Abstract Number: PB0554
Meeting: ISTH 2021 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical
Background: Individualizing treatment dose and frequency using pharmacokinetic (PK)-guided prophylaxis can help achieve and maintain targeted factor VIII (FVIII) trough levels in patients with hemophilia A.
Aims: To describe and compare clinical and resource use outcomes between patients with hemophilia A treated with or without PK-guided dosing using data from the Cost of Haemophilia in Europe: A Socioeconomic Survey (CHESS) II database.
Methods: CHESS II was a cross-sectional, retrospective, prevalence-based, burden-of-illness study using data collected across 8 European countries from November 2018 to July 2019. Patients eligible for this analysis were male, ≥18 years of age, and diagnosed with congenital hemophilia A of any severity. The clinical endpoints analyzed were annualized bleeding rate (ABR), joint deterioration (presence and number of problem/target joints), and occurrence of joint surgeries. Resource utilization endpoints included the number of hematologist consultations and bleed-related hospitalizations or emergency department admissions. Data were stratified according to the use of PK-guided dosing.
Results: A total of 155 patients on prophylactic treatment had available FVIII trough level data. Mean (SD) age was 38.6 (14.4) years. Of these, a specific FVIII trough level was targeted in 72 (46.5%) patients and 16 (22.2%) received PK-guided dosing. Patients receiving a PK-guided dose had a mean (SD) ABR of 1.6 (1.1) and a mean (SD) number of target joints of 0.6 (0.8) compared with 4.4 (2.8) and 1.2 (1.5), respectively, for patients whose dose was not PK-guided (Table 1). The mean (SD) number of hematologist consultations was 6.2 (2.9) for patients receiving PK-guided dosing versus 11.8 (5.7) for those who were not. A higher proportion of patients required hospitalization during their lifetime if they did not receive PK-guided dosing compared with those who did (Table 2).
| PK-guided dosing Yes (n=16) |
PK-guided dosing No (n=56) |
|
|---|---|---|
| ABR, mean (SD) | 1.6 (1.1) | 4.4 (2.8) |
| Presence of target joints, n (%) | 7 (43.8) | 30 (53.6) |
| Number of target joints, mean (SD) | 0.6 (0.8) | 1.2 (1.5) |
| Presence of problem joints, n (%) | 11 (68.8) | 29 (51.8) |
| Number of problem joints mean (SD) | 1.1 (0.9) | 0.9 (1.2) |
| ABR, annualized bleeding rate; NS, not significant; PK, pharmacokinetic | ||
ABR and joint health outcomes in patients with available PK information (n=72)
| PK-guided dosing Yes (n=16) |
PK-guided dosing No (n=56) |
|
|---|---|---|
| Proportion of patients needing hospitalization during their lifetime, n (%) | 10 (62.5) | 49 (87.5) |
| Proportion of patients needing hospitalization in the 12 months prior to data collection, n (%) | 7 (43.8) | 27 (48.2) |
| Details for hospitalizations occurring in the 12 months prior to data collection, mean (SD) | ||
| Number of day cases (n=72) | 0.2 (0.5) | 0.0 (0.2) |
| Number of days (n=4) | 1.5 (0.7) | 1.0 (0.0) |
| Number of ward hospitalizations (n=72) | 0.3 (0.4) | 0.8 (1.1) |
| Number of days on ward (n=31) | 5.2 (4.0) | 5.8 (4.1) |
| Number of ICU hospitalizations (n=72) | 0.1 (0.3) | 0.1 (0.3) |
| Number of days in ICU (n=5) | 1.0 (-) | 1.5 (0.6) |
| ICU, intensive care unit; NS, not significant; PK, pharmacokinetic | ||
Bleed-related hospitalizations in patients with available PK information (n=72)
Conclusions: This analysis provides real-world evidence suggesting a beneficial impact of PK-guided dosing for prophylaxis on clinical and health resource utilization outcomes.
To cite this abstract in AMA style:
Ferri Grazzi E, Burke T, O'Hara J, X Sun S. The Impact of PK-guided Prophylaxis on Clinical Outcomes and Resource Utilization in Hemophilia A Patients: Real-world Evidence From the CHESS II Study [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/the-impact-of-pk-guided-prophylaxis-on-clinical-outcomes-and-resource-utilization-in-hemophilia-a-patients-real-world-evidence-from-the-chess-ii-study/. Accessed December 10, 2023.« Back to ISTH 2021 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/the-impact-of-pk-guided-prophylaxis-on-clinical-outcomes-and-resource-utilization-in-hemophilia-a-patients-real-world-evidence-from-the-chess-ii-study/