Abstract Number: PB0900
Meeting: ISTH 2020 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical
Background: The Antihemophilic factor (recombinant) (rAHF) Hemophilia A outcome Database (AHEAD) study evaluates long-term effectiveness and safety outcomes in patients with hemophilia A receiving rAHF in routine clinical practice.
Aims: This 6-year interim analysis assessed age-stratified joint health in AHEAD study patients receiving either prophylactic (PRO) or on-demand (OD) rAHF.
Methods: AHEAD is an international, non-interventional, prospective, multicenter study (NCT02078427, started in 2011) including patients with moderate (factor VIII activity 1-5%) or severe (factor VIII < 1%) hemophilia A. Ethics committee approval and patients' informed consent were obtained. The primary objective for AHEAD is to describe joint health outcomes. In this analysis, joint health was assessed using the pain (score 0-3), bleeding (score 0-3), and physical exam (score 0-12) Gilbert scale parameters; higher scores for each category represent worsening conditions. Here we report results of the 6-year interim descriptive analysis in patients aged 2 to < 12, 12 to < 18, and ≥ 18 years with available Gilbert score data (data cutoff: 15 July 2019).
Results: At year 1, Gilbert score data from 52 children (2 to < 12 years; all PRO), 19 adolescents
(12 to < 18 years; all PRO), and 116 adults (≥ 18 years; PRO, n=86; OD, n=30) were available for analysis. In PRO children, average Gilbert scores remained consistently low throughout the observation period (Table 1); scores were lower in children and adolescents than in adults. Among adults aged ≥ 18 years, means and medians for average Gilbert scores were consistently lower in patients receiving rAHF PRO versus OD; this may represent a clinically meaningful difference. No new safety signals were observed for this 6-year interim analysis versus previous analyses.
| Age group (treatment), n | Year 1 | Year 2 | Year 3 | Year 4 | Year 5 | Year 6 |
| 2 to <12 years (PRO), n | 52 | 46 | 28 | 16 | 6 | 2 |
| Mean ± SD / median (range) | 0.8 ± 1.4 / 0.3 (0.0-8.0) | 0.6 ± 1.3 / 0.1 (0.0-8.0) | 0.5 ± 0.7 / 0.1 (0.0-2.0) | 0.3 ± 0.6 / 0.0 (0.0-2.0) | 0.4 ± 0.8 / 0.0 (0.0-2.0) | 0.2 ± 0.2 / 0.2 (0.0-0.3) |
| 12 to <18 years (PRO), n | 19 | 19 | 11 | 6 | 1 | 2 |
| Mean ± SD / median (range) | 0.8 ± 1.3 / 0.2 (0.0-5.0) | 0.9 ± 1.4 / 0.0 (0.0-4.0) | 0.9 ± 0.9 / 0.7 (0.0-3.0) | 1.8 ± 2.8 / 0.5 (0.0-7.0) | 0.0 / 0.0 | 0.0 ± 0.0 / 0.0 (0.0-0.0) |
| ≥18 years (PRO), n | 86 | 78 | 59 | 31 | 16 | 8 |
| Mean ± SD / median (range) | 2.7 ± 3.0 / 2.0 (0.0-14.5) | 2.5 ± 2.6 / 2.0 (0.0-13.0) | 2.9 ± 3.2 / 2.3 (0.0-17.0) | 2.1 ± 2.0 / 1.0 (0.0-6.3) | 1.7 ± 1.9 / 0.9 (0.0-6.0) | 1.4 ± 1.2 / 1.2 (0.0-3.2) |
| ≥18 years (OD), n | 30 | 28 | 18 | 8 | 5 | 2 |
| Mean ± SD / median (range) | 3.6 ± 2.7 / 3.0 (0.0-10.0) | 3.6 ± 2.9 / 3.3 (0.0-12.0) | 4.9 ± 2.6 / 4.9 (0.3-9.0) | 3.9 ± 2.8 / 4.2 (0.0-9.0) | 2.1 ± 2.2 / 1.4 (0.0-5.0) | 6.3 ± 5.3 / 6.3 (2.5-10.0) |
| *3 dimensions: pain, bleeding, physical exam. OD, on demand treatment; PRO, prophylactic treatment; rAHF, antihemophilic factor (recombinant). | ||||||
[Age-categorized average Gilbert score* across all joints over 6 years in patients with hemophilia A receiving rAHF PRO or OD]
Conclusions: These real-world data in patients with moderate and severe hemophilia A support early initiation of PRO rAHF as a treatment strategy to preserve joint health.
To cite this abstract in AMA style:
Ozelo MC, Santagostino E, Klamroth R, Guillet B, Khair K, Tsakiris DA, Botha J, Fernandez A, Tang L, Oldenburg J. Joint Health Outcomes in Patients with Hemophilia A Receiving Antihemophilic Factor (Recombinant) in a Real-World Setting: Results of a 6-Year Interim Analysis of the AHEAD International Study [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/joint-health-outcomes-in-patients-with-hemophilia-a-receiving-antihemophilic-factor-recombinant-in-a-real-world-setting-results-of-a-6-year-interim-analysis-of-the-ahead-international-study/. Accessed October 1, 2023.« Back to ISTH 2020 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/joint-health-outcomes-in-patients-with-hemophilia-a-receiving-antihemophilic-factor-recombinant-in-a-real-world-setting-results-of-a-6-year-interim-analysis-of-the-ahead-international-study/