Factor replacement for haemophilia – should cryoprecipitate be used?

Srivastava,

doi:10.1046/j.1365-2516.1999.00337.x

Cited by 23 publications

(18 citation statements)

References 18 publications

(17 reference statements)

Supporting

Mentioning

Contrasting

Order By: Relevance

“…The issues underpinning access to cryo have been reviewed [10]. Ensuring optimal FVIII yields through careful attention to blood processing and plasma thawing conditions is crucial, as these determine the supply of the product as well as the safety.…”

Section: Product Options – Local Productsmentioning

confidence: 99%

Safety and supply of haemophilia products: worldwide perspectives

Farrugia¹

2004

Haemophilia

View full text Add to dashboard Cite

The survival and well-being of people with haemophilia depends on the supply of safe therapeutic products. Safety and supply are entirely intertwined principles; in the absence of adequate amounts of coagulation products, safety measures may be compromised in order to enhance supply, leading to risks which may result in morbidity and mortality. As haemophilia therapy has emerged through the development of blood transfusion and plasma fractionation, the safety of the blood supply in general has had a strong effect on haemophilia care. Despite the gradual detachment of haemophilia care from blood transfusion through the use of recombinant products, the majority of the world's population with haemophilia in the developing world will be reliant on blood products for the foreseeable future. It is, therefore, important to continue efforts for a safe and sufficient blood supply worldwide. As such a blood supply develops, possibly in tandem with an independent plasma fractionation industry, the level of haemophilia care should improve with the gradual introduction of concentrates for the ultimate goal of covering all aspects of care. Constant vigilance for the threat of blood-borne pathogens should be linked to considerations of how these products are to be manufactured. This should be governed entirely by considerations of safety and pharmaceutical competence. Of equal importance is a governmental capacity to oversee the entry and maintenance of these products on the market. While it is not possible for all countries to have a regulatory authority of the same status as that of the developed countries, it is perfectly feasible to develop a set of basic principles which allow an assessment of basic product safety, quality and efficacy to be made.

show abstract

Section: Product Options – Local Productsmentioning

confidence: 99%

Safety and supply of haemophilia products: worldwide perspectives

Farrugia¹

2004

Haemophilia

View full text Add to dashboard Cite

show abstract

“…The average factor consumption among approximately 7000 registered haemophilic people is between 1000 and 2000 IU year −1 and that too in a highly inequitable distribution depending on the ability of each individual to pay for his treatment. Plasma and cryoprecipitate cost much less at $0.03–0.05 IU −1 and are certainly more affordable [10].…”

Section: Products For Factor Replacement In Developing Countriesmentioning

confidence: 99%

“…Most of them receive very little or no factor concentrates. Blood bank products continue to be used for factor replacement therapy [10]. As efforts are made to improve their care, it is necessary to develop more practical models for management of haemophilia in these countries.…”

mentioning

confidence: 99%

Factor replacement therapy in haemophilia – are there models for developing countries?

Srivastava

2003

Haemophilia

View full text Add to dashboard Cite

Successful models for factor replacement in severe haemophilia involves prophylactic or on-demand administration of large quantities (1500-9000 IU kg-1 year-1) of very high purity factor concentrates starting early in life. The prohibitive cost of these protocols make them completely impractical in developing countries where the quantity of factor used for replacement therapy is much lower and varies considerably (25-500 IU kg-1 year-1). At this level of treatment, as some joint damage is inevitable, the aim of therapy shifts to preventing disability and preserving reasonable joint function rather than perfect architecture. There are no carefully recorded data on long-term outcome of musculo-skeletal function on these doses. Appropriate studies are needed to document such data. With regard to products for factor replacement, economic compulsions lead to the use a variety of factor concentrates and blood-bank products for replacement therapy. Most patients in the developing world do not have access to adequate replacement therapy. Among the rest, some get limited quantities of plasma-derived concentrates while others use cryoprecipitate, fresh frozen plasma or even whole blood. Since the superiority of virus-inactivated purified factor concentrates in achieving the aims of replacement therapy is well established, the aim should be to provide this for all people with haemophilia. This can be achieved by production of such concentrates locally or by importing them. Different models are possible depending on the circumstances in each country.

show abstract

“…The major concern with the use of fresh-frozen plasma and cryoprecipitate is the risk of transmitted viruses by transfusion, especially HIV and hepatitis. [37] The development of clotting factor concentrates during the last three decades has revolutionized hemophilia care, and has had a major impact on patient morbidity and mortality. The introduction of plasma donor screening, viral inactivation methods, and DNA technology into the manufacturing of concentrates has minimized the problem of viral transmission with clotting factor concentrates.…”

Section: Clotting Factor Concentratesmentioning

confidence: 99%

Treatment Strategies in Children with Hemophilia

Petrini

2002

Pediatric Drugs

View full text Add to dashboard Cite

Hemophilia is an inherited bleeding disorder caused by quantitative or qualitative defects in the synthesis of factor VIII (FVIII) or factor IX (FIX). Clinically, it is divided into severe, moderate and mild disease depending on the levels of FVIII or FIX in the blood. The bleeding tendency is most pronounced and can start at a very young age in severe hemophilia, which is characterized by repeated hemorrhage into the joints and muscles. Without treatment, these episodes lead to severe arthropathy, and there is also a high risk of lethal cerebral hemorrhage. The treatment of bleeding symptoms requires the correction of the coagulation defect. Factor concentrates have been available for 30 years, initially with the development of cryoprecipitate, subsequently with increasingly purified plasma-derived forms, and ultimately with recombinant clotting factor concentrates. The advantage of this highly effective therapy has been subdued by the outbreak of HIV and Hepatitis C infections in patients with hemophilia treated with factor concentrates which did not have adequate viral inactivation steps in the purification process. Plasma-derived and recombinant factor concentrates are today considered to have a good safety profile, but are only available for a small group of hemophilia patients worldwide. A multidisciplinary team approach is important for early diagnosis, communication with the patient and parents, and to tailor the best treatment possible with the amount of clotting factor concentrates available. The main goal of hemophilia treatment is to prevent bleeding symptoms and allow normal integration in social life. In patients with severe hemophilia, this can best be achieved by early home treatment and primary prophylaxis. Future developments in gene therapy may transform severe hemophilia to a mild form, with no need for regular injections of clotting factor concentrates.

show abstract

Factor replacement for haemophilia – should cryoprecipitate be used?

Cited by 23 publications

References 18 publications

Safety and supply of haemophilia products: worldwide perspectives

Safety and supply of haemophilia products: worldwide perspectives

Factor replacement therapy in haemophilia – are there models for developing countries?

Treatment Strategies in Children with Hemophilia

Contact Info

Product

Resources

About