Personalized Prophylaxis with myPKFiTCE: A Real-World Cost-Effectiveness Analysis in Haemophilia A Patients
Abstract
:1. Introduction
2. Materials and Methods
2.1. Study Design and Data Source
2.2. Inclusion and Exclusion Criteria
2.3. Data Collection and Outcomes
2.4. Statistical Analysis
3. Results
4. Discussion
5. Conclusions
Author Contributions
Funding
Institutional Review Board Statement
Informed Consent Statement
Data Availability Statement
Acknowledgments
Conflicts of Interest
References
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N° of patients | 13 |
Sex, Male, N (%) | 13 (100.00%) |
Age, Median (Q1–Q3) | 14.00 (9.00–38.00) |
Weight, Kg—Median (Q1–Q3) | 50.00 (29.00–61.00) |
Height, cm—Median (Q1–Q3) | 161.00 (130.00–169.00) |
BMI (Kg/m2) | |
Underweight (<18.50) | 6 (46.15%) |
Normal weight (18.50–29.99) | 6 (46.15%) |
Overweight/Obesity (≥25.00) | 1 (7.69%) |
Blood Type | |
0 Rh− | 2 (15.38%) |
0 Rh+ | 5 (38.46%) |
A Rh− | 1 (7.69%) |
A Rh+ | 3 (23.08%) |
B Rh− | 1 (7.69%) |
B Rh+ | 1 (7.69%) |
Working status | |
Full-time employed | 3 (23.08%) |
Part-time employed | 1 (7.69%) |
Student | 7 (53.85%) |
Unemployed | 2 (15.38%) |
One Year Pre-myPKFiT | One Year Post-myPKFiT | Δ (%) | p-Value | |
---|---|---|---|---|
Total bleeds, N | 25 | 9 | −16 | |
Joint bleeds, N (%) * | 15 (60.00) | 4 (44.44) | ||
Patients with zero bleeds, N (%) | 2 (18.18) | 7 (63.64) | +5 (250.00) | 0.0588 |
All Patients | ||||
ABR | ||||
Median (Q1–Q3) | 2.00 (1.00–4.00) | 0.00 (0.00–1.00) | −2.00 (−100.00) | 0.0103 |
AJBR | ||||
Median (Q1–Q3) | 0.00 (0.00–3.00) | 0.00 (0.00–1.00) | - | 0.0271 |
Patients with ≥1 bleed | ||||
ABR | ||||
Median (Q1–Q3) | 4.00 (4.00–6.00) | 3.00 (1.00–4.00) | −1.00 (−25.00%) | 0.1088 |
AJBR | ||||
Median (Q1–Q3) | 3.00 (3.00–4.00) | 1.00 (1.00–2.00) | −2.00 (−66.67) | 0.1088 |
One Year Pre-myPKFiT | One Year Post-myPKFiT | Δ (%) | |
---|---|---|---|
HJHS Mean (±SD) | 8.45 (13.09) | 6.82 (11.19) | −1.64 (−19.40) |
Prophylaxis | |||
UI/Kg/Infusion Mean (±SD) | 36.18 (13.83) | 43.01 (13.46) | +6.83 (+18.88) |
N Infusion per week, Mean (±SD) | 2.91 (0.30) | 2.55 (0.52) | |
Annual prophylaxis consumption per patient, UI | 210,941.70 | 222,792.40 | +11,850.70 (+5.62) |
Treatment of bleeds | |||
UI/Kg/Infusion Mean (±SD) | 32.42 (9.43) | 32.97 (5.16) | +0.55 (+1.69) |
N of Infusion, Mean (±SD) | 2.80 (1.41) | 2.11 (1.17) | −0.69 (−24.60) |
Annual bleeding consumption per patient, UI | 11,450.00 | 3450.00 | −8000.00 (−69.87) |
Total consumption per patient, IU | 222,391.70 | 226,242.40 | +3850.70 (+1.73) |
Treatments | Total Costs (EUR) | Δ Costs (EUR) | ABR | Δ ABR | ICER (Euros per Bleed Avoided) |
---|---|---|---|---|---|
Pre-myPKFiT | 131,627.621 | - | 2.27 | - | - |
Post-myPKFiT | 139,022.458 | 7394.837 | 0.82 | −1.45 | 5099.89 |
Treatments | Total costs (EUR) | Δ costs (EUR) | AJBR | Δ AJBR | ICER |
Pre-myPKFiT | 131,627.621 | - | 1.36 | ||
Post-myPKFiT | 139,022.458 | 7394.837 | 0.36 | −1.00 | 7394.837 |
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Antonazzo, I.C.; Cortesi, P.A.; Zanon, E.; Pasca, S.; Morfini, M.; Santoro, C.; De Cristofaro, R.; Di Minno, G.; Cozzolino, P.; Mantovani, L.G. Personalized Prophylaxis with myPKFiTCE: A Real-World Cost-Effectiveness Analysis in Haemophilia A Patients. Medicina 2024, 60, 34. https://doi.org/10.3390/medicina60010034
Antonazzo IC, Cortesi PA, Zanon E, Pasca S, Morfini M, Santoro C, De Cristofaro R, Di Minno G, Cozzolino P, Mantovani LG. Personalized Prophylaxis with myPKFiTCE: A Real-World Cost-Effectiveness Analysis in Haemophilia A Patients. Medicina. 2024; 60(1):34. https://doi.org/10.3390/medicina60010034
Chicago/Turabian StyleAntonazzo, Ippazio Cosimo, Paolo Angelo Cortesi, Ezio Zanon, Samantha Pasca, Massimo Morfini, Cristina Santoro, Raimondo De Cristofaro, Giovanni Di Minno, Paolo Cozzolino, and Lorenzo Giovanni Mantovani. 2024. "Personalized Prophylaxis with myPKFiTCE: A Real-World Cost-Effectiveness Analysis in Haemophilia A Patients" Medicina 60, no. 1: 34. https://doi.org/10.3390/medicina60010034