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Pickard AS, Huynh L, Ivanova JI, et al. Value of transfusion independence in severe aplastic anemia from patients' perspectives - a discrete choice experiment. J Patient Rep Outcomes. 2018;2(1):13doi: 10.1186/s41687-018-0032-y.
Pickard, A. S., Huynh, L., Ivanova, J. I., Totev, T., Graham, S., Mühlbacher, A. C., Roy, A., & Duh, M. S. (2017). Value of transfusion independence in severe aplastic anemia from patients' perspectives - a discrete choice experiment. Journal of patient-reported outcomes, 2(1), 13. https://doi.org/10.1186/s41687-018-0032-y
Pickard, A Simon, et al. "Value of transfusion independence in severe aplastic anemia from patients' perspectives - a discrete choice experiment." Journal of patient-reported outcomes vol. 2,1 (2017): 13. doi: https://doi.org/10.1186/s41687-018-0032-y
Pickard AS, Huynh L, Ivanova JI, Totev T, Graham S, Mühlbacher AC, Roy A, Duh MS. Value of transfusion independence in severe aplastic anemia from patients' perspectives - a discrete choice experiment. J Patient Rep Outcomes. 2017;2(1):13. doi: 10.1186/s41687-018-0032-y. Epub 2018 Mar 01. PMID: 29757294; PMCID: PMC5934914.
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J Patient Rep Outcomes. 2017;2(1):13. doi: 10.1186/s41687-018-0032-y. Epub 2018 Mar 01.

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

Journal of patient-reported outcomes

A Simon Pickard, Lynn Huynh, Jasmina I Ivanova, Todor Totev, Sophia Graham, Axel C Mühlbacher, Anuja Roy, Mei Sheng Duh

Affiliations

  1. Second City Outcomes Research, Chicago, IL USA.
  2. 2Analysis Group, Inc., 111 Huntington Avenue, 14th Floor, Boston, MA 02199 USA.
  3. 3Analysis Group, Inc., New York, NY USA.
  4. 4Analysis Group, Inc., Menlo Park, CA USA.
  5. 5IGM Institute Health Economics and Health Care Management at Hochschule Neubrandenburg, Neubrandenburg, Germany.
  6. 6Novartis Pharmaceuticals Corporation, East Hanover, NJ USA.

PMID: 29757294 PMCID: PMC5934914 DOI: 10.1186/s41687-018-0032-y

Abstract

BACKGROUND: 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.

METHODS: 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 scores for transfusion frequency levels were reported.

RESULTS: Thirty patients completed the survey. Most were age ≥ 40 years (73.3%), female (70.0%), and from the US (86.7%). 33.3% underwent bone marrow transplant; 36.7% received iron chelation therapy. Patients largely agreed that transfusion independence would result in less burden on time and costs, greater control and quality of life, less fatigue (86.7% noted each) and less scheduling around medical appointments (83.3%). The DCE found highest relative importance for risk of bleeding (0.30), followed by risk of infection (0.28), fatigue (0.23), and frequency of transfusions (0.20). More frequent transfusions resulted in lower utility, particularly when increasing monthly transfusions frequency from 4 (0.57) to 8 (0.35).

CONCLUSIONS: Our study showed that higher utility was associated with fewer transfusions in SAA patients with insufficient response to immunosuppressive therapy. While risk of bleeding, risk of infection, and fatigue were more important for patient treatment preferences, frequency of transfusions was also important.

Keywords: Bone marrow failure; Discrete choice experiment; Preference; Severe aplastic anemia; Transfusions

Conflict of interest statement

All study materials have been reviewed and approved by the Ethical and Independent Review Services (E&I) Institutional Review Board (E&I study number 15128–01). No compensation was offered and informe

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