A systematic review of patient reported outcomes in phase II or III clinical trials of myelodysplastic syndromes and acute myeloid leukemia. (July 2018)

• Record Type: Journal Article
• Authors: Bryant, Ashley Leak; Drier, Sarah W.; Lee, Sejin; Bennett, Antonia V.
• Is Part Of: Leukemia research. Volume 70(2018)
• Page Start: 106
• Page End: 116
• Publication Date: 2018-07
• Subjects: patient reported outcomes -- quality of life -- acute myeloid leukemia -- myelodysplastic syndromes -- clinical trials -- phase 2/3; Leukemia -- Periodicals; Leukemia -- Periodicals; Leucémie -- Périodiques; Leukemia; Periodicals; Electronic journals; Electronic journals; 616.9941905
• DOI: 10.1016/j.leukres.2018.06.006
• Languages: English
• ISSNs: 0145-2126

Abstract

Highlights: Fatigue is a persistent and distressing symptom in adults with acute leukemia. Few MDS and AML drug trials have published patient reported outcome (PRO) data. Many of these trials had high rates of missing PRO data. PROs capture changes over time in health related quality of life in MDS/AML trials. Guidance documents describe how to implement PRO assessment in clinical trials. Abstract: The purpose of this systematic literature review was to identify clinical trials of MDS and AML that included patient-reported outcome (PRO) instruments, and to summarize the symptom and other health related quality of life (HRQOL) concepts most frequently assessed and the PRO instruments that were used. Sixteen manuscripts describing 14 distinct trials met all criteria (i.e., phase 2 or 3 clinical trial for MDS or AML which included PRO assessment) and were published between 1996-2017. In trials evaluating anemia, PRO scores showed significant improvement in relevant domains (e.g. fatigue, function) among patients identified as responders. In trials evaluating the impact of anti-cancer therapies, improvements the baseline to end of treatment were observed in physical functioning and HRQOL, however the rates of missing data in many of the trials was high or unreported. PRO instruments have the ability to capture changes over time in patients' function and well-being, and PRO instruments and guidance documents are available to support the assessment of HRQOL in AML/MDS clinical trials.