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 (Post-MPN AML Consortium)

摘要

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.