Abstract
SummaryBackgroundSeveral clinical trials in chronic phase (CP) chronic myeloid leukemia (CML) showed that early response to tyrosine kinase inhibitor (TKI) treatment results in an improved long-term survival and progression-free survival. This study assessed whether patients achieving early treatment response (ETR; partial cytogenetic response or BCR-ABL1 mRNA ≤10% at 3 months) in daily practice also have a long-term survival benefit.MethodsThe Retrospective Evaluation of Early response in CML for long-term Treatment outcome (R-EFECT), a multicenter, retrospective chart review, documented patients with newly diagnosed CML-CP starting first-line TKI therapy in routine clinical practice. The primary aim was to assess the 5‑year overall survival rate.ResultsOf the 211 patients from 12 centers across Austria (January 2004–May 2010), 176 (median age, 56 years) were included in the analysis. All patients received first-line therapy with imatinib. Overall, 136 patients (77.3%) achieved ETR (ETR+ group), whereas 40 (22.7%) did not reach ETR (ETR− group). The ETR+ group had higher 5‑year overall survival (92.5% vs. 77.5%, P = 0.018) and progression-free survival (95.6% vs. 87.5%, P = 0.06) rates compared with the ETR− group. As expected, more patients in the ETR− group were switched to another TKI. At the last contact, 120 patients were still on imatinib and 44 had switched to another TKI (25 to nilotinib, 15 to dasatinib, and 4 to bosutinib).ConclusionThe data are in line with randomized trials demonstrating that ETR is associated with improved survival and thus confirmed these results in patients treated in daily clinical routine.
Highlights
According to the European Leukemia Network (ELN) guidelines for chronic myeloid leukemia (CML), BCR-ABL1 transcript levels of ≤10% according to the international scale (IS, BCR-ABL1IS) in the peripheral blood or a partial cytogenetic response,
Several clinical trials have shown that patients with Chronic Myeloid Leukemia (CML) in chronic phase (CP) who achieve early molecular response (EMR, BCR-ABL1IS ≤10% at 3 months) with tyrosine kinase inhibitor (TKI) therapy have better long-term responses, overall survival (OS), and progression-free survival (PFS) [2,3,4,5,6,7,8,9,10]; these data were obtained in patients selected for clinical trials
The BCR-ABL1IS values were used for the analysis of early treatment response (ETR) in 102 of 176 patients (58%), cytogenetic responses in 41 patients (23%) and BCR-ABL1raw values in 33 patients (19%)
Summary
According to the European Leukemia Network (ELN) guidelines for chronic myeloid leukemia (CML), BCR-ABL1 transcript levels (addressed as BCR-ABL1 throughout) of ≤10% according to the international scale (IS, BCR-ABL1IS) in the peripheral blood or a partial cytogenetic response (pCyR),
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