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CC BY-NC-ND 4.0 · Thromb Haemost 2012; 107(06): 1072-1082
DOI: 10.1160/TH11-09-0642
Blood Coagulation, Fibrinolysis and Cellular Haemostasis
Schattauer GmbH

Experience of Advate rAHF-PFM in previously untreated patients and minimally treated patients with haemophilia A

Guenter Auerswald
1   Prof.-Hess-Kinderklinik, Bremen, Germany
,
Alexis A. Thompson
2   Children’s Memorial Hospital, Chicago, Illinois, USA
,
Michael Recht   *
3   The Hemophilia Center, Phoenix Children’s Hospital, Phoenix, Arizona, USA
,
Deborah Brown
4   University of Texas Health Science Center-Houston, Houston, Texas, USA
,
Raina Liesner
5   Great Ormond Street Hospital For Children, Haemophilia Centre, London, UK
,
Norma Guzmán-Becerra
6   Baxter Healthcare Corporation, Westlake Village, California, USA
,
Jacqueline Dyck-Jones
6   Baxter Healthcare Corporation, Westlake Village, California, USA
,
Bruce Ewenstein
6   Baxter Healthcare Corporation, Westlake Village, California, USA
,
Brigitt Abbuehl
7   Baxter Innovations GmbH, Vienna, Austria
› Author Affiliations Financial support: The work presented is a Baxter-sponsored clinical study.
Further Information

Publication History

Received: 15 September 2011

Accepted after major revision: 10 March 2012

Publication Date:
29 November 2017 (online)

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Summary

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®). Ondemand or prophylactic treatment regimens were determined at the discretion of the investigator. rAHF-PFM was also permitted for perioperative 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. Intraoperative (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. Nonserious, 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.

Keywords

Haemophilia A - previously untreated patients - paediatric - minimally treated patients - factor VIII - factor VIII inhibitor

* Currently at The Hemophilia Center, Oregon Health and Science University, Portland, OR, USA.


5 Currently in Portland, OR, USA.


 

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