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Open Access Highly Accessed Study protocol

Study protocol of the RAND-study: a multicenter, prospective cohort study investigating response and adherence to nilotinib treatment in chronic myeloid leukemia

Christel CLM Boons1*, Eleonora L Swart1, Lonneke Timmers1, Peter M van de Ven2, Jeroen JWM Janssen3 and Jacqueline G Hugtenburg14

Author Affiliations

1 Department of Clinical Pharmacology and Pharmacy, VU University Medical Center, De Boelelaan 1117, 1081 HV Amsterdam, The Netherlands

2 Department of Epidemiology and Biostatistics, VU University Medical Center, De Boelelaan 1118, 1081 HV Amsterdam, The Netherlands

3 Department of Hematology, VU University Medical Center, De Boelelaan 1117, 1081 HV Amsterdam, The Netherlands

4 The EMGO Institute for Health and Care Research, VU University Medical Center, Van der Boechorststraat 7, 1081 BT Amsterdam, The Netherlands

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BMC Cancer 2014, 14:247  doi:10.1186/1471-2407-14-247

Published: 8 April 2014

Abstract

Background

The antitumor drug nilotinib has a large inter- and intra individual variability in pharmacokinetics. Adherence to treatment may substantially influence plasma levels and has been recognized as the most important determinant of treatment failure in chronic myeloid leukemia (CML). A better understanding of the various factors contributing to the efficacy of treatment is essential for the development of interventions to optimize the treatment of chronic phase CML (CP-CML) with a protein kinase inhibitor like nilotinib.

Methods/Design

In this multicenter prospective observational cohort study 70 adult patients with CP-CML starting treatment with nilotinib will be followed up for at least 12 months. Response to treatment is evaluated after 3, 6 and 12 months. Adherence is primarily assessed by counting the daily intake of nilotinib capsules by means of a medication event monitoring system (MEMS). Before the start of nilotinib treatment and after 3, 6 and 12 months, patients are asked to fill in a comprehensive questionnaire including topics on quality of life, side effects, attitude towards disease and medication, the patients’ appreciation of information received about the medication, and discontinuation, and trough plasma levels of nilotinib are measured.

Discussion

The present study aims to get more insight into the efficacy of treatment with nilotinib and the various aspects that govern optimal response, of which adherence is a primary endpoint. We hypothesize that patients who experience inadequate response levels to nilotinib are less adherent. In addition, their plasma levels of nilotinib may be lower. We expect that our findings will be useful for health care professionals to support patients with the use of nilotinib in order to derive optimal benefit from their medication.

Trial registration

Netherlands Trial Registry NTR3992.

Keywords:
Chronic myeloid leukemia; Nilotinib; Treatment outcome; Response; Medication adherence; Patients’ experiences