Injectable Hypomethylating Agents for Management of Myelodysplastic Syndromes: Patients' Perspectives on Treatment

Background Until recently, patients with myelodysplastic syndromes (MDSs) could receive hypomethylating agents (HMAs) via intravenous (IV) or subcutaneous (SC) administration. An oral HMA was recently approved as an alternative to IV/SC administration. This study assessed the impact of IV/SC HMA on MDS patients, and their experience of, challenges with, and views about oral MDS treatment. Patients and methods We conducted an online cross-sectional survey among adult MDS patients (or caregivers as proxies) invited by 2 U.S. MDS patient advocacy groups. Patients were required to have received IV/SC HMA (i.e., azacitidine or decitabine) within 6 months of the survey. Results The survey was completed by 141 participants (120 patients, 21 caregiver proxies). Median patient age was 63.0 years, 53.9% were female, and 19.8%, 62.4%, and 17.7% had lower-, higher-, or unknown risk scores, respectively. HMA treatment received included SC azacitidine (37%), IV azacitidine (36%), and IV decitabine (27%). Among 89 IV HMA recipients, 74.2% and 69.7% reported treatment-related interference with their social and daily activities, respectively, and 66.3% reported pain related to treatment administration. Following an injection, SC HMA recipients reported pain (94.2%) and interference with daily (86.5%) and social (80.8%) activities. Among the 49.6% of patients who were working, 61.4% felt less productive due to treatment. Most (69.5%) MDS patients indicated they would prefer oral MDS treatment to IV/SC therapies. Conclusion Patients receiving IV/SC HMAs experienced pain/discomfort and interference with social and daily activities. The introduction of an oral HMA may alleviate some treatment challenges for MDS patients. Micro-Abstract This cross-sectional online survey assessed treatment challenges among myelodysplastic syndrome (MDS) patients (including caregivers as proxies) who recently received treatment with hypomethylating agents (HMAs) via infusion/injection. Patients experienced pain/discomfort, interference with daily and social activities, and productivity loss. Respondents indicated a preference for oral MDS treatment. The introduction of oral HMA therapy may alleviate some treatment challenges for MDS patients.