HEALTH STATE UTILITIES ASSOCIATED WITH TREATMENT FOR TRANSFUSION DEPENDENT ß-THALASSEMIA IN FRANCE
Author(s)
Louis Matza, PhD1, Adeline Durand, PhD2, Katie D. Stewart, MA1, Jennifer Drahos, BS, MA, MPH, MPhil, PhD2, Gabriela Vega-Hernandez, BSc, MSc2.
1Thermo Fisher Scientific, Bethesda, MD, USA, 2Vertex Pharmaceuticals, Inc., Boston, MA, USA.
1Thermo Fisher Scientific, Bethesda, MD, USA, 2Vertex Pharmaceuticals, Inc., Boston, MA, USA.
OBJECTIVES: Transfusion-dependent β-thalassemia (TDT) is a rare disease that affects the production of healthy red blood cells. Treatments for TDT include lifelong blood transfusions with iron chelation therapy, allogeneic hematopoietic stem cell transplant (allo-HSCT), and autologous-HSCT with gene therapy. Utilities associated with TDT-related health states were previously estimated in a vignette-based study in England. This study replicates that research in France to capture local preferences.
METHODS: Participants from the French general population in Paris valued eight health states in time trade-off interviews. The English study vignettes were translated into French and validated by a local clinical expert. Two vignettes described pre-transplant TDT with ongoing transfusions and iron chelation, three described the transplant year, and three described post-transplant health states. Graft-versus-host disease (GvHD), a possible complication of allo-HSCT, was included in two vignettes.
RESULTS: A total of 207 participants completed interviews (50.2% male; mean age 45.6 years). Mean (SD) utilities for pre-transplant states were 0.77 (0.20) for TDT with oral chelation and 0.65 (0.30) for TDT with subcutaneous chelation. Mean utilities for the transplant year were 0.66 (0.37) for autologous-HSCT, 0.58 (0.40) for allo-HSCT, and 0.48 (0.42) for allo-HSCT with acute GvHD. Post-transplant health states, utilities were 0.92 (0.08) for transfusion independence, 0.76 (0.20) for 60% transfusion reduction, and 0.72 (0.28) for transfusion independence with chronic GvHD. Patterns were similar to those observed in England, although French utilities tended to be higher. Differences between countries were relatively small (0.01 to 0.10), except for post allo-HSCT with chronic GvHD (difference of 0.21).
CONCLUSIONS: Utilities followed expected patterns, with more burdensome treatment processes and side effects associated with lower utilities. Differences between England and France highlight the value of vignettes for eliciting local preferences, particularly in rare diseases, and support the use of country-specific utilities in cost-utility models estimating the value of treatments for TDT.
METHODS: Participants from the French general population in Paris valued eight health states in time trade-off interviews. The English study vignettes were translated into French and validated by a local clinical expert. Two vignettes described pre-transplant TDT with ongoing transfusions and iron chelation, three described the transplant year, and three described post-transplant health states. Graft-versus-host disease (GvHD), a possible complication of allo-HSCT, was included in two vignettes.
RESULTS: A total of 207 participants completed interviews (50.2% male; mean age 45.6 years). Mean (SD) utilities for pre-transplant states were 0.77 (0.20) for TDT with oral chelation and 0.65 (0.30) for TDT with subcutaneous chelation. Mean utilities for the transplant year were 0.66 (0.37) for autologous-HSCT, 0.58 (0.40) for allo-HSCT, and 0.48 (0.42) for allo-HSCT with acute GvHD. Post-transplant health states, utilities were 0.92 (0.08) for transfusion independence, 0.76 (0.20) for 60% transfusion reduction, and 0.72 (0.28) for transfusion independence with chronic GvHD. Patterns were similar to those observed in England, although French utilities tended to be higher. Differences between countries were relatively small (0.01 to 0.10), except for post allo-HSCT with chronic GvHD (difference of 0.21).
CONCLUSIONS: Utilities followed expected patterns, with more burdensome treatment processes and side effects associated with lower utilities. Differences between England and France highlight the value of vignettes for eliciting local preferences, particularly in rare diseases, and support the use of country-specific utilities in cost-utility models estimating the value of treatments for TDT.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
PCR29
Topic
Patient-Centered Research
Topic Subcategory
Health State Utilities
Disease
Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)