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Therapeutic choices for patients with hemophilia and high-titer inhibitors

Kulkarni, Roshni; Aledort, Louis M.; Berntorp, Erik LU ; Brackman, Hans H.; Brown, Deborah; Cohen, Alan R.; Ewing, Nadia P.; Gringeri, Alessandro; Gruppo, Ralph and Hoots, Keith, et al. (2001) In American Journal of Hematology 67(4). p.240-246
Abstract
Effective treatment of bleeding episodes in hemophilia with high titer inhibitors (HTI) remains a challenge, despite the fact that the therapeutic armamentarium has expanded considerably over the past few years. Treatment safety has improved with the availability of porcine factor VIII (FVIII) and bypassing products such as recombinant factor VIIa (rFVIIa), and plasma-derived activated Prothrombin Complex Concentrates (aPCCs) that are virally inactivated. The major drawbacks of rFVIIa and aPCCs are their unpredictable hemostatic effect, lack of laboratory assays to monitor efficacy and dosing frequency, and the risk of thrombosis. The proceedings of a one-day workshop of physicians who specialized in treating patients with hemophilia held... (More)
Effective treatment of bleeding episodes in hemophilia with high titer inhibitors (HTI) remains a challenge, despite the fact that the therapeutic armamentarium has expanded considerably over the past few years. Treatment safety has improved with the availability of porcine factor VIII (FVIII) and bypassing products such as recombinant factor VIIa (rFVIIa), and plasma-derived activated Prothrombin Complex Concentrates (aPCCs) that are virally inactivated. The major drawbacks of rFVIIa and aPCCs are their unpredictable hemostatic effect, lack of laboratory assays to monitor efficacy and dosing frequency, and the risk of thrombosis. The proceedings of a one-day workshop of physicians who specialized in treating patients with hemophilia held in Vienna on May 13, 2000 have been summarized. In making a decision regarding the choice of product, physicians often consider the type of bleeding episode (life or limb threatening), age of the patient, volume of the reconstituted product, previous exposure to plasma derived products, cost, efficacy, and safety. For plasma naive patients, to achieve rapid hemostasis a majority of the panelists used porcine FVIII (for patients who lack porcine inhibitory antibodies) or rFVIIa. For patients previously treated with plasma derived factors, in addition to the above concentrates, aPCCs were recommended. Although no data exists regarding safety and efficacy, switching products was routinely practiced either because of availability or cost. Furthermore, the panelists were uncertain about the efficacy of bypassing agents in the prevention of joint disease in inhibitor patients. The workshop participants felt that future research offers the best solution to resolve some of the dilemmas faced by clinicians and may help individualise treatment in a hemophilia patient with a high titer inhibitor. (Less)
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publication status
published
subject
keywords
inhibitors, hemophilia, APCC, recombinant factor VIIa, immune tolerance induction
in
American Journal of Hematology
volume
67
issue
4
pages
240 - 246
publisher
John Wiley & Sons
external identifiers
  • pmid:11443636
  • scopus:0034940248
ISSN
0361-8609
DOI
10.1002/ajh.1123
language
English
LU publication?
yes
id
8729382d-277f-416a-ab34-0aea5b79530a (old id 1120892)
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2008-07-01 15:41:23
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2018-10-03 11:23:58
@article{8729382d-277f-416a-ab34-0aea5b79530a,
  abstract     = {Effective treatment of bleeding episodes in hemophilia with high titer inhibitors (HTI) remains a challenge, despite the fact that the therapeutic armamentarium has expanded considerably over the past few years. Treatment safety has improved with the availability of porcine factor VIII (FVIII) and bypassing products such as recombinant factor VIIa (rFVIIa), and plasma-derived activated Prothrombin Complex Concentrates (aPCCs) that are virally inactivated. The major drawbacks of rFVIIa and aPCCs are their unpredictable hemostatic effect, lack of laboratory assays to monitor efficacy and dosing frequency, and the risk of thrombosis. The proceedings of a one-day workshop of physicians who specialized in treating patients with hemophilia held in Vienna on May 13, 2000 have been summarized. In making a decision regarding the choice of product, physicians often consider the type of bleeding episode (life or limb threatening), age of the patient, volume of the reconstituted product, previous exposure to plasma derived products, cost, efficacy, and safety. For plasma naive patients, to achieve rapid hemostasis a majority of the panelists used porcine FVIII (for patients who lack porcine inhibitory antibodies) or rFVIIa. For patients previously treated with plasma derived factors, in addition to the above concentrates, aPCCs were recommended. Although no data exists regarding safety and efficacy, switching products was routinely practiced either because of availability or cost. Furthermore, the panelists were uncertain about the efficacy of bypassing agents in the prevention of joint disease in inhibitor patients. The workshop participants felt that future research offers the best solution to resolve some of the dilemmas faced by clinicians and may help individualise treatment in a hemophilia patient with a high titer inhibitor.},
  author       = {Kulkarni, Roshni and Aledort, Louis M. and Berntorp, Erik and Brackman, Hans H. and Brown, Deborah and Cohen, Alan R. and Ewing, Nadia P. and Gringeri, Alessandro and Gruppo, Ralph and Hoots, Keith and Leissenger, Cindy and Peerlinck, Katheliijne and Poon, Man-Chiu and Wong, Wing-Yen},
  issn         = {0361-8609},
  keyword      = {inhibitors,hemophilia,APCC,recombinant factor VIIa,immune tolerance induction},
  language     = {eng},
  number       = {4},
  pages        = {240--246},
  publisher    = {John Wiley & Sons},
  series       = {American Journal of Hematology},
  title        = {Therapeutic choices for patients with hemophilia and high-titer inhibitors},
  url          = {http://dx.doi.org/10.1002/ajh.1123},
  volume       = {67},
  year         = {2001},
}