Patient and Physician Preferences for Acute Myeloid Leukemia Maintenance Treatments Following Hematopoietic Stem Cell Transplantation

Lalit Saini; James D Griffin; Bhavik J Pandya; Manasee V Shah; Mo Zhou; Hongbo Yang; Yan Song; Deborah A Marshall

doi:10.2147/PPA.S421871

Patient and Physician Preferences for Acute Myeloid Leukemia Maintenance Treatments Following Hematopoietic Stem Cell Transplantation

Patient Prefer Adherence. 2023 Nov 6:17:2805-2819. doi: 10.2147/PPA.S421871. eCollection 2023.

Authors

Lalit Saini¹, James D Griffin², Bhavik J Pandya³, Manasee V Shah³, Mo Zhou⁴, Hongbo Yang⁴, Yan Song⁴, Deborah A Marshall⁵

Affiliations

¹ London Health Sciences Centre, London, Ontario, Canada.
² Dana-Farber Cancer Institute, Boston, MA, USA.
³ Astellas Pharma, Inc, Northbrook, IL, USA.
⁴ Analysis Group, Boston, MA, USA.
⁵ Cumming School of Medicine, University of Calgary, Calgary, Alberta, Canada.

Abstract

Purpose: 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.

Keywords: acute myeloid leukemia; discrete choice experiment; hematopoietic stem cell transplantation; maintenance treatment; quality of life; treatment preferences.

Grants and funding

This study was funded by Astellas Pharma, Inc.