Patient preferences for benefits, risks, and administration route of hypomethylating agents in myelodysplastic syndromes

Introduction/Background

Therapy with infused or injected hypomethylating agents (HMAs) may lead to higher treatment administration burden (i.e., local reaction, visit frequency and duration) vs. oral HMAs.

Objectives

To reveal preferences of US and Canadian patients with myelodysplastic syndromes (MDS) for HMAs’ benefits, risks, and administration burden through an online discrete-choice experiment (DCE).

Materials and Methods

Choice of DCE attributes and survey development were informed by literature review and interviews with clinicians, MDS patients, and caregivers serving as patient proxies, and patient advocacy groups (PAGs) representatives, including from AAMAC, AAMDS, and MDSF. DCE choice tasks were analyzed using random parameter logit models. Survey patients were recruited by the PAGs via their networks. To understand key preference drivers and how much patients were willing to trade between attributes, we calculated each attribute’s relative attribute importance (RAI) and marginal rates of substitution.

Results

184 respondents (including 158 patients; mean age, 67.2 years; male, 50.5%; White, 50.5%; US residents, 88%) completed the survey. MDS risk was low (34.8%), high (30.9%), or unknown (34.2%). RAI (in decreasing order) was as follows: risk of AML (40%), fatigue level (33%), number of visits (12%), mode of administration (6%), visit duration (5%), and administration frequency (4%). Assuming the same risk of AML transformation or level of fatigue, most respondents (76.6%) were predicted to switch to an oral pill if it were available to them.

Conclusion

Given equivalent effectiveness across HMAs, patients’ preferences for HMA administration method should be considered in treatment decision-making to minimize burden and facilitate adherence.

Micro-abstract

Patient preferences for characteristics of treatment of myelodysplastic syndrome with hypomethylating agents were explored using a discrete-choice experiment (DCE). Development of the DCE’s attributes and levels was informed by semi-structured interviews, with a survey deployed online. Patients prioritized clinical outcomes, preferred oral to parenteral administration, and desired a lower visit-related burden (fewer and shorter visits).

Authors A M Zeidan. J Tsai, M Karimi, J Schmier, S Jayade, E Zormpas, A Hassan, D Ruiters, C Anthony, K Hill, T Wert, M Botteman

Journal Clinical Lymphoma Myeloma & Leukemi

Therapeutic Area Oncology

Center of Excellence Patient-Centered outcomes

Year 2022

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