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Thromb Haemost 1998; 80(06): 912-918
DOI: 10.1055/s-0037-1615388
Letters to the Editor
Schattauer GmbH

Home Treatment of Mild to Moderate Bleeding Episodes Using Recombinant Factor VIIa (Novoseven) in Haemophiliacs with Inhibitors

Nigel S. Key
1   University of Minnesota Hemophilia Treatment Center, Minneapolis, Minnesota
,
Louis M. Aledort
2   Mount Sinai Hospital, New York, New York
,
Diana Beardsley
3   Yale Hemophilia Center, New Haven, Connecticut
,
Herbert A. Cooper
4   UNC Hospital at Chapel Hill, Chapel Hill, North Carolina
,
George Davignon
5   Pediatric Hematology/Oncology, UCSD Medical Center, San Diego, California
,
Bruce M. Ewenstein
6   Hematology/Oncology Division, The Children’s Hospital, Boston, Massachusetts
,
Gerald S. Gilchrist
7   Hemophilia Center, Mayo Clinic, Rochester, Minnesota
,
Joan C. Gill
8   Great Lakes Hemophilia Foundation, Wauwatosa, Wisconsin
,
Bertil Glader
9   Division of Hematology/Oncology, Stanford University Medical Center, Stanford, California
,
W. Keith Hoots
10   MD Anderson Cancer Center, Houston, Texas
,
C. Thomas Kisker
11   University of Iowa Hospitals and Clinics, Iowa City, Iowa
,
Jeanne M. Lusher
12   Children’s Hospital Michigan, Detroit, Michigan
,
Cathy G. Rosenfield
13   New England Medical Center, Boston, Massachusetts
,
Amy Shapiro
14   JW Riley Hospital for Children, Indianapolis, Indiana
,
Horace Smith
15   Division of Hematology/Oncology, The Children’s Memorial Hospital, Chicago, Illinois
,
Edwin Taft
16   Albany Medical College, Albany, New York, USA
› Author Affiliations
Further Information

Publication History

Received 04 May 1998

Accepted after revision 28 August 1998

Publication Date:
07 December 2017 (online)

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Summary

Objective. To assess the safety and efficacy of a fixed dose of recombinant activated factor VII (rFVIIa; NovoSeven™) in the home setting for mild to moderately severe joint, muscle, and mucocutaneous bleeding episodes in patients with haemophilia A or B with inhibitors. Design. Multicentre, open-label, single arm, phase III study of one year duration. Methods. Patients or their caregivers administered up to three doses of rFVIIa (90 μg/kg i.v.) at 3 h intervals within 8 h of the onset of a mild to moderate bleeding episode. Once the subject considered that rFVIIa had been “effective” with regard to haemostasis (after 1-3 injections), one further (maintenance) dose of rFVIIa was administered. Results. Of 60 patients enrolled, 56 experienced at least one bleed, and 46 completed the one year study. 614 of 877 bleeds (70%) were evaluable according to protocol definitions. Haemostasis was rated as “effective” in 92% (566/614) of evaluable bleeds after a mean of 2.2 injections. For successfully treated episodes, the time from onset of bleeding until administration of the first injection was 1.1 ± 2.0 h (mean ± SD). Twenty-four hours after initial successful response, haemostasis was reported as having been maintained in 95% of cases. Efficacy was comparable for muscle, joint and target joint, and muco-cutaneous bleeding episodes. In an intent-to-treat analysis of all 877 bleeding events, efficacy outcomes were equivalent to the evaluable bleeds, with an effective response in 88% of treated episodes. Treatment-related adverse events occurred in 32 (3% of all) bleeding episodes and consisted of re-bleeds/new bleeds in more than 50% (18/32) of these events. A single episode of superficial thrombophlebitis was the only thrombotic complication encountered, and there were no patient withdrawals due to adverse events. Development of FVII(a) antibodies could not be detected, and hypersensitivity reactions to rFVIIa were not reported. Conclusion. rFVIIa is effective and well tolerated when used in the home setting to treat mild to moderate bleeding episodes in patients with haemophilia A or B with inhibitors.