Abstract Number: PB0598
Meeting: ISTH 2021 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical
Background: The international Antihemophilic factor (recombinant) (rAHF) Hemophilia A (HA) outcome Database (AHEAD) study is assessing the long-term effectiveness and safety of rAHF in patients with HA in real-world clinical practice.
Aims: To report demographic and clinical characteristics at screening of the AHEAD Brazil patient subset.
Methods: AHEAD is a prospective, non-interventional, multicenter study (NCT02078427) that includes patients of any age with HA (factor VIII ≤5%). The study was approved in Brazil on July 14, 2015. Ethics committee approval and patients’ informed consent were obtained. The primary endpoint is joint health outcomes evaluated by Gilbert score (pain, 0-3; bleeding, 0-3; physical exam, 0-12). Investigators can choose Hemophilia Joint Health Score (HJHS) as an alternative to Gilbert Score. Secondary endpoints include annualized bleeding rates (ABRs; all bleeds), annualized joint bleeding rates (AJBRs), and safety endpoints. Here, we report demographic and clinical characteristics at screening from the safety analysis set (SAS) for patients in the AHEAD Brazil subset. These data were extracted from the 6th interim analysis of the AHEAD International study (data cutoff: July 15, 2019).
Results: The AHEAD Brazil SAS included 203 patients (median [range] age, 13.0 [0-43] years): 190 received prophylaxis (median [range] age, 14.0 [0-43] years), 2 received on-demand treatment (median [range] age, 12.0 [0-24] years), and 11 patients with inhibitors received immune tolerance induction (ITI; median [range] age, 12.0 [3-34] years). All patients were male (Table 1). At screening, bleeding events had occurred in 130 (68.4%) prophylaxis patients, 1 (50.0%) on-demand patient, and 4 (36.4%) patients receiving ITI (Table 2). Bleeding events recorded at screening had occurred up to 12 months prior to screening.
Prophylaxis (n=190) |
On-demand (n=2) |
ITI (n=11) |
Total (N=203) |
|
Ethnicity, n (%) | ||||
White | 98 (51.6) | 1 (50.0) | 6 (54.5) | 105 (51.7) |
Asian | 3 (1.6) | 0 (0.0) | 0 (0.0) | 3 (1.5) |
Black or African American | 27 (14.2) | 0 (0.0) | 3 (27.3) | 30 (14.8) |
Other | 62 (32.6) | 1 (50.0) | 2 (18.2) | 65 (32.0) |
Hemophilia A severity, n (%) | ||||
Factor VIII 1-5% | 35 (18.4) | 0 (0.0) | 2 (18.2) | 37 (18.2) |
Factor VIII <1% | 155 (81.6) | 2 (100) | 9 (81.8) | 166 (81.8) |
Prophylaxis | On-demand | ITI | |
Average Gilbert score (all joints), n | 35 | – | 1 |
Mean ± SD Median (range) |
1.7 ± 1.6 1.0 (0.0-5.0) |
– | 1.0 1.0 (1.0-1.0) |
HJHS: Global Gait Score, n | 86 | 0 | 8 |
Mean ± SD Median (range) |
1.6 ± 1.6 1.0 (0.0-4.0) |
– | 1.8 ± 1.5 1.0 (0.0-4.0) |
ABR, n | 190 | 2 | 11 |
Mean ± SD Median (range) |
3.3 ± 4.8 2.0 (0.0-30.0) |
5.0 ± 7.1 5.0 (0.0-10.0) |
4.0 ± 7.9 0.0 (0.0-26.0) |
AJBR,† n | 190 | 2 | 11 |
Mean ± SD Median (range) |
2.2 ± 3.9 1.0 (0.0-30.0) |
4.5 ± 6.4 4.5 (0.0-9.0) |
2.6 ± 5.6 0.0 (0.0-19.0) |
*Bleedings occurring during regimens of <90 days were not used for this analysis. Bleedings recorded at screening had occurred up to 12 months prior to screening. Patients with >1 regimen during a study year (switch patients) were counted for each regimen; bleeding events were only counted for the regimen during which they occurred. †Owing to missing information for joint/non-joint classification, AJBRs may be underestimated. |
Conclusions: The AHEAD Brazil subset, which is part of the larger AHEAD International study, included mostly patients receiving prophylaxis. Future analyses will investigate the clinical outcomes of patients in the AHEAD Brazil subset.
To cite this abstract in AMA style:
Cerqueira MH, Ferreira C, Lorenzato C, Carvalho LEM, C Oliveira de Oliveira L, Pinto I, Prezotti A, Norton J, Tang L, C Ozelo M. Demographic and Clinical Characteristics of Patients with Hemophilia A in Brazil: Real-world Data from the 6th Interim Analysis of the AHEAD International Study [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/demographic-and-clinical-characteristics-of-patients-with-hemophilia-a-in-brazil-real-world-data-from-the-6th-interim-analysis-of-the-ahead-international-study/. Accessed March 21, 2024.« Back to ISTH 2021 Congress
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