Pharmaceuticals Policy and Law - Volume 7, issue 0 - Journals

Abstract: Haemophilia is an x-chromosomal linked, hereditary lifelong disease which causes damages of bones and joints as well as early death. Per definition there are three different forms, the severe form (less than 1% of coagulation factor activity) demands regular substitution therapy with the missing coagulation factor. Haemophilia therapy developed since the 1960ies rapidly and today safe clotting factor concentrates are available, plasma – derived as well as recombinant. Nevertheless some 80% of the patients …affected do not receive treatment at all and only some 5 to 10% receive the so called "state of the art" treatment of their bleeding disorder. Non- viral- inactivated- clotting factor concentrates caused an epidemic of HIV/Aids within this population in the 1970ies and 1980ies, with deaths of some 50% (in some Haemophilia Treatment Centers even higher) of continuous treated patients. This fatal experience promoted research not only in gene- and biotechnology, but, despite of the early promises of the involved scientists and industries, a satisfying solution for Persons with Haemophilia is still missing. Today plasma derived clotting factor concentrates have to undergo various viral- inactivation- procedures, donor screening and pool- as well as final product testing is essential, strict legislation on blood safety issues became standard in most countries. Show more

Citation: Pharmaceuticals, Policy and Law, vol. 7, no. 0, pp. 119-123, 2006

Price: EUR 27.50

The physician's perspective on the use of immunoglobulins: Advantages and limits of the immunoglobulin therapy

Authors: Quinti, Isabella | Cagliuso, Maria | Fiorilli, Massimo

Article Type: Research Article

Abstract: Medical knowledge on the clinical investigation of human normal immunoglobulin for intravenous administration and treatment regimens are in a state of continuous development. Due to recent achievements, the question arises whether the currently established indications and their respective posologies might require modification to more adequately reflect current clinical practice with intravenous or subcutaneous immunoglobulins as substitutive therapy in patients affected by primary and secondary immunodeficiencies and as immunomodulators in patients affected by …autoimmune disorders. In addition, immunoglobulin therapies used "off label" have proved beneficial and other possible inclusion will be expected in the next future. In this review we will identify the clinical and immunological benefits and limits of imunoglobulin treatments and outline the physician's persepective on this topic. Show more

Citation: Pharmaceuticals, Policy and Law, vol. 7, no. 0, pp. 125-133, 2006

Price: EUR 27.50

Commitment to care: Delivering therapy to Alpha-1 patients

Authors: Hutt, Edward

Article Type: Research Article

Abstract: Plasma products are used to treat a number of serious inherited disorders, of which the best known is haemophilia. However, a number of other conditions exist at a similar frequency in the population but are much less well known: Alpha 1-Antitrypsin (AAT) deficiency or 'Alpha-1' is one of these. Alpha-1 is a life-threatening genetic disorder in some individuals who demonstrate clinical evidence of emphysema. Left untreated, this form of emphysema can be fatal. Historically, people with …Alpha-1 have been concerned with the critical short supply of the therapy. Recent product approvals will provide the desperately needed product, so people with Alpha-1 can focus more on their lives and less on when their therapy will be available. Show more

Citation: Pharmaceuticals, Policy and Law, vol. 7, no. 0, pp. 135-140, 2006

Price: EUR 27.50