BackgroundPosterior reversible encephalopathy syndrome (PRES) is an increasingly recognized serious complication of cyclosporine A (CSA) and tacrolimus (TAC) use in hematopoietic cell transplantation (HCT) recipients.ProcedureA retrospective study was carried out, including 84 cases of HCT for TM from January 2012 to January 2017. Eleven cases were diagnosed with PRES.ResultsThe cumulative incidence of PRES was 13.4% (95% confidence interval (CI) 9.7%‐17.2%). The median onset time of the symptoms was 63 [20, 143] days after transplantation. Lumber puncture found that CSF was normal. Univariate analysis showed that patients who received methylprednisolone (MP) (OR = 10.629 95% CI, 1.360‐83.071, P = 0.024), female patients (OR = 4.275, 95% CI, 1.154‐15.843, P = 0.032), patients who had severe hypertension (OR = 5.162, 95% CI, 1.042 to 25.559, P = 0.029) had significantly higher risks of PRES. Multivariate analysis showed that severe hypertension (hazard ratio [HR], 12.793; 95% CI, 1.477 to 110.813; P  = 0.021), and Pesaro class 3 (HR, 3.367; 95% CI, 1.210 to 9.368; P  =  0.020) were associated with PRES.ConclusionsThe severe hypertension is an independent risk factor for PRES post‐HCT in children with thalassemia major. Patients of Pesaro class 3 may benefit from optimum control of blood pressure post‐HCT for prophylaxis of PRES.

Pure red cell aplasia (PRCA) associated with cytomegalovirus (CMV) and Epstein-Barr virus (EBV) infection is uncommon. Here, we describe the clinical features and management of seven cases of Chinese children with PRCA associated with viral infections. The patients presented with pallor on admission. Blood cell counts and marrow smears showed anemia, reticulocytopenia, and aplasia of erythroblasts. Serological investigation and DNA polymerase chain reactions for CMV were positive in four patients and those tests for EBV were positive in other three patients. All patients received blood transfusion, corticosteroids treatment, and ganciclovir injection. Two patients had a complete response and one had a partial response after the treatments. The other three patients had a complete response to second-line therapies, including high-dose methylprednisolone, cyclosporin A, and intravenous immunoglobulin. Only one patient had no response to the therapies. Our results indicated that it might be important to combine immunosuppressive drugs with an antiviral drug in the management of PRCA associated with CMV and EBV infection.

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BackgroundAcute lymphoblastic leukemia (ALL) is an aggressive malignant disorder of lymphoid progenitor cells in both children and adults. Although improvements in contemporary therapy and development of new treatment strategies have led to dramatic increases in the cure rate in children with ALL, the relapse rate remains high and the prognosis of relapsed childhood ALL is poor. Molecularly targeted therapies have emerged as the leading treatments in cancer therapy. Multi-cytotoxic drug regimens have achieved success, yet many studies addressing targeted therapies have focused on only one single agent. In this study, we attempted to investigate whether the effect of the mammalian target of rapamycin (mTOR) inhibitor rapamycin is synergistic with the effect of focal adhesion kinase (FAK) down-regulation in the treatment of ALL.MethodsThe effect of rapamycin combined with FAK down-regulation on cell proliferation, the cell cycle, and apoptosis was investigated in the human precursor B acute lymphoblastic leukemia cells REH and on survival time and leukemia progression in a non-obese diabetic/severe combined immunodeficiency (NOD/SCID) mouse model.ResultsWhen combined with FAK down-regulation, rapamycin-induced suppression of cell proliferation, G0/G1 cell cycle arrest, and apoptosis were significantly enhanced. In addition, REH cell-injected NOD/SCID mice treated with rapamycin and a short-hairpin RNA (shRNA) to down-regulate FAK had significantly longer survival times and slower leukemia progression compared with mice injected with REH-empty vector cells and treated with rapamycin. Moreover, the B-cell CLL/lymphoma-2 (BCL-2) gene family was shown to be involved in the enhancement, by combined treatment, of REH cell apoptosis.ConclusionsFAK down-regulation enhanced the in vitro and in vivo inhibitory effects of rapamycin on REH cell growth, indicating that the simultaneous targeting of mTOR- and FAK-related pathways might offer a novel and powerful strategy for treating ALL.