In this commentary, we review the principles utilized in a recent systematic review of the use of haemophilia products carried out in Sweden as part of an HTA. We suggest that ranking haemophilia related interventions with the standard interventions of therapeutics and public health in CUA comparisons is inappropriate. Given that haemophilia treatment is a form of blood replacement therapy, we propose that such comparisons should be made with

Selumetinib the interventions of mainstream blood transfusion. We suggest that unequivocally effective treatments such as haemophilia therapies should be assessed differently from mainstream interventions, that new methodologies are required for these kinds of diseases and that evidence of a societal willingness to support people with rare disorders

needs to be recognized when reimbursement policies are developed. “
“Knowledge regarding the management of orthopaedic surgery in patients with rare bleeding disorders (RBDs) is limited. GS-1101 molecular weight Retrospective data collection and analysis of 35 orthopaedic procedures (6 minor and 29 major) carried out in 22 patients with RBD between 1982 and 2013. These surgeries were performed using heterogeneous regimens of hemostatic therapy, except for seven procedures performed with no hemostatic treatment in four patients with mild factor deficiency. Of the 28 procedures carried out with hemostatic treatment, nine (32%) were performed using replacement therapy with dosages of concentrates of the deficient factor aimed to achieve perioperative plasma levels judged to be compatible with hemostasis; three (11%) using factor replacement therapy associated with fresh frozen plasma (FFP); four (14%) using recombinant activated factor VII; four (14%) using virus inactivated plasma alone; three (11%) using virus inactivated plasma associated with desmopressin; one (4%) using FFP alone; and four (14%)

procedures using tranexamic acid alone. Bleeding complications occurred in 7 of 35 procedures (20%) involving five patients. Prophylaxis of venous thromboembolism was performed only in one case with no excessive bleeding, MCE but two patients not on thromboprophylaxis developed superficial thrombophlebitis. A satisfactory control of hemostasis was achieved in most patients. In some of those characterized by mild factor deficiency (FVII, FXI) hemostatic treatment could be avoided in some instances. The control of hemostasis combined with an adequate surgical technique is needed for the successful outcome of orthopaedic surgery in RBDs that requires the involvement of specialized haemophilia centres. “
“Summary.  Worldwide, children with haemophilia suffer from limitations in performing activities of daily living. To measure such limitations in adults a disease-specific instrument, the Haemophilia Activities List (HAL), was created in 2004.