米托蒽醌、依托泊苷、阿糖胞苷方案治疗伴一系或多系病态造血的儿童急性髓系白血病的疗效

目的 探讨寻求伴一系或多系病态造血的儿童急性髓系白血病治疗方案,以期在短时间内达到完全缓解,为长期无病生存打下基础.方法 对DA或HA标准方案治疗1、2个疗程,无效的10例一系或多系病态造血的急性髓性白血病患儿采用自身对照的方法 .第2疗程化疗均采用米托蒽醌、依托泊苷、阿糖胞苷(MEA)方案,并辅以抗感染、成分输血、细胞因子应用等治疗.结果 10例患儿MEA方案治疗1个疗程,治疗中骨髓抑制期最长达19 d.中性粒细胞绝对值0～0.08×109 L-1,血小板(6～10)×109 L-1,白细胞升至3.5×109 L-1时 ,骨髓原始细胞8例下降至0.05以下,血小板恢复正常,Hb升高,8例获完全缓解,2例无效.结论 用MEA方案治疗伴一系或多系病态造血的急性髓系白血病儿童患者疗效较好.     

儿童系统性红斑狼疮合并脑后部可逆性脑病综合征4例并文献复习

目的 分析儿童系统性红斑狼疮（SLE）合并脑后部可逆性脑病综合征（PRES）的临床特征,提高对本病的认识。方法 报告北京协和医院诊断的4例儿童SLE合并PRES的临床资料,在PubMed数据库检索相关病例行文献复习,分析儿童SLE合并PRES的临床表现、影像学检查、治疗及预后情况。结果 ①4例SLE患儿均伴有狼疮性肾炎,从确诊SLE至出现PRES的时间1~63个月,出现PRES时1例正在接受甲泼尼龙及环磷酰胺(CTX)冲击治疗。PRES均以惊厥、头痛起病,并伴血压升高。②PubMed数据库检索到11例SLE合并PRES的儿童病例,结合本文报道的4例,15例进入分析。女性14例。年龄最小8岁。从确诊SLE至出现PRES的间隔中位时间为6个月（1个月至8年）。15例出现PRES时均有惊厥发作,10例伴头痛,7例呕吐,9例意识丧失,7例视力障碍。15例均有血压升高。12例有狼疮性肾炎。治疗SLE予甲泼尼龙或CTX冲击治疗分别为4和3例,予羟氯喹2例,予环孢素和利妥昔单抗各1例,PRES分别发生在免疫抑制剂治疗后的2 d至4年。15例行头颅MRI检查示大脑后循环皮质下白质受累为主。12例予降血压治疗,10例予抗惊厥药物短期治疗。9例SLE处于活动期,继予糖皮质激素和免疫抑制剂治疗;6例非活动期的SLE患儿减停糖皮质激素及免疫抑制剂。15例神经系统症状均恢复,随访均未遗留神经系统后遗症。9例复查头颅MRI示颅内病变完全或基本消失。结论 儿童SLE合并PRES主要表现为惊厥、头痛、意识障碍和视觉障碍。对于有狼疮性肾炎的SLE患儿血压升高时,尤其同时予大剂量糖皮质激素或CTX等免疫抑制剂治疗时,应警惕PRES的发生。早期诊断和治疗PRES预后较好。     

多种免疫抑制剂联合治疗儿童急性再生障碍性贫血

目的观察多种免疫抑制剂联合治疗儿童急性再生障碍性贫血(再障,AAA)的疗效,探索治疗AAA的有效方法。方法采用大剂量甲泼尼龙、抗淋巴细胞球蛋白(ALG)、环孢素、大剂量丙种球蛋白4种免疫抑制剂联合,并辅以粒系集落刺激因子、输血等治疗AAA 13例,并对其临床资料进行回顾分析。结果13例中基本治愈9例,缓解、进步各1例,无效2例(其中死亡1例);基本治愈9例白细胞达4×109/L,时间为(24.60±8.86)d,血红蛋白恢复至正常时间为(152.22±68.88)d,血小板达80×109/L,时间为(125.55±55.76)d。治疗过程中7例发生感染,2例出现ALT和AST增高,1例有低蛋白血症和水肿,2例出现肉眼血尿。1例牙龈明显增生,脱牙。结论多药联合免疫抑制剂是治疗小儿AAA一种安全有效的治疗方法。     

系统性红斑狼疮合并血栓性血小板减少性紫癜1 例分析

目的探讨系统性红斑狼疮(SLE)合并血栓性血小板减少性紫癜(TTP)的临床特点、诊断和治疗。方法回顾分析1例重症SLE合并TTP患儿的临床资料,并复习相关文献。结果患儿,女,13岁,以肾病综合征起病,诊断重症SLE、狼疮性肾炎。初治好转后再现头痛、血小板减少、贫血、急性肾损伤及发热。血涂片破碎红细胞2%,诊断合并TTP。经激素冲击、血浆置换、免疫抑制剂等综合性治疗后病情缓解。结论 SLE合并TTP较为少见,病死率高,早期识别、合理治疗可改善预后。     

合并过敏性鼻炎对儿童系统性红斑狼疮病情及治疗的影响

目的探讨过敏性鼻炎(AR)及其干预对儿童系统性红斑狼疮(JSLE)病情和治疗用药的影响。方法收集96例确诊的JSLE患儿临床资料,按是否合并过敏性鼻炎或其他过敏性疾病分为过敏性鼻炎组(44例)和非鼻炎过敏组(20例),以及非过敏组(32例),过敏性鼻炎组又随机分为鼻炎干预组和非干预组(各22例)。所有患儿均进行规范JSLE治疗。比较各组患儿的SLE疾病活动性指数(SLEDAI)和激素、免疫抑制剂用药情况。结果合并过敏性鼻炎组和非鼻炎过敏组的JSLE患儿治疗前的SLEDAI评分、糖皮质激素日累积剂量、免疫抑制剂用药种数均高于非过敏组(P0.05),而过敏性鼻炎组和非鼻炎过敏组间的差异无统计学意义(P0.05)。治疗1个月后,JSLE合并过敏性鼻炎患儿干预组的SLEDAI评分及激素日累积剂量低于非干预组(P0.05),两组患儿免疫抑制剂用药种数的差异无统计学意义(P0.05);治疗3个月及6个月后,干预组的SLEDAI评分、激素日累积剂量、免疫抑制剂使用种类均少于非干预组(P0.05)。结论 JSLE合并AR等过敏性疾病可能对SLE病情及治疗产生不利影响;针对AR给予干预有利于病情控制。     

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近20年来,有关儿童血液肿瘤性疾病诊断和治疗的研究进展较快,但仍有些血液病的治疗十分困难,如急性重型再生障碍性贫血(SAA)、骨髓增生异常综合征(MDS)、慢性免疫性血小板减少性紫癜(ITP),其研究进步稍慢。现将其近年来国内外进展综述如下。1急性重型再生障碍性贫血SAA是一种十分严重的非肿瘤性骨髓造血衰竭性血液病,在无治疗下患者常在1年内死于感染或出血。目前普遍认为SAA是一种由免疫异常介导的疾病。对于这样一种严重的疾病目前常用的治疗手段包括雄性激素、免疫抑制剂和异体造血干细胞移植,由于前者疗效缓慢,有效率低而常被作为…     

慢性难治性特发性血小板减少性紫癜的治疗进展

慢性难治性特发性血小板减少性紫癜(ITP)诊断条件如下:(1)曾经用标准剂量糖皮质激素(泼尼松1～2mg/kg·d)治疗无效,(2)血小板计数<30×109/L,(3)ITP病程在6个月以上,(4)无引起血小板减少的其它原因。近年来关于慢性难治性ITP的治疗已取得了一些进展,现综述如下:1静脉抗Rh(D)免疫球蛋白Sajid等[1]用静脉抗Rh(D)免疫琢蛋白治疗难治性和复发性ITP并与脾切除治疗进行比较,共23例病人,其中12例用静脉抗Rh(D)免疫球蛋白治疗,平均年龄8.9岁,男女之比为1∶1,11例作了脾切除,平均年龄13岁,男女之比为1∶1.2,治疗前平均血小板计数为9×109/L,…     

以血液系统损害为首发症状的儿童系统性红斑狼疮

目的探讨以血液系统改变为首发或主要表现的儿童SLE的临床特点、治疗方案及预后。方法对2005年6月-2011年6月收治以血液系统改变为主并最终确诊为SLE的38例患儿进行回顾性分析。其血液学改变按白细胞改变、贫血及血小板减少进行分析,并随访6～40个月。结果本组患儿血液系统改变中贫血28例(73.7%)、白细胞改变24例(63.2%)、血小板减少15例(39.5%),距确诊SLE的平均时间为8.5(0～24)个月。针对患儿不同症状选择免疫治疗,本组30例患儿SLE基本无活动,6例轻度活动,2例中度活动,无重度活动。15例PLT减少患儿中10例恢复正常,5例PLT维持在安全水平,8例已停药观察。16例自身免疫性溶血性贫血患儿中13例未再出现溶血发作,死亡1例;另2例患儿间断有轻度溶血发作。2例SLE相关再生障碍性贫血治疗显效。1例SLE相关纯红细胞再生障碍性贫血得到有效控制。结论儿童SLE很隐匿,初期常表现为血液系统损害,值得重视,对治疗效果欠佳患儿或青春期前后女童应警惕SLE,延长随访期限,评估病情以选择治疗方案和疗程。     

异基因造血干细胞移植治疗儿童范可尼贫血5例并文献复习

目的初步探讨异基因造血干细胞移植(HSCT)治疗范可尼贫血(FA)的疗效,为探索更加优化移植方案提供依据。方法回顾性分析2012年6月-2016年12月我院收治5例FA患儿进行HSCT的临床资料并复习相关文献。5例患儿中2例行非血缘相合HSCT治疗,3例行单倍体HSCT治疗。预处理方案以氟达拉滨(Flu)、低剂量环磷酰胺(CTX)、抗人胸腺/T淋巴细胞免疫球蛋白(ATG-G/F)为主干,根据移植前输血总量、是否合并白血病,在主干基础上±白消安(Bu)或±全身照射(TBI)。5例患儿回输CD34~+细胞中位计数为8.46(5.46~15.29)×10~6/kg,单个核细胞(MNC)中位计数为13.07(8.33~14.26)×10~8/kg。采用他克莫司和吗替麦考酚酯联合预防移植物抗宿主病(GVHD)。随访中位时间40.7(15~42)个月。结果 5例患儿HSCT预处理过程中,除1例合并严重消化道黏膜反应,其余耐受性尚可;中性粒细胞恢复中位时间10(8~13)d,血小板恢复中位时间16(12~61)d,无原发性植入失败发生;移植后3例发生移植物排斥,分别通过停用全部免疫抑制剂、回输供者干细胞后恢复为完全供者型;4例发生不同程度急性GVHD,3例需升级为二线免疫抑制治疗控制病情,2例发展为慢性GVHD;随访至2016年12月,2例无事件存活,2例存在慢性GVHD,目前病情控制理想,1例死亡。4例存活患儿移植后血细胞未再检测出FA相关基因突变,生长发育同正常同龄儿童,目前尚未发现合并实体肿瘤。结论对于缺乏同胞相合供者的FA患者,其他类型HSCT治疗采用"Flu+低剂量CTX+ATG±Bu或±TBI"预处理方案耐受性尚可,无原发性植入失败发生,但加用Bu或TBI对预处理相关毒性及远期预后的影响还需要深入研究;移植物排斥和GVHD仍是影响患儿生存主要因素,探索个体化、优化HSCT方案治疗FA的临床研究已成为必然。     

幼年型粒单核细胞白血病单倍体相合造血干细胞移植1例

目的探讨单倍体相合造血干细胞移植治疗幼年型粒单核细胞白血病(JMML)的可行性。方法 1岁6个月JMML患儿,行单倍体相合造血干细胞移植。采用Bu/Cy+Flu+ALG方案预处理及CSA+MMF+MTX方案预防移植物抗宿主病(GVHD)。结果于移植+10 d粒系植入成功(1.2×109/L),移植+14 d血小板植入成功(260×109/L),造血初步恢复。移植+21 d查植入证据为100%嵌合。患儿移植后反复出现Ⅰ～Ⅱ度GVHD(皮肤型),给予免疫抑制剂治疗后好转。至今已生存14个月,未见复发。结论单倍体相合造血干细胞移植可能是治愈JMML的可行方法。     

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??Objective??To summarize the efficacy and safety of arsenic trioxide combined with chemotherapy for children with recurrent/refractory neuroblastoma. Methods??Retrospective analysis was made in seven cases of neuroblastoma treated with traditional chemotherapy combined with venous arsenic trioxide after diagnosis with poor prognosis or the condition deteriorated. Results??Among the seven patients treated with chemotherapy combined with venous arsenic trioxide for 3 to 7 courses??4 were effective and 3 were ineffective. Patient 3 was treated with arsenic trioxide combined with following regimen after tumor was not resected totally??and patient 5??6 and 7 were given arsenic trioxide combined with preoperative chemotherapy. Patient 3 and patient 5 achieved complete remission after combination chemotherapy??and patient 6 and patient 7 showed decreased tumor volume and tumor marker. Patient 1??2 and 4 were given arsenic trioxide after failure of conventional chemotherapy. Metastasis in patient 1 disappeared at first??but the disease relapsed after 11 months. The tumor responded poorly and increased in size and number in patient 2 and patient 4. Patient 1 the use of arsenic trioxide was stopped in patient 1 because of prolonged QT interval??0.51s????who returned normal after symptomatic treatment. Five patients are still alive and they have lived for 3.5 y??3.0 y??1.2 y??1.0 y and 0.7 y??respectively. Conclusion??Venous arsenic trioxide combined with chemotherapy can be a choice of treatment for recurrent/refractory neuroblastoma??whose effect and safety requires further investigation.     

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??Objective??To study the current status of common blood purification therapy for childhood-onset severe systemic lupus erythematosus??SLE?? in China. Methods??The questionnaire was discussed and set by Pediatric Blood Purification Specialist Committee??the data of 127 hospitalized children with blood purification therapy in 22 units were collected and analyzed from January 1??2012 to December 31??2017. Results????1??Among the 127 cases??including 28 males and 99 females??between 4 to 16 years old. 63 cases were treated by plasma exchange??PE?? for 180 times??41 cases were treated by DNA immunoadsorption??DNA-IAS?? for 106 times??11 cases were treated by hemodialysis??HD?? for 112 times??12 cases were treated by hemoperfusion??HP?? for 32 times. ??2??PE and DNA-IAS can alleviate the clinical symptoms effectively??causing the systemic lupus erythematosus disease activity index??SLEDAI????ANA titer??antidouble-stranded DNA antibodies??immunoglobulin to decrease and complement to increase significantly. HD for children with renal insufficiency was effective??HP can eliminate inflammatory factors and relieve clinical symptoms of children. The remission rates of PE??DNA-IAS??HD and HP were 87.30%??87.80%??72.73% and 75.00% respectively. Conclusion??PE and DNA-IAS therapy can remove the immune substances in the blood of children with severe SLE quickly and relieve the disease??HD therapy is mainly used for children with severe edema and renal insufficiency??HP therapy can eliminate inflammatory factors effectively and improve clinical symptoms. For children with severe SLE??different blood purification mode should be selected according to the difference of the children’s condition.     

Immune thrombocytopenia and hemolytic anemia as a presenting manifestation of Hodgkin disease

A very unusual clinical presentation of Hodgkin disease with immune thrombocytopenia and autoimmune hemolytic anemia is reported. A 6.5-year-old boy presented with thrombocytopenia, Coombs' positive hemolytic anemia, and multiple small posterior cervical lymph nodes. After a course of high-dose methylprednisolone therapy with a diagnosis of Evans syndrome, complete response for thrombocytopenia and partial response for anemia was achieved. Six weeks later there was a sudden increase in the size of left posterior cervical lymph nodes and a biopsy was compatible with Hodgkin disease, mixed cellularity type. The child was successfully treated with chemotherapy and radiation therapy. He has been off therapy for 28 months and has no clinical or laboratory evidence of autoimmune cytopenia. A combination of immune thrombocytopenia and autoimmune hemolytic anemia may be associated with Hodgkin disease. The recognition of this clinical picture as a complication of Hodgkin disease has important implications. This complication appeares to be managed best by the definitive treatment of Hodgkin disease.     

IMMUNE THROMBOCYTOPENIA AND HEMOLYTIC ANEMIA AS A PRESENTING MANIFESTATION OF HODGKIN DISEASE

A very unusual clinical presentation of Hodgkin disease with immune thrombocytopenia and autoimmune hemolytic anemia is reported. A 6.5-year-old boy presented with thrombocytopenia, Coombs' positive hemolytic anemia, and multiple small posterior cervical lymph nodes. After a course of high-dose methylprednisolone therapy with a diagnosis of Evans syndrome, complete response for thrombocytopenia and partial response for anemia was achieved. Six weeks later there was a sudden increase in the size of left posterior cervical lymph nodes and a biopsy was compatible with Hodgkin disease, mixed cellularity type. The child was successfully treated with chemotherapy and radiation therapy. He has been off therapy for 28 months and has no clinical or laboratory evidence of autoimmune cytopenia. A combination of immune thrombocytopenia and autoimmune hemolytic anemia may be associated with Hodgkin disease. The recognition of this clinical picture as a complication of Hodgkin disease has important implications. This complication appeares to be managed best by the definitive treatment of Hodgkin disease.     

Successful treatment of refractory immune hemolysis following unrelated cord blood transplant with Campath-1H

Immune-mediated hemolytic anemia is a well-recognized complication of hematopoietic stem cell transplantation. We report on a 6-year-old boy with X-linked adrenoleukodystrophy who developed severe delayed alloimmune hemolytic anemia associated with immune-mediated neutropenia and thrombocytopenia following major ABO incompatible unrelated cord blood transplantation. The patient's cytopenias were refractory to treatment with corticosteroids, cyclosporine, intravenous immune globulin, rituximab, and pentostatin. After one course of Campath-1H his hematologic parameters normalized, suggesting that the compound may be an effective therapy for complex immunohematologic disorders complicating hematopoietic stem cell transplantation. The case also emphasizes the importance of T-cells in transplant associated immune cytopenias.     

Necrotizing polyarteritis nodosa-like vasculitis in a child with systemic lupus erythematosus

A 10-year-old child presented with prolonged fever, lymphadenopathy, weight loss, oral ulcers, alopecia and parotitis. She later developed arterial thrombosis, poly-serositis, nephritis, myocarditis, sacro-ilitis, autoimmune hemolytic anemia and refractory thrombocytopenia. Though anti-dsDNA was negative, she was diagnosed to have systemic lupus erythematosus (SLE). Terminally, she had pulmonary symptoms and succumbed to her illness. The autopsy showed lupus nephritis-Class II, polyserositis, myocarditis, inflammatory myositis, immune mediated vasculitis involving renal, coronary, pancreatic, adrenal, dermal and intramuscular arteries, and pulmonary hemorrhages and edema.     

Evans syndrome in childhood: pathophysiology, clinical course, and treatment

Evans syndrome is defined as the simultaneous or sequential occurrence of Coombs' positive hemolytic anemia and immune thrombocytopenia without known underlying etiology. Ten cases of Evans syndrome were seen at our hospital over the past decade; three patients died. Two cases are described in detail and demonstrate the chronic, refractory nature of this condition. Most patients have required corticosteroid therapy and splenectomy, but further therapy (e.g., intravenous gammaglobulin, danazol, cyclophosphamide) is usually necessary because of recurrent autoimmune hemolytic anemia, thrombocytopenia, or both. There is a substantial risk for development of other autoimmune problems and hypogammaglobulinemia. A number of defects in humoral immunity have been described in Evans syndrome; different antibodies are directed against platelets and red blood cells. Cellular immunity is probably abnormal, but a distinct pattern of immunoregulatory disturbance has not been identified.     

Intravenous gamma globulin for thrombocytopenia in children with Evans syndrome

We describe three patients with Evans syndrome (immune hemolytic anemia and immune thrombocytopenia) who were refractory to conventional therapy, including steroids and splenectomy in all of the patients, vincristine in two, and cyclophosphamide in one. The patients were then treated with modified intravenous gamma globulin 0.4 g/kg/day for 5 consecutive days. Two patients failed to respond, but the third had a clinical remission after gamma globulin therapy. Given the usual chronic and relapsing course of Evans syndrome and the poor response to conventional therapy, we recommend that high dose i.v. gamma globulin be considered an alternative therapy in the management of these patients.     

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??Objective??To investigate the changes and clinical significance of serum ferritin??SF??levels in children with systemic lupus erythematosus??SLE??. Methods??The serum ferritin levels of 58 children with SLE were measured by Chemiluminescence before and after treatment??and serum ferritin levels were measured in 30 normal children. Results??The serum ferritin levels were significantly higher in children with SLE than those in healthy controls as well as in active children than in inactive children with SLE.And the serum ferritin leveis were significantly decreased after treatment??Moreover?? the serum levels of ferritin were positively correlated with anti-ds-DNA antibody levels and SLEDAI??SLE Disease Activity Index??in children with SLE. Conclusion??The serum ferritin levels in children with SLE are significantly higher than those in normal children. Serum ferritin levels aree positively correlated with disease activity in children with SLE.Therefore serum ferritin levers can be a useful laboratory marker to diagnose SLE in children.