The natural history and treatment outcome of blast phase BCR-ABL- myeloproliferative neoplasms.
We analyzed the outcomes of 74 patients diagnosed with BCR-ABL(-) myeloproliferative neoplasms in blast phase receiving induction chemotherapy (55%), low-intensity therapy (16%), stem cell transplantation (SCT; 3%), or supportive care (26%). Median survival from the date of blastic transformation was 5 months. Patients receiving supportive therapy had a median survival of 6 weeks. Complete remission with or without blood recovery was achieved in 46% of patients receiving induction chemotherapy, but remissions were not durable with a median progression-free survival of only 5 months. Eight patients received SCT either as first therapy or after responding to antileukemia therapy. These patients had a markedly superior survival, with 73% alive at a median follow-up of 31 months. JAK2V617F kinetics were assessed in 16 patients: 0 of 4 negative patients became positive at transformation, and among 12 positive patients, 1 had an increase in JAK2V617F% at transformation, 7 had a substantial decrease, and 4 had stable levels. Myeloproliferative neoplasm blast phase is associated with a dismal prognosis. Responses to chemotherapy can be achieved but are not durable. Long-term survivors had all received SCT either as first therapy or in first remission.
Department of Leukemia, University of Texas M. D. Anderson Cancer Center, Houston, TX 77030, USA.
Constantine S
CS
Tam
Roberto M
RM
Nussenzveig
Uday
U
Popat
Carlos E
CE
Bueso-Ramos
Deborah A
DA
Thomas
Jorge A
JA
Cortes
Richard E
RE
Champlin
Stefan E
SE
Ciurea
Taghi
T
Manshouri
Sherry M
SM
Pierce
Hagop M
HM
Kantarjian
Srdan
S
Verstovsek
D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000970:Antineoplastic Agents / Q000627:therapeutic use; D001752:Blast Crisis / Q000209:etiology / Q000235:genetics* / Q000473:pathology / Q000628:therapy; D005260:Female; D016313:Genes, abl; D006801:Humans; D053614:Janus Kinase 2 / Q000235:genetics; D015470:Leukemia, Myeloid, Acute / Q000209:etiology / Q000235:genetics* / Q000473:pathology / Q000628:therapy; D008297:Male; D008875:Middle Aged; D009154:Mutation; D009196:Myeloproliferative Disorders / Q000209:etiology / Q000235:genetics* / Q000473:pathology / Q000628:therapy; D012189:Retrospective Studies; D033581:Stem Cell Transplantation; D015996:Survival Rate; D016896:Treatment Outcome
D000970:Antineoplastic Agents; C507924:JAK2 protein, human; D053614:Janus Kinase 2