上海儿童医学中心WT-99方案诊治儿童肾母细胞瘤临床报告

目的：改善儿童肾母细胞瘤预后。方法：对1998年10月-2001年10月住院明确诊断为肾母细胞瘤及肾肉瘤的20例病人采用外科手术、内科化疗、选择性放疗,病理科、影像学科协作诊断综合治疗（即上海儿童医学中心WT-99方案）。按方案中条件根据分期及其他危险因素进行分组,并按分组给予不同药物组合和强度的化疗。Ⅰ期及Ⅱ期病理分型预后良好型的不放疗,估计手术不能完全切除时给予2个疗程术前化疗。结果：全组20例,年龄7个月至12岁。病理分类预后良好型14例,预后不良型3例;透明细胞肉瘤2例,横纹肌肉瘤样1例。临床结合病理分期为Ⅰ期5例,Ⅱ期5例,Ⅲ期6例,Ⅳ期3例,Ⅴ期1例。全组20例中获完全缓解18例（90％）,2例初治失败,缓解后复发1例。无病生存时间平均27个月17例（11-45个月）,占85％,目前均已停药。结论：所采用多专业联合诊断治疗工作模式及上海儿童医学中心WT-99诊治方案对儿童肾母细胞瘤有效。     

小儿肿瘤协会儿童肾母细胞瘤WT-2003协作方案初步报告

目的 探讨建立儿童肿瘤协作组可能性，改善我国儿童肾母细胞瘤预后。方法 在中国小儿肿瘤协会下建立儿童肿瘤协作组，制定儿童肾母细胞瘤WT-2003协作方案，方案包括诊断与分期标准，分组标准、各组包括外科手术、内科化疗、选择性放疗的治疗规则。协作组WT-2003方案先在上海儿童医学中心试行20例，证明安全性和有效性后2003年起在协作组内应用。根据患儿分组术后接受18-24周不同强度化疗。方案 规定Ⅰ期及Ⅱ期病理分型为预后良好型不放疗，其余病例治疗均包含有手术、不同强度的放疗和化疗，估计手术不能完全切除时给予2个疗程术前化疗。结果 15家医院参加协作组，7家医院上报汇总资料，累计病例52例。最大年龄12岁，最小6个月，中位29个月。病理分型为预后良好型（FH）35例，预后不良型（UFH）8例，透明细胞肉瘤（CS）5例，肾未分化肉瘤（US）2例，横纹肌样肉瘤（RS）1例，典型肾母细胞瘤特征影像学诊断1例（拒绝手术）。分期：Ⅰ期17例，Ⅱ期11例，Ⅲ期17例，Ⅳ期6例，Ⅴ期1例。48例获完全缓解，占92．3％。目前无肿瘤生存45例，占本组病例的86．5％，时间为3～72个月，中位13个月。结论 中国儿童肿瘤多中心协作临床研究有可行性，通过这一方式可较快积累病例，使治疗更合理，提高我国儿童肿瘤整体诊治水平，本组患儿所采用诊断治疗方案有效，安全性好。     

单中心儿童肾肿瘤SCMC WT-99方案临床报告

目的改善儿童肾母细胞瘤预后。方法对1998年10月至2007年12月住院明确诊断为肾母细胞瘤及肾肉瘤患儿,采用外科手术、内科化疗、选择性放疗,病理科、影像学科协作诊断综合治疗的上海儿中心WT-99方案。按方案中条件根据分期及其他危险因素进行分组,并按分组给予不同药物组合和强度的化疗。Ⅰ期及Ⅱ期病理分型预后良好型不放疗。估计手术不能完全切除时给予2个疗程术前化疗。结果全组54例,年龄3个月至13岁。病理分类预后良好型39例,预后不良型4例,透明细胞肉瘤5例,横纹肌肉瘤样1例,分型困难或未分化型5例。临床结合病理分期为Ⅰ期19例,Ⅱ期7例,Ⅲ期19例,Ⅳ7例,Ⅴ期2例。54例中获完全缓解51例(94.4%),3例初治失败。缓解后复发9例。随访1～104个月,中位31.5个月,估计5年无病生存率(EFS)和总生存率(SR)分别为75.7%和83.9%。结论多专业联合诊断治疗工作模式及儿中心WT-99方案对儿童肾母细胞瘤有效。     

儿童肾母细胞瘤国际及国内诊治方案解读

肾母细胞瘤(Wilms tumor,WT)是儿童最常见的肾恶性肿瘤。COG(Children's Oncology Group,儿童肿瘤协作组)和SIOP(International Society of Paediatric Oncology,国际儿童肿瘤学会)两大儿童肿瘤研究的国际协作组织分别形成了各自的诊疗方案:COG推荐手术优先,然后行化疗与放射治疗等辅助治疗; SIOP强调术前化疗,然后行手术与放射治疗。目前该两大协作组发布的肾母细胞瘤患者总体生存率均达到90%。中国抗癌协会小儿肿瘤专业委员会及中华医学会小儿外科学分会泌尿外科学组在COG和SIOP方案的基础上也先后发表了诊疗建议和共识。本文重点介绍COG方案和SIOP方案的异同,主要包括组织学分类、临床分期、危险度分组、治疗方案以及复发后治疗等方面;进而解读中国儿童肾母细胞瘤诊疗共识,分析中国的诊疗现状,供广大临床医师参考和借鉴。     

化疗对于肾母细胞瘤肺转移灶的疗效观察

目的 观察肾母细胞瘤(WT)肺转移灶的转归,探讨不同化疗方案的疗效。方法 回顾性分析2012年至2020年就诊并规律随访的WT肺转移患儿的临床资料,分析肿瘤肺转移灶对不同化疗的应答差异。结果 20例WT肺转移患儿,男11例、女9例,中位年龄38.0(20.0～5.8)月;其中预后良好型17例,预后不良型3例;单肺转移13例,双肺转移7例;初诊时肿瘤分期Ⅰ期1例,Ⅱ期5例,Ⅲ期3例,Ⅳ期9例,2例患儿外院首诊分期不明;12例术前发现肺转移,8例术后发现;合并其他远处转移者8例;化疗后转移应答为8例完全缓解,4例部分缓解,4例稳定,4例进展。采用有序logistic回归分析进行多因素分析,结果显示化疗方案CCCG-WT-2016的偏回归系数为-2.05,并呈现出0.05检验水准的显著性(Z=-2.03,P=0.043),采用CCCG-WT-2016方案化疗患儿的肺转移灶转移应答优于NWTS-5方案。结论 采用WT-2016方案化疗的患儿转移应答明显优于采用NWTS-5方案化疗的患儿。     

儿童肾母细胞瘤基因突变的研究进展

肾母细胞瘤是是小儿泌尿系统最常见的恶性肿瘤.该文综述了肾母细胞瘤发生及预后相关基因方面的研究进展,其中WT1基因、CTNNB1基因及WTX基因与该肿瘤的发生相关,MYCN基因、P53基因与SIX1基因可能与预后相关.     

小儿双侧肾母细胞瘤的治疗

小儿双侧肾母细胞瘤的治疗中山医科大学附属第一医院小儿外科研究室（５１００８０）刘唐彬，赖炳耀，谢家伦，刘文旭，李桂生中山医科大学病理教研组周慕珩我院１９６０年１月～１９９１年６月共收治小儿肾母细胞瘤１４８例，全部经手术及组织学诊断证实，其中双侧患者６...     

62例儿童肾母细胞瘤的组织学分类

本文介绍作者于1985年对肾母细胞瘤提出的4类8型及第3次NWTS对肾母细胞瘤的组织分类法。62例有随访结果的肾母细胞瘤,按1985年提出的标准,高分化型存活率为58.6％,低分化型为21.2％。近10年有随访资料的肾母细胞瘤40例,按NWTS的标准,良好组织类型的生存率为60.6％,不良组织类型的生存率仅14.3％。指出NWTS的分类法与怍者过去提出的分类法有类似之处,且更易为临床各科医师掌握及应用。     

儿童复发肾母细胞瘤的治疗及预后相关因素分析

目的 探讨儿童复发肾母细胞瘤的治疗方案及影响预后的相关因素.方法 回顾性分析2006年4月至2019年12月间上海交通大学医学院普外科/肿瘤外科收治的23例复发肾母细胞瘤患者资料,从肿瘤分期、病理分型、术中情况、术后放疗情况、术后复发时间及复发部位、复发后治疗以及预后等方面进行统计分析.结果 Ⅱ期及Ⅲ期病例在复发时间(...     

HIT 2000 方案治疗3 岁以下儿童髓母细胞瘤疗效分析

目的评价分析3岁儿童髓母细胞瘤HIT 2000 方案的治疗效果及预后。方法回顾分析采用HIT 2000 方案治疗的3岁髓母细胞瘤患儿的临床资料。结果共纳入38 例3岁髓母细胞瘤患儿,完全缓解8 例、部分缓解5 例、疾病稳定6 例、疾病进展或复发19 例。5 年无事件生存率、总体生存率分别为(43.9±8.8)%、(49.5±9.1)%;M0组和M+组,DMB/MBEN组和CMB、LC/A 组,SHH组和G4组之间3 年或5 年无事件生存率和总体生存率差异均有统计学意义(P 均 0.05);而R0组和R+ 组、放疗组和未放疗组之间的5 年无事件生存率和总体生存率差异均无统计学意义(P 均0.05)。13 例患儿行放疗,放疗期间均未出现放射性脑坏死及继发第二肿瘤,1 例出现放射性肺损伤。所有患儿均化疗,化疗期间均出现不同程度的骨髓抑制,肝功能损害3 例,口腔黏膜溃疡5 例,经对症处理后预后良好。没有患儿因放、化疗而死亡。结论 HIT2000 方案治疗3 岁以下儿童MB疗效较好,疾病预后与M分期、病理分型及分子分型有关,化疗耐受可,可作为3 岁以下儿童MB治疗方案之一。     

A single-institution Wilms' tumor and localized neuroblastoma series

Since January 1980, 120 children affected by Wilms' tumor have been treated at Bambino Gesù, mostly with multimodality treatment according to Société Internationale d'Oncologie Pediatrique (SIOP) protocols, including chemotherapy, surgery and radiotherapy in selected cases. This treatment approach emphasizes the role of preoperative (neoadjuvant) chemotherapy as opposed to the approach favored by the National Wilms' Tumor Study, which is focused on optimizing postoperative chemotherapy after primary surgery. Thus, using SIOP guidelines, staging occurs at the time of surgery, after chemotherapy administration. These differences will constitute the baseline for a comparison between the two experiences. Bilaterality, nephroblastomatosis, partial nephrectomy in unilateral Wilms' tumor and thrombosis of the vena cava are the main topics discussed. For the present study, the analysis was restricted to 98 consecutive cases diagnosed until December 1999, for whom at least 24 mo of follow-up is available. The more recent experience of treating resectable neuroblastoma in cooperative studies dates back to 1979, when the first Italian Cooperative Group Neuroblastoma protocol was introduced. This experience was continued within the frame of the first Localized Neuroblastoma European Study Group protocol (LNESG 94), and will be compared to North American Cooperative Group approaches and outcomes. Preoperative evaluation of surgical risk factors, intraoperative complications and their management, and long-term outcome will be discussed.     

Needle biopsy and preoperative chemotherapy for massive unilateral and bilateral Wilms' tumor

Massive unilateral and bilateral Wilms' tumors present complex therapeutic questions. We report five cases in which we used radiological investigations, needle biopsy, and preoperative chemotherapy followed later by definitive surgery.     

Epidural metastasis in chemoresistant Wilms' tumor with perilobar nephroblastomatosis

An 8-year-old boy with vertebral and epidural metastases was diagnosed with Wilms' tumor associated with perilobar nephroblastomatosis (NB) based on histologic examination. During combined chemotherapy with vincristine, actinomycin D, doxorubicin, and cyclophosphamide (NWTS-3 J protocol), a rapid increase in tumor size was observed. The treatment was replaced with etoposide and carboplatin (JET regimen). A transient response was sustained for 5 months during this chemotherapy. However, regrowth of the tumor was observed and the patient died 11 months after the initial chemotherapy.     

Management of Wilms' tumor and neuroblastoma

Wilms' tumor and neuroblastoma resemble each other in usual presentation and by their occurrence in young children. However they represent a dichotomy in advances in treatment. In Wilms' tumor, survival of patients has dramatically improved as a result, of multimodal therapy, whereas in neuroblastoma little advance in treatment has occurred. Development of rational chemotherapeutic regimens through better understanding of drugs and of cell cycle kinetics as well as investigation of other modalities of therapy, i.e. immunotherapy, etc., may be helpful in improving survival in the years to come.     

Wilms' tumor and neuroblastoma

Significant differences exist between the European and North American treatment protocols for Wilms' tumor and neuroblastoma. There are variations in biopsy technique, timing and extent of initial surgery, chemotherapy protocols and dosage routines, as well as the type of salvage therapy. With the consolidation of the two major North American study groups into a single entity (Children's Oncology Group), the European and North American study groups represent the only remaining large-scale venues for treatment comparison. It is important to study and understand the variation in treatment protocols in order to maintain an open forum of scientific investigation that will lead to improving the care and outcome of children with cancer. It is anticipated that the unification of the North American groups will lead to greater interest and scientific cooperation with the European study group. This paper will serve as a forum for such a discussion at a local level.     

畸胎瘤样肾母细胞瘤治疗体会及文献回顾

目的 探讨儿童畸胎瘤样肾母细胞瘤临床诊断和治疗方法,提高临床对该病的认识.方法 回顾我院2007年至2016年间诊治的4例儿童畸胎瘤样肾母细胞瘤,男2例,女2例,年龄2～40个月,平均年龄21个月;原发灶:右肾3例,左肾1例;根据COG分期为Ⅰ、Ⅱ、Ⅲ、Ⅳ期(肺及骨髓)各1例,分析其临床表现、诊断及治疗方法.结果 4例患儿均行肾脏肿瘤根治性切除术,其中Ⅲ、Ⅳ期患儿术前行化疗再行根治术;3例术后辅以化疗,术后复发1例(化疗不敏感);3例早中期患儿预后可(2例随访时间超9年,1例随访3年),1例Ⅳ患儿再次手术后化疗加放疗中.结论 儿童畸胎瘤样肾母细胞瘤是肾母细胞瘤的罕见病理类型,临床症状通常难与经典肾母细胞瘤鉴别,主要依靠术前影像学及病理诊断,患侧肾脏肿瘤根治性切除术为主要治疗手段,早中期TWT患儿预后良好,至于保肾肿瘤切除术在该类型肾母细胞瘤的应用,暂未见文献报道,本组病例数较少,也未涉及.对于晚期TWT患儿,可能因为其独特的病理特征,其预后可能比经典肾母细胞瘤更差.     

CCG7942/POG9354方案治疗儿童外周型原始神经外胚层肿瘤的临床研究

目的 研究儿童外周型原始神经外胚层肿瘤(pPNET)的临床表现、诊断、治疗及生存情况.方法 回顾性分析2008 年10 月至2013 年10 月收治的10 例pPNET 患儿完整的临床资料,其中3 例存在转移,7 例无转移.7 例无转移患儿采用美国儿童癌症协作组CCG7942 治疗方案,3 例有转移患儿采用美国儿童肿瘤协作组POG9354 治疗方案.治疗后采用WHO 实体瘤疗效评定标准和通用不良反应术语标准对相关毒副反应进行评价.结果 采用CCG7942 方案治疗的7 例患儿中,完全缓解4 例,稳定1 例,进展2 例,复发2 例;采用POG9354 方案治疗的3 例患儿中,完全缓解1 例,进展2 例,复发2 例,死亡2 例.目前7 例无转移患儿的生存期为5~60 个月,无事件生存率为71%;3 例有转移患儿的生存期为13~25 个月,无事件生存率为33%.所有患儿除均有4 级骨髓抑制的毒性损害外,其他毒性反应如消化道、肝肾功能多为1~2 级损害.结论 CCG7942方案对治疗没有转移的pPNET 患儿有效、安全;存在转移的pPNET 患儿即使采用POG9354 方案疗效仍然欠佳,需要研究新的治疗方案以提高疗效.     

Treatment and prognosis of 32 patients with bilateral Wilms' tumor

During the 29-year period 1955–1983, 32 children with bilateral Wilms' tumor were treated at the Hospital for Sick Children, Toronto (HSC) and the Royal Children's Hospital, Melbourne (RCH). Those whose tumors presented synchronously (n=22) had a good prognosis: 15 (68%) are alive and disease-free 12 to 204 months post-diagnosis with a median follow-up of 96 months. There were no deaths due to metastases. The asynchronous group (n=10) had a poor prognosis: only 3 are disease-free survivors, 2 long-term and 1 who has survived 17 months following diagnosis of the contralateral tumor. Five of the 7 deaths were due to metastases, possibly related to the immunosuppressive effect of the additional chemo/radiotherapy given for the separate treatment of both tumors. The poor prognosis could not be attributed to extent of disease or unfavourable histology as only 2 were stage IV at diagnosis of the second tumor and none had anaplastic histology. We recommend renal ultrasound and abdominal computerized axial tomography for an accurate diagnosis of synchronous Wilms' tumors, particularly in children at high risk for bilaterality, and for prolonged follow-up to detect asynchronous disease. Offprint requests to: R. M. Filler at the above address in Canada     

Wilms' tumor metastatic to the mediastinum

We report two children with mediastinal metastasis of Wilms' tumor. One case presented with a right hilar mass and the other with a posterior mediastinal mass. In each instance, no additional radiologic evidence of metastasis was found. Metastasis of Wilms' tumor to the mediastinum is uncommon, usually occurring with radiologic evidence of either additional metastases or direct cephalad growth of the primary tumor.Presented at the Members Miscellany, 23rd Annual Meeting, The Society for Pediatric Radiology, 18–20 April 1980, Salt Lake City, Utah, USA     

肾母细胞瘤患儿p73基因的表达及其甲基化研究

目的：探讨肾母细胞瘤患儿血液中p73基因的转录表达、启动子甲基化状态及二者之间关系。方法：收集45例肾母细胞瘤患儿为病例组,以健康体检或因其他原因就诊的性别、年龄匹配的15例（排除肿瘤等恶性疾病）儿童为对照组。采集两组儿童外周血,运用实时荧光定量PCR（qRT-PCR）和甲基化特异性PCR (MSP)法检测p73基因转录表达水平及其启动子甲基化状态,并分析病例组中p73基因的表达及甲基化与临床资料的关系,及p73基因的甲基化对其转录表达的影响。结果：病例组中p73 mRNA的相对表达量（3.2±0.9）高于对照组（1.6±1.1）,差异有统计学意义（P<0.01）;病例组p73基因甲基化阳性率（20%）低于对照组 (73%),差异有统计学意义（P<0.01）。病例组中甲基化p73 mRNA的相对表达量大于非甲基化p73 mRNA的相对表达量（P<0.01）,且大于对照组中甲基化p73 mRNA的相对表达量（P<0.01）;非甲基化p73 mRNA的相对表达量在两组间差异无统计学意义（P=0.810）。结论：肾母细胞瘤外周血中p73基因启动子的异常甲基化是其基因表达调节方式之一,并与肾母细胞瘤的发生发展有关;发生甲基化的p73基因在肾母细胞瘤中有可能充当癌基因的角色,转录水平的过度表达与其甲基化状态有关。