. The authors concluded that these findings support assessing HRQoL in routine diagnostic practice and incorporating patient-reported data into the evaluation of pretreatment fitness.

Toward Patient-Centered Medicine 

“There’s no doubt about the prognostic value of the patient-reported HRQoL: several studies underlined the pivotal role of this measure in hematologic and solid tumours,” Massimo Di Maio, president of the Italian Society of Medical Oncology, told Medscape News Europe. He cited a 20-year-old lung cancer study on the same topic to emphasize that the importance of the patient voice is not new to the scientific community. Di Maio, head of oncology at the University of Turin, A.O.U. Città della Salute e della Scienza di Torino (Ospedale Molinette, Turin, Italy), is also first author of the European Society for Medical Oncology Clinical Practice Guideline on the role of PRO. According to Di Maio, who was not involved in the EORTC AML21 study, the results add new evidence confirming the pivotal role of PRO across the continuum of cancer care, including the initial prognostic evaluation.

Despite the strong scientific evidence supporting its prognostic value, however, the pretreatment assessment of HRQoL has not yet been incorporated into clinical practice. “I do not think time is the main barrier. Administering just a few PRO items may be enough to provide meaningful prognostic insights. Rather, the challenge lies in organization and mindset,” said Efficace, emphasizing patients’ willingness to report on their health status and noting that these assessments can be completed quickly. “One very positive message is that QoL considerations have recently been clearly recognized in the ELN recommendations for fitness assessment in AML,” Efficace said optimistically.

As for including patient-reported HRQoL among the endpoints of clinical trials, Di Maio said that it is not realistic to always use this measure as a primary endpoint, but that it could serve as a co-primary endpoint, particularly when progression-free survival is the primary endpoint. “Even if it is a secondary endpoint, it should still be given due weight in the interpretation of the study results and from a regulatory perspective,” he said.

Finally, one concern raised by some regulators is the subjective nature of patient-reported QoL. For Di Maio, the fact that it remains so strongly associated with prognosis confirms that what patients report corresponds to the clinical course of the disease and is closely tied to clinical outcomes. “Indirectly, these data also reflect this aspect and thus address at least in part this criticism,” he concluded. 

The study received support from Janssen Pharmaceuticals.

Efficace reported receiving fees for consultancy from AbbVie, Kyntra Bio, Novartis, and GSK and receiving research funding (to his institution) from Daiichi Sankyo and Otsuka. Di Maio reported receiving fees for consultancy or advisory board participation from AstraZeneca, Johnson & Johnson, Roche, Novartis, Merck, Amgen, GSK, Viatris, Ipsen, Astellas, and Resilience; and institutional grants for participation in clinical trials from Beigene, Exelixis, MSD, Pfizer, Roche, and Daiichi Sankyo. 

Cristina Ferrario is a molecular biologist and former researcher in molecular oncology at three institutes in Milan. She has a master’s degree in communication and health from the University of Milan and a master’s degree in cancer genetics from the University of Pavia. She has worked as a science journalist for more than 20 years.