Proposed criteria for response assessment in patients treated in clinical trials for myeloproliferative neoplasms in blast phase (MPN-BP): formal recommendations from the post-myeloproliferative neoplasm acute myeloid leukemia consortium

Leuk Res. 2012 Dec;36(12):1500-4. doi: 10.1016/j.leukres.2012.08.013. Epub 2012 Aug 28.

Abstract

Leukemic transformation (LT) of a myeloproliferative neoplasm (MPN) is associated with a dismal prognosis and no medical therapies have shown a survival improvement in patients with MPN in blast phase (MPN-BP). Effective therapies for the treatment of MPN-BP are a serious unmet need. Consensus response criteria do not exist for the treatment of patients with MPN-BP and this is necessary for the uniformed reporting of treatment response in clinical trials. We have identified relevant MPN and MPN-BP features in order to define treatment response categories that reflect hematological, clinical, pathological, cytogenetic and molecular changes after therapeutic intervention. We plan to validate these proposed response criteria within multi-centered clinical trials.

Publication types

  • Practice Guideline

MeSH terms

  • Blast Crisis / diagnosis*
  • Blast Crisis / pathology
  • Blast Crisis / therapy
  • Blood Cells / drug effects
  • Blood Cells / pathology
  • Bone Marrow / drug effects
  • Bone Marrow / pathology
  • Clinical Trials as Topic
  • Genetic Markers
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Karyotyping
  • Leukemia, Myeloid, Acute / diagnosis*
  • Leukemia, Myeloid, Acute / pathology
  • Leukemia, Myeloid, Acute / therapy
  • Myeloablative Agonists / therapeutic use*
  • Myeloproliferative Disorders / diagnosis*
  • Myeloproliferative Disorders / pathology
  • Myeloproliferative Disorders / therapy
  • Spleen / drug effects
  • Spleen / pathology
  • Transplantation Conditioning
  • Treatment Outcome

Substances

  • Genetic Markers
  • Myeloablative Agonists