Value of transfusion independence in severe aplastic anemia from patients' perspectives - a discrete choice experiment

Journal of Patient-reported Outcomes
A Simon PickardMei Sheng Duh

Abstract

Aplastic anemia is a rare, serious blood disorder due to bone marrow failure to produce blood cells. Transfusions are used to reduce risk of bleeding, infection and relieve anemia symptoms. In severe patients, transfusions may be required more than once/week. It is unclear from the patient perspective the impact that transfusions have on quality of life. This study aimed to elicit patient preferences for attributes associated with severe aplastic anemia (SAA) treatment, including transfusion independence. An online discrete choice experiment (DCE) was conducted among patients with SAA who experienced insufficient response to immunosuppressive therapy and transfusion dependence for ≥3 months in the past 2 years. Recruitment occurred through the Aplastic Anemia and Myelodysplastic Syndromes International Foundation and referrals from clinical sites in the US and France. Respondents chose between hypothetical treatment pairs characterized by a common set of attributes: transfusions frequency, fatigue, risk of infection, and risk of serious bleeding. Conditional logit model with effects coding was used to estimate part-worth utilities for different attribute levels and the relative importance of each attribute. Predicted utility sc...Continue Reading

References

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Citations

Apr 6, 2021·Journal of the Endocrine Society·Keziah CookRebecca J Brown

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Methods Mentioned

BETA
MDS

Software Mentioned

SAS
Sawtooth Software

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