Emicizumab prophylaxis among infants and toddlers with severe hemophilia A and inhibitors—a single-center cohort
Barg A. A., Avishai E., Budnik I. A., Levy-Mendelovich S., Barazani T. B., Kenet G., Livnat T.
Pediatric Blood and Cancer
Vol.66, Issue11, Num.e27886
Опубликовано: 2019
Тип ресурса: Статья
Аннотация:
Background: Emicizumab is a bispecific antibody that bridges factor IXa and factor X to restore hemostasis in patients with hemophilia A (HA). Its efficacy and safety have been proven in multicenter trials. However, real world data regarding its use in very young children are currently lacking. Ancillary test results for monitoring emicizumab's hemostatic effect and their clinical correlations are scarce. Methods: Children with HA and inhibitors treated by emicizumab were prospectively followed at our center. Laboratory follow-up included rotational thromboelastometry (ROTEM) and thrombin generation (TG), prior to and during treatment. Results: Eleven children whose median age was 26 months were treated by emicizumab and followed for a median of 36 weeks. During follow-up, none experienced hemarthrosis or any other spontaneous bleeds. For 7/11 patients, emicizumab prophylaxis was sufficient to maintain hemostasis without additional supplemental therapy. Only 4/11 patients were occasion
Ключевые слова:
emicizumab; hemophilia A; inhibitors; ROTEM; thrombin generation
emicizumab; recombinant blood clotting factor 7a; activated partial thromboplastin time; Article; blood sampling; blood transfusion; child; clinical article; clinical outcome; cohort analysis; drug efficacy; drug safety; female; follow up; hemarthrosis; hemophilia A; hemostasis; human; infant; iron deficiency anemia; laboratory test; loading drug dose; maintenance drug dose; male; minor surgery; pediatric patient; preschool child; priority journal; prophylaxis; prospective study; thrombin time; thromboelastography; treatment duration
Язык текста: Английский
ISSN: 1545-5017
Barg A. A.
Avishai E.
Budnik I. A. Ivan Aleksandrovich 1983-
Levy-Mendelovich S.
Barazani T. B.
Kenet G.
Livnat T.
Барг А. А.
Авишаи Е.
Будник И. А. Иван Александрович 1983-
Левy-Менделович С.
Баразани Т. Б.
Кенет Г.
Ливнат Т.
Emicizumab prophylaxis among infants and toddlers with severe hemophilia A and inhibitors—a single-center cohort
Текст визуальный непосредственный
Pediatric Blood and Cancer
John Wiley & Sons
Vol.66, Issue11 Num.e27886
2019
Статья
emicizumab hemophilia A inhibitors ROTEM thrombin generation
emicizumab recombinant blood clotting factor 7a activated partial thromboplastin time Article blood sampling blood transfusion child clinical article clinical outcome cohort analysis drug efficacy drug safety female follow up hemarthrosis hemophilia A hemostasis human infant iron deficiency anemia laboratory test loading drug dose maintenance drug dose male minor surgery pediatric patient preschool child priority journal prophylaxis prospective study thrombin time thromboelastography treatment duration
Background: Emicizumab is a bispecific antibody that bridges factor IXa and factor X to restore hemostasis in patients with hemophilia A (HA). Its efficacy and safety have been proven in multicenter trials. However, real world data regarding its use in very young children are currently lacking. Ancillary test results for monitoring emicizumab's hemostatic effect and their clinical correlations are scarce. Methods: Children with HA and inhibitors treated by emicizumab were prospectively followed at our center. Laboratory follow-up included rotational thromboelastometry (ROTEM) and thrombin generation (TG), prior to and during treatment. Results: Eleven children whose median age was 26 months were treated by emicizumab and followed for a median of 36 weeks. During follow-up, none experienced hemarthrosis or any other spontaneous bleeds. For 7/11 patients, emicizumab prophylaxis was sufficient to maintain hemostasis without additional supplemental therapy. Only 4/11 patients were occasion