Patient‐reported outcomes in acute leukemia: a roadmap for future research

To the Editor: The study presented by Leunis and colleagues (1) provides one of the very few evidence available on the impact of therapy on quality of life (QoL) in patients with acute myeloid leukemia (AML). The authors assessed QoL in a cohort of 92 AML survivors enrolled in previous clinical trials at the Erasmus University and compared it with that of the general population. Comparison was made using the EORTC QLQ-C30 and analyses were adjusted by age and gender, thus providing confidence in the final results. Of note, differences between AML survivors and general population in fatigue and dyspnea severity were clinically meaningful. Likewise, other QoL aspects of AML survivors, such as physical and social functioning, were also clinically meaningful worse than their peers in the general population. This is an important finding as it should further raise physicians’ awareness that, although being ‘successfully’ treated for their disease, these patients still have major consequences from their treatments. This finding underscores the need to keep improving follow-up care programs for this patient population. In their study, allogeneic hematopoietic stem cell transplantation was found to be associated with poorer QoL; however, given the relatively small sample size, analyses to disentangle the real influence of specific treatments received by these patients on QoL outcomes were limited. In any case, this data seem to be substantiated by some previous evidence. As appropriately noted by the authors, given the progress made in patients with AML, including the outstanding achievements in some AML subtypes (i.e., acute promyelocytic leukemia) (2), time is now mature to move toward a more patient-centered care approach in this population. It has to be acknowledged, in fact, that assessment of QoL in patients with leukemia has been a neglected issue for many years and very little evidence is available on this patient population (3). One of the most solid data in this area of research, being replicated in various studies, seems to indicate that better long-term QoL outcomes can be expected for AML patients treated with conventional chemotherapy, compared to those who received bone marrow transplantation (allogeneic or autologous) (4, 5). This is broadly in line with findings reported by Leunis and colleagues in their article. Including a QoL assessment in a clinical study is, in essence, the only way of obtaining evidence-based data on the effect of a treatment from the patient’s view. QoL assessment in patients with cancer provides data that are unique. This is now well documented by several studies in patients with solid tumors showing the independent prognostic value of QoL parameters for survival outcomes (6, 7). These data seem to emerge also in patients with AML. Deschler and colleagues recently showed that pretreatment patients’ self-report of fatigue severity is an independent predictor of survival in a series of 195 elderly patients diagnosed with AML and myelodysplastic syndromes (MDS) (8). This evidence, although preliminary, provides an additional rationale for the importance of collecting QoL data in patients with AML. We thus encourage the scientific community to start including QoL assessment as an outcome measure in future AML clinical trials to better understand pro and cons of different treatment strategies. It is now recognized that a comprehensive evaluation of treatment effectiveness should include a patient-reported outcome (PRO) assessment to capture patients’ perceptions of symptoms, functioning, and general well-being (9). Also, PROs are now well accepted endpoints by major regulatory stakeholders (10). Several international guidelines are now available to help investigators in the planning, conducting, and reporting QoL in clinical trials, such as those recently published by the CONSORT Group (11), and these will likely to help implementation of QoL in future studies. There are excellent examples in patients with solid tumors of how inclusion of QoL in a clinical trial setting has greatly contributed to changes in clinical practice (12), and it is hoped that robust QoL data will be soon generated from AML trials to better inform clinical decision-making.