Prognostic factors and survival outcomes in patients with chronic myeloid leukemia in blast phase in the tyrosine kinase inhibitor era: Cohort study of 477 patients

Preetesh Jain, Hagop M. Kantarjian, Ahmad Ghorab, Koji Sasaki, Elias J. Jabbour, Graciela Nogueras Gonzalez, Rashmi Kanagal-Shamanna, Ghayas C. Issa, Guillermo Garcia-Manero, K. C. Devendra, Sara Dellasala, Sherry Pierce, Marina Konopleva, William G. Wierda, Srdan Verstovsek, Naval G. Daver, Tapan M. Kadia, Gautam Borthakur, Susan O'Brien, Zeev EstrovFarhad Ravandi, Jorge E. Cortes

Research output: Contribution to journalArticlepeer-review

106 Scopus citations


BACKGROUND: Outcomes in patients with chronic myeloid leukemia in blast phase (CML-BP) are historically dismal. Herein, the authors sought to analyze the characteristics, prognostic factors, and survival outcomes in patients with CML-BP in the tyrosine kinase inhibitor (TKI) era. METHODS: A total of 477 patients with CML-BP were treated with a TKI at some point during the course of their CML. Cox proportional hazard models identified characteristics that were predictive of survival. Overall survival and failure-free survival were assessed. Optimal cutoff points for specific parameters were identified using classification and regression tree (CART) analysis. RESULTS: The median age of the patients was 53 years (range, 16-84 years) and 64% were male. Approximately 80% of patients initially were diagnosed in the chronic phase of CML at a median of 41 months (range, 0.7-298 months) before transformation to CML-BP. De novo CML-BP occurred in 71 patients. Approximately 72% of patients received TKI therapy before CML-BP. The initial therapy for CML-BP included a TKI alone (35%), a TKI with chemotherapy (46%), and non-TKI therapies (19%). The median overall survival was 12 months and the median failure-free survival was 5 months. In multivariate analysis, myeloid immunophenotype, prior TKI, age ≥58 years, lactate dehydrogenase level ≥1227 IU/L, platelet count < 102 K/μL, no history of stem cell transplantation, transition to BP from chronic phase/accelerated phase, and the presence of chromosome 15 aberrations predicted for a significantly increased risk of death. Achievement of major hematologic response and/or complete cytogenetic response to first-line treatment was found to be predictive of better survival. The combination of a TKI with intensive chemotherapy followed by stem cell transplantation appeared to confer the best outcome. CONCLUSIONS: Patients with CML-BP continue to pose a therapeutic challenge, have dismal outcomes, and require newer treatment approaches. Cancer 2017;123:4391-402.

Original languageEnglish (US)
Pages (from-to)4391-4402
Number of pages12
Issue number22
StatePublished - Nov 15 2017
Externally publishedYes


  • blast phase (BP)
  • bosutinib
  • chronic myeloid leukemia (CML)
  • dasatinib
  • imatinib
  • nilotinib
  • ponatinib
  • tyrosine kinase inhibitors (TKI)

ASJC Scopus subject areas

  • Oncology
  • Cancer Research


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