scholarly journals Long-Term Expression of Human Coagulation Factor VIII in a Tolerant Mouse Model Using the φC31 Integrase System

2012 ◽  
Vol 23 (4) ◽  
pp. 390-398 ◽  
Author(s):  
Christopher L. Chavez ◽  
Annahita Keravala ◽  
Jacqueline N. Chu ◽  
Alfonso P. Farruggio ◽  
Vanessa E. Cuéllar ◽  
...  
Keyword(s):  
Mouse Model ◽  
Factor Viii ◽  
Coagulation Factor ◽  
Coagulation Factor Viii ◽  
Φc31 Integrase ◽  
Tolerant Mouse

Coagulation factor VIII levels are associated with long-term survival – interactions with gender in a large hospital-based cohort

2010 ◽  
Vol 122 (11-12) ◽  
pp. 334-340 ◽  
Author(s):  
Florian M. Kovar ◽  
Claudia L. Marsik ◽  
Christian Joukhadar ◽  
Thomas Perkmann ◽  
Helmuth Haslacher ◽  
...  
Keyword(s):  
Factor Viii ◽  
Coagulation Factor ◽  
Coagulation Factor Viii ◽  
Large Hospital ◽  
Term Survival ◽  
Long Term Survival

Therapy of Coagulation Factor VIII Autoantibodies with Long-term Extracorporeal Protein A Adsorption and Immunosuppression

1997 ◽  
Vol 19 ◽  
pp. 39-42 ◽  
Author(s):  
Behrouz Mansouri Taleghani ◽  
Ralf Grossmann ◽  
Franz Keller ◽  
Dieter Wiebecke
Keyword(s):  
Factor Viii ◽  
Protein A ◽  
Coagulation Factor ◽  
Coagulation Factor Viii

scholarly journals Prediction of long-term survival and coagulation factor VIII levels: interactions with gender in a large hospital-based cohort

Critical Care ◽  
2009 ◽  
Vol 13 (Suppl 1) ◽  
pp. P435
Author(s):  
F Kovar ◽  
C Marsik-Strasser ◽  
C Joukhadar ◽  
T Schickbauer ◽  
P Kyrle ◽  
...  
Keyword(s):  
Factor Viii ◽  
Coagulation Factor ◽  
Coagulation Factor Viii ◽  
Large Hospital ◽  
Term Survival ◽  
Long Term Survival

RECOVERY TEST AND ITS ROLE IN ASSESSMENT OF LONG-TERM PROPHYLAXIS EFFECTIVENESS IN HEMOPHILIA A PATIENTS

2017 ◽  
Author(s):  
Т. Андреева ◽  
И. Лавриченко ◽  
В. Константинова ◽  
А. Ким ◽  
О. Крашенинникова ◽  
...  
Keyword(s):  
Factor Viii ◽  
Hemophilia A ◽  
Coagulation Factor ◽  
Recombinant Factor Viii ◽  
Coagulation Factor Viii ◽  
Recovery Test ◽  
Fviii Activity ◽  
In Vivo Recovery

Введение. Гемофилия А (ГФА) — сцепленное с Х-хромосомой рецессивное врожденное заболевание, обусловленное недостаточностью VIII фактора свертывания крови (FVIII). Главным признаком заболевания является склонность к кровотечениям. Клинические проявления ГФА, как правило, коррелируют с уровнем активности FVIII. Единственным эффективным лечением гемофилии в настоящее время является заместительная терапия препаратами дефицитного фактора. Материалы и методы. Проведен ретроспективный анализ результатов обследования 28 пациентов (14 детей в возрасте от 3 до 17 лет и 14 взрослых в возрасте от 21 до 57 лет) с тяжелой формой ГФА. Все пациенты находились на длительной профилактической терапии препаратами FVIII — как рекомбинантными, так и плазматическими. Для подбора оптимального режима профилактики пациенту с ГФА проводили тест восстановления (in vivo recovery) — определение активности FVIII в двух пробах крови; определяли также прирост показателя восстановления (incremental recovery), активированное частичное тромбопластиновое время и каолиновое время. Результаты. Проведена оценка теста восстановления фактора свертывания VIII при применении плазматических и рекомбинантного препаратов фактора VIII у пациентов с тяжелой гемофилией А; изучена корреляция теста восстановления с фармакодинамическими показателями, характеризующими состояние коагуляции. Заключение. Ввиду наличия внутрииндивидуальных и межиндивидуальных колебаний фармакокинетических показателей препаратов фактора свертывания VIII у пациентов с ГФА рекомендуется проведение теста восстановления как при назначении препарата фактора свертывания VIII, так и в процессе длительной профилактики с целью контроля за терапией и ее оптимизации в случае необходимости. Introduction. Hemophilia A (HРA) is a recessive X-linked congenital disease caused by the defi ciency of VIII coagulation factor (FVIII). Hemorrhagic tendency is the main sign of the disease. As a rule, clinical manifestations of HPA correlate with level of FVIII activity. At present time substitution therapy with medication of defi cit factor is the only eff ective treatment for hemophilia. Materials and methods. We performed a retrospective study analysis of 28 patients (14 children aged from 3 to 17 years and 14 adults aged from 21 to 57 years) examination with severe HРA. All patients received prolonged preventive therapy with recombinant factor VIII products. To select the optimal prophylaxis mode for patients with HPA we performed a recovery test (in vivo recovery), determination of FVIII activity in two blood samples, determined index of incremental recovery, activated partial thromboplastin time and kaolin time. Results. We assessed recovery test for coagulation factor VIII using plasma and recombinant factor VIII products in patients with severe haemophilia A and studied correlation of recovery test with pharmacodynamic parameters that characterized coagulation state. Conclusion. Due to intra-individual and inter-individual fl uctuations in pharmacokinetic parameters of recombinant factor VIII products in patients with HPA we recommend to perform a recovery test both at appointment of coagulation factor VIII product and also during of long-term prophylaxis for monitoring and optimization of therapy.

scholarly journals Gene Therapy in Hemophilia: Recent Advances

2021 ◽  
Vol 22 (14) ◽  
pp. 7647
Author(s):  
E. Carlos Rodríguez-Merchán ◽  
Juan Andres De Pablo-Moreno ◽  
Antonio Liras
Keyword(s):  
Gene Therapy ◽  
Adverse Events ◽  
Factor Viii ◽  
Coagulation Factor ◽  
Cost Savings ◽  
Factor Ix ◽  
Clotting Factors ◽  
Advanced Therapies ◽  
Potential Benefits

Hemophilia is a monogenic mutational disease affecting coagulation factor VIII or factor IX genes. The palliative treatment of choice is based on the use of safe and effective recombinant clotting factors. Advanced therapies will be curative, ensuring stable and durable concentrations of the defective circulating factor. Results have so far been encouraging in terms of levels and times of expression using mainly adeno-associated vectors. However, these therapies are associated with immunogenicity and hepatotoxicity. Optimizing the vector serotypes and the transgene (variants) will boost clotting efficacy, thus increasing the viability of these protocols. It is essential that both physicians and patients be informed about the potential benefits and risks of the new therapies, and a register of gene therapy patients be kept with information of the efficacy and long-term adverse events associated with the treatments administered. In the context of hemophilia, gene therapy may result in (particularly indirect) cost savings and in a more equitable allocation of treatments. In the case of hemophilia A, further research is needed into how to effectively package the large factor VIII gene into the vector; and in the case of hemophilia B, the priority should be to optimize both the vector serotype, reducing its immunogenicity and hepatotoxicity, and the transgene, boosting its clotting efficacy so as to minimize the amount of vector administered and decrease the incidence of adverse events without compromising the efficacy of the protein expressed.

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