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Acquired hemophilia A (AHA) is a severe auto‐immune bleeding disorder. Treatment of AHA is burdensome and optimal management is still unresolved. Therefore a retrospective nationwide multi‐center cohort study (1992‐2018) was performed to evaluate clinical presentation and treatment efficacy and safety of AHA in the Netherlands. Multivariate logistic and Cox regression analysis was used to study independent associations between patient characteristics and clinical outcomes. A total of 143 patients (median age 73 years; 52.4% male) were included with a median follow‐up of 16.8 months (IQR 3.6‐41.5 months). First‐line immunosuppressive treatment was mostly steroid monotherapy (67.6%), steroids/cyclophosphamide (11.9%) and steroids/rituximab (11.9%), with success rates of 35.2%, 80.0% and 66.7% respectively, P < .05. Eventually 75% of patients achieved complete remission (CR). A high anti‐FVIII antibody titer, severe bleeding and steroid monotherapy were associated with lower CR rates. Infections, the most important adverse event, occurred significantly more often with steroid combination therapy compared to steroids alone (38.7% vs 10.6%; P = .001). Overall mortality was 38.2%, mostly due to infections (19.2%) compared to 7.7% fatal bleeds. Advanced age, underlying malignancy and ICU admission were predictors for mortality. This study showed that AHA is characterized by significant disease‐related and treatment‐related morbidity and mortality. A high anti‐FVIII titer, severe bleeding and steroid monotherapy were associated with a lower CR rate. The efficacy of steroid combination therapies however, was overshadowed by higher infection rates and infections represented the most important cause of death. The challenging and delicate balance between treatment effectivity and safety requires ongoing monitoring of AHA and further identification of prognostic markers.

ARTICLES 1525 Qualità of Life IntroductionImmune thrombocytopenia (ITP) is an autoimmune disorder characterized by isolated thrombocytopenia (peripheral blood platelet count <100x10 9 /L) in the absence of other disorders associated with thrombocytopenia.1 Only 20-25% of children with ITP will develop chronic disease, currently defined as thrombocytopenia <100x10 9 /L lasting for more than 12 months. 1 In the case of no or mild bleeding the management consists of careful observation and restriction of activities that carry a risk of severe bleeding, regardless of platelet count. 2Severe bleeding, occurring in only 3-6% of children, 3 requires treatment with corticosteroids, intravenous immunoglobulin or anti-D immunoglobulin, either alone or in combination.Despite the transient and often benign course of the disease, many clinicians observe that ITP has a significant impact on health-related quality of life (HRQoL). 4 Recently published management guidelines state that HRQoL issues should be taken into account while making decisions on management in childhood ITP.2,5 However, these statements are based on clinical experience rather than results of research since HRQoL studies in childhood ITP are scarce. Recently, several clinical studies addressing HRQoL in children with ITP have been performed, 6-11 but large prospective studies with longitudinal generic as well as disease-specific HRQoL measurements are lacking.For this reason we decided to study HRQoL as part of a prospective study in children with newly diagnosed ITP: the TIKI study (Therapy with or without Intravenous Immunoglobulin for Kids with acute ITP), a multicenter randomized clinical trial to determine whether early administration of intravenous immunoglobulin can prevent a chronic course of the disease. In this study, children receive either a single dose of intravenous immunoglobulin or careful observation and treatment only in the case of severe bleeding. The final results of the primary outcome of this study are awaited and have not been published yet. The aim of the current HRQoL study was to relate generic as well as disease-specific HRQoL scores of children with newly diagnosed ITP to type of treatment, bleeding severity and clinical course of the disease, to analyze changes in HRQoL scores over time and to compare generic HRQoL scores of children with newly diagnosed ITP with already published data from a reference group of Dutch school children. 12 Methods PatientsChildren aged 3 months to 16 years with newly diagnosed ITP, a platelet count below 20x10 9 /L and with mild to moderate bleeding were eligible for inclusion in the TIKI study. Despite its generally transient and benign course, childhood immune thrombocytopenia has a large impact on health-related quality of life. Recently published guidelines state that quality of life should be taken into account while making decisions on management in childhood immune thrombocytopenia. We, therefore, assessed healthrelated quality of life in children with newly diagnosed immune thrombocytopeni...