Drugs
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Emicizumab (Hemlibra®), a recombinant, humanized, bispecific monoclonal antibody, restores the function of missing activated factor VIII (FVIII) by bridging FIXa and FX to facilitate effective haemostasis in patients with haemophilia A. Subcutaneous emicizumab is approved in several countries, including in the USA and Japan, for the routine prophylaxis of bleeding episodes in patients with haemophilia A with or without FVIII inhibitors. ⋯ Emicizumab was also associated with beneficial effects on health-related quality of life and health status, and was generally well tolerated. In view of its convenient route of administration and versatile dosage regimens (maintenance dose of once every 1, 2 or 4 weeks), emicizumab provides an effective and generally well-tolerated alternative to conventional FVIII replacement products for the prophylaxis of bleeding episodes in patients with haemophilia A, regardless of the presence or absence of inhibitors.
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Lusutrombopag (Mulpleta®), an orally bioavailable, small molecule thrombopoietin receptor agonist, is approved for the treatment of thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo a procedure. In placebo-controlled phase 3 clinical trials, lusutrombopag significantly increased the proportion of patients who did not require a platelet transfusion prior to the procedure or rescue therapy for bleeding up to 7 days after the scheduled procedure. ⋯ Lusutrombopag is well tolerated, with headache being the most common adverse reaction in lusutrombopag recipients in clinical trials. Thus, lusutrombopag represents a promising emerging therapeutic option for the treatment of thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo a procedure.