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5188 Development of the Treatment Preference in Myelodysplasia Questionnaire (TPMQ) for Clinicians (mTPMQ), Carers (cTPMQ), and Patients (pTPMQ)

Program: Oral and Poster Abstracts
Session: 906. Outcomes Research—Myeloid Malignancies: Poster III
Hematology Disease Topics & Pathways:
Clinical Practice (Health Services and Quality)
Monday, December 11, 2023, 6:00 PM-8:00 PM

Anoop K Enjeti, MBBS FRCPA MD MRCP1, Chun Yew Fong, MBBS, FRACP, FRCPA, PhD2, Francesco Castaldi, PhD MSc3*, Taliesha Paine, PhD3*, Donna Collett4* and Robert Morlock5*

1Calvary Mater Hospital, Waratah, NSW, AUS
2Austin Hospital, Heidelberg, Australia
3Otsuka Australia Pharmaceutical Pty Ltd, Chatswood, NSW, Australia
4Valeur Consulting Pty Ltd, Sydney, Australia
5YourCareChoice, Ann Arbor, MI, Ann Arbor, MI

Objective: Myelodysplastic syndrome (MDS) is a disease of the hematopoietic stem cells and leads to an increased risk of developing acute myeloid leukemia (AML).[1] The injectable hypomethylating agents (HMA) azacitdine and decibtabine have been shown to be superior to best supportive care in improving cytopenias in intermediate to high-risk patients.[2, 3] These injectable HMAs regimens are commonly administered in a healthcare facility, with patients traveling to the facility on consecutive days. The recent availability of an oral form of decitabine, ASTX 727 (a fixed-dose combination of decitabine and the cytidine deaminase inhibitor cedazuridine), may significantly impact patient, carer, and clinician choices. To assess preference for an injectable vs. orally administered HMA, three questionnaires were developed based on qualitative input from clinicians, patients, and carers: the Treatment Preference in Myelodysplasia Questionnaire (TPMQ) for clinicians (mTPMQ), carers (cTPMQ), and patients (pTPMQ).

Methods: This was a non-interventional, cross-sectional qualitative interview study consisting of interviews with patients with MDS who were eligible for or were being/had been treated with injectable azacitidine, their carers and their treating clinicians. Data were collected from qualitative mixed method interviews composed of concept elicitation and cognitive debriefing. Two sites were identified to enrol participants. During concept elicitation, participants were asked about MDS and treatment options to best understand the constructs of interest relative to their specific circumstance and preferences. Cognitive debriefing occurred after participants reviewed a draft of the questionnaire. Based on the interviews, the questionnaires were revised to ensure the constructs of importance were captured and the questionnaires, response options, and instructions were all well understood.

Results: 15 participants completed interviews (5 clinicians for the mTPMQ, 5 patients for the pTPMQ, and 5 carers for the cTPMQ). Concepts identified as important included across groups included: easier to be compliant, less travel time, less time in the clinic, less painful, costs and convenience (Table 1).

The mTPMQ underwent 3 revisions. Overall, clinicians indicated the directions, items and response options were understandable and clear. Comments that significantly modified the questionnaire concerned splitting the last question from 1 to 2 questions, depending on which treatment the patient would be receiving.

Patient participants thought the pTPMQ captured concepts of interest and was easy to understand. Modifications were made to the questionnaire either as a direct result of patient participant comments (e.g., changed skip directions to "turn to page X, question X"), study team discussions of the interviewer's comments and notes and making items consistent between questionnaires.

Carer participants thought the cTPMQ captured the constructs of interest and the questions were clear and the questionnaire was easy to answer. Modifications were made to the questionnaire as a direct result of carer participant comments (e.g., changed "We spend less time in the clinic" to "It means less time in the clinic for the patient"), study team discussions of the interviewer's comments and notes (e.g., added "It makes it easier for the patient to continue treatment") and making items consistent between questionnaires.

Conclusions: The findings of the participant interviews suggest the concepts assessed in the assessments are complete. Respondents could respond to items and talk about all aspects assessed in the questionnaires in meaningful ways and were able to comprehend the instructions and response options on a categorical scale. The TPMQs are based on direct input from the population of interest and assess the constructs of importance to each group.

  1. Bell, J.A., et al., Clin Lymphoma Myeloma Leuk, 2018. 18(4): p. e157-e166.
  2. Kantarjian, H., et al., Cancer, 2006. 106(8): p. 1794-803.
  3. Fenaux, P., et al., Lancet Oncol, 2009. 10(3): p. 223-32.

Disclosures: Enjeti: Servier: Honoraria; Otsuka: Honoraria, Speakers Bureau; Pfizer: Honoraria; Jazz: Honoraria; RACE oncology: Honoraria; Astellas: Honoraria; AbbVie: Honoraria, Speakers Bureau. Fong: AbbVie, Pfizer, Servier: Speakers Bureau; AbbVie, Astellas, RACE oncology, Jazz, Pfizer, Otsuka, Servier: Honoraria. Castaldi: Otsuka Australia Pharmaceutical Pty Ltd: Current Employment. Paine: Otsuka Australia Pharmaceutical Pty Ltd: Current Employment. Collett: Otsuka Australia Pharmaceutical Pty Ltd: Consultancy, Honoraria; Valeur Consulting Pty Ltd: Current Employment, Membership on an entity's Board of Directors or advisory committees. Morlock: Otsuka, Novartis, Heron Therapeutics, Replimune, Horizon Therapeutics, Syndax, Arthrosi, Pfizer: Consultancy.

*signifies non-member of ASH