A multicenter retrospective evaluation of Chronic Myeloid Leukemia (CML) therapy in Austria assessing the impact of early treatment response on patient outcomes in a real-life setting : R-EFECT study.

Abstract

BACKGROUND:

Several 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.

METHODS:

The 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.

RESULTS:

Of 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).

CONCLUSION:

The 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.

• Authors: Andreas L Petzer, Wolfgang R Sperr, Veronika Buxhofer-Ausch, Thamer Sliwa, Stefan Schmidt, Richard Greil, Albert Wölfler, Petra Pichler, Clemens Dormann, Sonja Burgstaller, Christoph Tinchon, Alois Lang, Florian Goebel, Shanow Uthman, Niklas Muenchmeier, Peter Valent
• Journal: Wiener klinische Wochenschrift (Wien Klin Wochenschr) Vol. 132 Issue 15-16 Pg. 415-422 (Aug 2020) ISSN: 1613-7671 [Electronic] Austria
• PMID: 32533444 (Publication Type: Journal Article, Multicenter Study)

Chemical References

• Antineoplastic Agents
• Dasatinib

Topics

• Adolescent
• Adult
• Aged
• Aged, 80 and over
• Antineoplastic Agents (therapeutic use)
• Austria
• Dasatinib (therapeutic use)
• Female
• Humans
• Leukemia, Myelogenous, Chronic, BCR-ABL Positive (diagnosis, drug therapy, genetics)
• Leukemia, Myeloid, Chronic-Phase (drug therapy)
• Male
• Middle Aged
• Retrospective Studies
• Treatment Outcome
• Young Adult