Patient-Reported Outcomes in Myelodysplastic Syndromes and MDS/MPN Overlap Syndromes: Stepping Onto the Stage with Changing Times

Abstract

Quality of life (QOL) and symptom burden are important measures captured by patient-reported outcomes (PROs). Myelodysplastic and myelodysplastic/myeloproliferative (MDS/MPN) neoplasm overlap syndromes are notable for significant morbidity and mortality, including a wide spectrum of physical and psychosocial effects. Thus, the development and application of PROs can provide meaningful information to facilitate communication and assist in follow up care. Disease-specific measures can more accurately reflect the full breadth of functional restrictions and symptoms. While traditional endpoints include remission, relapse, and survival rates, adoption of PROs in myeloid neoplasm clinical trials can facilitate drug approval. Integration of PROs in myeloid neoplasms is an important measure to capture QOL and symptoms, which can improve disease management.

Keywords: HRQOL; MDS; MDS/MPN; Myelodysplastic syndromes; Patient-reported outcomes; Pro.