Efficacy of Rasburicase for critically ill children with advanced Burkitt′s lymphoma / 中华实用儿科临床杂志

Objective:To explore the efficacy and safety of Rasburicase therapy in critically ill children su-ffering from advanced Burkitt′s lymphoma.Methods:A retrospective analysis of children with advanced Burkitt′s lymphoma was admitted to Pediatric Intensive Care Unit, the First Affiliated Hospital of Sun Yat-Sen University, from January 2015 to May 2020 and accepted treatment.According to the uric acid-lowering therapies, patients were divided into 2 groups, namely Rasburicase group (Group R) and traditional treatment group (Group T), to compare the effects of hypouricemic treatment and the prognosis between the 2 groups.Results:Twenty-nine children with advanced Burkitt′s lymphoma were included in this study, with 13 cases (44.83%) of stage Ⅲ and 16 cases (55.17%) of stage Ⅳ.Abdominal mass/ abdominal distension (13 cases, 44.83%) and abdominal pain (7 cases, 24.14%) were the main reasons of initial medical visit attendance.The most common primary tumor site was abdominal/ pelvic cavity (21 cases, 72.41%), followed by head or neck (6 cases, 20.69%). There were 15 cases in Group R and 14 cases in group T. No significant differences in serum creatinine, lactate dehydrogenase and uric acid were detected between the 2 groups (all P>0.05). The proportion of serum uric acid recovery rate of 24 hours and 72 hours after initial treatment in Group R were significantly higher than those in T group (85.71% vs.25.00%, 100.00% vs.25.00%, all P<0.01). Although there were no obvious differences in the incidence of tumor lysis syndrome between the 2 groups (33.33% vs.64.29%, P=0.096), the incidence of acute renal injury, renal replacement therapy requirement, serious complications and the 28 day mortality in Group R were remarkably lower than those in Group T (33.33% vs.85.71%, 13.33% vs.64.29%, 20.00% vs.78.57%, 0 vs.35.71%, all P< 0.05). Conclusions:Rasburicase can effectively reduce the serum uric acid level and decrease the incidence of acute kidney injury and other severe complications, thus improving the prognosis of children experiencing advanced Burkitt′s lymphoma.

Predictive value of four pediatric scores of critical illness and mortality on evaluating mortality risk in pediatric critical patients / 中华危重病急救医学

Objective To assess the performance of pediatric clinical illness score (PCIS), pediatric risk of mortality scoreⅢ(PRISMⅢ), pediatric logistic organ dysfunction score 2 (PELOD-2), and pediatric multiple organ dysfunction score (P-MODS) in predicting mortality in critically ill pediatric patients. Methods The data of critically ill pediatric patients admitted to Pediatric Intensive Care Unit (PICU) of First Affiliated Hospital of Sun Yat-Sen University from August 2012 to May 2017 were retrospectively analyzed. The gender, age, basic diseases, the length of PICU stay were collected. The children were divided into survival group and non-survival group according to the clinical outcome during hospitalization. The variables of PCIS, PRISMⅢ, PELOD-2, and P-MODS were collected and scored. Receiver operating characteristic (ROC) curve was plotted, the efficiency of PCIS, PRISMⅢ, PELOD-2, and P-MODS for predicting death were evaluated by the area under ROC curve (AUC). Hosmer-Lemeshow goodness of fit test was used to evaluate the fitting degree of each scoring system to predict the mortality and the actual mortality. Results Of 461 critically ill children, 35 children were excluded because of serious data loss, hospital stay not exceeding 24 hours, and death within 8 hours after admission. Finally, a total of 426 pediatric patients were enrolled in this study. 355 pediatric patients were survived, while 71 were not survived during hospitalization, with the mortality of 16.7%. There was no significant difference in gender, age, underlying diseases or length of PICU stay between the two groups. PCIS score in non-survival group was significantly lower than that of survival group [80 (76, 88) vs. 86 (80, 92)], and PRISMⅢ, PELOD-2 and P-MODS scores were significantly increased [PRISMⅢ: 16 (13, 22) vs. 12 (10, 15), PELOD-2: 6 (5, 9) vs. 4 (2, 5), P-MODS: 6 (4, 9) vs. 3 (2, 6), all P < 0.01]. ROC curve analysis showed that the AUCs of PCIS, PRISMⅢ, PELOD-2, and P-MODS for predicting death of critical ill children were 0.649, 0.731, 0.773, and 0.747, respectively. Hosmer-Lemeshow test showed that PCIS predicted the mortality and the actual mortality in the best fitting effect (χ2= 7.573, P = 0.476), followed by PELOD-2 and P-MODS (χ12 = 9.551, P1= 0.145; χ22 = 10.343, P2= 0.111), while PRISMⅢ had poor fitting effect (χ2= 43.549, P < 0.001). Conclusions PRISMⅢ, PELOD-2 and P-MODS can discriminate between survivors and moribund patients well, and assessing the condition of critically ill pediatric patients with relatively accuracy. PCIS was the best fitting effect in predicting mortality and actual mortality, followed by PELOD-2 and P-MODS, while PRISMⅢ had poor fitting effect.

Analysis on novel mutations in GAA gene of a Chinese family with two siblings affected with juvenile onset form glycogen storage disease Ⅱ / 重庆医学

Objective To identify a novel pathogenicity mutation of acid alpha‐glucosidase(GAA) gene in a Chinese family with two siblings affected with juvenile onset form glycogen storage disease Ⅱ(GSD Ⅱ) .Methods The clinical and family data of two siblings presenting recurrent respiratory tract infections ,respiratory failure associated with systemic muscle weakness ,were an‐alyzed and diagnosed with GSD Ⅱ by detecting alpha‐1 ,4‐glucosidase activity .DNA was extracted from peripheral blood of the proband ,younger brother and his parents .All 20 exons and the intron‐exon splice sites of GAA gene were amplified by polymerase chain reaction (PCR) .Mutations were detected by direct sequencing the PCR products .Results The younger brother was found to be compound heterozygous for two mutations in the GAA gene :c .1216G>A (p .Asp406Asn) missense mutation in the exon 8 from his father and c .1935C>A (p .Asp645Glu) missense mutation in the exon 14 from his mother .Conclusion The compound hetero‐zygous c .1216G>A and c .1935C>A mutations caused the juvenile onset form GSD Ⅱ characterized by dyspnea and cardiac hyper‐trophy .The novel c .1216G>A mutation may be related to the juvenile onset form GSD Ⅱ .

Bone marrow-derived mesenchymal stem cells regulate the function of Th17/Treg in peripheral blood of severe asthmatic children / 中国病理生理杂志

AIM: To investigate the regulatory function of bone marrow-derived mesenchymal stem cells (MSCs) on T helper 17 cells (Th17) and regulatory T cells (Treg) in peripheral blood of severe asthmatic children . METHODS:MSCs were isolated , cultured and identified in vitro.MSCs digested with mitomycin were cocultured with T lymphocytes (TLC) at different ratios (1∶1, 1∶2, 1∶10 and 1∶20) from severe asthmatic children for 72 h.The prolifera-tion of TLC was measured by CCK-8 method.In the coculture system of the 1∶2 ratio and the single TLC system , the super-natant levels of interleukin-17 (IL-17) and transforming growth factor-β(TGF-β) were measured by ELISA.The mRNA expression of retinoic acid-related orphan nuclear receptor C (RORC) and forkhead box protein 3 (Foxp3) in TLC was de-tected by qRT-PCR.RESULTS:After cocultured with MSCs , the proliferation of TLC decreased significantly in a dose-dependent manner (P0.05).CONCLUSION: MSCs suppresses Th17 polarization of naive peripheral blood CD 4 +T cells and matures Th17 cells secreting IL-17, which may ef-fectively revise Th17/Treg imbalance of asthma .