Effectiveness of autologous hematopoietic stem cell transplantation in the treatment of high-risk neuroblastoma in children: a single-center clinical study北大核心CSCD

Objective To investigate the effectiveness of high-dose chemotherapy combined with autologous hematopoietic stem cell transplantation (ASCT) in the treatment of children with high-risk neuroblastoma (NB). Methods A retrospective analysis was performed on 29 children with high-risk NB who were admitted to Shanghai Children's Hospital and were treated with high-dose chemotherapy combined with ASCT from January 2013 to December 2021, and their clinical features and prognosis were analyzed. Results Among the 29 children treated by high-dose chemotherapy combined with ASCT, there were 18 boys (62%) and 11 girls (38%), with a median age of onset of 36 (27, 59) months. According to the International Neuroblastoma Staging System, 6 children (21%) had stage III NB and 23 children (79%) had stage IV NB, and the common metastatic sites at initial diagnosis were bone in 22 children (76%), bone marrow in 21 children (72%), and intracalvarium in 4 children (14%). All 29 children achieved reconstruction of hematopoietic function after ASCT. After being followed up for a median time of 25 (17, 45) months, 21 children (72%) had continuous complete remission and 8 (28%) experienced recurrence. The 3-year overall survival rate and event-free survival rate were 68.9% ±16.1% and 61.4%±14.4%, respectively. Presence of bone marrow metastasis, neuron-specific enolase ≥370 ng/mL and positive bone marrow immunophenotyping might reduce the 3-year event-free survival rate (P<0.05). Conclusions Children with high-risk NB who have bone marrow metastasis at initial diagnosis tend to have a poor prognosis. ASCT combined with high-dose chemotherapy can effectively improve the prognosis of children with NB with a favorable safety profile. © 2023 Xiangya Hospital of CSU. All rights reserved.     

Detection of RAS gene mutation and its clinical significance in children with acute lymphoblastic leukemia北大核心CSCD

Objective To investigate the mutation rate of the RAS gene and its clinical significance in children with acute lymphoblastic leukemia. Methods A retrospective analysis was performed on the medical data of 120 children with newly diagnosed acute lymphoblastic leukemia, who were admitted to the Third Affiliated Hospital of Zhengzhou University from January 2015 to January 2020 and underwent next-generation sequencing. The clinical and molecular features were analyzed. The impact of RAS gene mutation on the overall survival rate was evaluated in these children. Results Among the 120 children, 35 (29.2%) had RAS gene mutation, 30 (25.0%) had KRAS gene mutation, and 5 (4.2%) had both NRAS and KRAS gene mutations. All NRAS mutations and 71% (25/35) of KRAS mutations were located at the 12th and 13th codons. RAS gene mutation was detected in 35 (33.3%) out of 105 children with B-lineage acute lymphoblastic leukemia, but it was not detected in those with acute T lymphocyte leukemia. Of all the children, 11 (9.2%) were lost to follow-up, and among the 109 children followed up, 16 (14.7%) died. The children with RAS gene mutation had a significantly lower 2-year overall survival rate than those without RAS gene mutation (P<0.05). The prognosis of children with RAS gene mutation combined with WT1 overexpression and WBC>50×109/L at diagnosis was worse (P<0.05). Conclusions RAS gene mutation is commonly observed in children with B-lineage acute lymphoblastic leukemia and may have an adverse effect on prognosis. © 2022 Xiangya Hospital of CSU. All rights reserved.     

胆道闭锁术中胆汁流量及胆汁成分的观察分析

Objective To assess the prognostic values of intraoperative bile flow and bile components in patients with biliary atresia. Methods The intrahepatic bile of 27 children with biliary atresia (BA) were collected intraoperatively. The bile collected from 17 children with choledochal cysts in surgery were studied as the control group. Bile flow was monitored in 7 cases. The concentrations of phospholipid, total protein (TP), bile acid (TBA), the total bilirubin (TBIL), direct bilirubin (DBIL),total cholesterol (TC), aspartate aminotransferase (AST), alanine aminotransferase ( ALT), γ- glutamine (GGT),alkaline phosphatase (ALP) in bile were measured. Patients with BA were followed for 1-6 months post-operatively. The information collected during the follow-up included the time when the jaundice disappeared,the blood biochemistry and onset of cholangitis. Results The bile flow of children with BA were significantly lower than that in the control group (P<0. 05). The bile flow of children with BA whose operative age > 60 days or preoperative blood ALT > 150 U/L were significantly lower than that in the control group (P<0. 05). The jaundice-relieving rate 2 months post-operatively in children with BA whose bile flow ≥0. 19 ml/min was significantly higher than that in patients whose bile flow <0. 19 ml/min (P<0. 05). The concentrations of phospholipid,TBA and DBIL in bile of children with BA were significantly lower than those in the control group (P<0. 05). The concentration of ALT in bile of patients with BA was significantly higher than that in the control group (P<0. 01 ). The concentrations of AST and TC in bile of children with BA whose jaundice have been relieved before the third month postoperatively were significantly lower than those in with persistent jaundice (P<0. 05). Conclusions The correlation between the intraoperative bile flow and the prognosis is significant in children with BA: the faster the bile flow, the better of prognosis. This suggested that the intraoperative bile flow can effectively predict the long-term prognosis of BA after Kasai operation. The change of bile components in bile of BA indicated that the hepatic function of BA is poor, the lower concentrations of phospholipid,TBA and DBIL in bile may be due to poor hepatic function. The correlations between the concentrations of AST and TC in bile and prognosis were significant:, the lower of the concentration of AST and TC, the better of prognosis.     

胆道闭锁术中胆汁流量及胆汁成分的观察分析

Objective To assess the prognostic values of intraoperative bile flow and bile components in patients with biliary atresia. Methods The intrahepatic bile of 27 children with biliary atresia (BA) were collected intraoperatively. The bile collected from 17 children with choledochal cysts in surgery were studied as the control group. Bile flow was monitored in 7 cases. The concentrations of phospholipid, total protein (TP), bile acid (TBA), the total bilirubin (TBIL), direct bilirubin (DBIL),total cholesterol (TC), aspartate aminotransferase (AST), alanine aminotransferase ( ALT), γ- glutamine (GGT),alkaline phosphatase (ALP) in bile were measured. Patients with BA were followed for 1-6 months post-operatively. The information collected during the follow-up included the time when the jaundice disappeared,the blood biochemistry and onset of cholangitis. Results The bile flow of children with BA were significantly lower than that in the control group (P<0. 05). The bile flow of children with BA whose operative age > 60 days or preoperative blood ALT > 150 U/L were significantly lower than that in the control group (P<0. 05). The jaundice-relieving rate 2 months post-operatively in children with BA whose bile flow ≥0. 19 ml/min was significantly higher than that in patients whose bile flow <0. 19 ml/min (P<0. 05). The concentrations of phospholipid,TBA and DBIL in bile of children with BA were significantly lower than those in the control group (P<0. 05). The concentration of ALT in bile of patients with BA was significantly higher than that in the control group (P<0. 01 ). The concentrations of AST and TC in bile of children with BA whose jaundice have been relieved before the third month postoperatively were significantly lower than those in with persistent jaundice (P<0. 05). Conclusions The correlation between the intraoperative bile flow and the prognosis is significant in children with BA: the faster the bile flow, the better of prognosis. This suggested that the intraoperative bile flow can effectively predict the long-term prognosis of BA after Kasai operation. The change of bile components in bile of BA indicated that the hepatic function of BA is poor, the lower concentrations of phospholipid,TBA and DBIL in bile may be due to poor hepatic function. The correlations between the concentrations of AST and TC in bile and prognosis were significant:, the lower of the concentration of AST and TC, the better of prognosis.     

抗胸腺细胞球蛋白治疗儿童再生障碍性贫血的临床研究

目的 总结以抗胸腺细胞球蛋白(ATG)为主的免疫抑制疗法治疗儿童再生障碍性贫血时,ATG的实施方法、不良反应防治和长期随访措施等与远期疗效的相关性.方法 儿童再生障碍性贫血35例,其中极重型再生障碍性贫血(VSAA)6例,急性再生障碍性贫血(SAA)11例,慢性重型SAA8例,中型再生障碍性贫血(MAA)10例.ATG治疗期间措施包括:治疗前清除感染灶;积极防治过敏反应;密切观察和处理因ATG相关血小板减少所致严重出血和免疫抑制所致严重感染;积极防治血清病;重视长期随访中的治疗措施与质量.结果 ATG治疗后,所有病例均出现60%以上的淋巴细胞绝对计数下降.平均随访28个月,总有效率为77.14%(27/35),显效率为57.14%(20/35).VSAA、SAA和MAA间疗效无明显差异.ATG不良反应观察结果:①48.6%出现轻度类过敏反应;②血清病发生率42.9%,平均病程3.6 d;③9例(25.7%)外周血小板(BPC)计数＜10×109/L;④8例(22.9%)在ATG治疗后1个月内发生感染;⑤未发生ATG治疗相关死亡.Genzyme和Fresenius两种不同ATG制剂,在疗效和ATG相关不良反应发生率方面差异均无统计学意义.结论 ATG治疗儿童SAA和MAA疗效显著,但需积极预防和控制ATG不良反应,避免治疗相关死亡.长期辅助治疗和提高随访质量,也是确保疗效的重要环节.

Abstract：

Objective To evaluate the efficacy of antithymocyte globulin (ATG) based immunosupression therapy for childhood aplastic anemia, to reduce the adverse effects and to observe the long-term outcome. Method Thirty-five children with aplastic anemia (AA) were enrolled in this study.Six of the cases had very severe AA (VSAA), 11 had severe AA (SAA)-Ⅰ, 8 had SAA-Ⅱ and 10 had moderate AA (MAA). All these patients were treated with ATG plus Cyclosporin A (CSA). The following measures were taken during the ATG therapy: infection of the patients had been controlled before ATG treatment. Comprehensive anti-allergic measures were implemented. Close attention was paid to the hemorrhage related with platelet reduction caused by ATG and severe infection of the patients. Result Shortly after the ATG usage, all the patients had a significant decrease of absolute peripheral lymphoblast count by more than 60 percent. With a mean follow-up time of 28 months, the total effective rate was 77.14％ ( 27/35), significant response rate was 57.14％(20/35). There was no significant difference among VSAA, SAA and MAA groups in the response rate. Adverse reactions included the following:① 48.6％ (17/35) patients presented mild anaphylactoid reaction during the first day of ATG treatment; ②42.9％(15/35) cases presented serum sickness 5-11 days after the last dose of ATG with a mean duration of 3. 6 days, all the patients were cured effectively with methylprednisolone; ③25.7％ (9/35)patient's peripheral blood platelet count was reduced, might be caused by ATG, to below 10 × 109/L, but no patient had severe hemorrhagic complication after platelet transfusion was performed; ④22.9％ ( 8/35 ) of patients got infection within a month after ATG therapy, including 3 cases with clinical septicemia, all the 3 cases recovered after antibiotics treatment. There was no ATG treatment-related death in this series. Conclusion ATG is a very effective therapy for children with SAA and MAA. Comprehensive measures are needed to prevent and handle the side effects to avoid treatment-related death. Long-term supportive therapy and proper follow up contribute to the favourable outcomes of the patients.     

发育性髋关节脱位患儿术后股骨骨密度的变化及临床意义

Objective To analyze the risks and treatments of post-operative fracture by assessing the femoral bone mineral density in the children with developmental dislocation of hip. Methods The Norland XR46 bone densitomer system (USA) was employed and dual X-ray absorptiometry was employed to measure the bilateral femoral density of 92 children with developmental dislocation of hip pre-operatively and,2,4 and 6 months post-operatively. Results All 92 children demonstrated significant femoral bone mineral density reduction on the operated side 2 months posts-operatively (from 0. 5178 ± 0. 0829 to 0. 2878 ± 0. 0674). They recovered to different degree after 4 months after removing plaster and internal fixation plate (the average recoved to 0. 4927 ± 0. 1073). Conclusions The mineral density of the femur in children who underwent operative reduction for developmental dislocation of the hip joint decreased significantly 2 months post-operatively. It recoverd after the removal of the plaster. The femoral bone density during this period was lower than normal and it is susceptible to fracture.     

A pilot study on the effects of early use of valproate sodium on neuroinflammation after traumatic brain injury北大核心CSCD

Objective To study the effect of early use of sodium valproate on neuroinflammation after traumatic brain injury (TBI). Methods A total of 45 children who visited in Xuzhou Children's Hospital Affiliated to Xuzhou Medical University from August 2021 to August 2022 were enrolled in this prospective study, among whom 15 healthy children served as the healthy control group, and 30 children with TBI were divided into a sodium valproate treatment group and a conventional treatment group using a random number table (n=15 each). The children in the sodium valproate treatment group were given sodium valproate in addition to conventional treatment, and those in the conventional group were given an equal volume of 5% glucose solution in addition to conventional treatment. The serum concentrations of nucleotide-binding oligomerization domain-like receptor protein 3(NLRP3), high-mobility group box 1 (HMGB1), tumor necrosis factor-α (TNF-α), and interleukin-1β (IL-1β) were measured in the healthy control group on the day of physical examination and in the children with TBI on days 1, 3, and 5 after admission. Glasgow Outcome Scale-Extended (GOS-E) score was evaluated for the children with TBI 2 months after discharge. Results Compared with the healthy control group, the children with TBI had significantly higher serum concentrations of NLRP3, HMGB1, TNF- α, and IL-1β on day 1 after admission (P<0.017). The concentration of NLRP3 on day 5 after admission was significantly higher than that on days 1 and 3 after admission in the children with TBI (P<0.017). On days 3 and 5 after admission, the sodium valproate treatment group had a significantly lower concentration of NLRP3 than the conventional treatment group (P<0.05). For the conventional treatment group, there was no significant difference in the concentration of HMGB1 on days 1, 3, and 5 after admission (P>0.017), while for the sodium valproate treatment group, the concentration of HMGB1 on day 5 after admission was significantly lower than that on days 1 and 3 after admission (P<0.017). On day 5 after admission, the sodium valproate treatment group had a significantly lower concentration of HMGB1 than the conventional treatment group (P<0.05). For the children with TBI, the concentration of TNF-α on day 1 after admission was significantly lower than that on days 3 and 5 after admission (P<0.017). On days 3 and 5 after admission, the sodium valproate treatment group had a significantly lower concentration of TNF-α than the conventional treatment group (P<0.05). The concentration of IL-1β on day 3 after admission was significantly lower than that on days 1 and 5 after admission (P<0.017) in the children with TBI. On days 3 and 5 after admission, the sodium valproate treatment group had a significantly lower concentration of IL-1β than the conventional treatment group (P<0.05). The GOS-E score was significantly higher in the sodium valproate treatment group than that in the conventional treatment group 2 months after discharge (P<0.05). Conclusions Early use of sodium valproate can reduce the release of neuroinflammatory factors and improve the prognosis of children with TBI. © 2023 Xiangya Hospital of CSU. All rights reserved.     

儿童急性单核细胞性白血病63例疗效分析

目的 评估儿童急性单核细胞性白血病(AML-M5)的疗效,探讨异基因造血干细胞移植(allo-HSCT)治疗儿童AML-M5的指征.方法 对AML-M5患儿进行回顾性分析,与同期非AML-M5的AML进行比较,采用Kaplan-Meier曲线评估患儿的无事件生存(EFS)率和总生存(OS)率,COX回归模型评估预后因素.结果 ①75例AML-M5患儿中12例放弃治疗;63例纳入分析,其中2例化疔第2天死亡,61例患儿2个疗程完全缓解(CR)率为73.8%(45例),5年EFS为(34.5±6.8)%,与同期117例非AML-M5、非急性早幼粒细胞性白血病(AML-M3)的AML患儿(51.0±4.9)%的5年EFS相比差异有统计学意义(P＜0.01).②多因素分析显示患儿年龄≥10岁、1疗程后骨髓原幼稚细胞比例≥15%、2疗程后未CR是独立危险因素.③20例1疗程后骨髓原幼稚细胞比例≥15%的AML-M5患儿选择allo-HSCT者(n=5)预后优于单纯化疗者(n=15)[(60.0±21.9)%vs.(7.3±7.1)%,P=0.024].结论 AML-M5患儿预后明显差于其他AML;化疗1疗程后骨髓原幼稚细胞比例≥15%患儿选择allo-HSCT能明显提高生存率;目前依据尚不足以提示1疗程后骨髓原幼稚细胞比例＜15%患儿应选择非血缘相关供体移植治疗.

Abstract：

Objective To evaluate the outcomes of childhood acute monocytic leukemia (AML-M5) and explore the indications of allogeneic hematopoietic stem cell transplantation (allo-HSCT) for children with AML-M5. Method Seventy-five AML-M5 patients and 201 non-AML-M5 AML patients were enrolled in this retrospective analysis. Event-free survival (EFS) and overall survival (OS) rates were estimated by Kaplan-Meier method and prognostic factors were evaluated by COX regression with SPSS.Result ①Twelve patients gave up treatment after confirmed diagnosis. Two patients died on the second day after chemotherapy. Of the 61 patients, 73. 8％ (45/61) achieved complete remission (CR) after two courses of chemotherapy. The 5-year EFS rate was 34. 5％ ±6. 8％. But of the 117 non-AML-M5/M3 AML patients,the 5-year EFS rate was 51.0％ ±4. 9％. ② Multivariate analysis showed that age ≥ 10 y, the proportion of bone marrow blast cell counts≥ 15％ after the first induction therapy, not CR after two courses of chemotherapy were risk factors for the long-term prognosis. ③ Of the 20 patients whose bone marrow blast cell counts≥ 15％ after the first induction therapy, 5 patients who choose allo-HSCT had a better OS than the other 15 patients who choose chemotherapy only ( 60. 0％ ± 21.9％ vs. 7. 3％ ± 7. 1％, P = 0. 024 ).Conclusion Children with AML-M5 had a poorer prognosis than the other AML patients; patients whose bone marrow blast cell counts ≥ 15％ after the first induction therapy chose allo-HSCT had a better prognosis. At present, there is no enough evidence to support that patients whose bone marrow blast cell counts ＜ 15％ after the first induction therapy should choose unrelated donor for allo-HSCT.     

Acute hemorrhagic edema of infancy: the experience of a large tertiary pediatric center in Israel

Background:Acute hemorrhagic edema of infancy (AHEI) is a rare leukocytoclastic vasculitis of the small vessels occurring at a young age and considered as a benign self-limited disease.Due to its low prevalence,there are limited data on the presentation and complications of this disease.Methods:All computerized files of children who were hospitalized at a tertiary pediatric center due to AHEI over a 10 year period were reviewed.Clinical,laboratory and histopathological data were collected.Results:Twenty-six patients were included in our study,accounting for 0.7 cases per 1000 admissions of children aged 2 years or less.Mean age was 12.9 months.More than two thirds of the children had preceding symptoms compatible with a viral infection.Upon admission,all patients presented with typical findings of a rash and edema.Edema was most profound over the lower extremities (73％).Concomitant viral or bacterial infections were found in six children.Skin biopsy was performed in six patients revealing leukocytoclastic vasculitis.Thirteen children (50％) had systemic involvement including joint involvement (n=9),gastrointestinal hemorrhage (n=4),microscopic hematuria (n=1) and compartment syndrome of the limb (n=1).The latter was diagnosed in a patient with familial Mediterranean fever.Conclusions:Our largest data series highlighted what is known regarding clinical and histological findings in children with AHEI.However,contrary to what was previously reported,we found a higher rate of systemic involvement.Although AHEI is a rare entity,pediatricians should be familiar with its presentation,management and our reported complications.     

Clinical features of children with colorectal polyps and the efficacy of endoscopic treatment: an analysis of 1 351 cases北大核心CSCD

Objective To study the clinical features of children with colorectal polyps and the efficacy of endoscopic treatment. Methods A retrospective analysis was performed on the medical data of 1 351 children with colorectal polyps who were admitted and received colonoscopy and treatment in the past 8 years, including clinical features and the pattern and outcomes of endoscopic treatment. Results Among the 1 351 children, 893 (66.10%) were boys and 981 (72.61%) had an age of 2-<7 years, and hematochezia (1 307, 96.74%) was the most common clinical manifestation. Of all the children, 89.27% (1 206/1 351) had solitary polyps, and 95.77% (1 290/1 347) had juvenile polyps. The polyps were removed by electric cauterization with hot biopsy forceps (6 cases) or high-frequency electrotomy and electrocoagulation after snare ligation (1 345 cases). A total of 1 758 polyps were resected, among which 1 593 (90.61%) were pedunculated and 1 349 (76.73%) had a diameter of <2 cm. Postoperative complications included bleeding in 51 children (3.77%), vomiting in 87 children (6.44%), abdominal pain in 14 children (1.04%), and fever in 39 children (2.89%), while no perforation was observed. The children aged <3 years had the highest incidence rates of postoperative bleeding and fever (P<0.0125), and the children with a polyp diameter of ≥2 cm had significantly higher incidence rates of postoperative bleeding, vomiting, and fever (P<0.05). Conclusions Solitary polyps, pedunculated polyps, and juvenile polyps are common types of pediatric colorectal polyps. Electric cauterization with hot biopsy forceps or high-frequency electrotomy and electrocoagulation after snare ligation can effectively remove colorectal polyps in children, with good efficacy and few complications. Younger age and larger polyp diameter are associated with a higher risk of postoperative bleeding. © 2022 Xiangya Hospital of CSU. All rights reserved.     

免疫抑制治疗获得性重型再生障碍性贫血患儿疗效分析

目的 分析免疫抑制治疗儿童获得性重型再生障碍性贫血(severe aplastic anemia,SAA)的近远期疗效.方法 回顾性分析2000年1月至2006年6月在我院应用联合免疫抑制治疗的获得性重型再生障碍性贫血患儿.112例患儿随机分3组:Ⅰ组(26例):单用环孢素A(CSA)组;Ⅱ组(30例):CSA+丙种球蛋白[400 mg/(kg·d)×5 d];Ⅲ组(56例):兔抗胸腺细胞球蛋白(R-ATG)[3～5 mg(kg·d)×5 d]+CSA.所有患儿治疗均同时加用司坦唑醇或丙酸睾丸酬.CSA血药浓度调整到谷浓度100 ug/L以上,峰浓度300 ug/L以上.结果Ⅰ组免疫抑制治疗的总反应率为26.92%;Ⅱ组免疫抑制治疗的总反应率为33.33%;Ⅲ组免疫抑制治疗的总反应率为62.5%,明显高于Ⅰ组(P=0.001);比较Ⅰ组与Ⅱ组的总反应率差异无统计学意义.Ⅰ、Ⅱ和Ⅲ组5年总生存率分别为(20.50±15.41)%、(39.77±9.77)%和(66.27±6.84)%.结论对无HLA匹配同胞供者的重型获得性再生障碍性贫血患儿ATG联合CSA是最理想的治疗方法 .     

Rabbit Antithymocyte Globulin Treatment in Childhood Acquired Severe Aplastic Anemia

Acquired severe aplastic anemia (SAA) is a life threatening bone marrow failure characterized by pancytopenia and hypocellular bone marrow. Matched sibling donor is not available for majority of the patients and many children receive immunosuppressive therapy (IST). Although horse antithymocyte globuline (ATG) is the preferred option, our patients received rabbit ATG; since horse ATG is not available in Turkey. We reviewed the medical records of children with SAA who were treated with rabbit ATG, cyclosporine, and granulocyte colony stimulating factor (GCSF) between 2006 and 2012. Fifteen children with SAA aged between 1.5 and 17 years received rabbit ATG as first line treatment. Only two of them showed partial response and the others did not give any response at 3rd, 6th, and 12th months after the first course of IST. The second course of ATG was given to 8 of the patients; Rabbit ATG at the same dosage was used for 3 of them, and others were given horse ATG. None of the patients responded to the second course of ATG. Invasive fungal infection (IFI) which was seen in 80% of the patients was the most significant problem. Overall survival rate was 60%. The median time between the diagnosis and initiation of IST was 57 (range; 29–144) days. This delay might be significantly contributed to unresponsiveness. In our series, the use of rabbit ATG was not effective for these patients as first line treatment modality. Response rate was very low and the incidence of fungal infections was very high in the SAA patients who received rabbit ATG.     

儿童再生障碍性贫血免疫抑制剂治疗疗效及相关因素分析

目的：探讨免疫抑制疗法（IST）对儿童再生障碍性贫血（AA）的疗效及其影响疗效的相关因素。方法：对2003年2月至2009年11月住院接受IST治疗的、可进行疗效评估的110例AA患儿的临床资料进行回顾性分析。110例患儿中,重型AA（SAA）83例,非重型 AA（非SAA）27例。前者采用抗胸腺细胞球蛋白（ATG）联合环孢素（CSA）及泼尼松、雄激素四联治疗,后者采用CSA联合泼尼松、雄激素三联治疗。结果：SAA与非SAA组的总有效率分别为69.9%和70.4%。单因素分析显示病程、骨髓CD34+细胞比例、CD4+CD25+调节性T细胞比例与疾病严重程度相关,但与预后无关。治疗有效组患儿年龄、病程、骨髓CD3+、CD8+细胞比例显著低于治疗无效组（P<0.05）。多因素分析显示年龄＞10岁、骨髓CD8+细胞比例＞25%的患儿治疗失败的风险分别是对应组的3.36倍和3.59倍。结论：IST治疗儿童AA疗效确切。年龄、病程、CD3+、CD8+ T细胞水平与IST的疗效相关。     

免疫抑制疗法治疗儿童再生障碍性贫血疗效分析

目的：探讨免疫抑制疗法(IST)治疗儿童再生障碍性贫血(AA)的疗效、安全性及影响疗效的主要因素。方法：对2007年1月至2010年12月接受IST治疗的55例重型再生障碍性贫血（SAA）及51例慢性再生障碍性贫血（CAA）患儿的临床资料进行回顾性分析。结果：① 在CAA患儿中,抗胸腺球蛋白（ATG）联合环孢素A（CsA）治疗组总有效率明显高于CsA单独治疗组(80％ vs 44％,P40%、治疗前无重症感染以及有G-CSF早期治疗反应的患儿治疗效果较好,而治疗效果与AA分型、年龄等指标无关。结论：ATG+CsA联合治疗是治疗儿童AA的一种安全有效的方法;病程长短、有无严重感染、骨髓造血面积及G-CSF早期治疗反应是影响疗效的主要因素。     

免疫抑制治疗儿童重型再生障碍性贫血54例疗效分析

目的 探讨免疫抑制治疗儿童重型再生障碍性贫血（severe aplasia anemia,SAA）的疗效。方法 回顾性分析我院1997年1月—2003年6月儿童重型再生障碍性贫血54例,应用环孢素A（cyclosporine,CSA）和抗胸腺细胞球蛋白（antithymie globlin,ATG）为主的免疫抑制治疗,其中应用CSA联合ATG治疗31例（称为CSA联合ATG组）,应用CSA治疗23例（称为CSA组）,比较两组的治疗有效率、复发率、不良反应和无病生存率。结果两组的分型和极重型患者资料具有可比性。CSA联合ATG组和CSA组起效时间平均分别为2．5个月和3．5个月,两组有效率分别为81％（25／31）和52％（12／23）（x^2=4．962,P〈0．05）。治疗有效者共37例,CSA联合ATG组和CSA组的复发率分别为8％（2／25）和50％（6／12）（xc^2=6．143,P〈0．05）。两组患者不良反应的发生率差异无统计学意义。所有患者随访至少1年,CSA联合ATG组和CSA组1年以上生存率分别为81％（25／31）和52％（12／23）;随访超过2年者共47例,CSA联合ATG组和CSA组2年以上生存率分别为74％（20／27）和50％（10／20）（P〈0．01）。结论免疫抑制治疗儿童重型再生障碍性贫血的疗效肯定,而CSA联合ATG治疗重型再生障碍性贫血的疗效更优于单用CSA。     

抗胸腺细胞球蛋白联合环孢菌素A治疗儿童再生障碍性贫血疗效及相关因素分析

目的探索采用抗胸腺细胞球蛋白(ATG)联合环孢菌素A(CSA)的免疫抑制疗法(IST)治疗儿童再生障碍性贫血(再障)的疗效及其相关影响因素,为进一步提高临床疗效提供参考依据。方法共40例再障患儿(重型再障28例,依赖成分输血的慢性再障12例)接受ATG联合CSA治疗。统计分析治疗前病程和外周血三系下降程度;ATG治疗后外周血淋巴细胞绝对计数(ALC)下降程度、血清病发生率及不同制剂ATG治疗等临床因素与远期疗效的相关性。结果中位随访时间19(9～44)个月,总有效率和显效率分别为78%和45%。疗效相关统计分析显示:(1)ATG治疗后2周内,ALC下降幅度≥2×109/L者的有效率明显高于下降幅度<2×109/L者,两组总有效率分别为89%和54%(P<0.05)。(2)从确诊再障到接受ATG治疗,病程≤6个月者有效率明显高于病程>6个月者,两组总有效率分别为92%和53%(P<0.05)。(3)采用两种ATG制剂(美国Genzyme或德国Fresenius)各治疗18例和22例,两组疗效差异无显著性(P>0.05)。结论 ATG联合CSA的免疫抑制治疗是儿童再障的有效疗法,目前常用的两种ATG制剂,均能获得显著疗效。再障确诊后早期治疗有助于提高疗效。治疗期间密切观察ALC下降程度,对于ALC下降不明显者,是否需要适当增加ATG剂量,有待进一步研究论证。     

ALG为主的免疫抑制剂治疗儿童重型再生障碍性贫血的疗效及相关因素

目的通过回顾性分析临床资料,探讨重型再生障碍性贫血(SAA)患儿以抗淋巴细胞球蛋白(ALG)为主的免疫抑制治疗(IST)疗效及相关因素。方法54例重型再生障碍性贫血、并接受ALG治疗患儿的临床资料进行分析。结果基本治愈24例(44.4%),缓解12例(22.2%),明显进步4例(7.4%),无效14例(25.9%),总有效率(74.1%)。典型的血清病多在治疗后7~14 d内出现。随访的54例患儿中1例出现骨髓增生异常综合征(MDS)样病态造血。结论ALG作为重要免疫抑制剂治疗SAA疗效肯定,治疗前患者外周血ANC计数、骨髓涂片中淋巴细胞的比例以及检测CsA有效浓度可能对疗效判断有提示意义。IST后出现重度感染多为预后不良的重要因素之一。     

再生障碍性贫血患儿骨髓核素显像特点及其临床意义分析

目的 应用99mTc-硫胶体全身骨髓显像检测再生障碍性贫血（再障）患儿全身骨髓显影特点,探讨其在再障诊治中的价值。方法　对2009年1月至2012年1月青岛大学医学院附属医院小儿血液科收治的45例再障患儿（再障组）及15名对照组进行99mTc-硫胶体全身骨髓显像。99mTc-硫胶体注射放射剂量10～15 MBq/kg,分析骨髓核素显像的特异性、敏感性及不同类型再障患儿骨髓显像特点和临床转归的相关性。结果　再障组45例骨髓显像中活性减低33例（73.3%,包括0级11例、1级22例）,表现为全身骨髓显像有不同程度受抑,其特点是全身骨髓显影不良、显影骨髓总量减少;2级12例（26.7%）,骨髓活性正常。45例再障组临床诊断为急性再障（SAA）组19例、慢性再障（CAA）组26例,其中SAA组骨髓显像表现为中央＋外周骨髓均抑制者15例,仅中央骨髓抑制2例;CAA组中央＋外周骨髓均抑制6例,仅中央周骨髓抑制6例,仅外周抑制4例;两组骨髓显像抑制部位差异有统计学意义（χ2= 10.37,P＜0.05）;提示慢性再障骨髓显像受抑程度较轻,可仅有中央或外周骨髓的抑制并多存在灶状增生。对再障患儿骨髓显像与早期治疗反应进行统计分析,结果示不同骨髓显像分级间疗效差异有统计学意义（χ2=12.49,P＜0.05）,骨髓显像0级组有效率为36.3%,较1级组、2级组（77.3%、83.4%）疗效差。结论　全身骨髓显影不良、显影骨髓总量减少及显像特点有助于对儿童再障的诊断、分型及预后判断。骨髓显像与临床转归和预后关系仍需进一步扩大病例研究。     

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??Abstract?? Objective To investigate the characteristics of 99mTcmsulfur collid bone marrow scintigraphy in childhood aplastic anemia ??AA?? and its clinical significance.Methods A total of 45 children were newly diagnosed with AA??including 19 cases of serious aplastic anemia ??SAA?? and 26 cases of chronic aplastic anemia ??CAA??.Bone marrow scintigraphy using 99mTcmsulfur collid 10??15 MBq/kg was performed in 45 patients and 15 controls.The difference of bone marrow scintigraphy was analysed. The relationship between laboratory indices and clinical response was investigated. Results According to the bone marrow imaging results??grade 2 of marrow activities was defined as normal. Totally 33 patients??33/45??73.3%?? had abnormal marrow activities??and in 15 controls only 3??3/15??20%??.Most AA patients showed 99mTcmsulfur collid uptake decrease. CAA patients had higher marrow activities than SAA patients ??P??0.05??.In the 33 patients who had abnromal marrow activities??less then 2 grade????SAA and CAA patients showed different 99mTcmsulfur collid uptake in the peripheral and the center of bone marrow??and CAA had focals whose uptake was increasing.After treatment of 6 months??45 AA patients were investigated the relationship between the grade of marrow activities and the therapeutic effect??including improved??inefficacy??death??.There showed significantly difference??P??0.05?? between them. Conclusion The 99mTcmsulfur collid bone marrow imaging is helpful to the diagnosis and prognosis of AA??but further study is needed to judge its relationship with the curative effect and prognosis.     

环胞菌素A治疗儿童再生障碍性贫血疗效研究

探讨环胞菌素 A(CSA)治疗儿童再生障碍性贫血 (再障 )的方法 ,疗效和疗效相关因素。方法 :应用 CSA对 34例儿童再障行免疫抑制治疗 (IS) ;部分重型再障(SAA)加用抗胸腺细胞球蛋白 (ATG)或大剂量免疫球蛋白 (HDIG) ,均以雄性激素作为辅助治疗。结果 :基本治愈 4例 ,缓解 1 1例 ,明显进步 8例 ,总有效率为 6 7.6 5 % ;其中 2 9例 SAA总有效率为 6 5 .5 2 % ,1 8例慢性重型再障 (SAA- )有效率为5 5 .5 6 % ;1 1例急性再障 (SAA- )总有效率达 81 .82 % ,单项资料对比分析结果显示SAA的年龄 ,性别和治疗前外周血象等因素与 CSA有效率无关 ;但病程较短者 (<1 2个月 )有效率较高 ;CSA与 ATG等行联合 IS则有效率可明显提高。结论 :CSA为治疗儿童再障的有效方法之一 ,SAA拟选用联合 IS,雄性激素为 CSA的有效辅助治疗