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To analyze the clinical features and prognosis of 28 children with myelodysplastic syndrome (MDS) and to screen the high risk factors affecting the prognosis so as to provide the new ideas for standard of clinical diagnosis and therapy.The clinical data of 28 children with newly diagnosed MDS treated in our hospital from March 1994 to July 2016 were analyzed retrospectively, the features of disease onset and the results of laboratory examination were summarized, all MDS children were followed up, the prognosis and the high risk factors affecting the prognosis were evaluated.In all 28 MDS children, the ratio of male to female was 1.8∶1, the incidence of MDS was observed in boys, while the low incidence of MDS was found in older children. The clinical manifestations were mainly the decrease of three series blood cells in 16 cases (57.14%), other cases presented simple anemia (7.1%), simple thrombocytopenia (7.1%), neutropenia with anemia (14.29%), and anemia with thrombocytopenia (14.28%).The bone marrow image showed mainly hyperplasia (82.14%), and the pathological hematopoiesis, moreover the manifistation of pathological hematopoiesis was different in forma and degree; the bone marrow biopsy showed the typical abnormal localization of immature precursor(ALIP) accepted for 33.33%; the chromosome karyotype detection showed the detected rate of chronosome abnormality was 41.18%. The median follow-up time was 1.75 years. 5 children with MDS received the hematopoietic stem cell transplantation (HSCT), among them 1 dead and 4 maintained CCR; Out of other 23 patients no-received HSCT, 7 cases given up treatment after confirmed diagnosis, 16 cases received the chemotherapy (2 cases given up treatment after CR, 5 cases transformed into AML, 3 cases relapsed, 3 cases maintained CCR), 11 cases dead, 9 cases failed to be followed up. The 5-years OS rate and EFS rate in all patients were predicted as (38.2±11.3)% and (35.3±11.3)%,respectively, among them, the OS and EFS rates of patients received the HSCT allo superior to those of patients did not received HSCT ［(80.0±17.9）% vs.(22.8±11.5）%］ (P0.05) and ［(80.0±17.9)% vs (17.5±11.1)%］(P0.05). Analysis showed that in addition to receiving the HSCT(P0.05), platelet decrease in peripheral blood(P0.01), the age, sex, existance of micromegakaryocytes in bone marrow and progressive MDS or no influenced not on the prognosis(P0.05).The children MDS is rare and easy to be misdiagnosis, moreover displays more high heterogeneity and poor prognosis, thereby the early diagnosis is crucial, in addition, the system of prognosis evaluation is imperative to be perfected. The HSCT may be the effective method for curative treatment of childhood MDS.

We aim to investigate the efficacy and safety of the treatment with fully matched allogeneic hematopoietic stem cell transplants for children with severe aplastic anemia (SAA) in the first prospective trial in China. Six SAA children received allogeneic hematopoietic stem cell transplantation combined with chemotherapy. Five patients had successful engraftment while the sixth child regained normal peripheral blood counts consistent with spontaneous autologous hematopoiesis. Mean duration of follow-up was 2.75 years, and survival was 83%(5/6). The results indicated that allogeneic hematopoietic stem cell transplantation is a good option for the treatment of children with severe aplastic anemia.

Objective Cotransplantion of Mesenchymal stem cells(MSCs)with UCB-HSC promote the engraftment, accelerate the recovery of hematopoiesis and alleviate the severity of graft versus host disease(GVHD). Cases and methods 18 cases were treated with UD-UCBT (15 cases) and RD-UCBT (3 cases) .of them, acute lymphoblastic leukemia (high risk) 2 cases, acute myeloblastic leukemia 4 cases, chronic myeloblastic leukemia 3 cases, β-thalassemia major 8 cases and, mucopolysaccharidosis 1 case. HLA matched (6/6) in 3 cases, one locus (5/6) were dismatched in 12 cases and two HLA loci (4/6) in 3 cases. UCB was transfused into recipients according to the regular method. The MSCs were from auto-BM in 3 cases, from allogenic bone marrow of parents 4 cases, from allogenic related bone marrow 10 cases, and cord blood MSCs 1 case. The MSCs were transfused by IV (14 cases) or IBM (4 cases) injection. The conditioning regimens modified on the basis of Bu/CY + Fluda + ATG. GVHD was prevented according to the regular method in our department. Infused NCs dose of UCB was 6.77×107/kg, CD34+ÆÞsG5.58×105/kg; allo-MSCs 4.03×106/kg, auto-MSCs 8.83×106/kg. MSCs origin were detected. Results Ten cases were full donor engrafted, the time to get ANC ≥0.5×109/L and PLT ≥2.0×1010/L were on the 13.9th day and the 30.8th day. CFU-MIX, MSCs of recipients in Co-transplantation groups recovered gradually from 14th day to 28th day. MSCs origin were detected in 4 cases, all were recipients. Conclusion MSCs co-transplantation accelerated HSCs engraftment and hematopoiesis recovery, the time to get ANC ≥0.5×109/L and PLT ≥2.0×1010/L were faster than those of single CBT and UD-BMT. MSCs co-transplantation cannot promote engraftment in CBT of β-thalassemia major. Co-transplantation of MSCs accelerated reconstitution of hematopoietic cells and MSCs.