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A case-control study was conducted among Italian children treated with a stem cell transplant (SCT). Cases (n = 43) were allogeneic recipients with osteonecrosis, and the controls (n = 129) were matched to the corresponding cases on the basis of survival, SCT center and date of transplant. Univariate analysis showed that older age at SCT (OR 1.39; 95% CI 1.24-1.57), total body irradiation (TBI) (OR 5.73; 95% CI 2.38-13.83), chronic graft-versus-host disease (GvHD) (OR 4.31; 95% CI 2.05-9.07), and duration of steroid treatment after SCT (OR 1.118; 95% CI 1.034-1.209) were statistically correlated with osteonecrosis. However, multivariate analysis revealed that the only factors that were significantly associated with osteonecrosis were older age at SCT (p = 0.0001), TBI (p = 0.001) and chronicGvHD (p = 0.001).

Background: In Fanconi anemia bone marrow failure is the major cause of morbidity and mortality and hematopoietic stem cell transplantation represents the only curative treatment. Liver disease, in terms of elevated liver function tests, as well as benign and malignant liver tumors, occurs especially in case of androgen treatment. We report a unique case of a child with Fanconi anemia with FANCD2 mutation who developed neonatal cryptogenic liver cirrhosis and bone marrow failure. The child successfully underwent sequential liver transplantation and hematopoietic stem cell transplantation in the first 2 years of life. Nineteen months after hematopoietic stem cell transplantation and 30 months after liver transplantation, the patient is clinically well with normal hematopoietic function and excellent liver function. Conclusion: This is the first FA patient who successfully received sequential LT and HSCT highlighting that successful sequential transplantation is feasible in Fanconi anemia patients. [ABSTRACT FROM AUTHOR]

Despite advances in graft-versus-host-disease (GVHD) treatment, it is estimated that overall survival (OS) at 2 years for hematopoietic cell transplantation (HCT) recipients who experience steroid-resistant GVHD is 10%. Among recent therapeutic approaches for GVHD treatment, mesenchymal stromal cells (MSCs) hold a key position. We describe a multicenter experience of 11 pediatric patients diagnosed with acute or chronic GVHD (aGVHD, cGVHD) treated for compassionate use with GMP-grade unrelated HLA-disparate donors'' bone marrow-derived MSCs, expanded in platelet-lysate (PL)-containing medium. Eleven patients (aged 4-15 years) received intravenous (i.v.) MSCs for aGVHD or cGVHD, which was resistant to multiple lines of immunosuppression. The median dose was 1.2 × 106/kg (range: 0.7-3.7 × 106/kg). No acute side effects were observed, and no late side effects were reported at a median follow-up of 8 months (range: 4-18 months). Overall response was obtained in 71.4% of patients, with complete response in 23.8% of cases. None of our patients presented GVHD progression upon MSC administration, but 4 patients presented GVHD recurrence 2 to 5 months after infusion. Two patients developed chronic limited GVHD. This study underlines the safety of PL-expanded MSC use in children. MSC efficacy seems to be greater in aGVHD than in cGVHD, even after failure of multiple lines of immunosuppression. [Copyright &y& Elsevier]