Results from a large multinational clinical trial (guardian™3) using prophylactic treatment with turoctocog alfa in paediatric patients with severe haemophilia A: safety, efficacy and pharmacokinetics

Haemophilia ◽  
2013 ◽  
Vol 19 (5) ◽  
pp. 698-705 ◽  
Author(s):  
R. Kulkarni ◽  
F. A. Karim ◽  
S. Glamocanin ◽  
D. Janic ◽  
V. Vdovin ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Prophylactic Treatment ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Paediatric Patients ◽  
Multinational Clinical Trial

Surgery with emicizumab prophylaxis for two paediatric patients with severe haemophilia A with inhibitors

2021 ◽  
Author(s):  
Marguerite Lockhart ◽  
Brigitte Tardy‐Poncet ◽  
Aurélie Montmartin ◽  
Pauline Noyel ◽  
Sandrine Thouvenin ◽  
...  
Keyword(s):  
Haemophilia A ◽  
Severe Haemophilia ◽  
Paediatric Patients

Break-through bleeding in relation to pharmacokinetics of Factor VIII in paediatric patients with severe haemophilia A

Haemophilia ◽  
2017 ◽  
Vol 24 (1) ◽  
pp. 120-125 ◽  
Author(s):  
X. Cheng ◽  
P. Li ◽  
Z. Chen ◽  
N. Zhang ◽  
Y. Zhen ◽  
...  
Keyword(s):  
Factor Viii ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Paediatric Patients

Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B

1992 ◽  
Vol 232 (1) ◽  
pp. 25-32 ◽  
Author(s):  
I. M. NILSSON ◽  
E. BERNTORP ◽  
T. LÖFQVIST ◽  
H. PETTERSSON
Keyword(s):  
Prophylactic Treatment ◽  
Haemophilia A ◽  
Severe Haemophilia

Human-Cl Rhfviii Effectively and Safely Prevents Bleeding Episodes in Previously Treated Adult Patients with Severe Haemophilia A

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 1132-1132
Author(s):  
Sigurd Knaub ◽  
Toshko Lissitchkov ◽  
Kingsley Hampton ◽  
Mario Von Depka ◽  
Savita Rangarajan ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Research Funding ◽  
Prophylactic Treatment ◽  
Hemophilia A ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Open Label ◽  
Bleeding Rate ◽  
Previously Treated

Abstract Abstract 1132 The main purpose of this prospective, multi-center, open-label phase 3 study was to assess the efficacy of prophylactic treatment with Human-cl rhFVIII, the first human cell-line derived recombinant FVIII, in previously treated patients (PTPs) with severe haemophilia A. Patients were to receive 30–40 international units (IU) FVIII of Human-cl rhFVIII per kg every other day for 6 months. Efficacy of preventing and treating bleeds were judged using objective criteria taking the monthly bleeding rate and the number of infusions needed to manage a break-through bleed into account. In-vivo recovery (IVR) was determined at the beginning of the study and after 3 and 6 months. FVIII:C was measured by validated chromogenic (CHR) and one-stage (OS) assays in a central laboratory, which also assigned drug potencies. Inhibitor activity was determined using the Nijmegen modification of the Bethesda assay before the first administration and at defined intervals thereafter. Thirty-two patients between 18 and 75 years of age were enrolled from 11 centres in Europe and treated prophylactically for 6.0±0.9 months (mean ± SD) with a mean prophylactic dose of 32.8 IU/kg. Sixteen patients never bled, 11 patients bled once and 5 more than once. The mean total and spontaneous monthly bleeding rate was 0.188±0.307 and 0.095±0.211, respectively. Efficacy of the prophylactic treatment was “excellent” in all patients for spontaneous BEs and “excellent” or “good” in all patients but one for all types of bleeds. All treatments of bleeds were rated as “excellent” (71.4%) or “good” (28.6%). The IVR at baseline was 2.6±0.5 % per IU/kg for the CHR and 2.2±0.5 % per IU/kg for the OS assay and remained stable during the study. A total of 2921 infusions were given in the study. Human-cl rhFVIII was well tolerated and no patient experienced a related serious adverse event. No FVIII inhibitors were detected. Conclusion: The data indicate that Human-cl rh FVIII is safe and efficacious in preventing and treating bleeds in PTPs with severe haemophlia A. Disclosures: Knaub: Octapharma AG: Employment. Lissitchkov:Octapharma AG: PI Other. Tuddenham:College London: Consultancy, Employment, Gene therapy for hemophilia A, Gene therapy for hemophilia A Patents & Royalties, Research Funding. Collins:Octapharma AG: Consultancy. Oldenburg:d and e: Baxter, Bayer, Biotest, CSL-Behring, Grifols, Inspiration, NovoNordisk, Octapharma, Pfizer e: Biogen IDec, Swedish Orphan Biovitrum: Honoraria, Research Funding. Bichler:Octapharma AG: Employment.

Safety and efficacy of a glycoPEGylated rFVIII (turoctocog alpha pegol, N8-GP) in paediatric patients with severe haemophilia A

2017 ◽  
Vol 117 (09) ◽  
pp. 1705-1713 ◽  
Author(s):  
Sandrine Meunier ◽  
Jayanthi Alamelu ◽  
Silke Ehrenforth ◽  
Hideji Hanabusa ◽  
Faraizah Abdul Karim ◽  
...  
Keyword(s):  
Half Life ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Older Children ◽  
Open Label ◽  
Paediatric Patients ◽  
Effective Prophylaxis ◽  
History Of ◽  
Previously Treated Patients ◽  
Previously Treated

SummaryTuroctocog alfa pegol (N8-GP, Novo Nordisk, Bagsværd, Denmark), an extended half-life glycoPEGylated recombinant factor VIII (rFVIII), is being developed for prophylaxis and treatment of bleeds in haemophilia A patients. pathfinder™5 is a multinational, open-label, single-arm trial to assess safety, efficacy and pharmacokinetics of N8-GP in paediatric (<12 years), previously treated patients. Boys with severe haemophilia A (<1 % FVIII), no history of inhibitors and previously treated with FVIII products (>50 exposure days [ED] for patients aged 0–5 years [younger cohort]; >150 ED for patients aged 6–11 years [older cohort]) were included. For prophylaxis, N8-GP was dosed at 50–75 IU/kg twice weekly; bleeds were treated with 20–75 IU/kg. Half-life was estimated for the patients’ previous FVIII product and for N8-GP. Sixty-eight patients received N8-GP; none developed inhibitors and no other concerns were identified. Median annualised bleeding rate was 1.95 (1.94 and 1.97 in the younger and older cohorts, respectively). Twenty-nine patients (42.6 %; 15 younger and 14 older children, respectively) did not report any bleeding while on N8-GP prophylaxis; 39 patients (57.4 %; 19 younger and 20 older children, respectively) reported 70 bleeds (all mild/moderate). N8-GP treatment was successful for 78.6 % of bleeds in all patients, 80.0 % in younger and 77.5 % in older patients. Most bleeds (80.0 %) were treated with ≤2 injections. Half-life ratio between N8-GP and the patients’ previous FVIII product was 1.85. N8-GP was well tolerated and provided effective prophylaxis and treatment of bleeds in paediatric patients with severe haemophilia A.Trial registered at www.clinicaltrials.gov (NCT01731600).Supplementary Material to this article is available online at www.thrombosis-online.com.

Impact of treatment regimen with moroctocog alfa (AF‐CC) on bleeding rates in paediatric patients with severe haemophilia A

Haemophilia ◽  
2020 ◽  
Vol 26 (5) ◽  
Author(s):  
Mark P. Smith ◽  
Jeremy Rupon ◽  
Yasser Wali ◽  
Hala Rimawi ◽  
Joan Korth‐Bradley ◽  
...  
Keyword(s):  
Treatment Regimen ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Paediatric Patients
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