Reshaping Leukemia Treatment and QOL Through Improved PROM Data Integration

Integration of patient-reported outcome measures (PROMs) needs to improve to provide a more holistic view of patients’ care experiences in the settings of hematologic malignancies, such as acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS), according to a recent review of clinical trials published in Hematology Reports.¹ These data are known to provide an unfiltered look at the impact of modern therapies, as patients can report on their health status directly.

This call to action is a sharp contrast to the use of typical measures of a treatment’s success, such as overall survival (OS) and disease-free survival, which can fail to adequately capture the daily reality of patient life during and after treatment.

The researchers conducted a comprehensive narrative and qualitative literature review of MEDLINE and the Cochrane Library for studies published between 2013 and 2025. Keywords searched were patient-reported outcomes, ALL, AML, CLL, CML, and MDS and medical subject headings searched were Disease-Free Survival, Leukemia, Lymphoid; Leukemia, Myeloid; Myelodysplastic Syndromes; Patient Reported Outcome Measures; Survival Rate; and Progression-Free Survival. Studies not published in English were excluded.

Giving Voice to the Patients

Fifteen randomized controlled trials that compared PROMs and traditional survival data were ultimately included. To synthesize how diverse patient lives could be affected by different treatment regimens, 9 studies used the EORTC QLQ-C30; 3 studies used the EQ-5D; 2 studies each used the SF-36, FACT-Leu, and EORTC QLQ-CLL16; and 1 used the PedsQL to measure PROMs.

The analysis was structured around symptoms, symptom burden, physical function, emotional health, social/role function, and global health status measurements that were assessed at baseline, while on treatment, and post treatment or during survivorship.

Overall, one of the more striking findings was that a patient’s self-reported quality of life (QOL) can be a powerful predictor of clinical success. For example, in older patients with AML, FACT-Leu scales, health-related QOL, and physical functioning scores were shown to be independent prognosticators of OS.

The authors explain that this may suggest that how a patient feels may be as important as actual markers of disease. “PROs provide a reasonable strategy for evaluating the treatment impact of complex diseases such as cancer,” they said, “where patients may suffer from chronic pain, nausea, fatigue, sexual dysfunction, and emotional effects such as anxiety for which objective physiologic measures may be limited in scope.”

The review also detailed how newer, targeted therapies are performing compared to traditional chemotherapy. In a phase 3 trial for acute lymphoblastic leukemia that compared inotuzumab ozogamicin (IO) vs investigator's choice of chemotherapy (NCT01564784),² there was superior clinical efficacy and a better QOL with the CD22-directed antibody-drug conjugate compared with standard chemotherapy. Similarly, in another phase 3 trial of patients with chronic lymphocytic leukemia (NCT02242942), use of venetoclax and obinutuzumab provided early relief of disease-related symptoms without impairing patient overall QOL in older “unfit” individuals. The most common symptom reported among the patients included in the 15 studies was fatigue, and this was evident across MDS and all types of leukemia.

Real-World Implications

The authors explain that there are practical implications to these findings. Integrating PROMs into routine care can help health care providers facilitate healthy dialogues with their patients that may serve to improve patient satisfaction and influence the overall quality of care. Personalized adjustments to therapy are also possible because these discussions can help clinicians distinguish disease symptoms from the adverse effects of the treatment.

The future of oncology may lie in a more compassionate, equitable approach, with the authors emphasizing, “The growing international recognition of the patient’s perspective in ensuring equitable, compassionate, and outcome-driven care cannot be overstated and may be best achieved through identifying ways in which practitioners may better utilize PROMs in clinical research and practice.”

They also acknowledge gaps that require further exploration. Outside references in psychiatric oncology and behavioral immunology could potentially back up their review’s findings regarding the link between depression, neurobiology, and cancer progression. Further, broader studies on financial toxicity could provide necessary context for the socioemotional scores seen in global health assessments.

References

1. Chan B, Bala E, Villalona S, Karp J, Leahy A, Lai C. A review of patient-reported outcomes and clinical outcomes in acute and chronic myeloid and lymphoid leukemias. Hematol Rep. 2026;18(1):15. doi:10.3390/hematolrep18010015
2. A study of inotuzumab ozogamicin versus investigator's choice of chemotherapy in patients with relapsed or refractory acute lymphoblastic leukemia. ClinicalTrials.gov. Updated January 9, 2019. Accessed March 4, 2026. https://clinicaltrials.gov/study/NCT01564784