Beneficial effect of prophylactic emicizumab on Thai hemophilia A with and without inhibitor: a case series report
Prophylactic emicizumab for Thai hemophilia A
Keywords:
Hemophilia, Hemophilia with inhibitor, Emicizuab, Annual bleeding rate, ProphylaxisAbstract
Abstract:
Background: Nonfactor replacement of emicizumab has shown an effectiveness in preventing bleeding episodes
among patients with hemophilia with and without inhibitor.
Objective: A retrospective evaluation of patients
with severe hemophilia A with and without inhibitor receiving prophylactic emicizumab was conducted.
Subjects and methods: Five patients with severe hemophilia A with and without inhibitor aged > 12 years experiencing at least 5 bleeding episodes during the previous 6-month period, were enrolled. The standard loading dose of prophylactic emicizumab at 3 mg/kg weekly was given for 4 weeks, followed by the maintenance dose at 1.5 mg/kg weekly (n = 1) or 6 mg/kg every 4 weeks (n = 4). The occurrence of bleeding episode was monitored.
Results: Five enrolled patients whose ages ranged from 13 to 28 years, were enrolled. Two patients had high inhibitor while the remaining three patients had no inhibitor. During a 3-year treatment, the zero annual bleeding rate was 12 of 15 (80%) accumulative patient-treatment years among 5 studied patients and additional 1 episode of annual bleeding rate was 3 of 15 (20%), which were markedly decreased compared with those of the pretreatment
period. A total of 3 bleeding episodes included one each occurring at the iliopsoas muscle, lower gastro-intestine
tract and knee hemarthrosis with multiple abrasion from motorcycle accident. All were responsive to the
additional recombinant factor VIIa and factor VIII concentrate for patients with and without inhibitor accordingly. The musculoskeletal outcome was markedly improved in terms of pain, joint motion, muscle strength, functional status and physical fitness. Hemophilia Joint Health Score (HJHS) was also improved among 2 patients evaluated by this score.
Conclusion: The standard dose of emicizumab has shown effectiveness in decreasing the annual bleeding rates among Thai patients with hemophilia A with and without inhibitor.
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