Economic and Humanistic Burden of Moderate and Severe Hemophilia A and B in Spain: Real-World Evidence Insights from the CHESS II Study
**Background:** Hemophilia is a congenital disorder characterized by deficiency or absence of clotting factor VIII in hemophilia A (HA) or clotting factor IX in hemophilia B (HB), resulting in frequent, repeated, and prolonged spontaneous or traumatic bleeding into joints or soft tissue. Severity is...
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Columbia Data Analytics, LLC
2024-05-01
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| Series: | Journal of Health Economics and Outcomes Research |
| Online Access: | https://doi.org/10.36469/001c.92369 |
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| author | Carmen Peral Alfonso De Lossada Juste Nadia Lwoff Nataly Espinoza-Cámac Miguel Ángel Casado Tom Burke Jose Alvir Sheena Thakkar Enrico Ferri Grazzi |
| author_facet | Carmen Peral Alfonso De Lossada Juste Nadia Lwoff Nataly Espinoza-Cámac Miguel Ángel Casado Tom Burke Jose Alvir Sheena Thakkar Enrico Ferri Grazzi |
| author_sort | Carmen Peral |
| collection | DOAJ |
| description | **Background:** Hemophilia is a congenital disorder characterized by deficiency or absence of clotting factor VIII in hemophilia A (HA) or clotting factor IX in hemophilia B (HB), resulting in frequent, repeated, and prolonged spontaneous or traumatic bleeding into joints or soft tissue. Severity is classified by the patient's baseline level of clotting factor activity as mild (>5%-40%), moderate (1%-5%), or severe (<1%). In Spain, there is limited information on the societal economic burden of disease.
**Objective:** To estimate the economic and humanistic burden of disease in adult patients with non-inhibitor moderate and severe HA and HB in Spain.
**Methods:** Spanish data from the CHESS II study (2018-2020) on patients' clinical characteristics, health-related quality of life (HRQoL) and hemophilia-related healthcare resource utilization were analyzed. Economic burden was determined by estimating condition-related annual per-patient direct (medical and nonmedical) and indirect costs, stratified according to hemophilia type and severity and presented as 2022 Euros. HRQoL was assessed via the EQ-5D-5L.
**Results:** Of 341 patients in the Spanish CHESS II cohort, 288 patients met the inclusion criteria: 181 had HA (37% \[n = 66\] moderate and 63% \[n=115\] severe) and 107 had HB (26% \[n = 28\] moderate and 74% \[n = 79\] severe). Mean annual direct cost was higher in HB than in HA, and higher in severe than in moderate patients, resulting in an annual cost/patient of €17 251 (moderate HA), €17 796 (moderate HB), €116 767 (severe HA) and €206 996 (severe HB). The main direct cost component in all groups except moderate HA was factor replacement therapy. Mean per-patient indirect cost was €4089 (moderate HA), €797 (moderate HB), €8633 (severe HA) and €8049 (severe HB). Finally, the mean total cost (direct and indirect) for moderate and severe patients were €91 017 (HA) and €163 924 (HB). EQ-5D-5L \[SD\] scores were lower in patients with severe HA (0.77 \[0.18\]) and severe HB (0.70 \[0.22\]) compared with patients with moderate HA (0.81 \[0.15\]) and moderate HB (0.86 \[0.17\]).
**Conclusions:** Independently of the type of hemophilia, greater condition severity was associated with increased costs and a decrease in HRQoL. |
| format | Article |
| id | doaj-art-5efc18f288da4291bf3ac5dd4d52055e |
| institution | Kabale University |
| issn | 2327-2236 |
| language | English |
| publishDate | 2024-05-01 |
| publisher | Columbia Data Analytics, LLC |
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| series | Journal of Health Economics and Outcomes Research |
| spelling | doaj-art-5efc18f288da4291bf3ac5dd4d52055e2025-02-10T16:13:21ZengColumbia Data Analytics, LLCJournal of Health Economics and Outcomes Research2327-22362024-05-01111Economic and Humanistic Burden of Moderate and Severe Hemophilia A and B in Spain: Real-World Evidence Insights from the CHESS II StudyCarmen PeralAlfonso De Lossada JusteNadia LwoffNataly Espinoza-CámacMiguel Ángel CasadoTom BurkeJose AlvirSheena ThakkarEnrico Ferri Grazzi**Background:** Hemophilia is a congenital disorder characterized by deficiency or absence of clotting factor VIII in hemophilia A (HA) or clotting factor IX in hemophilia B (HB), resulting in frequent, repeated, and prolonged spontaneous or traumatic bleeding into joints or soft tissue. Severity is classified by the patient's baseline level of clotting factor activity as mild (>5%-40%), moderate (1%-5%), or severe (<1%). In Spain, there is limited information on the societal economic burden of disease. **Objective:** To estimate the economic and humanistic burden of disease in adult patients with non-inhibitor moderate and severe HA and HB in Spain. **Methods:** Spanish data from the CHESS II study (2018-2020) on patients' clinical characteristics, health-related quality of life (HRQoL) and hemophilia-related healthcare resource utilization were analyzed. Economic burden was determined by estimating condition-related annual per-patient direct (medical and nonmedical) and indirect costs, stratified according to hemophilia type and severity and presented as 2022 Euros. HRQoL was assessed via the EQ-5D-5L. **Results:** Of 341 patients in the Spanish CHESS II cohort, 288 patients met the inclusion criteria: 181 had HA (37% \[n = 66\] moderate and 63% \[n=115\] severe) and 107 had HB (26% \[n = 28\] moderate and 74% \[n = 79\] severe). Mean annual direct cost was higher in HB than in HA, and higher in severe than in moderate patients, resulting in an annual cost/patient of €17 251 (moderate HA), €17 796 (moderate HB), €116 767 (severe HA) and €206 996 (severe HB). The main direct cost component in all groups except moderate HA was factor replacement therapy. Mean per-patient indirect cost was €4089 (moderate HA), €797 (moderate HB), €8633 (severe HA) and €8049 (severe HB). Finally, the mean total cost (direct and indirect) for moderate and severe patients were €91 017 (HA) and €163 924 (HB). EQ-5D-5L \[SD\] scores were lower in patients with severe HA (0.77 \[0.18\]) and severe HB (0.70 \[0.22\]) compared with patients with moderate HA (0.81 \[0.15\]) and moderate HB (0.86 \[0.17\]). **Conclusions:** Independently of the type of hemophilia, greater condition severity was associated with increased costs and a decrease in HRQoL.https://doi.org/10.36469/001c.92369 |
| spellingShingle | Carmen Peral Alfonso De Lossada Juste Nadia Lwoff Nataly Espinoza-Cámac Miguel Ángel Casado Tom Burke Jose Alvir Sheena Thakkar Enrico Ferri Grazzi Economic and Humanistic Burden of Moderate and Severe Hemophilia A and B in Spain: Real-World Evidence Insights from the CHESS II Study Journal of Health Economics and Outcomes Research |
| title | Economic and Humanistic Burden of Moderate and Severe Hemophilia A and B in Spain: Real-World Evidence Insights from the CHESS II Study |
| title_full | Economic and Humanistic Burden of Moderate and Severe Hemophilia A and B in Spain: Real-World Evidence Insights from the CHESS II Study |
| title_fullStr | Economic and Humanistic Burden of Moderate and Severe Hemophilia A and B in Spain: Real-World Evidence Insights from the CHESS II Study |
| title_full_unstemmed | Economic and Humanistic Burden of Moderate and Severe Hemophilia A and B in Spain: Real-World Evidence Insights from the CHESS II Study |
| title_short | Economic and Humanistic Burden of Moderate and Severe Hemophilia A and B in Spain: Real-World Evidence Insights from the CHESS II Study |
| title_sort | economic and humanistic burden of moderate and severe hemophilia a and b in spain real world evidence insights from the chess ii study |
| url | https://doi.org/10.36469/001c.92369 |
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