Clinicians, Caregivers, Patients: MDS Treatment Preferences Explored

A recent study published in eJournal of Haematology introduces the Treatment Preference Myelodysplasia Questionnaires (TPMQ), a set of questionnaires designed to evaluate treatment preferences in myelodysplastic syndromes (MDS) from the perspectives of clinicians, caregivers, and patients. The research highlights the importance of factors such as convenience and emotional distress in shaping treatment decisions.¹

As part of this study, the 15 participants discussed their understanding of MDS and treatment options | Image Credit: sabelskaya-stock.adobe.com

The study aimed to develop the TPMQ for clinicians (mTPMQ), carers (cTPMQ), and patients (pTPMQ). These tools are designed to evaluate preferences for injectable vs orally administered hypomethylating agent (HMA) treatments. HMA injectable agents azacitidine and decitabine have been shown to be superior to best supportive care in improving cytopenias in intermediate to high-risk patients.^2,3 They are available in multiple dosing options, including intravenous and subcutaneous. However, it is hypothesized that some patients who are candidates for HMA treatment refuse therapy, discontinue therapy, or request discontinuation of therapy due to the inconvenience of the treatment regimen and its clinical and demographic factors.⁴ An oral HMA recently became available to treat MDS in some countries, including the US and Canada. Having the option of oral therapy may significantly affect patient, caregiver, and clinician choices.

This noninterventional, cross-sectional qualitative interview study involved 15 participants: 5 clinicians, 5 caregivers, and 5 patients, all located in Australia. The interviews were divided into 2 parts: concept elicitation and cognitive debriefing. During concept elicitation, participants discussed their understanding of MDS and treatment options to identify the constructs of interest. Cognitive debriefing involved reviewing draft questionnaires to ensure clarity and appropriateness of questions, instructions, and response options.

For clinicians, key factors influencing treatment preference included efficacy, tolerance, manageability, patient preference, compliance, and hesitancy to switch treatments. Cognitive debriefing led to revisions such as adding specific details like (subcutaneous) after Azacitidine and (oral) after Decitabine/Cedazuridine, and splitting questions based on treatment type.

Caregiver interviews revealed that factors like convenience, manageability, clinic time, emotional distress, and costs influenced their treatment preferences. Modifications based on caregiver feedback included changing phrasing to clarify responses, such as "We spend less time in the clinic" to "It means less time in the clinic for the patient."

Patient interviews highlighted similar factors to carers, emphasizing convenience, manageability, clinic time, emotional distress, and treatment burden on caregivers. Cognitive debriefing confirmed that the questionnaires were easy to understand and captured relevant concepts. Modifications included adjusting skip directions and ensuring consistency across questionnaires.

"The mTPMQ, cTPMQ, and pTPMQ were developed with direct input from clinicians, caregivers, and patients to assess the key concepts of interest related to the preference for the treatment of MDS to be used in further studies, including clinical research to investigate patient, carer, and clinician preference for the treatment of MDS."

The study noted that although previous research has explored patient and caregiver preferences, a validated tool for MDS treatment preferences has yet to be developed. The newly developed TPMQs are designed to fill this gap and can be used in future clinical trials to further investigate preferences for HMA treatments, the authors emphasized. These tools will help capture treatment preferences' strengths and reasons, potentially improving treatment adherence and patient outcomes.

References

1. Morlock R, Fong C, Castaldi F, Paine T, Collett D, Enjeti A. Development of the treatment preference in myelodysplasia questionnaire for clinicians, carers, and patients. eJHaem. 2024;5(3):535-540. doi:10.1002/jha2.930

2. Kantarjian H, Issa JP, Rosenfeld CS, et al. Decitabine improves patient outcomes in myelodysplastic syndromes: results of a phase III randomized study. Cancer. 2006;106(8):1794-1803. doi:10.1002/cncr.21792

3. Fenaux P, Mufti GJ, Hellstrom-Lindberg E, et al. Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study. Lancet Oncol. 2009;10(3):223-232. doi:10.1016/S1470-2045(09)70003-8

4. Corman S, Joshi N, Wert T, Kale H, Hill K, Zeidan AM. Under-use of hypomethylating agents in patients with higher-risk myelodysplastic syndrome in the United States: a large population-based analysis. Clin Lymphoma Myeloma Leuk. 2021;21(2):e206-e211. doi:10.1016/j.clml.2020.10.013