Advancing Effective Clinical Trial Designs for Myelofibrosis

Overview

Journal Hematol Oncol Clin North Am

Specialties Hematology
Oncology

Date 2021 Mar 1

PMID 33641878

Authors

Heidi E Kosiorek

Amylou C Dueck

Affiliations

Soon will be listed here.

Abstract

Design features of phase I, II, and III clinical trials of pharmaceutical interventions in myelofibrosis (MF) are discussed. Model-assisted and model-based designs for phase I trials are useful for maximizing therapeutic benefit and include novel approaches to dose escalation. Trials in MF have shifted to accommodate new challenges following approval of JAK inhibitor therapies. Standardized response criteria exist; however, alternative measures of response when evaluating newer agents may be needed. Noninferiority and other adaptive designs can be used to incorporate design changes over time. Patient-reported outcomes, including quality-of-life and symptom assessment, should be included as outcome measures.

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