Response and adherence to Nilotinib in daily practice (RAND-study): An in-depth observational study of patients with chronic myeloid leukemia treated with Nilotinib

CCLM Boons, Lonneke Timmers, Peter Westerweel, Eleonora Swart, Harry Hendrikse, Jacqueline Hugtenburg

Research output: Contribution to journalArticleAcademicpeer-review


Background: In chronic myeloid leukemia (CML), oral treatment needs to be taken indefinitely in the majority of patients and specifically for nilotinib, the necessity of a twice daily, fasted schedule is an extra burden. Non-adherence to CML treatment may influence plasma levels and has been recognized as a determinant of treatment failure in CML. Objectives: To gain insight into adherence with the use of nilotinib in daily clinical practice and its influence on exposures and treatment outcome. Methods: A multicentre prospective observational study conducted between August 2013-April 2017. CML patients treated with nilotinib were followed for twelve months. Achievement of a major molecular response (MMR) within the first twelve months of nilotinib treatment was assessed. Adherence was measured by three methods: medication event monitoring system (MEMS) (proportion of days covered [PDC]), pill count (adherence rate [AR]), and self-reported Medication Adherence Report Scale (MARS-5). Nilotinib trough plasma concentrations were measured at baseline, three, six, and twelve months. Results: Sixty-eight patients (57.0 ± 15.0 years;48% female) participated. At baseline, 29 patients were newly starting nilotinib (subpopulation- A) and 39 were already on treatment with nilotinib (subpopulation-B) with a median treatment duration of 39 months (range 3-92). The overall 1-year MMR rate ranged from 44-75%. Median PDC and AR were 99.6 and 100.2 in subpopulation-A and 97.6 and 98.1 in subpopulation-B, respectively. Three and five patients, respectively, had PDC and AR
Original languageEnglish
Pages (from-to)125-126
Number of pages2
JournalPharmacoepidemiology and Drug Safety
Publication statusPublished - 2019

Cite this