scholarly journals Modelling lifelong effects of different prophylactic treatment strategies for severe haemophilia A

Haemophilia ◽  
2016 ◽  
Vol 22 (5) ◽  
Author(s):  
K. Fischer ◽  
D. Lewandowski ◽  
M. P. Janssen
Keyword(s):  
Prophylactic Treatment ◽  
Treatment Strategies ◽  
Haemophilia A ◽  
Severe Haemophilia

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.

scholarly journals PB1842 ROLE OF THROMBOELASTOGRAPHY IN THE MONITORING OF PROPHYLACTIC TREATMENT IN PATIENTS WITH SEVERE HAEMOPHILIA A

HemaSphere ◽  
2019 ◽  
Vol 3 (S1) ◽  
pp. 842
Author(s):  
V. Krasivska ◽  
O. Stasyshyn ◽  
M. Semerak ◽  
O. Tushnytskyi
Keyword(s):  
Prophylactic Treatment ◽  
Haemophilia A ◽  
Severe Haemophilia

Epidemiology and Treatment of Patients with Haemophilia in Austria—Update from the Austrian Haemophilia Registry

2018 ◽  
Vol 39 (03) ◽  
pp. 284-293 ◽  
Author(s):  
Judit Rejtő ◽  
Sylvia Reitter-Pfoertner ◽  
Sylvia Kepa ◽  
Clemens Feistritzer ◽  
Michael Grundbichler ◽  
...  
Keyword(s):  
Prophylactic Treatment ◽  
Hiv Infections ◽  
Epidemiological Data ◽  
Treatment Modalities ◽  
Hiv Positive ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Haemophilia B ◽  
Immunodeficiency Virus ◽  
Mild Haemophilia

AbstractThe Austrian Haemophilia Registry collects epidemiological data on patients with haemophilia, on treatment modalities and potential side effects. The Registry covers more than 85% of the assumed total number of haemophilia patients in Austria. This report summarizes data on 753 patients: 84.3% (635) have haemophilia A and 15.7% (118) have haemophilia B. Patients' median age is 34 years (range: 1–93 years). Of the total cohort, 39.0% (294) patients have severe haemophilia, 11.3% (85) moderate haemophilia, and 49.4% (372) mild haemophilia. Of the patients with severe haemophilia, 38.4% (113) have been infected with hepatitis C virus (HCV) and 12.6% (37) are human immunodeficiency virus (HIV) positive. Overall, 10.6% (67) of patients with haemophilia A and 1.7% (2) of those with haemophilia B have had an inhibitor in their history. Among patients with severe haemophilia, 68.4% (201) receive prophylaxis and 28.6% (84) receive on-demand therapy. There are 65.0% (191) patients with severe haemophilia who are treated with recombinant products. In conclusion, most patients with severe haemophilia receive prophylactic treatment. HCV and HIV infections are still important issues in the Austrian haemophilia population.

Effects of prophylactic treatment regimens in children with severe haemophilia: a comparison of different strategies

Haemophilia ◽  
2002 ◽  
Vol 8 (s2) ◽  
pp. 43-46 ◽  
Author(s):  
H. M. Van Den Berg ◽  
K. Fischer ◽  
J. G. Van Der Bom ◽  
G. Roosendaal ◽  
E. P. Mauser-Bunschoten
Keyword(s):  
Prophylactic Treatment ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Treatment Regimens

Consequences of switching from prophylactic treatment to on-demand treatment in late teens and early adults with severe haemophilia A: the TEEN/TWEN study

Haemophilia ◽  
2013 ◽  
Vol 19 (5) ◽  
pp. 727-735 ◽  
Author(s):  
M. J. Manco-Johnson ◽  
J. Sanders ◽  
N. Ewing ◽  
N. Rodriguez ◽  
M. Tarantino ◽  
...  
Keyword(s):  
Prophylactic Treatment ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
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