Pediatr Transplant
November 2021
Introduction: The only curative treatment for severe aplastic anemia in children is an allogeneic stem cell transplant; however, few patients have a matched related or unrelated donor. Haploidentical stem cell transplantation (haplo-SCT) using bone marrow (BM) and peripheral blood stem cells (PBSC) has been recently described as effective and safe. In this study, we retrospectively report the outcome of twelve pediatric patients who underwent haplo-SCT using only PBSC.
View Article and Find Full Text PDFIntroduction: Thrombotic thrombocytopenic purpura (TTP) is characterized by a reduction in the von Willebrand cleavage protein ADAMTS-13, mainly as a consequence of autoimmunity. Plasma exchange (PEx) is standard, achieving complete remission (CR) in 77-83% of cases, but rates are variable depending on ADAMTS-13 activity and relapse is frequent in patients with <10%. Thus, an effective front-line immunosuppressive treatment is needed.
View Article and Find Full Text PDFObjective And Importance: Thrombotic thrombocytopenic purpura (TTP) is a life-threatening disorder that needs prompt diagnosis and treatment. Front-line therapy consists of plasma exchange (PEx) and steroids, but, in some instances, this is not enough to achieve a complete and sustained response.
Clinical Presentation: We report four cases of TTP treated with low-dose rituximab, PEx, and a short course of steroids with an excellent outcome.