Meeting unmet needs in inhibitor patients.
For patients with haemophilia, the development of inhibitors complicates treatment, and inhibitor patients may thus have a range of unmet needs. Although successful inhibitor eradication will render patients responsive to factor replacement therapy, with potentially beneficial effects on long-term outcomes, this may not always be possible. Physicians treating inhibitor patients should aim to achieve reliable control of bleeding episodes, and the prevention of joint disease should also be a priority. Patients with high-titre inhibitors require therapy with bypassing agents--recombinant activated factor VII (rFVIIa) or a plasma-derived activated prothrombin complex concentrate (pd-APCC)--for the treatment of bleeding. When treating joint haemorrhage in inhibitor patients, both aggressive treatment of intercurrent joint bleeds and prophylaxis should be considered, although evidence is needed as to whether prophylaxis with bypassing agents can significantly delay/prevent the development of osteochondral changes in patients with inhibitors. Despite physicians' best efforts, joint disease may ultimately occur in inhibitor patients, and in such instances optimizing treatment, of both early and late stages, is important. There is no single therapeutic modality for dealing with the various treatment challenges posed by inhibitor patients, but overall goals should be to improve quality of life, with the provision of cost-effective care that aims to maintain physical function.
Blanchette
V S
VS
Division of Hematology/Oncology, The Hospital for Sick Children, Department of Pediatrics, University of Toronto, Toronto, ON, Canada. victor.blanchette@sickkids.ca
Manco-Johnson
M J
MJ
D019774:Blood Coagulation Factor Inhibitors / Q000037:antagonists & inhibitors / Q000378:metabolism; D001779:Blood Coagulation Factors / Q000627:therapeutic use*; D019468:Disease Management; D006467:Hemophilia A / Q000150:complications / Q000188:drug therapy*; D002836:Hemophilia B / Q000150:complications / Q000188:drug therapy*; D006470:Hemorrhage / Q000517:prevention & control*; D006801:Humans; D007592:Joint Diseases / Q000517:prevention & control*; D013585:Synovitis / Q000628:therapy
D019774:Blood Coagulation Factor Inhibitors; D001779:Blood Coagulation Factors