Background Childhood acute lymphoblastic leukemia (cALL) involves the aberrant expression of lncRNAs. This study aimed to clarify the mechanisms of Lnc-TIM3 in cALL. Procedure The expression of Lnc-TIM3 in cALL bone marrow, cell lines were analyzed by quantitative real-time PCR (qRT-PCR). Cell migration, invasion were evaluated by transwell. CCK8 assays, soft agar colony formation, flowcytometry was used to measure the proliferation ability. The xenografts of zebrafish were analyzed to explore the roles of Lnc-TIM3 in vivo. The ceRNA regulatory mechanism of Lnc-TIM3 was evaluated by dual luciferase reporter assay. The protein levels of TIM3 were measured by western blot assay. Results Lnc-TIM3 was up-regulated in both cALL born marrow and cell lines. Lnc-TIM3 knockdown down-regulated TIM3 expression in cALL cells and remarkably restrained cALL progression both in vitro and in vivo. The phenotypic characteristics induced by Lnc-TIM3 knockdown were significantly reversed by miR-5094 inhibitor, and the downstream pathway was the TIM3/GAL9 autocrine stimulatory loop. Conclusion Lnc-TIM3 can sponge miR-5094 to up-regulate the expression of TIM3, thus promoting the development of cALL.

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare hematologic neoplasm derived from plasmacytoid dendritic cell precursors. The malignancy was characterized by cutaneous and bone marrow involvement and leukemic spread，predominantly involving elderly patients. Pediatric cases of BPDCN are much fewer reported in the literature, making the management of pediatric BPDCN challenging. We report a congenital BPDCN patient who manifested with neutropenia and nodular skin lesions. Whole-exome sequencing suggests the presence of kmt2c gene duplication. She died four months after diagnosis. This case report reminds clinicians, especially neonatologists, to consider the possibility of BPCDN when finding neonates present with rash(such as purplish nodules, bruiselike macules) and a cytopenia. In addition, this study suggests that the KMTC2 gene may play a vital role in the pathogenesis of BPDCN.

Background: Hemophagocytic lymphohistiocytosis (HLH) frequently is a fatal disorder of immune system, mainly manifesting as uninterrupted fever, hepatosplenomegaly, pancytopenia, hemophagocytosis, central nervous system (CNS) involvement, and elevation of HLH related biomarkers. Hematopoietic stem cell transplantation (HSCT) is the sole curative therapy for this disease. Methods: Retrospective study of outcomes in patients who underwent HSCT in a single center. Results: Eight patients (four males), with a median age at diagnosis was 3.3 years (range, 1.2 to 13.4) and at transplantation was 2.9 years (range, 0.8 to 13.0), received standard HLH chemotherapy before HSCT. Two patients were in active disease (AD). All received busulfan-based myeloablative conditioning (MAC) regimen. Four (50%) patients received peripheral blood stem cells (PBSC), four (50%) received umbilical cord blood (UCB). Two (25%) patients who received UCB first underwent second transplantation. Three (37.5%) patients had acute Graft-versus-host disease (GVHD), and one (12.5%) had chronic GVHD. Only one (12.5%) patient died due to multiple organ dysfunction syndrome (MODS). Conclusion: Active disease and mixed chimerism were the fatal prognostic factors. Graft failure was a part of the significant disadvantages of UCB. In combination with standard HLH therapy, HSCT could be an effective means of prolonging survival and curing pediatric HLH. To some extent, UCB is a good option in the absence of PBSC.