BACKGROUND:Allogeneic hematopoietic stem cell transplantation is currently the most effective method for the radical treatment of thalassemia major,but only half of patients can find compatible bone marrow or peripheral blood stem cells.Sib-derived umbilical cord blood stem cells have different characteristics from bone marrow and peripheral blood stem cells,and are a potential alternative source of hematopoietic stem cells for transplantation in patients with thalassemia major.OBJECTIVE:To investigate the therapeutic effect of human leukocyte antigen matched sibling fresh umbilical cord blood transplantation in the treatment of β-thalassemia major in children.METHODS:Forty-eight children with β-thalassemia major,including 28 males and 20 females,with a median age of 4 years old,were selected from Nanfang Hospital of Southern Medical University from June 2010 to June 2020.All of them received fresh cord blood transplantation from human leukocyte antigen matched sibling.Transplantation conditioning adopted a myeloablative regiment without anti-thymocyte globulin.A combination of cyclosporine A and mycophenolate mofetil with or without short-range methotrexate was administered for graft-versus-host disease.RESULTS AND CONCLUSION:(1)The median infused doses of total nucleated cells and CD34+cells were 8.17×107/kg and 2.40×105/kg,respectively in 48 children.The median follow-up time after cord blood transplantation was 98 months,and 44 cases were successfully engrafted.The median time to neutrophil and platelet engraftment was 28 and 31 days,respectively.Among them,37 cases were found to be donor-type complete chimerism detected as evidence of implantation after transplantation,7 cases were found to be stable mixed chimerism.(2)Among the 44 children with successful implantation,four patients developed acute graft-versus-host disease,and were scored as grade Ⅰ(n=2)and grade Ⅱ(n=2).All the affected organs were skin,and no chronic graft-versus-host disease occurred.(3)After umbilical cord blood transplantation,cytomegalovirus infection and activation occurred in 5 of the 48 cases,sepsis in 12 cases,invasive fungal disease in 3 cases,stomatitis in 21 cases,hemorrhagic cystitis in 8 cases,and hepatic vein occlusion in 1 case.(4)Among 48 children,47 patients survived;1 died of severe pneumonia combined with acute heart failure 28 days after transplantation;43 survived without disease;3 had primary implantation failure,and 1 had pancytopenia after transplantation.The 5-year probabilities of overall survival and disease-free survival were 98％and 89％,respectively.The cumulative incidence of transplant-related deaths at 1 year was 2.1％.(5)The above results indicate that human leukocyte antigen matched sibling fresh umbilical cord blood transplantation is effective in the treatment of β-thalassemia major in children with a low incidence of graft-versus-host disease.

BACKGROUND:Allogeneic hematopoietic stem cell transplantation is currently the most effective method for the radical treatment of thalassemia major,but only half of patients can find compatible bone marrow or peripheral blood stem cells.Sib-derived umbilical cord blood stem cells have different characteristics from bone marrow and peripheral blood stem cells,and are a potential alternative source of hematopoietic stem cells for transplantation in patients with thalassemia major.OBJECTIVE:To investigate the therapeutic effect of human leukocyte antigen matched sibling fresh umbilical cord blood transplantation in the treatment of β-thalassemia major in children.METHODS:Forty-eight children with β-thalassemia major,including 28 males and 20 females,with a median age of 4 years old,were selected from Nanfang Hospital of Southern Medical University from June 2010 to June 2020.All of them received fresh cord blood transplantation from human leukocyte antigen matched sibling.Transplantation conditioning adopted a myeloablative regiment without anti-thymocyte globulin.A combination of cyclosporine A and mycophenolate mofetil with or without short-range methotrexate was administered for graft-versus-host disease.RESULTS AND CONCLUSION:(1)The median infused doses of total nucleated cells and CD34+cells were 8.17×107/kg and 2.40×105/kg,respectively in 48 children.The median follow-up time after cord blood transplantation was 98 months,and 44 cases were successfully engrafted.The median time to neutrophil and platelet engraftment was 28 and 31 days,respectively.Among them,37 cases were found to be donor-type complete chimerism detected as evidence of implantation after transplantation,7 cases were found to be stable mixed chimerism.(2)Among the 44 children with successful implantation,four patients developed acute graft-versus-host disease,and were scored as grade Ⅰ(n=2)and grade Ⅱ(n=2).All the affected organs were skin,and no chronic graft-versus-host disease occurred.(3)After umbilical cord blood transplantation,cytomegalovirus infection and activation occurred in 5 of the 48 cases,sepsis in 12 cases,invasive fungal disease in 3 cases,stomatitis in 21 cases,hemorrhagic cystitis in 8 cases,and hepatic vein occlusion in 1 case.(4)Among 48 children,47 patients survived;1 died of severe pneumonia combined with acute heart failure 28 days after transplantation;43 survived without disease;3 had primary implantation failure,and 1 had pancytopenia after transplantation.The 5-year probabilities of overall survival and disease-free survival were 98％and 89％,respectively.The cumulative incidence of transplant-related deaths at 1 year was 2.1％.(5)The above results indicate that human leukocyte antigen matched sibling fresh umbilical cord blood transplantation is effective in the treatment of β-thalassemia major in children with a low incidence of graft-versus-host disease.

【Objective】 To evaluate the safety and efficacy of the collection of peripheral blood stem cells (PBSCs) in pediatric patients with thalassemia major (TM) weighing 20 kg or less. 【Methods】 PBSCs collection data of 170 pediatric patients with TM weighing 20 kg or less from January 2013 to December 2020 in our center were reviewed. Safety was assessed by the occurrence of adverse events during apheresis procedures, and efficacy was evaluated by the number of CD34+ cells collected. 【Results】 A total of 171 PBSCs procedures were performed on 170 patients with TM weighing 20 kg or less, with a median age of (4.98±1.53) years and a median weight of (17.30±2.18) kg. The probability of collecting at least 1×10⁶ CD34+ cells/kg during a single course of apheresis was 99.41% (169/170), with a median (5.88±4.23) ×10⁶ CD34+ cells collected per kg of weight of the recipient. A minimum pre-apheresis hemoglobin (Hb) of 60 g/L in patients with TM weighing 20 kg or less was safe and feasible. The most common adverse event of G-CSF mobilization in TM patients is bone pain, with the incidence of 7.65% (13/170), which was higher than that of healthy children donors in our center. The most common adverse events during the collection were pain at the puncture site of the femoral vein (6.47%, 11/170) and low pressure of the fluid (2.92%, 5/170). And no serious complications related to PBSCs mobilization, central venous catheter(CVC)placement or the apheresis procedure occurred. 【Conclusion】 PBSCs collection by COM.TEC blood cell separator in children weighing 20 kg or less is safe and efficacious.

Objective To investigate the value of endoscopic papillary incision combined with balloon di-lation in the treatment of choledocholithiasis with periampullary diverticula(PAD). Methods A total of 118 pa-tients with choledocholithiasis and PAD admitted to our hospital from January 2015 to January 2017 were retrospec-tively analyzed.According to the operation method,all patients were divided into two groups:sEST+EPBD group (60 cases)and EST group(58 cases).The success rate of ERCP and the success rate of one-step stone removal, the changes of serum TBil and DBil before and after operation,and the incidence of postoperative complications were compared between the two groups. Results The successful rates of ERCP and stone removal in sEST+EPBD group were significantly higher than those in EST group(P < 0.05). The levels of serum TBil and DBil in both groups were significantly lower than those before operation(P<0.05).The overall incidence of postoperative com-plications in sEST + EPBD group was significantly lower than that in EST group(P < 0.05). Conclusions The treatment of choledocholithiasis with PAD in patients with endoscopic papillary small incision and balloon dilation treatment effect is significant,which can significantly improve the success rate of ERCP and success rate of one-time stone,benefitting the prognosis of patients.

Objective To design and synthesize compounds with protein tyrosine kinase(PTK)inhibitory activity with L029 as the lead compound. Methods L029 derivatives were designed and synthesized from L029 by reduction and/or substitution with the 3-dimethylamino-1-propyl,methyl acetate,methyl propionate in its active H and other sites. PTK activity was measured by enzyme-linked immunosorbent assay(ELISA). The inhibitory rate was calculated to screen out the compounds with PTK inhibitory activity. Re-sults Five target compounds were synthesized and their structures were confirmed by 1H NMR and MS. Three compounds T2,T3 and T5 were screened out with strong PTK inhibitory activity. Conclusion The synthetic routes of the target compounds are simple with mild reaction condition,and 3 compounds show strong inhibitory activity by ELISA. These results can provide reference for the further design and synthesis of this kind of molecules.

BACKGROUND:Cytokines play an important role in the occurrence and development of graft-versus-host disease, but there is a current lack of reports on the association between cytokines and graft-versus-host disease after al ogeneic hematopoietic stem cel transplantation for treatment ofβ-thalassemia major.
OBJECTIVE:To investigate the association between cytokines and graft-versus-host disease after al ogeneic hematopoietic stem cel transplantation forβ-thalassemia major.
METHODS:We observed the dynamic variation of interleukin 6, interleukin 8, interleukin 12, tumor necrosis factor-αand macrophage migration inhibitory factor in 11 children withβ-thalassemia major before onset of graft-versus-host disease, when graft-versus-host disease occurred, at days 4 and 7 after onset of graft-versus-host disease, and when graft-versus-host disease disappeared.
RESULTS AND CONCLUSION:There was a significant difference in serum levels of interleukin-6, interleukin-12, tumor necrosis factor-α, macrophage migration inhibitory factor in different time points, and the highest levels of different cytokines appeared when graft-versus-host disease occurred, fol owed by those at 7 days after
graft-versus-host disease. There was a significant difference in serum levels of interleukin-8 in different time points, and the highest level appeared at 4 days after graft-versus-host disease. The dynamic expression of interleukin-6, interleukin-8, interleukin-12, tumor necrosis factor-α, macrophage migration inhibitory factor can estimate the immune function ofβ-thalassemia major patients who develops graft-versus-host disease after al ogeneic hematopoietic stem cel transplantation, and can be used as the immunobiology indicators for the early diagnosis of graft-versus-host disease.

This study was aimed to find the relationship between lung function injured characteristic of ulcerative colitis (UC) and endoscopic index. It will verify and enrich Chinese medicine theory to guide clinical diagnosis and treatment. UC patients without respiratory system diseases were selected between September 2009 and March 2011 from several triple-A hospitals in China. Furthermore, disease history, lung function, colonoscopy and histopathology were taken. The results showed that among 171 enrolled cases, there were statistical differences in endoscopic index of residual-function and diffusion-function between the abnormal group and the normal group (P< 0.05). The resid-ual-function group was r = 0.003. There were no statistical differences in ventilatory-function and small-airway-function in the abnormal group. It was concluded that there were different types of injured lung function in UC. A-mong them, the abnormalities of residual-function and diffusion-function had strong correlation with the intestinal damage degree. The change on residual-function was more obvious.

Pediatrics internship is a bridge that brings medical theory into clinical reality.Cultivate systematic clinical thinking during internship is the key to the growth of clinical doctors.Good outcome has been achieved among these eight-year medical students by using teaching methods such as case discussions,lectures,teacher instructions,which can cultivate proper clinical thinking and make students more active.