Abstract Number: PB0785
Meeting: ISTH 2020 Congress
Background: AHA is a rare hemorrhagic disease caused by autoantibodies against the FVIII. The response to immunosuppressive therapy (IST) is high (60-80%) but the time to obtain it can be long, exposing the patient at risk of fatal bleeding.
Aims: We describe an AHA cohort in which we evaluated the impact of some parameters on response, time to response (TTR) and overall survival (OS).
Methods: Data were collected from the clinical charts. Response criteria: complete response (CR) FVIII > 50%, undetectable inhibitor, absence of bleeding; partial response (PR) FVIII > 50%, persistent inhibitor; no response (NR) FVIII < 50%, persistent inhibitor.
Results: 61 patients were diagnosed over a period of 41 years (median follow-up 26.6 months). Tables 1-2 show the baseline characteristics of the patients and bleedings. 50/61 patients (82%) underwent hemostatic therapy: rFVIIa (52%); aPCC (38%) with no significant differences in the response rate (RR) (rFVIIa 92.3% vs aPCC 100%). No thromboembolic events occurred. 60/61 patients underwent first-line IST: corticosteroids (76.6%); corticosteroids-cyclophosphamide (13.4%). The RR after 4 weeks was 53%: CR 38%, PR 15%, NR 28%. At the last follow-up: CR 61%, PR 5%, NR 29%. The 1-year OS was 85%. The bleeding mortality rate was 3%. We evaluated: the impact of FVIII < 1% and inhibitor > 5 BU, at diagnosis, on TTR and that of Hb level, FVIII:C, inhibitor titer, age, sex, related disorders, on the response and OS. None of these was statistically significant on TTR and response. Age < 70 years (p=0.011), achievement of response (p = 0.002) and post-partum (p= 0.001) improved the OS.
Conclusions: Our cohort is the largest as single center with a long follow up, a highest 1-year OS and a lowest mortality described. We highlight obtaining a response improves the OS. It would be useful to identify response predictors to drive therapy and shorten TTR.
|Bleeding as a trigger for diagnosis n. (%)||61 (100)|
|Time from bleeding event to diagnosis|
|Median (days-range)||31 (0-264)|
|Cause of bleeding episodes n (%)|
|Site of bleedings n (%)|
|Mucosal and skin||3 (5)|
[Bleeding at diagnosis]
|Patients n (%)||61 (100)|
|Median age at diagnosis (range)||71 ( 18-89)|
|Male: female n (%)||26/35 (42.6%/57.4%)|
|Median FVIII activity (U dL- range)||1.5 ( < 0.25- 42.5)|
|Median Inhibitor titer ( BU- range)||10.95 (1.06-1188)|
|Median hb at diagnosis (g/dL-range)||11 (4.9- 15.2)|
[Demographics characteristics and related disorders at diagnosis]
To cite this abstract in AMA style:Porrazzo M, Ferretti A, Baldacci E, Aprile S, Barone F, Abbruzzese R, Chistolini A, Mazzucconi MG, Santoro C. Real Life Experienceon 61 Acquired Haemophilia A (AHA) Patients from a Single Center [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/real-life-experienceon-61-acquired-haemophilia-a-aha-patients-from-a-single-center/. Accessed January 28, 2022.
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