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

Study protocol of the RAND-study: a multicenter, prospective cohort study investigating response and adherence to nilotinib treatment in chronic myeloid leukemia - Descarga este documento en PDF. Documentación en PDF para descargar gratis. Disponible también para leer online.

BMC Cancer

, 14:247

Clinical oncology


BackgroundThe 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-DesignIn 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.

DiscussionThe 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 registrationNetherlands Trial Registry NTR3992.

KeywordsChronic myeloid leukemia Nilotinib Treatment outcome Response Medication adherence Patients’ experiences AbbreviationsASATAspartate transaminase

BMQBeliefs about Medicines Questionnaire

IPQBrief Illness Perception Questionnaire

CRFCase Report Form

CMLChronic Myeloid Leukemia

CP-CMLChronic Phase Chronic Myeloid Leukemia

CCyRComplete Cytogenetic Response

CHRComplete Hematological Response

CMRComplete Molecular Response

DBSDried Blood Spot

MMRMajor Molecular Response

MRECMedical Research Ethics Committee

WMOMedical Research Involving Human Subjects Act

MARSMedication Adherence Rating Scale

MEMSMedication Event Monitoring System

Ph+Philadelphia positive

RQ-PCRReal-time Quantitative-Polymerase Chain Reaction assay

SIMSSatisfaction with Information about Medicines Scale

TKITyrosine kinase inhibitors.

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Autor: Christel CLM Boons - Eleonora L Swart - Lonneke Timmers - Peter M van de Ven - Jeroen JWM Janssen - Jacqueline G Hugten


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