title: 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.
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.
authors.affiliation: Tisch Cancer Institute, Mount Sinai School of Medicine, New York, NY, USA. john.mascarenhas@mssm.edu
authors.forename: John
authors.initials: J
authors.lastname: Mascarenhas
authors.forename: Mark L
authors.initials: ML
authors.lastname: Heaney
authors.forename: Vesna
authors.initials: V
authors.lastname: Najfeld
authors.forename: Elizabeth
authors.initials: E
authors.lastname: Hexner
authors.forename: Omar
authors.initials: O
authors.lastname: Abdel-Wahab
authors.forename: Raajit
authors.initials: R
authors.lastname: Rampal
authors.forename: Farhad
authors.initials: F
authors.lastname: Ravandi
authors.forename: Bruce
authors.initials: B
authors.lastname: Petersen
authors.forename: Gail
authors.initials: G
authors.lastname: Roboz
authors.forename: Eric
authors.initials: E
authors.lastname: Feldman
authors.forename: Nikolai
authors.initials: N
authors.lastname: Podoltsev
authors.forename: Dan
authors.initials: D
authors.lastname: Douer
authors.forename: Ross
authors.initials: R
authors.lastname: Levine
authors.forename: Martin
authors.initials: M
authors.lastname: Tallman
authors.forename: Ronald
authors.initials: R
authors.lastname: Hoffman
mesh_terms: D001752:Blast Crisis / Q000175:diagnosis* / Q000473:pathology / Q000628:therapy; D001773:Blood Cells / Q000187:drug effects / Q000473:pathology; D001853:Bone Marrow / Q000187:drug effects / Q000473:pathology; D002986:Clinical Trials as Topic; D005819:Genetic Markers; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007621:Karyotyping; D015470:Leukemia, Myeloid, Acute / Q000175:diagnosis* / Q000473:pathology / Q000628:therapy; D019653:Myeloablative Agonists / Q000627:therapeutic use*; D009196:Myeloproliferative Disorders / Q000175:diagnosis* / Q000473:pathology / Q000628:therapy; D013154:Spleen / Q000187:drug effects / Q000473:pathology; D019172:Transplantation Conditioning; D016896:Treatment Outcome
chemical_list: D005819:Genetic Markers; D019653:Myeloablative Agonists