A Multicenter Study of Recombinant Factor VIII (RecombinateTM) in Previously Treated Patients with Hemophilia A

White, G C; Courter, S; Bray, Gordon L.; Lee, M; Gomperts, Edward D.

doi:10.1055/s-0038-1656030

Cited by 130 publications

(131 citation statements)

References 45 publications

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“…11 A prospective open label multicenter study was then conducted to compare the pharmacokinetics of rFVIII with a plasma-derived FVIII (pdFVIII) and to assess the safety and long-term home-treatment efficacy with rFVIII for individuals with hemophilia A. 12 Sixty-nine patients with hemophilia A (67 severe, 2 moderate) participated in a trial for a median of 3.7 (1.0 to 5.7) years. At study entry, 44 were HIV seropositive and 25 were HIV seronegative.…”

Section: Recombinant Factor Viii: Recombinate/bioclate ®mentioning

confidence: 99%

The Use of Recombinant Factor VIII Products in Previously Treated Patients with Hemophilia A: Pharmacokinetics, Efficacy, Safety, and Inhibitor Development

Lee

2002

Semin Thromb Hemost

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Studies in previously treated patients (PTPs) have been conducted for the three recombinant factor VIII (rFVIII) concentrates currently licensed. Kogenate, has shown a recovery of 2.7% per IU/kg and a half-life of 15.8 hours. A prospective 5-year study in 58 patients showed that 82% of bleeds resolved with one treatment; the average dose was 25 IU/kg. For Recombinate, the recovery was 2.4% per IU/kg and the half-life was 14.7 hours. In 69 patients, the average dose used to treat a bleed was 27.5 IU/kg, and 91% of responses were categorized as good or excellent. B-domain depleted rFVIII, Refacto, has shown a recovery of 2.7% per IU/kg and a half-life of 14 hours. In 113 PTPs, the average dose used was 29.5 IU/kg, and 71% of bleeds responded to one treatment. All three recombinant products have been shown to be safe and effective in very similar dosing regimens. In view of the good recoveries, it is probable that smaller doses, such as 20 IU/kg, could be recommended to treat a bleed effectively with one dose, given that dosing regimens were previously established using intermediate products. Clearly, this would have considerable financial advantages.

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Section: Recombinant Factor Viii: Recombinate/bioclate ®mentioning

confidence: 99%

The Use of Recombinant Factor VIII Products in Previously Treated Patients with Hemophilia A: Pharmacokinetics, Efficacy, Safety, and Inhibitor Development

Lee

2002

Semin Thromb Hemost

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“…Studies have also shown that rFVIII and pdFVIII have similar in vivo half-life and recovery [4], with some data suggesting that mean peak response and recovery values are greater for rFVIII than pdFVIII [9]. The benefits of prophylaxis have been demonstrated in several studies [10,11] in which early onset prophylaxis appears to prevent joint damage [12].…”

mentioning

confidence: 99%

“…The safety and efficacy of rFVIII has been demonstrated in a number of clinical trials in previously treated and previously untreated patients, and in long-term and home treatment settings [4][5][6][7][8]. Studies have also shown that rFVIII and pdFVIII have similar in vivo half-life and recovery [4], with some data suggesting that mean peak response and recovery values are greater for rFVIII than pdFVIII [9].…”

mentioning

confidence: 99%

Comparative effectiveness of plasma‐derived and recombinant FVIII concentrates in the on‐demand and prophylactic treatment of patients with haemophilia A

Pollmann¹,

Richter²,

Siegmund³

2004

Haemophilia

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Administration of the deficient coagulation factor VIII (FVIII) is a well-established and effective treatment used in patients with haemophilia A. While plasmaderived FVIII (pdFVIII) concentrates have been used successfully for many years, technological advances have enabled the large-scale production of recombinant FVIII (rFVIII), which provides enhanced viral safety and a supply not limited by the availability of donor plasma [1,2]. The adoption of prophylactic treatment regimens in many countries has also provided haemophilia patients with therapeutic benefits through sustaining adequate FVIII levels and reducing the frequency of bleeds. These benefits are especially important in patients with severe disease or those susceptible to haemophilic arthropathy [3].The safety and efficacy of rFVIII has been demonstrated in a number of clinical trials in previously treated and previously untreated patients, and in long-term and home treatment settings [4][5][6][7][8]. Studies have also shown that rFVIII and pdFVIII have similar in vivo half-life and recovery [4], with some data suggesting that mean peak response and recovery values are greater for rFVIII than pdFVIII [9]. The benefits of prophylaxis have been demonstrated in several studies [10,11] in which early onset prophylaxis appears to prevent joint damage [12]. However, age at onset of bleeding is an important consideration, as prophylaxis has no benefit in a non-bleeding child [13].In our haemophilia centre in Mü nster, we initially treat bleeds in young patients with on-demand therapy (after a bleed), and subsequently change to continuous prophylaxis as bleeding episodes accumulate (> 6 joint bleeding episodes per year or recurrence of long-lasting, severe joint bleeds). We describe here our experience over a 1-year period with patients who were switched from pdFVIII to rFVIII after 6 months during either an on-demand or continuous prophylaxis home treatment regimen. These observational data provide a comparison of pdFVIII and rFVIII efficacy that is interesting in the current environment in which the relative merits of different FVIII therapies are increasingly questioned.Data were extracted from diary records for 27 patients with severe haemophilia A (<0.01 U mL )1 FVIII activity). Of these patients, 12 received FVIII treatment on-demand and the remaining 15 received FVIII by continuous prophylaxis. For each type of concentrate, on-demand treatment was provided using a dose of 25 IU kg )1 and continuous prophylaxis involved up to three times per week a dose of 25 IU kg )1 . The date of FVIII administration, dose, product name and lot number were recorded together with the location of the bleed and the treatment regimen (on-demand or continuous prophylaxis). Efficacy was assessed according to the number of infusions required during on-demand or continuous prophylactic treatment, number of breakthrough bleeds and on-demand infusions required during continuous prophylaxis, and total FVIII consumption. A number of different pdFVIII and rFVIII concentrates were...

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“…VIII lub cz. IX komórki jajnika chomika chińskiego lub nerki zarodka chomika [5,6]. Ostatnio pojawił się rekombinowany koncentrat cz.…”

Section: Rodzaje Koncentratów Cz VIII I Ixunclassified

“…Zawierał albuminę ludzką do stabilizacji białka końcowego, jak również białka zwierzęce i ludzkie w podłożu hodowlanym. Preparat taki nazwano koncentratem rekombinowanym pierwszej generacji [5]. Kolejnym ważnym etapem w procesie produkcji czynników krzepnięcia było wprowadzenie cukru jako stabilizatora zamiast albuminy.…”

Section: Rodzaje Koncentratów Cz VIII I Ixunclassified

Jak wybrać koncentrat czynnika krzepnięcia dla dziecka chorego na hemofilię?

Klukowska¹

2014

Acta Haematologica Polonica

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Leczenie chorych na hemofilię opiera się na substytucji niedoborowego czynnika krzepnięcia krwi. W ciężkiej postaci hemofilii, aby zapobiec krwawieniom do stawów oraz rozwojowi zmian degeneracyjnych, metodą z wyboru w leczeniu, zwłaszcza dzieci, jest profilaktyczne podawanie koncentratu czynnika krzepnięcia [1][2][3]. Wcześnie wprowadzona profilaktyka -przed pojawieniem się zmian a c t a h a e m a t o l o g i c a p o l o n i c a 4 5 ( 2 0 1 4 ) 4 9 -5 3 i n f o r m a c j e o a r t y k u l e Historia artykułu: Słowa kluczowe: hemofilia rekombinowane koncentraty czynnika VIII i IX osoczopochodne koncentraty czynnika VIII i IX dzieci Keywords: Haemophilia Recombinant factor VIII and IX concentrates Plasma-derived factor VIII and IX concentrates Children a b s t r a c t The introduction of the coagulation factor VIII and IX concentrates has changed the treatment of haemophilia patients dramatically. The concentrates were produced from pooled human plasma in the beginning. During the 1990s of the 20th century, recombinant factor VIII was accessed, then factor IX was introduced. The modern methods of elimination and inactivation of viruses led to the development of safe plasma-derived products. However, minimal risk of viral transmission is still greater by plasma-derived than recombinant concentrates. The efficacy of both products is very good in treatment of bleeds as well as in surgery and prophylaxis. The pharmacokinetics of plasma-derived and recombinant factor VIII is similar. Nevertheless, the recovery of recombinant factor IX is lower than plasma-derived, therefore the doses of recombinant factor IX should be 30% higher. The half-life of both factor IX products is almost the same. In developed countries recombinant coagulation factor concentrates are recommended treatment of choice for patients with haemophilia and almost all children are being treated with them.

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A Multicenter Study of Recombinant Factor VIII (RecombinateTM) in Previously Treated Patients with Hemophilia A

Cited by 130 publications

References 45 publications

The Use of Recombinant Factor VIII Products in Previously Treated Patients with Hemophilia A: Pharmacokinetics, Efficacy, Safety, and Inhibitor Development

The Use of Recombinant Factor VIII Products in Previously Treated Patients with Hemophilia A: Pharmacokinetics, Efficacy, Safety, and Inhibitor Development

Comparative effectiveness of plasma‐derived and recombinant FVIII concentrates in the on‐demand and prophylactic treatment of patients with haemophilia A

Jak wybrać koncentrat czynnika krzepnięcia dla dziecka chorego na hemofilię?

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