Advancing Effective Clinical Trial Designs for Myelofibrosis

Heidi E. Kosiorek, Amylou C. Dueck

Research output: Contribution to journalReview articlepeer-review


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.

Original languageEnglish (US)
Pages (from-to)431-444
Number of pages14
JournalHematology/Oncology Clinics of North America
Issue number2
StatePublished - Apr 2021


  • Adaptive designs
  • Biostatistics
  • Clinical trials
  • Endpoints
  • Myelofibrosis
  • Phase I design
  • Quality of life
  • Symptoms

ASJC Scopus subject areas

  • Hematology
  • Oncology


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