Abstract
Achievement of deep molecular response following treatment with a tyrosine kinase inhibitor (TKI) allows for treatment-free remission (TFR) in many patients with chronic myeloid leukemia (CML). Successful TFR is defined as the achievement of a sustained molecular response after cessation of ongoing TKI therapy. The phase 3 ENESTPath study was designed to determine the required optimal duration of consolidation treatment with the second-generation TKI, nilotinib 300 mg twice-daily, to remain in successful TFR without relapse after entering TFR for 12 months. The purpose of this Italian ‘patient’s voice CML’ substudy was to evaluate patients’ psycho-emotional characteristics and quality of life through their experiences of stopping treatment with nilotinib and entering TFR. The purpose of the present contribution is to early present the study protocol of an ongoing study to the scientific community, in order to describe the study rationale and to extensively present the study methodology. Patients aged ≥18 years with a confirmed diagnosis of Philadelphia chromosome positive BCR-ABL1+ CML in chronic phase and treated with front-line imatinib for a minimum of 24 months from the enrollment were eligible. Patients consenting to participate the substudy will have quality of life questionnaires and in-depth qualitative interviews conducted. The substudy will include both qualitative and quantitative design aspects to evaluate the psychological outcomes as assessed via patients’ emotional experience during and after stopping nilotinib therapy. Randomization is hypothesized to be a timepoint of higher psychological alert or distress when compared to consolidation and additionally any improvement in health-related quality of life (HRQoL) due to nilotinib treatment is expected across the timepoints (from consolidation, to randomization, and TFR). An association is also expected between dysfunctional coping strategies, such as detachments and certain personality traits, and psychological distress and HRQoL impairments. Better HRQoL outcomes are expected in TFR compared to the end of consolidation. This substudy is designed for in-depth assessment of all potential psycho-emotional variables and aims to determine the need for personalized patient care and counselling, and also guide clinicians to consider the psychological well-being of patients who are considering treatment termination.NCT number: NCT01743989, EudraCT number: 2012-005124-15
Highlights
Chronic myeloid leukemia (CML) is a hematopoietic disorder, characterized by the rearrangement of the fusion oncogene BCRABL1 that encodes an oncoprotein with dysregulated ABL1 tyrosine kinase activity [1,2,3]
Clinical practice guidelines for CML have so far not included psycho-emotional issues related to stopping tyrosine kinase inhibitors (TKIs) treatment and attempting treatment-free remission (TFR) [7, 26]
Few studies have been conducted to investigate the emotional and psychological ramifications of involvement in CML TFR trials, data relating to treatment efficacy, safety, and interruption are becoming increasingly significant
Summary
Chronic myeloid leukemia (CML) is a hematopoietic disorder, characterized by the rearrangement of the fusion oncogene BCRABL1 that encodes an oncoprotein with dysregulated ABL1 tyrosine kinase activity [1,2,3]. The CML patients’ voice study is designed as a mixed method study that uses both quantitative and qualitative approaches to explore patients’ psychological outcomes, health-related quality of life, and their experiences of being involved in the ENESTPath trial and discontinuation of nilotinib, with a particular focus on the time when patients completed the consolidation phase and approached the TFR phase. The objectives of this substudy include assessment of personality traits, psychological distress, the strategies that patients adopt for coping during the consolidation phase of the ENESTPath trial and HRQoL from the consolidation to the TFR phase. The emotional experience of patients who are not eligible for randomization and have to withdraw from the substudy will be explored
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