Real‐world experience on the tolerability and safety of emicizumab prophylaxis in paediatric patients with severe haemophilia A with and without FVIII inhibitors

Haemophilia ◽  
2021 ◽  
Author(s):  
Eman Hassan ◽  
Lancashire Jonathan ◽  
Motwani Jayashree
Keyword(s):  
Real World ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Paediatric Patients

scholarly journals Long-term clinical and economic outcomes in previously untreated paediatric patients with severe haemophilia A: A nationwide real-world study with 700 person-years

Haemophilia ◽  
2018 ◽  
Vol 24 (3) ◽  
pp. 436-444 ◽  
Author(s):  
K. Vepsäläinen ◽  
P. Riikonen ◽  
R. Lassila ◽  
M. Arola ◽  
P. Huttunen ◽  
...  
Keyword(s):  
Real World ◽  
Economic Outcomes ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Paediatric Patients

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

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

scholarly journals Clinical experience with moroctocog alfa (AF-CC) in younger paediatric patients with severe haemophilia A: Two open-label studies

Haemophilia ◽  
2018 ◽  
Vol 24 (4) ◽  
pp. 604-610 ◽  
Author(s):  
L. Rusen ◽  
K. Kavakli ◽  
J. Korth-Bradley ◽  
F. Huard ◽  
P. Rendo ◽  
...  
Keyword(s):  
Clinical Experience ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Open Label ◽  
Paediatric Patients
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