The cost associated with a clinical condition can be categorized into three main categories: direct, indirect, and intangible. Direct costs are items that are more closely related to the disease and its treatment (e.g. drug treatment, hospitalizations, surgery, physiotherapy), indirect costs that are related to productivity varation due to the disease and its management, and intangible costs that refer to the consequences of a disease and of a medical treatment on individuals' quality of life.
What is the rationale for a new concentrate of a Factor IX (FIX) Extended Half Life? The reduction of the Annual Bleeding Ratio (ABR) and the prevention of further hemarthrosis in the target joints through weekly infusions. Other objectives are: the improvement of the joint status and the quality of life thanks to the possibility of reducing the frequency of prophylactic infusions, and the reduction of the risk of inhibitors. Nonacog beta pegol N9-GP (Refixia, Novonordisk) is an increased half-life recombinant FIX (rFIX) that meets all these needs. Refixia is produced in Chinese hamster ovary (CHO) cells by a serum-free manufacturing process.
COVID19 has dramatically modified several aspects of medical care, this is particularly true for patients affected by a chronic, rare disease like haemophilia A and B. Several issues and concerns have been raised during the course of the current pandemic, which include: the relations between physicians and patients; the management of replacement therapies and prophylaxis, with the prompt development of ad hoc recommendations by National and International Societies involved in haemophilia care; advice about vaccines against Sars-CoV2 and their administration; the development of telemedicine and its widespread use. The current presentation describes each of these specific aspects, currently still involving the “new and next future” management of haemophilia.
Thrombotic microangiopathies (TMAs) are a wide spectrum of diseases characterized by thrombocytopenia, microangiopathic hemolytic anemia and widespread ischemic damage due to microvascular thrombosis. TMAs could be life-threatening unless promptly recognized and treated, hence demand a rapid differential diagnosis and initiation of proper therapy, especially in the light of the new targeted therapies available nowadays. Thrombotic thrombocytopenic purpura (TTP) is a rare and severe primary TMA, caused by the congenital or acquired deficiency of ADAMTS13, the von Willebrand factor-cleaving protease.
P. M. Mannucci
Haematological complications caused by autoimmune mechanisms which are associated with haemorrhages, thrombosis, or both, have been described after vaccinations against various infectious, viral or bacterial diseases. The most frequent examples occur after vaccines administered to young children (e.g. against measles/mumps/chickenpox/rubella), after vaccination against diphtheria/tetanus/whooping cough, but also in adults after polio, hepatitis B, influenza and pneumococcus vaccines. Although rare in absolute terms, the most frequent complications are autoimmune thrombocytopenia accompanied by haemorrhagic manifestations.