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4872 Understanding What Matters to Myelodysplastic Syndrome Patients – a Study of Preferences for Treatments with Hypomethylating Agents

Program: Oral and Poster Abstracts
Session: 903. Health Services and Quality–Myeloid Malignancies: Poster III
Hematology Disease Topics & Pathways:
MDS, Clinical Practice (Health Services and Quality), Chronic Myeloid Malignancies, Diseases, Myeloid Malignancies, Human
Monday, December 12, 2022, 6:00 PM-8:00 PM

Amer M. Zeidan, MD1, Sophie (jui-Hua) Tsai, MD, MS2*, Milad Karimi, PhD, MS3*, Jordana Schmier, MA2*, Sayeli Jayade, MPH2*, Evangelos Zormpas, PhD, MS4*, Audrey Hassan5*, Desiree Ruiters6*, Cindy Anthony7*, Kala Hill, MSc8*, Tim Wert9* and Marc Botteman, MS, MA2*

1Department of Internal Medicine, Yale School of Medicine and Yale Cancer Center, New Haven, CT
2OPEN Health Evidence and Access, Bethesda, MD
3OPEN Health Evidence and Access, Rotterdam, Netherlands
4OPEN Health Evidence and Access, York, United Kingdom
5The Myelodysplastic Syndromes (MDS) Foundation, Inc, Yardville, NJ
6Aplastic Anemia and MDS International Foundation, Rockville, MD
7Aplastic Anemia & Myelodysplasia Association of Canada, Ontario, Canada
8Taiho Oncology, Princeton
9Taiho Oncology, Princeton, NJ

Introduction/Background: Hypomethylating agents (HMAs) are the standard of care for the treatment of myelodysplastic syndrome (MDS). Understanding how patients with MDS perceive and value different HMA characteristics provides critical treatment selection insight for healthcare providers.

Objectives: To understand HMA preferences for United States (US) and Canadian patients with MDS. Given HMAs' treatments’ similar efficacy and safety profiles, we also investigated how HMA administration burden can play a role in patients’ preferences when all other factors are equal.

Materials and Methods: We used the discrete-choice experiment (DCE) method to conduct a preference study in collaboration with patient organizations in the US and Canada (i.e., MDS Foundation, the Aplastic Anemia and MDS International Foundation, and the Aplastic Anemia and Myelodysplasia Association of Canada). The study was comprised of two Phases: (1) a qualitative Phase (i.e., DCE survey design) and (2) a quantitative Phase (i.e., DCE survey implementation). In Phase 1, literature review, interviews with clinician, patient and caregiver, and input from the patient organizations informed the DCE survey design. Qualitative interviews were analyzed through content analysis. In Phase 2, survey participants answered a series of DCE choice tasks to indicate their preference between different hypothetical HMA profiles that varied in characteristics (i.e., attributes). Respondents were also asked to indicate their preferences by choosing between HMA profiles mirroring real-life options, all associated with the same risk of AML transformation and level of fatigue but differing in mode of and frequency administration: IV HMAs requiring 5 days or 7 days of treatment session each month, SC HMA with 5 sessions each month, oral HMA and no treatment. Survey results were analyzed using random parameter logit models. The relative importance of attributes (RAI) was calculated, which is the contribution (expressed in percentages) of each attributes relative to all attributes in patient preferences. A larger RAI percentage score shows that the attribute has a larger effect on how the respondents make their decisions. All patient respondents were recruited through the patient organizations’ networks.

Results: 16 Phase 1 respondents completed the interview and 184 Phase 2 respondents (including 158 patients and 26 caregivers serving as patient proxies) completed the DCE survey. Interview respondents in Phase 1 valued efficacy attributes (i.e., risk of AML transformation and fatigue) and expressed the treatment burdens associated with injectable treatments (such as injection site reaction, stressful experience of damaged veins, burden of travelling to clinic, etc.). The Phase 2 survey participants had a mean age of 67.2 years old and 88% were from the US. About one-third of the survey participants did not know their MDS risk, 35% were low risk and 31% were high risk. Most (56%) participants were currently receiving treatment, while 24% had received treatment but discontinued and the remaining 20% had not received treatment. The attributes’ importance rankings (via RAI) were risk of AML transformation (40%), fatigue level (33%), number of visits (12%), mode of administration (6%), visit duration (5%), and administration frequency (4%). When HMA treatments were assumed to be associated with the same risk of AML and level of fatigue but to differ in terms of mode and frequency administration, 77% of respondents were predicted to switch to an oral pill if available to them. When considering real-life profiles, 77% and 88% of participants considered the oral option to be the best choice and no treatment to be the worst one, respectively.

Conclusion: MDS patients demonstrated strong preferences for HMA with lesser treatment burden (i.e., oral pill) when such agents are with similar treatment efficacy and safety profiles. Understanding patients’ treatment preferences can facilitate shared decision making towards optimal management of MDS.

Disclosures: Zeidan: BMS, AbbVie, Takeda, Novartis, Aprea, Amgen, Otsuka, Gilead, Kura, Loxo Oncology, Geron, Mendus, Tyme: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees. Tsai: Insmed Inc, Taiho Oncology, Bayer Pharmaceuticals, Merck & Co., Janssen Pharmaceuticals: Research Funding; OPEN Health Evidence and Access: Current Employment. Karimi: Kite, Abbvie, AstraZeneca, Bayer Pharmaceuticals, Taiho Oncology: Research Funding; OPEN Health Evidence and Access: Current Employment. Schmier: AbbVie, AstraZeneca, Bayer, Bristol Myers Squibb, Ferring, Mallinckrodt, Merck & Co., Taiho Oncology, Takeda, Trefoil: Research Funding; OPEN Health Evidence and Access: Current Employment. Jayade: OPEN Health Evidence and Access: Current Employment; Taiho Oncology, Bayer, Bristol Myers Squibb: Research Funding. Zormpas: Taiho Oncology: Research Funding; OPEN Health Evidence and Access: Ended employment in the past 24 months. Hassan: Taiho Oncology: Research Funding. Ruiters: Taiho Oncology: Research Funding. Anthony: Alexion, Bristol Myers Squibb, Novartis, Sobi, Taiho Oncology, Takeda: Other: Patient support, Research Funding. Hill: Merck & Co.: Current Employment; Taiho Oncology: Ended employment in the past 24 months, Research Funding. Wert: Taiho Oncology: Current Employment, Research Funding. Botteman: Taiho Oncology, Bayer Pharmaceuticals, Merck & Co., AstraZeneca: Research Funding; OPEN Health Evidence and Access: Ended employment in the past 24 months.

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