Patient Preference and Adherence (Nov 2023)
Patient and Physician Preferences for Acute Myeloid Leukemia Maintenance Treatments Following Hematopoietic Stem Cell Transplantation
Abstract
Lalit Saini,1 James D Griffin,2 Bhavik J Pandya,3 Manasee V Shah,3 Mo Zhou,4 Hongbo Yang,4 Yan Song,4 Deborah A Marshall5 1London Health Sciences Centre, London, Ontario, Canada; 2Dana-Farber Cancer Institute, Boston, MA, USA; 3Astellas Pharma, Inc, Northbrook, IL, USA; 4Analysis Group, Boston, MA, USA; 5Cumming School of Medicine, University of Calgary, Calgary, Alberta, CanadaCorrespondence: Bhavik J Pandya, Director, Oncology Health Economics and Clinical Outcomes Research, Astellas Pharma, Inc, 1 Astellas Way, Northbrook, IL, 60062, USA, Tel +1 847 226-0066, Email [email protected]: This study assessed and compared preferences for treatment attributes of maintenance therapies post-hematopoietic stem cell transplantation (HSCT) in patients with acute myeloid leukemia (AML) and in physicians who treat these patients.Patients and Methods: Patients with AML post HSCT and physicians from the United States, United Kingdom, Canada, and Australia (physicians only) completed a web-based discrete choice experiment (DCE). The DCE used inputs identified via a targeted literature review and qualitative interviews to ascertain relevant treatment attributes and associated levels. Six treatment attributes were selected (chance of 2-year relapse-free survival, quality of life [QoL], risk of serious infections, risk of nausea, chance of achieving transfusion independence, and duration of hospitalization annually), each with three or four levels. The experimental design included 36 choice tasks that presented a pair of hypothetical treatment profiles with varying attribute levels; participants chose a preferred treatment for each choice task. Choice tasks were divided into three blocks of 12 tasks each in the patient survey and 4 blocks of 9 tasks each in the physician survey; survey participants were randomly assigned to one of the blocks. Random parameter logit regression models were used to assess the impact of stated attributes on preferences for maintenance treatment post HSCT.Results: Surveys from 84 patients and 149 physicians were assessed. For patients, QoL was the most important attribute, followed by duration of hospitalization and chance of 2-year relapse-free survival. For physicians, chance of 2-year relapse-free survival was the most important attribute, followed by QoL and risk of serious infections.Conclusion: Differences in how patients and physicians valued post-HSCT maintenance treatment attributes were identified. These differences suggest that patient-centered decision-making may help physicians choose maintenance treatments for patients with AML post HSCT that better meet their treatment needs and improve their treatment satisfaction.Plain Language Summary: Patients with acute myeloid leukemia (AML) are often treated with chemotherapy followed by stem cell transplantation (SCT). Following SCT, evidence suggests that some patients may benefit from maintenance therapy. In this study, researchers surveyed patients and physicians who treat patients with AML to understand what maintenance treatment characteristics they most valued following SCT. Characteristics evaluated during the survey were chance of survival without relapse, impact on quality of life, risk of serious infections, risk of nausea, chance of being free of needing blood transfusions, and impact on annual hospital length of stay. For patients, the most important maintenance treatment characteristics were better quality of life and fewer hospital days annually. For physicians, the most important maintenance treatment characteristic was a higher chance of survival without relapse. These results show differences in maintenance treatment preferences between patients and physicians, suggesting that patient-centered decision-making could be useful when selecting a maintenance treatment for AML after SCT that meets patients’ treatment needs and improves their treatment satisfaction. Keywords: acute myeloid leukemia, hematopoietic stem cell transplantation, maintenance treatment, discrete choice experiment, quality of life, treatment preferences
