Abstract
INTRODUCTION. Three tyrosine-kinase inhibitors (TKIs, imatinib, nilotinib [NIL] and dasatinib) are approved for first-line treatment of CP CML. Clinical trials, with stringent inclusion/exclusion criteria and management, may be not fully representative of the daily clinical practice. Thus, it is important to retrieve information on the safety and efficacy of TKIs in the real-life setting. Observational studies are particularly useful for these evaluations.
AIM. To investigate the safety and efficacy of NIL as first line treatment of newly diagnosed CP CML pts in the real-life setting
METHODS. Prospective, multicenter, observational study. Inclusion criteria: CP CML within 6 months from diagnosis; no prior treatment with TKIs; written informed consent. Comorbidities (of any kind and severity) were not exclusion criteria. NIL was used according to prescribing information (initial dose 300 mg BID). There was no formal indication for dose modifications, management of adverse events, and response monitoring.
RESULTS. Between 06/2013 and 11/2015, 123 pts were enrolled in 27 Italian Centers; median age: 51 yrs; Sokal high-risk pts: 19%; EUTOS high-risk pts: 7%. Despite Median follow-up: 18 months (updated results will be presented). At the last contact, of evaluable pts with at least 12 months of follow-up, 78% and 43% of pts were in MR3 and MR4, respectively. At the last contact, all but one pt were alive. 113 (89%) pts were still on study (on NIL treatment); in the remaining pts, NIL discontinuation occurred in: 9 pts for failure/suboptimal response, including 1 progression to blast phase; 2 pts for toxicity; 1 pt for a CML-unrelated death, 2 pts for other reasons.
CONCLUSION. These preliminary data in real-life CP CML pts treated with NIL first-line showed that: 1) despite the absence of exclusion criteria for comorbidities, the median age of enrolled pts (51 years) was similar to that reported in clinical trials, and inferior to the median age of CML pts in population based-registries; 2) NIL use was not limited to intermediate or high-risk pts; 3) efficacy was similar to that reported in clinical trials, with very low rates of progression to advanced phase and high molecular response rates.
Gugliotta: Novartis: Consultancy, Honoraria; Bristol Myers Squibb: Consultancy, Honoraria; Incyte: Consultancy, Honoraria. Castagnetti: Incyte: Consultancy, Honoraria; Novartis: Consultancy, Honoraria; Pfizer: Consultancy, Honoraria; Bristol-Myers Squibb: Consultancy, Honoraria. Abruzzese: Novartis: Consultancy; Incyte: Consultancy; BMS: Consultancy; Pfizer: Consultancy. Breccia: Pfizer: Consultancy; Bristol Myers Squibb: Consultancy; Incyte: Consultancy; Novartis: Consultancy. Iurlo: Novartis: Consultancy, Honoraria; Bristol Myers Squibb: Consultancy, Honoraria; Pfizer: Consultancy, Honoraria. Tiribelli: Novartis: Consultancy, Honoraria; Bristol Myers Squibb: Consultancy, Honoraria; Incyte: Consultancy, Honoraria. Crugnola: BMS: Honoraria; Celgene: Honoraria; Novartis: Honoraria. Bonifacio: Incyte: Membership on an entity's Board of Directors or advisory committees; Pfizer: Membership on an entity's Board of Directors or advisory committees; Novartis: Membership on an entity's Board of Directors or advisory committees, Research Funding; Bristol-Myers Squibb: Membership on an entity's Board of Directors or advisory committees. Soverini: Incyte Biosciences: Consultancy; Novartis: Consultancy; Bristol-Myers Squibb: Consultancy. Foà: Janssen: Consultancy, Speakers Bureau; Celgene: Consultancy, Speakers Bureau; Novartis: Consultancy, Speakers Bureau; Abbvie: Consultancy, Speakers Bureau; Gilead: Consultancy, Speakers Bureau; Amgen: Consultancy, Speakers Bureau; BMS: Consultancy, Speakers Bureau; Roche: Consultancy, Speakers Bureau; Sandoz: Consultancy, Speakers Bureau. Martinelli: PFIZER: Consultancy; JOHNSON & JOHNSON: Consultancy; CELGENE: Consultancy; ROCHE: Consultancy; ARIAD/INCYTE: Consultancy; AMGEN: Consultancy. Pane: Novartis: Honoraria, Speakers Bureau. Saglio: Pfizer: Consultancy, Honoraria; Ariad: Consultancy, Honoraria; Roche: Consultancy, Honoraria; BMS: Consultancy, Honoraria; Novartis: Consultancy, Honoraria; Incyte: Consultancy, Honoraria. Baccarani: Novartis: Consultancy, Honoraria, Speakers Bureau; Bristol-Myers Squibb: Honoraria, Speakers Bureau; Pfizer: Honoraria, Speakers Bureau; Incyte ARIAD: Consultancy, Honoraria, Speakers Bureau. Rosti: Pfizer: Research Funding, Speakers Bureau; Novartis: Research Funding, Speakers Bureau; Bristol Myers Squibb: Research Funding, Speakers Bureau; Incyte: Research Funding, Speakers Bureau.
Author notes
Asterisk with author names denotes non-ASH members.
