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

Discontinuing early prophylaxis in severe haemophilia leads to deterioration of joint status despite low bleeding rates

2016 ◽  
Vol 115 (05) ◽  
pp. 931-938 ◽  
Author(s):  
Annelies Nijdam ◽  
Wouter Foppen ◽  
Piet de Kleijn ◽  
Evelien P. Mauser-Bunschoten ◽  
Goris Roosendaal ◽  
...  
Keyword(s):  
Functional Limitations ◽  
Objective Assessment ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
On Demand ◽  
Recommended Treatment ◽  
Objective Monitoring ◽  
Joint Health ◽  
Observational Cohort

SummaryProphylaxis is the recommended treatment for children with severe haemophilia A, but whether prophylaxis should be continued in adulthood is still under debate. Previous studies with limited follow-up have suggested that some patients may be able to stop prophylaxis in adulthood, while maintaining good joint health. This single-centre observational cohort study examined patients with severe haemophilia A born 1970–1988 without inhibitor development, and assessed the long-term consequences of discontinuing prophylaxis. Patient-initiated changes in prophylaxis, including all switches to on-demand treatment lasting a minimum of two consecutive weeks, were recorded from the time self-infusion began until the last evaluation. Sixty-six patients were evaluated at a median age of 32.4 years: 26 of patients had stopped prophylaxis for a median of 10 years, 15 had interrupted prophylaxis and 59 had continued prophylaxis. Annual joint bleeding rate (AJBR), Haemophilia Joint Health Score (HJHS-2.1; 0–124 points), radiological Pettersson score (0–78 points) and Haemophilia Activities List score (HAL; 100–0 points) were compared between patients who stopped and patients who continued prophylaxis. Although self-reported bleeding rates and functional limitations were similar in both groups (AJBR: 1.5 vs 1.2 and HAL: 84 vs 84 for those who stopped and continued prophylaxis, respectively), objective assessment of joint status showed increased arthropathy after 10 years of on-demand treatment in patients who stopped prophylaxis compared with those who continued (HJHS: 23 vs. 14 and Pettersson: 16 vs 5, respectively; P< 0.01). These results support continuation of long-term prophylaxis in adults and demonstrate the need for objective monitoring of joint status.Trial registration: Dutch Trial Registry number 3098; UTN U1111–1121–7069.

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
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