P1608: REAL-WORLD INSIGHTS ON THE MANAGEMENT OF IMMUNE-MEDIATED THROMBOTIC THROMBOCYTOPENIC PURPURA (ITTP) WITH CAPLACIZUMAB IN BELGIUM

• DOI: 10.1097/01.hs9.0000973308.93798.cb
• Publication Date: 2023-08-08T16:37:23Z
• Authors: Daan Dierickx; Thierry Connerotte; Julie Dallemagne; Ann De Becker; Ine Moors; Sylvia Snauwaert; Anne Sonet; Koen Theunissen; Dimitri Breems; Bernard De Prijck; Aurélie Jaspers; Catherine Lambert; Bert Heyrman; Joris Hautekiet; Sofie Lyna; Chantal Maertens; Vanessa Delrieu
• Keywords: ADAMTS13

Topic: 32. Platelet disorders Background: Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a rare, life-threatening disorder, caused by autoantibody-mediated deficiency of disintegrin and metalloproteinase with thrombospondin type 1 motif, member 13 (ADAMTS13). Real-world data from Germany, France, the United Kingdom confirm that caplacizumab allows rapid recovery iTTP. Caplacizumab has been commercialized reimbursed in Belgium since September 1, 2019. on treatment iTTP patients are lacking. Aims: To provide real-world use Belgium. Methods: This retrospective study analyzed hospital chart review for (≥18 years old) who were hospitalized treated Belgian hospitals (September 2019, to January 15, 2022). Patients included if they had initiated ended within period. Patient demographics, characteristics, efficacy safety outcomes, healthcare resource utilization presented. identify differences disease management, analyses performed 2 subgroups: first diagnosed episodes relapse episodes. Results: A total 39 (25 diagnoses, 14 episodes) identified 33 (18–89 (Table). Females developed 21 males 18 episodes, higher proportion females than episode subgroup (71.4% vs 28.6%). Mean (standard deviation [SD]) number doses per was 39.2 (14.7) 31.9 (8.4) quickly after admission (2.7 days) (0.4 days). generally same day as diagnosis confirmation ADAMTS13 activity-testing (level <10%); centers started before due delayed results. (SD) duration stay 16.0 (10.3) days 9.1 (2.9) Exacerbation observed 2/39 (5.1%) occurred 6/39 (15.4%) Treatment interrupted at time both exacerbations because there lack symptoms ongoing disease; patient did not have ADAMTS13-testing interruption level <10%. Of 6 relapse, 3 short (~7 between termination their reported relapse; stopped these platelet count normalization symptom resolution, however, No refractory or passed away. Bleeding events 3/25 (12.0%) 2/14 (14.3%) respectively. Summary/Conclusion: combination therapeutic plasma exchange immunosuppressive therapy. Although testing well established diagnosis, our results show additional should be used optimize guide discontinuation. Exacerbations relapses may avoided tailored activity. Our line other published data. Disease management international ISTH guidelines front-line therapy.Keywords: ADAMTS13, Platelet, Thrombotic (TTP)