Controversies regarding the prophylactic management of adults with severe haemophilia A

Valentino, Leonard A.

doi:10.1111/j.1365-2516.2009.02159.x

Cited by 17 publications

(16 citation statements)

References 68 publications

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“…While reaching more advanced age, persons with hemophilia are developing medical and surgical conditions, such as cardiovascular diseases and cancers, that were previously uncommon [17,18,51-53]. These diseases represent novel causes of morbidity and mortality, whose management represents a challenge for hemophilia caregivers [54,55].…”

Section: Treatment Of Hemophilia: the Presentmentioning

confidence: 99%

Past, present and future of hemophilia: a narrative review

Franchini

Mannucci

2012

Orphanet J Rare Dis

210

196

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Over the past forty years the availability of coagulation factor replacement therapy has greatly contributed to the improved care of people with hemophilia. Following the blood-borne viral infections in the late 1970s and early 1980, caused by coagulation factor concentrates manufactured using non-virally inactivated pooled plasma, the need for safer treatment became crucial to the hemophilia community. The introduction of virus inactivated plasma-derived coagulation factors and then of recombinant products has revolutionized the care of these people. These therapeutic weapons have improved their quality of life and that of their families and permitted home treatment, i.e., factor replacement therapy at regular intervals in order to prevent both bleeding and the resultant joint damage (i.e. primary prophylaxis). Accordingly, a near normal lifestyle and life-expectancy have been achieved. The main current problem in hemophilia is the onset of alloantibodies inactivating the infused coagulation factor, even though immune tolerance regimens based on long-term daily injections of large dosages of coagulation factors are able to eradicate inhibitors in approximately two-thirds of affected patients. In addition availability of products that bypass the intrinsic coagulation defects have dramatically improved the management of this complication. The major challenges of current treatment regimens, such the short half life of hemophilia therapeutics with need for frequent intravenous injections, encourage the current efforts to produce coagulation factors with more prolonged bioavailability. Finally, intensive research is devoted to gene transfer therapy, the only way to ultimately obtain cure in hemophilia.

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Section: Treatment Of Hemophilia: the Presentmentioning

confidence: 99%

Past, present and future of hemophilia: a narrative review

Franchini

Mannucci

2012

Orphanet J Rare Dis

210

196

View full text Add to dashboard Cite

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“…Paradoxically, limited evidence suggests that those patients who permanently discontinued prophylaxis tended to have a milder bleeding pattern than those who continued prophylaxis [25]. According to one reference counselling for adolescents and young adults about the consequences of abandoning primary prophylaxis is essential to prevent complications [28].…”

Section: Summary Of Findings On Treatmentmentioning

confidence: 99%

The History and Evolution of the Clinical Effectiveness of Haemophilia Type A Treatment: A Systematic Review

Castro

Briceño

Casas

et al. 2012

Indian J Hematol Blood Transfus

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First evidence of cases of haemophilia dates from ancient Egypt, but it was when Queen Victoria from England in the 19th century transmitted this illness to her descendants, when it became known as the ''royal disease''. Last decades of the 20th century account for major discoveries that improved the life expectancy and quality of life of these patients. The history and evolution of haemophilia healthcare counts ups and downs. The introduction of prophylactic schemes during the 1970s have proved to be more effective that the classic on-demand replacement of clotting factors, nevertheless many patients managed with frequent plasma transfusions or derived products became infected with the Human Immunodeficiency Virus (HIV) and Hepatitis C virus during the 1980s and 1990s. Recombinant factor VIII inception has decreased the risk of blood borne infections and restored back longer life expectancies. Main concerns for haemophilia healthcare are shifting from the pure clinical aspects to the economic considerations of long-term replacement therapy. Nowadays researchers' attention has been placed on the future costs and cost-effectiveness of costly long-term treatment. Equity considerations are relevant as well, and alternative options for less affluent countries are under the scope of further research. The aim of this review was to assess the evidence of different treatment options for haemophilia type A over the past four decades, focusing on the most important technological advances that have influenced the natural course of this ''royal disease''.

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“…Recent observations suggest protective effect of early start of prophylaxis on inhibitor development [5,6]. At present the feasibility of indefinite extension of prophylaxis in adulthood has been intensively discussed [7,8]. Substantial progress has been achieved also in the treatment of haemophilia with inhibitors, including the availability of effective bypassing agents and the adoption of immune tolerance induction (ITI) as a first-choice therapy for newly developed inhibitors [9].…”

Section: Introductionmentioning

confidence: 99%

Special lectures in haemophilia management

2010

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Summary. During the last two decades major advances have been achieved in the management of haemophilia. Modern approaches aimed at preventing the recurrent bleedings and their sequelae have been widely adopted. Major challenges of intensive treatment regimens employed today, such a short half life of haemophilia therapeutics with a need for frequent injections and the risk of inhibitor, encourage further development towards the production of factor concentrates with prolonged efficacy and reduced immunogenicity. Intensive research work on gene therapy aimed at ultimate cure of haemophilia by the restoration of missing factor FVIII (FVIII) and factor IX (FIX) production is ongoing. The current issues of gene therapy and mechanisms, modifying the host immune response to the FVIII and FIX transgene material and the coagulation factors expressed are the topic of the Arosenius lecture by Katherine High. Despite an extensive research on mechanisms leading to inhibitor development, the real reason of these serious complications of haemophilia therapy still remains unclear. Alessandro Gringeri will discuss the immunogenicity of plasma derived FVIII (pd FVIII) and recombinant FVIII (rFVIII) concentrates as one of potential, treatment related, and probably ÔmodifiableÕ risk factors for inhibitor development. The SIPPET study -a new prospective, randomised study aimed to reveal real incidence of inhibitors in patients treated with either pdFVIII or rFVIII will be presented.

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Controversies regarding the prophylactic management of adults with severe haemophilia A

Cited by 17 publications

References 68 publications

Past, present and future of hemophilia: a narrative review

Past, present and future of hemophilia: a narrative review

The History and Evolution of the Clinical Effectiveness of Haemophilia Type A Treatment: A Systematic Review

Special lectures in haemophilia management

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