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Nilotinib dose‐optimization in newly diagnosed chronic myeloid leukaemia in chronic phase: final results from ENEST xtnd
Author(s) -
Hughes Timothy P.,
Munhoz Eduardo,
Aurelio Salvino Marco,
Ong Tee Chuan,
Elhaddad Alaa,
Shortt Jake,
Quach Hang,
Pavlovsky Carolina,
Louw Ver J.,
Shih LeeYung,
Turkina Anna G.,
Meillon Luis,
Jin Yu,
Acharya Sandip,
Dalal Darshan,
Lipton Jeffrey H.
Publication year - 2017
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/bjh.14829
Subject(s) - nilotinib , medicine , adverse effect , rash , clinical endpoint , nausea , clinical trial , oncology , myeloid leukemia , imatinib
Summary The Evaluating Nilotinib Efficacy and Safety in Clinical Trials–Extending Molecular Responses ( ENEST xtnd) study was conducted to evaluate the kinetics of molecular response to nilotinib in patients with newly diagnosed chronic myeloid leukaemia in chronic phase and the impact of novel dose‐optimization strategies on patient outcomes. The ENEST xtnd protocol allowed nilotinib dose escalation (from 300 to 400 mg twice daily) in the case of suboptimal response or treatment failure as well as dose re‐escalation for patients with nilotinib dose reductions due to adverse events. Among 421 patients enrolled in ENEST xtnd, 70·8% (95% confidence interval, 66·2–75·1%) achieved major molecular response ( BCR ‐ ABL 1 ≤ 0·1% on the International Scale) by 12 months (primary endpoint). By 24 months, 81·0% of patients achieved major molecular response, including 63·6% (56 of 88) of those with dose escalations for lack of efficacy and 74·3% (55 of 74) of those with dose reductions due to adverse events (including 43 of 54 patients with successful re‐escalation). The safety profile of nilotinib was consistent with prior studies. The most common non‐haematological adverse events were headache, rash, and nausea; cardiovascular events were reported in 4·5% of patients (grade 3/4, 3·1%). The study was registered at clinicaltrials.gov (NCT01254188).