Pharmacotherapy of haemophilia A.
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Pharmacotherapy of haemophilia A. / Gringeri, Alessandro; Muça-Perja, Myrvet; Mangiafico, Lucia; Mackensen von, Sylvia.
In: EXPERT OPIN BIOL TH, Vol. 11, No. 8, 8, 2011, p. 1039-1053.Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review
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TY - JOUR
T1 - Pharmacotherapy of haemophilia A.
AU - Gringeri, Alessandro
AU - Muça-Perja, Myrvet
AU - Mangiafico, Lucia
AU - Mackensen von, Sylvia
PY - 2011
Y1 - 2011
N2 - INTRODUCTION: Haemophilia A is due to factor VIII (FVIII) deficiency. The main treatment is replacement therapy with FVIII concentrates. However, these concentrates carried a high risk of blood-borne viral infections and still have a high risk of inducing anti-FVIII inhibitors. AREAS COVERED: An overview of products available and therapeutic options for haemophilia A management in order to help in decision making. A literature search using Medline with the keywords: 'haemophilia', 'factor VIII', 'therapy', 'inhibitor', 'concentrate', 'bleeding', 'prophylaxis', 'on demand', 'plasma-derived', 'recombinant', 'coagulation factors', 'immunotolerance' was performed. The years 1960 - 2010 are included. EXPERT OPINION: Progress in management of patients with haemophilia A has allowed increased life expectancy and quality of life. There is evidence that prophylaxis prevents or, at least, slows down arthropathy development when started early in childhood. FVIII concentrates have achieved high levels of blood-borne pathogen safety. However, treatment is frequently complicated by development of FVIII-neutralizing inhibitors, which prevent control of bleeding and predispose to a high morbidity and mortality risk. Bypassing agents are effective in bleeding treatment in a high percentage of cases. Prophylaxis with bypassing agents and their use in combination are offering opportunities in management of inhibitor patients. More evidence is necessary to understand how to prevent and manage this complication.
AB - INTRODUCTION: Haemophilia A is due to factor VIII (FVIII) deficiency. The main treatment is replacement therapy with FVIII concentrates. However, these concentrates carried a high risk of blood-borne viral infections and still have a high risk of inducing anti-FVIII inhibitors. AREAS COVERED: An overview of products available and therapeutic options for haemophilia A management in order to help in decision making. A literature search using Medline with the keywords: 'haemophilia', 'factor VIII', 'therapy', 'inhibitor', 'concentrate', 'bleeding', 'prophylaxis', 'on demand', 'plasma-derived', 'recombinant', 'coagulation factors', 'immunotolerance' was performed. The years 1960 - 2010 are included. EXPERT OPINION: Progress in management of patients with haemophilia A has allowed increased life expectancy and quality of life. There is evidence that prophylaxis prevents or, at least, slows down arthropathy development when started early in childhood. FVIII concentrates have achieved high levels of blood-borne pathogen safety. However, treatment is frequently complicated by development of FVIII-neutralizing inhibitors, which prevent control of bleeding and predispose to a high morbidity and mortality risk. Bypassing agents are effective in bleeding treatment in a high percentage of cases. Prophylaxis with bypassing agents and their use in combination are offering opportunities in management of inhibitor patients. More evidence is necessary to understand how to prevent and manage this complication.
KW - Animals
KW - Humans
KW - Blood Coagulation Factors/therapeutic use
KW - Hemophilia A/drug therapy
KW - Animals
KW - Humans
KW - Blood Coagulation Factors/therapeutic use
KW - Hemophilia A/drug therapy
M3 - SCORING: Journal article
VL - 11
SP - 1039
EP - 1053
JO - EXPERT OPIN BIOL TH
JF - EXPERT OPIN BIOL TH
SN - 1471-2598
IS - 8
M1 - 8
ER -
