How I manage patients with inherited haemophilia A and B and factor inhibitors

Abstract

Development of inhibitors to coagulation factor VIII or IX is still the most challenging complication in haemophilia care. 'Bypassing agents' may be used to treat a bleed but the eradication of the inhibitor by immune tolerance induction (ITI) is the main objective in the treatment of a patient with haemophilia who has developed neutralizing antibodies. Several options exist for ITI and the patient may be at 'good' or 'bad risk' for successful outcome with different regimens. This paper offers a review of current regimens to be considered in the treatment of a bleed in a patient with an inhibitor but the main focus is the aspects of different choices in the management of the child or the adult with severe or mild forms of haemophilia A or B, who has developed an inhibitor. There are also some final outlooks on new and emerging treatment possibilities.

Outpatient minor oral surgery in patients with hemophilia: A case series of 23 patients

“…In our series, there were no cases of postoperative infection. The results obtained in our series were similar to those reported by other authors (4-13). At present, the coagulation factor concentrates undergo viral inactivation and consequently the most severe complication related to the hemophilia treatment is developing anti-FVIII and FIX inhibitors (2).…”

“…At present, the coagulation factor concentrates undergo viral inactivation and consequently the most severe complication related to the hemophilia treatment is developing anti-FVIII and FIX inhibitors (2). The incidence of inhibitor development in patients with severe hemophilia A is of approximately 30% and of 2-4% in patients with severe hemophilia B (13). Inhibitors were not detected in any of the patients included in our series.…”

Emicizumab: Review of the literature and critical appraisal

“…In addition to lack of complete efficacy, the burden of chronic, lifelong intravenous injections and the economic burden of the disease and its treatment make haemophilia an attractive target for innovative and potentially disruptive treatment strategies. Moreover, approximately one third of patients with severe haemophilia A will develop an alloimmune response against the infused clotting factor which then inhibits the function of the administered factor concentrate increasing the likelihood of uncontrollable bleeding and increasing the morbidity of the disease …”

“…Moreover, approximately one third of patients with severe haemophilia A will develop an alloimmune response against the infused clotting factor which then inhibits the function of the administered factor concentrate increasing the likelihood of uncontrollable bleeding and increasing the morbidity of the disease. 20 Innovative strategies to meet these unmet needs have emerged and include pursuit of a genetic treatment that might be administered once in a lifetime that results in long-term, persistent FVIII expression to mitigate the need for regular infusions of clotting factor concentrates by achieving a level high enough to eliminate the risk of bleeding in patients with an active lifestyle. 21,22 Other approaches include non-factor treatments which are administered subcutaneously at infrequent intervals.…”

Gastric Cancer, Hemophilia A and Angiodysplasia - An Unreported Association with Clinical Implications in a Patient with Digestive Hemorrhage

“…Treatment of patients with hemophilia and factor inhibitors aims to eradicate the inhibitor by immune tolerance induction (29). For bleeding episodes, factor eight inhibitor bypassing activity (FEIBA), an activated prothrombin complex concentrate and activated recombinant factor VII are the only available drugs (21).…”