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Adenovirus (AdV) infection in the course of allogeneic stem cell transplantation (SCT) is associated with high transplant-related morbidity and mortality. Disseminated AdV disease is lethal in most instances. Early detection of AdV infection and identification of patients carrying a high risk of disseminated disease therefore remain a major challenge. In(More)
BACKGROUND Hepatic veno-occlusive disease is a leading cause of morbidity and mortality after haemopoietic stem-cell transplantation (HSCT). We aimed to assess whether defibrotide can reduce the incidence of veno-occlusive disease in this setting. METHODS In our phase 3 open-label, randomised controlled trial, we enrolled patients at 28 European(More)
Mutations in DOCK8 result in autosomal recessive Hyper-IgE syndrome with combined immunodeficiency (CID). However, the natural course of disease, long-term prognosis, and optimal therapeutic management have not yet been clearly defined. In an international retrospective survey of patients with DOCK8 mutations, focused on clinical presentation and(More)
Mutations of FoxP3 result in the disturbance of FoxP3 expression and lack of functional CD4 ϩ CD25 high regulatory T cells in humans, causing immune dysregulation, polyendocrinopathy, enteropathy, X-linked (IPEX) syndrome. The only curative approach for IPEX syndrome, which is fatal within the first year of life in many cases, is allogeneic hematopoietic(More)
Viral infections after allogeneic hematopoietic stem cell transplantation (HSCT) are important complications associated with high morbidity and mortality. In this setting, reactivations of persisting latent viral pathogens from donor and/or recipient cells play a central role whereas the sterile environment of transplant units renders new infections less(More)
Thalassemia major and sickle cell disease are the two most widely disseminated hereditary hemoglobinopathies in the world. The outlook for affected individuals has improved in recent years due to advances in medical management in the prevention and treatment of complications. However, hematopoietic stem cell transplantation is still the only available(More)
Diamond-Blackfan anemia (DBA) is a rare, inherited, pure red cell aplasia, associated with congenital anomalies 1 and predisposition to cancer. 2 Despite remarkable advances in our understanding of the underlying patho-genic mechanisms, 3 first-line treatment still relies on repeated red blood cell (RBC) transfusions and/or corti-costeroid therapy. 4 While(More)
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