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Background and objectivesLack of suitable donors and regimen related toxicity are major barriers for hematopoietic stem cell transplantation (HSCT) in patients with sickle cell disease (SCD). The aim of the study is the assessment of efficacy and toxicity of Treosulfan-based conditioning regimen for SCD also when alternative donors such as mismatched unrelated donor and haploidentical donor are employed.MethodsWe report our single-center experience: 11 patients with SCD received HSCT with a Treosulfan/Thiotepa/Fludarabine/Anti-thymoglobulin conditioning regimen between 2010 and 2015. The donor was a matched sibling donor (n= 7), a haploidentical parent (n= 2), a matched unrelated donor (n= 1) or a mismatched unrelated donor (n=1). The haploidentical and mismatched unrelated donor grafts were manipulated by removing TCRαβ and CD19 positive cells.ResultsAll patients survived the procedure and achieved stable engraftment. Stable mixed chimerism was observed in 5/11 patients. Grade III–IV regimen related toxicity was limited to mucositis and no grade III–IV graft-versus-host disease (GvHD) occurred. No SCD manifestation was observed post transplant and cerebral vasculopathy improved in 3/5 evaluable patients. Organ function evaluation showed no pulmonary, cardiac or renal toxicity but gonadal failure occurred in 1/4 evaluable patients.ConclusionOur data suggest that Treosulfan is associated with low toxicity and may be employed also for unrelated and haploidentical donor HSCT.

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Background: Immune thrombocytopenia (ITP) is an acquired immune-mediated disorder characterized by isolated thrombocytopenia. Pediatric ITP patients are prone to develop autoantibodies such as antithyroglobulin (TG) and antithyroperoxidase (TPO), even in the absence of clinical signs of autoimmune disease. The aim of this multicenter retrospective study was to evaluate (1) the prevalence of positivity of antithyroid antibodies (TPO and TG) in a large cohort of pediatric patients with chronic ITP; (2) the role of autoimmune thyroiditis as a prognostic factor for chronicity of ITP.Procedure: For this retrospective study, we collected data from patients diagnosed as affected by chronic ITP between 2011 and 2014 in six centers belonging to the Italian Association of Pediatric Haematology and Oncology (AIEOP). Results:From the analysis of data, we found a significantly higher prevalence of antithyroid antibodies in children with chronic ITP (11.6%) than in the pediatric population (1.2%-1.3%).No correlation has been found between the platelet count and the prevalence of positive antithyroid antibodies at any detection time of the study. Conclusions:The results of our study demonstrated that (1) the prevalence of positivity for antithyroid antibodies (anti-TPO and anti-TG) in pediatric patients with chronic ITP results is significantly higher than in the pediatric population; (2) autoimmune thyroiditis does not seem to play a role as a prognostic factor for chronicity of ITP in pediatric patients. K E Y W O R D Santithyroglobulin (TG), antithyroperoxidase (TPO), autoimmune diseases, autoimmune thyroidi- tis, immune thrombocytopeniaRecently, an International Working Group (IWG) of experts established a platelet count less than 100 000/mm 3 as the threshold for diagnosis. 3 According to the IWG, ITP has been classified as newly diagnosed (within 3 months from diagnosis), persistent (between 3 and 12 months) and chronic (lasting for more than 12 months). 3 Female gender, older age at presentation, absence of previous infection or vaccination, insidious onset, higher platelet count at presentation, positivity for antinuclear antibodies (ANA), and unresponsiveness to a single dose of intravenous human immunoglobulins are considered the predictors of the chronic course of the disease. 4 The platelet count is not completely related with the bleeding risk and the clinical