Abstract Number: PB0975
Meeting: ISTH 2020 Congress
Background: Inhibitors development in haemophilia patients is a severe complication. The Immune tolerance (ITI) is the only way to eradicate inhibitors. In February 2019, a novel recombinant, humanized, bispecific antibody (emicizumab) was licensed in Argentina for the prevention of bleeding in hemophilia A patients with inhibitors, but wasn´t available until June/19.
Aims: Gain legal access to this drugs (it was not available in Argentina at that moment)
Methods: Clinical data from a 8 years old boy with severe haemophilia A, with inhibitor that failed two ITI.
Results: He was diagnosed at 8 month and after 18 ED he developed a high title factor VIII inhibitor. He underwent two ITI : first at 21 month old, required a central catheter (Port-a-cath). .After 14 months, the portal was removed due to infection. He required a second central catheter and after 43 months he stopped the ITI by failure of eradication of inhibitor (44UB). Second ITI was made at 6 years old and after 13 months, the ITI was stopped (12UB). He started prophylaxis with rFVIIa 90-120 ug/Kg , 3 times per week.
During bypass prophylaxis, the child had a mean of 2 episodes of bleeding by month with prolonged hospitalization. He had synovial hiperthrophy in both ankles. He missed several days of school, and had a lot of pain episodes, requiring treatment with morphine.
After judicial action, in March 2019 he started receiving emicizumab. After 11 months of treatment his health improved, he stopped having bleeding episodes and hospital admissions, resumed school and currently has the normal life of an 8 years´child .One tooth extraction was required, no factor supplementation and had not bleeding episodes. Also shows improvement in both ankles by ultrasonography.
Conclusions: It´s the first successful case of treatment with emicizumab in Argentina, after a hard push the authorities.
To cite this abstract in AMA style:Sliba G, Martinez M, Nally P, Morales F, Dolabella G, Nally F. Impact of a New Drug in the Real Child´s Life [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/impact-of-a-new-drug-in-the-real-childs-life/. Accessed September 29, 2023.
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