A pragmatic patient-reported outcome strategy for rare disease clinical trials: application of the EORTC item library to myelodysplastic syndromes, chronic myelomonocytic leukemia, and acute myeloid leukemia

• Main Authors: Jill A. Bell (Author), Aaron Galaznik (Author), Farrah Pompilus (Author), Sara Strzok (Author), Rafael Bejar (Author), Fatima Scipione (Author), Robert J. Fram (Author), Douglas V. Faller (Author), Stefan Cano (Author), Patrick Marquis (Author)
• Format: Book
• Published: SpringerOpen, 2019-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Abstract Background Novel, pragmatic, patient-centered strategies are needed to ensure fit-for-purpose patient-reported outcomes (PRO) instruments in clinical trial research for rare diseases such as myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), and chronic myelomonocytic leukemia (...