Experience of advate rahf-pfm in previously untreated patients and minimally treated patients with haemophilia a

Guenter Auerswald, Alexis A. Thompson, Michael Recht, Deborah Brown, Raina Liesner, Norma Guzmán-Becerra, Jacqueline Dyck-Jones, Bruce Ewenstein, Brigitt Abbuehl

Research output: Contribution to journalArticle

22 Scopus citations

Abstract

We report a prospective trial of 55 previously untreated patients (PUPs) and minimally treated patients (MTPs) with severe/moderately severe haemophilia A (baseline factor VIII [FVIII] ≤2%) treated with a single FVIII replacement product. It was the objective of this study to evaluate the immunogenicity, efficacy, and safety of rAHF-PFM (Advate ®). On-demand or prophylactic treatment regimens were determined at the discretion of the investigator. rAHF-PFM was also permitted for peri-operative management. There were 633 bleeding episodes (BEs), including 517 treated, and 466 rated for efficacy. Haemostatic efficacy was considered excellent/good in 93% of 466 rated treatments. Of 517 treated BEs, 463/517 (90%) were managed with one (356/517 [69%]) or two infusions (107/517 [21%]). There were 27 surgeries. Intraoper-ative (n=22) and postoperative (n=25) haemostatic efficacies were considered excellent or good in 100% of rated surgeries. Related serious adverse events (SAEs) were inhibitor development in 16/55 (29.1%) subjects who received at least one infusion of rAHF-PFM. Non-serious, related adverse events (AEs) were few in number (14 in eight subjects). The odds ratio (OR [95% Confidence Interval, CI]) of developing inhibitors was significantly higher in subjects with a family history of inhibitor (4.95 [1.29-19.06]), non-Caucasian ethnicity (4.18, [1.18-14.82]), and intensive treatment at high dose (4.5 [1.05-19.25]) within ≤20 exposure days (EDs). In conclusion, rAHF-PFM was safe and effective for the management and perioperative coverage of PUPs/ MTPs with severe/moderately severe haemophilia A. This report supports previous findings from studies in which family history of inhibitor, non-Caucasian ethnicity, and high intensity treatment were associated with high risk of inhibitor development.

Original languageEnglish (US)
Pages (from-to)1072-1082
Number of pages11
JournalThrombosis and Haemostasis
Volume107
Issue number6
DOIs
StatePublished - Jun 2012

Keywords

  • Factor viii
  • Factor viii inhibitor
  • Haemophilia a
  • Minimally treated patients
  • Paediatric
  • Previously untreated patients

ASJC Scopus subject areas

  • Hematology

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    Auerswald, G., Thompson, A. A., Recht, M., Brown, D., Liesner, R., Guzmán-Becerra, N., Dyck-Jones, J., Ewenstein, B., & Abbuehl, B. (2012). Experience of advate rahf-pfm in previously untreated patients and minimally treated patients with haemophilia a. Thrombosis and Haemostasis, 107(6), 1072-1082. https://doi.org/10.1160/TH11-09-0642