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Introduction <p>Preferences between treatment characteristics can be understood and quantified by combining discrete choice experiment (DCE) and time trade-off (TTO) (DCE<sub>TTO</sub>) methods. The goal is to elicit preferences for people with hemophilia&#xa0;A (PWHA) and quantify impacts of treatment attribute changes on patient health utility with DCE<sub>TTO</sub>.</p> Methods <p>A total of 119 PWHA from a hemophilia treatment center and the National Bleeding Disorders Foundation Community Voices in Research were recruited. Their sociodemographics, clinical characteristics, and EQ-5D-5L scores were gathered. Treatment attributes for the DCE survey were based on the core outcome set for hemophilia gene therapy (coreHEM). For DCE<sub>TTO</sub>, 10-, 15-, and 20-year life durations were used. In total 115 PWHA completed 12 DCE and DCE<sub>TTO</sub> tasks, which were analyzed using conditional logistic models.</p> Results <p>Moderately burdensome treatment (24% treat &gt; once/week) was reported by 67%. Mean EQ-5D-5L visual analogue score was 75; mean utility score was 0.684. All DCE attributes were significant; treatment administration was most important (2–3 intravenous [IV] infusions/week vs. a single IV infusion with 10-year durability, regression coefficient [RC], − 1.99), followed by mental health (always vs. no concern, RC, − 1.37), annual bleeding frequency (≥ 5 vs. none, RC, − 0.73), and chronic pain (yes vs. no, RC, − 0.36). For DCE<sub>TTO</sub>, multiple IV infusions weekly and multiple monthly subcutaneous (SQ) injections were associated with annualized utility decrements (0.046 vs. 10-year durability, 0.044 vs. 5-year; 0.037 vs. 10-year, 0.030 vs. 5-year, respectively).</p> Conclusion <p>All coreHEM outcomes are important for treatment choices of PWHA. One-time IV infusion with 5- to 10-year durability can provide important patient utility gains over lifelong repeated SQ injections or IV infusions.</p>

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Estimating the Impact of Hemophilia A Treatment Administration on Patient Health Utility: Combining a Discrete Choice Experiment with Time Trade-Off Method

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年3月24日

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  • Melody S. Benton,
  • Dongzhe Hong,
  • Mark W. Skinner,
  • Louis P. Garrison,
  • Milad Karimi,
  • Er Chen,
  • Henry Mead,
  • Lizheng Shi

摘要

Introduction

Preferences between treatment characteristics can be understood and quantified by combining discrete choice experiment (DCE) and time trade-off (TTO) (DCETTO) methods. The goal is to elicit preferences for people with hemophilia A (PWHA) and quantify impacts of treatment attribute changes on patient health utility with DCETTO.

Methods

A total of 119 PWHA from a hemophilia treatment center and the National Bleeding Disorders Foundation Community Voices in Research were recruited. Their sociodemographics, clinical characteristics, and EQ-5D-5L scores were gathered. Treatment attributes for the DCE survey were based on the core outcome set for hemophilia gene therapy (coreHEM). For DCETTO, 10-, 15-, and 20-year life durations were used. In total 115 PWHA completed 12 DCE and DCETTO tasks, which were analyzed using conditional logistic models.

Results

Moderately burdensome treatment (24% treat > once/week) was reported by 67%. Mean EQ-5D-5L visual analogue score was 75; mean utility score was 0.684. All DCE attributes were significant; treatment administration was most important (2–3 intravenous [IV] infusions/week vs. a single IV infusion with 10-year durability, regression coefficient [RC], − 1.99), followed by mental health (always vs. no concern, RC, − 1.37), annual bleeding frequency (≥ 5 vs. none, RC, − 0.73), and chronic pain (yes vs. no, RC, − 0.36). For DCETTO, multiple IV infusions weekly and multiple monthly subcutaneous (SQ) injections were associated with annualized utility decrements (0.046 vs. 10-year durability, 0.044 vs. 5-year; 0.037 vs. 10-year, 0.030 vs. 5-year, respectively).

Conclusion

All coreHEM outcomes are important for treatment choices of PWHA. One-time IV infusion with 5- to 10-year durability can provide important patient utility gains over lifelong repeated SQ injections or IV infusions.