Jacek Toporski

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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)
OBJECTIVE Hematopoietic stem cell transplantation (HSCT) is curative in several life-threatening pediatric diseases but may affect children and their families inducing depression, anxiety, burnout symptoms, and post-traumatic stress symptoms, as well as post-traumatic growth (PTG). The aim of this study was to investigate the co-occurrence of different(More)
BACKGROUND Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative treatment available for severe combined immunodeficiency (SCID); although, there is a high incidence of severe infections and an increased risk of graft-versus host-disease (GvHD) with HSCT. Early intervention is a crucial prognostic factor and a HLA-haploidentical(More)
AIM Oral mucositis is a common and debilitating side effect of haematopoietic stem cell transplantation. Our study investigated parents' and children's experiences of oral mucositis treatment and whether the parents' perceptions accurately reflected the children's views. METHODS We analysed 71 questionnaires completed by the parents of children who had(More)
BACKGROUND Mucopolysaccharidosis VI (MPS VI) is an autosomal recessive progressive multiorgan disorder due to mutation in the gene encoding the enzyme Arylsulfatase B (ARSB). Dysfunctional ARSB causes lysosomal accumulation of glycosaminoglycans (GAG). Currently, enzyme replacement therapy (ERT) is preferred to hematopoietic stem cell transplantation (SCT)(More)
BACKGROUND Childhood pre-B acute lymphoblastic leukemia (ALL) is a bone marrow (BM) derived disease, which often disseminates out of the BM cavity, where malignant cells to a variable degree can be found circulating in the peripheral blood (PB). Normal pre-B cells are absolutely dependent on BM stroma for survival and differentiation. It is not known(More)
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