Health-related quality of life and symptom assessment in randomized controlled trials of patients with leukemia and myelodysplastic syndromes: What have we learned?

• Inclusion of patient-reported outcomes (PROs) in randomized controlled trials (RCTs) of patients with leukemia and myleodysplastic syndromes (MDS) is feasible.
• PROs in RCTs of patients with leukemia and MDS can provide unique information to facilitate clinical decision-making.
• Disease specific PRO measures are being developed for patients with leukemia and MDS.
• The number of RCTs with a PRO component in patients with leukemia and MDS is growing.

Health-related quality of life (HRQOL) and other patient-reported outcomes (PROs) are crucial for a comprehensive evaluation of treatment effectiveness. A systematic review of randomized controlled trials (RCTs) with a PRO endpoint conducted in patients with leukemia and myelodysplastic syndromes (MDS) was performed. Eligible studies were evaluated independently, according to a pre-defined coding scheme, by two reviewers. Thirteen RCTs, enrolling overall 3380 patients were identified. There were four RCTs involving acute myeloid leukemia patients (AML), one with acute lymphoid leukemia (ALL), five with chronic lymphocytic leukemia (CLL) and three with MDS. Six RCTs accurately documented PRO methodology assessment and were thus considered likely to robustly inform clinical decision-making. Of these, three RCTs dealt with AML, two with CLL, one with MDS. A growing number of RCTs in leukemia and MDS have included a PRO component in recent years. Inclusion of PROs in RCTs is feasible and can provide unique information to facilitate clinical decision-making.