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 Blood, 1 September 2008, Vol. 112, No. 5, pp. 1628-1637.
Prepublished online as a Blood First Edition Paper on June 19, 2008; DOI 10.1182/blood-2008-02-138230.

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CLINICAL TRIALS AND OBSERVATIONS

The natural history and treatment outcome of blast phase BCR-ABL– myeloproliferative neoplasms

Constantine S. Tam1, Roberto M. Nussenzveig1, Uday Popat2, Carlos E. Bueso-Ramos3, Deborah A. Thomas1, Jorge A. Cortes1, Richard E. Champlin2, Stefan E. Ciurea2, Taghi Manshouri1, Sherry M. Pierce1, Hagop M. Kantarjian1, and Srdan Verstovsek1

Departments of1 Leukemia, 2 Stem Cell Transplantation and Cellular Therapy, and 3 Hematopathology, University of Texas M. D. Anderson Cancer Center, Houston

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.


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[Abstract] [Full Text] [PDF]


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