Feasibility of Real-World Assessment of Patient-Reported Outcomes in Acute Myeloid Leukemia By Cytogenetic Risk and Treatment Status

Background

Prior research has demonstrated the benefit of gathering patient-reported-outcomes (PRO's) in oncology practice for clinical outcomes, health resource utilization and quality of life. The FDA guidance on Core Patient Reported Outcomes in Cancer Clinical Trials further highlights the importance of PRO's for capturing disease, treatment, and functional impacts of oncology therapy. For patients with Acute Myeloid Leukemia (AML), disease mortality and survey compliance pose particular challenges to gathering PRO data.

Methods

The study cohort was derived from users of Patient Reported Outcomes mobile platform (PROmpt®), an application for remote patient reporting and provider symptom management that is employed in routine care in 30+ integrated cancer treatment centers in the United States. Patients age 18+, with confirmed AML diagnosis from March, 2021 to May, 2022 were enrolled in the platform at treatment initiation and provided with weekly surveys. Weekly assessments included symptom burden (PRO-CTCAE), function impact (PROMIS 4A), and overall quality of life (EORTC QLQ C29, C30). Clinical, biomarker, and treatment data were obtained from electronic medical record structured data. Patients were stratified by cytogenetic risk (Poor vs Favorable/Intermediate/Unknown) and treatment type (Curative Therapy vs Maintenance Therapy). Baseline demographics and characteristics were assessed. Symptom burden, functional impact and quality-of-life impact were described longitudinally

Results

The overall cohort (n=65) had a median age of 69 (range 20-87). Among the persons with reported frailty status (n=42), there were 11 (26%) Fit, 21 (50%) (Intermediate Fit) and 10 (24%) Frail. Across all unique patient-weeks, the most frequent symptoms reported were fatigue (19%), decreased appetite (15%), general pain (14%) and nausea (13%). From Weeks 1 to 12, the average number of symptoms reported per patient per week ranged from 1.3 to 2.7, the averages collectively decreasing over time. Patients receiving Maintenance Therapy were directionally older and less fit than patients receiving Curative Therapy, mean age 72 vs 67 years and Intermediate-Fit/Frail 72 vs 50%, respectively. Over 12 weeks of treatment, PROMIS 4A Composite Function Scores were also consistently higher among patients receiving Maintenance Therapy compared to patients receiving Curative Therapy (Figure 1). In contrast, patients with Poor Cytogenetic Risk showed similar age and fitness status compared to patients with Favorable, Intermediate or Unknown Cytogenetic Risk. However, patients with Poor Cytogenetic Risk consistently reported higher PROMIS 4A Composite Function Scores over the 12 weeks of treatment compared to those with Favorable, Intermediate, or Unknown Cytogenetic Risk (Figure 2).

Conclusion

This study demonstrates the feasibility of gathering PRO's with real-world data among a cohort of patients with AML using a remote symptom monitoring platform, across baseline prognostic and therapeutic factors. Although results are limited due to small sample sizes, higher levels in overall function for patients on Maintenance Therapy and those with Poor Cytogenetic Risk underscore the value of routine follow-up, which should include PRO assessment throughout the entire therapeutic process.

Galaznik:Carevive Systems: Current Employment, Current equity holder in private company.