US experience with recombinant factor VIIa for surgery and other invasive procedures in acquired haemophilia: analysis from the Hemostasis and Thrombosis Research Society Registry

Haemophilia ◽  
2015 ◽  
Vol 22 (1) ◽  
pp. e18-e24 ◽  
Author(s):  
A. D. Ma ◽  
C. M. Kessler ◽  
H. A. B. Al-Mondhiry ◽  
R. Z. Gut ◽  
D. L. Cooper
Keyword(s):  
Recombinant Factor Viia ◽  
Factor Viia ◽  
Research Society ◽  
Invasive Procedures ◽  
Acquired Haemophilia ◽  
Thrombosis Research

Use of Recombinant Factor VIIa (rFVIIa) for Acute Bleeding Episodes in Acquired Hemophilia: Final Analysis From the Hemostasis and Thrombosis Research Society (HTRS) Registry AH Study

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 4624-4624 ◽  
Author(s):  
Alice D. Ma ◽  
Craig M. Kessler ◽  
Hamid A B Al-Mondhiry ◽  
Margaret Fisher ◽  
Robert Z. Gut ◽  
...  
Keyword(s):  
Total Dose ◽  
Recombinant Factor Viia ◽  
Factor Viia ◽  
Final Analysis ◽  
Research Society ◽  
Blood Components ◽  
Acquired Hemophilia ◽  
Thrombosis Research ◽  
Bleeding Episodes ◽  
White Patients

Abstract Abstract 4624 Introduction: Acquired hemophilia (AH) is a rare disorder marked by the development of autoantibodies to factor VIII. Patients typically present with bleeding and a prolonged aPTT that does not correct with mixing with normal plasma. Recombinant factor VIIa (rFVIIa) is the only FDA approved bypassing agent for treatment of bleeding in AH. The Hemostasis and Thrombosis Research Society Registry was established as an IRB-monitored web-based platform with informed consent in 1999 to support the society's research needs and monitor rFVIIa use after its FDA approval. AH surveillance was initiated in October 2006. Methods: Data on bleeding episodes entered between January 2004-November 2011 were analyzed. For each rFVIIa-treated bleed, the initial dose, total dose, mean dose per infusion, number of doses, and treatment duration were calculated. Results: Of 166 registered AH patients, 110 had bleeding episodes reported. Of 237 bleeds, 17 (7%) occurred in patients aged ≤40, 54 (23%) in ages 41–60, and 166 (70%) with age >60. The most common sites were subcutaneous (40%), mucosal (32%), muscle (20%) and joint (11%). Subcutaneous bleeds were more commonly reported in females (55% vs. 40% males) and white patients (44% vs. 27% black). Mucosal bleeds were more common in black patients (49% vs. 25%) and muscle bleeds more common in white patients (14% vs. 23%). There were 139 (59%) rFVIIa-treated bleeds (89 rFVIIa alone, 50 rFVIIa plus other agents/blood components); 127 were treated with rFVIIa first-line. There were 75 episodes (43 patients) treated with other hemostatic agents or blood components only, 21 episodes (18 patients) recorded with no treatment for the episode, and 2 episodes (2 patients) with no treatment data recorded. For rFVIIa-treated episodes, 71 were in males and 68 females; 101 were Caucasian and 30 were black. Mean (median) age at bleeding was 67 (69) years. rFVIIa-treated bleeds were spontaneous (95), traumatic (30), surgical/procedure-related (7), dental (2) or other (4). Median (IQR) initial rFVIIa dose was 90 (88–100) mcg/kg, and average dose per injection was 90 (88–99). The total dose per episode was 334 (166–1383) mcg/kg administered as 3 (2–14) injections over 1 (0–2.75) day. Median (IQR) data for rFVIIa dosing by treatment sequence, bleed location and type is described below: Efficacy of rFVIIa, physician-rated for each regimen, was reported as “bleeding stopped” in 117 (85%) episodes; “bleeding slowed” in 15 (11%) episodes (stopped with other agents in 3 episodes); “no improvement” in 5 (4%) episodes (no bleed stop date identified in 4, stopped with other agent in 1), and was not documented in 1. Considering only the 4 rFVIIa treatment failures where bleeding stopped after switching to another agent, overall rFVIIa efficacy was 97%. The only thromboembolic event was transient neurologic symptoms in a 31-year-old post-partum patient after 110 doses of 90 mcg/kg every 2 hours. The neurologist reported it most likely related to eclampsia and vasculitis. Conclusions: The HTRS registry final analysis represents the 2nd largest data set in AH. While subcutaneous bleeding as a first bleed location was uncommon outside of Caucasians, it represents the most common bleed location of recorded bleeds in all race/ethnicity groups. As this registry was originally intended in part to track the safety of rFVIIa, the proportion of bleeds treated with rFVIIa (59%) and associated data derived from those bleeds may be somewhat biased and selective. Nevertheless, they are certainly indicative that rapid and safe hemostasis can be achieved with rFVIIa in an aging population with AH where thrombogenicity is of concern. Disclosures: Ma: Novo Nordisk Inc.: Consultancy, Speakers Bureau. Kessler:Novo Nordisk: Consultancy, Research Funding. Fisher:Novo Nordisk Inc.: Employment. Gut:Novo Nordisk Inc.: Employment. Cooper:Novo Nordisk Inc.: Employment.

Thrombelastographic monitoring of recombinant factor VIIa in acquired haemophilia

Haemophilia ◽  
2008 ◽  
Vol 14 (4) ◽  
pp. 736-742 ◽  
Author(s):  
H. DEHMEL ◽  
S. WERWITZKE ◽  
A. TRUMMER ◽  
A. GANSER ◽  
A. TIEDE
Keyword(s):  
Recombinant Factor Viia ◽  
Factor Viia ◽  
Acquired Haemophilia

US Experience with Recombinant Factor VIIa (rFVIIa) for Surgery in Acquired Hemophilia (AH): Analysis From the Hemophilia and Thrombosis Research Society (HTRS) Registry

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 3372-3372
Author(s):  
Hamid A B Al-Mondhiry ◽  
Alice D. Ma ◽  
Craig M. Kessler ◽  
Margaret Fisher ◽  
Robert Z. Gut ◽  
...  
Keyword(s):  
Surgical Procedures ◽  
Factor Viia ◽  
Package Insert ◽  
Research Society ◽  
Factor Vii ◽  
Orthopedic Procedures ◽  
Case Report Form ◽  
Filter Placement ◽  
Thrombosis Research ◽  
Post Surgery

Abstract Abstract 3372 Background: AH is a rare disorder caused by autoantibodies against factor VIII (FVIII). Patients can present with peri-operative bleeding, and a prolonged aPTT that doesn't correct with 1:1 mixing after 2 hours (37°C). The Hemostasis and Thrombosis Research Society (HTRS) Registry was established to support the society's research needs and monitor the safety of recombinant activated factor VII (rFVIIa). In October 2006, the FDA approved rFVIIa for the treatment of bleeding and prevention of bleeding during surgery in AH at 70–90 mcg/kg every 2–3 hours. In 2007, a new case report form (CRF) for capturing information about surgeries was added. Methods: Data on treatment during surgical procedures was collected from the HTRS registry surgical CRF. For each rFVIIa-treated surgery, the initial dose, total dose per procedure (prior to and post surgery), average infused dose, number of doses, and treatment duration were calculated. Queries were issued to verify any procedures lacking treatment data or where treatment was listed as “none”. Efficacy was assessed on a 4 point scale and an investigator assessed adequacy of hemostasis on the “planned regimen” immediately and at 24 and 72 hours. Results: Of 166 registered AH patients, 36 patients underwent 58 surgeries. There were 24 (43%) rFVIIa-treated procedures (17 rFVIIa only). The mean (range) age of all patients undergoing surgery was 69 (14–89), and for rFVIIa-treated patients was 77.8 (28–89). rFVIIa-treated patients were mostly female (71%) and Caucasian (59%). Approximately 33% (19/58) of all surgeries were elective, including 54% (13/24) of rFVIIa-treated surgeries. The most common procedures overall were central line placement (12), endoscopy (12) and orthopedic (5). rFVIIa-treated surgeries included: central venous access (6), endoscopy (5), incision, drainage and grafting of hand hematomas (3), AVM embolization (2), orthopedic procedures (2) cholecystectomy (1), colon biopsy (1), catheter removal (1), exchange of prostate brachytherapy implant (1), venipuncture (1) and IVC filter placement (1). rFVIIa was used pre-op in 19 procedures and post-surgery in 15. In 9/24 procedures (8/24 pre-op), a single rFVIIa dose was used. Median (range) for rFVIIa treatment is described in the table below: rFVIIa efficacy was rated as excellent/good in 17 (77.3%), fair/partially effective in 2 (9.1%), and poor/ineffective in 3 (13.6%). For those rated fair/partially effective, one received only one pre-op rFVIIa dose of 75 mcg/kg with antifibrinolytics (no post-surgery treatment) and the other received 1 pre-op and 2 post-op rFVIIa 106 mcg/kg doses at 2 hour intervals with no other treatments. For those rated poor/ineffective, one received rFVIIa and PRBCs only, one received rFVIIa 81 mcg/kg initially every 4–6 hrs then plasma-derived activated prothrombin complex concentrate (pd-aPCC) then rFVIIa, and one had resolution of a neck hematoma with rFVIIa (subsequently switched to pd-aPCC) but could not maintain an airway and was transferred to hospice. No thromboembolic events were reported in rFVIIa-treated patients. Two patients underwent 6 follow-up procedures during ongoing post-op rFVIIa-treatment. Eleven patients used hemostatic agents other than rFVIIa in 12 surgical procedures. These included use of pd- aPCC in 5 cases (3 pre-op; 2 pre-op and post-op) and use of desmopressin in 2 cases, and recombinant FVIII in 3 cases. Blood products (FFP, PRBCs) were used post-op in 2 cases. Thirteen patients underwent 16 surgeries with no reported treatment. Conclusions: rFVIIa provided adequate hemostasis for almost all rFVIIa-treated AH surgeries at doses largely conforming to the package insert. There were no safety concerns and no thromboembolic complications reported in this population of older patients. Lack of hemostatic therapy reported for some procedures may reflect surgery as a presenting AH symptom, surgery during ongoing treatment not requiring additional treatment, or surgery performed following immune suppression/tolerization. Disclosures: Ma: Novo Nordisk Inc.: Consultancy, Speakers Bureau. Kessler:Novo Nordisk: Consultancy, Research Funding. Fisher:Novo Nordisk Inc.: Employment. Gut:Novo Nordisk Inc.: Employment. Cooper:Novo Nordisk Inc.: Employment.

The use of recombinant factor VIIa in a patient with acquired haemophilia A undergoing surgery

1995 ◽  
Vol 6 (2) ◽  
pp. 125-128 ◽  
Author(s):  
H. A. Doughty ◽  
A. Northeast ◽  
L. Sklair ◽  
T. Roques ◽  
A. E. Young ◽  
...  
Keyword(s):  
Recombinant Factor Viia ◽  
Factor Viia ◽  
Haemophilia A ◽  
Acquired Haemophilia
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