Similar outcome of patients with chronic myeloid leukemia treated with imatinib in or out of clinical trials

Musa Yilmaz, Hagop Kantarjian, Elias Jabbour, Susan O'Brien, Gautam Borthakur, Srdan Verstovsek, Guillermo Garcia-Manero, Farhad Ravandi, Jan Burger, Sherry Pierce, Alfonso Quintas-Cardama, Jorge Cortes

Research output: Contribution to journalArticlepeer-review

6 Scopus citations


Introduction Outcomes of CML-CP patients treated in clinical trials are frequently perceived to be not representative of those treated outside of clinical trials. Patients and Methods We investigated the outcomes of patients receiving imatinib outside of a clinical trial (off protocol) or in a clinical trial (on protocol) for CML-CP. Results We identified 65 patients treated with imatinib off protocol and 71 patients treated on protocol with standard-dose imatinib. The overall complete cytogenetic response (CCyR) rate was 83% for patients treated on and off protocol. CCyR rates 12 months after initiation of imatinib were not statistically different (61% vs. 66%, respectively; P =.15). Patients treated off protocol had similar rates of overall major molecular response (72% vs. 73%) compared with the patients treated on protocol. The 5-year event-free survival rates were 84% and 86% for off and on protocol patients, respectively. There was also no significant difference in 5-year transformation free survival (94% vs. 96%) and overall survival (96% vs. 90%). Conclusion These results suggest that patients with CML treated outside of a clinical trial might have the same excellent outcome as those treated in a clinical trial provided they are followed with the same rigor.

Original languageEnglish (US)
Pages (from-to)693-699
Number of pages7
JournalClinical Lymphoma, Myeloma and Leukemia
Issue number6
StatePublished - Dec 2013
Externally publishedYes


  • CML
  • Imatinib
  • Outside of a clinical trial
  • Prognosis
  • TKI

ASJC Scopus subject areas

  • Hematology
  • Oncology
  • Cancer Research


Dive into the research topics of 'Similar outcome of patients with chronic myeloid leukemia treated with imatinib in or out of clinical trials'. Together they form a unique fingerprint.

Cite this