Neuroblastome surrénalien bilatéral et syndrome de Pepper : à propos de quatre observations

4S neuroblastoma with bilateral adrenal involvement is defined by small primitive tumors (stage 1 or 2) with disseminated disease restricted to the liver, skin, and/or bone marrow. Children are less than one year old. These tumors are rare and of multicentric origin. PATIENTS AND METHODS: Our multicentric study analyzed four children less than four months old at diagnosis. RESULTS: All had a favourable histology, with normal MYC-N copy number, and one case had a diploid tumor. The four patients had first supportive care at the beginning, but three cases received chemotherapy because of progressive disease, with liver radiotherapy in two cases because of massive hepatomegaly; three cases had surgery (unilateral adrenal resection in two cases and bilateral in one case) and one had only a biopsy. Surgery was the only treatment in one case. One patient relapsed 17 months after initial treatment and was treated with intensive chemotherapy and stem cell rescue. The outcome is favorable for the four patients, without evidence of recurrent disease. CONCLUSION: Children with 4S neuroblastoma with bilateral adrenal tumors have a good prognosis. Treatment should be the less aggressive as possible. The group with favorable prognostic parameters should have supportive care if spontaneous regression occurs. But we have to treat with chemotherapy neonates with massive hepatomegaly and children with one or more unfavorable prognostic factors (unfavorable histology, high MYC-N copy number).     

Success of chemotherapy and a liver transplant in a pediatric patient with hepatic angiosarcoma: A case report

Hepatic angiosarcoma is an extremely rare diagnosis in children, with fewer than 50 pediatric cases reported in the literature worldwide. This aggressive vascular sarcoma carries a very dismal prognosis and is known to be resistant to radiation, chemotherapy, and other vascular‐targeted agents. Complete surgical resection is felt to provide the best chance for long‐term survival. In patients with tumors not amenable to resection, a liver transplant can be considered. However, very few such transplants have been reported, given that they remain controversial due to high cancer recurrence and mortality post‐transplant. Herein, we report the unique case of a 2‐year‐old child with localized hepatic angiosarcoma not amendable to resection who successfully underwent a liver transplant and received chemotherapy with six cycles of doxorubicin, docetaxel, and ifosfamide.     

小儿炎性肌纤维母细胞瘤诊断与治疗的探讨

目的探讨小儿炎性肌纤维母细胞瘤(IMT)的诊断与治疗方法。方法分析和总结我院收治的5例手术病理确诊为IMT患儿的临床表现、实验室检查、影像学检查、病理学诊断、外科治疗方法及随访情况等。结果 5例IMT患儿中,2例肿瘤位于膀胱,3例肿瘤分别位于颈部、腹部及右下肺,行肿瘤完整切除术,其中膀胱及右下肺肿瘤患儿术后随访2～8个月未见肿瘤复发,腹部肿瘤患儿未能随访;1例膀胱肿瘤活检,明确诊断后家长放弃治疗;1例颈部肿瘤复发,初期经化疗肿瘤消失,后出现颈部肿瘤转移及复发,再次手术及化疗,后期无法有效控制肿瘤增长,最终家长放弃治疗。结论小儿IMT是一种罕见的软组织肿瘤,无特异性表现,确诊依靠病理学检查。手术切除是有效的治疗方法,但存在复发和转移可能,术后需长期随访,对于复发者化疗有一定疗效,反复复发者预后不良。     

The role of neoadjuvant chemotherapy in children with malignant solid tumors

Pediatric surgeons play a critical role in diagnosing, staging, and treating malignant solid tumors in children. Over the years, the surgical management of the primary tumor site has evolved from an aggressive en-bloc resection at diagnosis to a more tailored surgical approach, often affecting definitive local control after the delivery of neoadjuvant therapy, as currently directed by many solid tumor protocols. In fact, inappropriate upfront resection can lead to unnecessary short- and long-term morbidity, an incomplete resection, and may be associated with a delay in the initiation of the systemic chemotherapy that is critical to the treatment of gross or occult metastatic disease. Therefore, it is important for the pediatric surgeon, as a member of the multidisciplinary team involved in the care of these children, to understand the indications for and implications of neoadjuvant therapy in the treatment of pediatric solid tumors. Here we review the current management of childhood solid tumors focusing on the role of neoadjuvant therapy.     

儿童颅骨肿物的诊断和治疗

目的 探讨儿童颅骨肿物的常见病理类型、临床特点和治疗方法。方法 回顾性分析25例手术治疗的儿童颅骨肿物病例，对其临床症状、影像学资料、治疗方法以及生存情况等进行分析研究。结果 全切除手术15例，近全切除手术6例，部分切除手术3例，1例行活检手术。随访20例患儿，85％的患儿手术后基本恢复了正常的生活，2例生活需要照顾，随访期间死亡的患儿1例。结论 儿童颅骨肿物的诊断和治疗相对有别于成人。积极的手术治疗可以达到明确诊断，延缓神经功能缺损的发展，延长患儿的生存期以及美容等目的。     

Supratentorial primitive neuroectodermal tumor: a single center experience and comparison with the literature

Supratentorial primitive neuroectodermal tumors (stPNETs) are malignant tumors. We saw within three years six children with stPNETs. In four of the six children radical resection could be achieved. All had craniospinal irradiation and chemotherapy according to the HIT-91 protocol. The two children with incomplete resection died due to tumor progression after 7 and 10 months. Two of the 4 children with complete tumor resection had local relapses 8 months after diagnosis and died after 14 and 18 months. One child had a diffuse meningeal relapse 12 months after diagnosis. Despite (high-dose) systemic chemotherapy and intraventricular mafosfamide, he died 21 months after diagnosis due to tumor although remission could be achieved. Only one child is still in remission 86 months after diagnosis.     

Atypical teratoid/rhabdoid tumors of the central nervous system: clinical,radiographic and pathologic features

INTRODUCTION: Atypical teratoid/rhabdoid tumors (ATT/RT) of the central nervous system (CNS) are uncommon malignancies of childhood with an aggressive course and a uniformly fatal outcome. METHODS: The medical records, radiographic images and pathologic files at the Rainbow Babies and Childrens Hospital over the previous 6 years were retrospectively reviewed. RESULTS: Eight children underwent surgery for CNS ATT/RT at our institution since 1996. There were 6 boys and 2 girls. Median age at presentation was 21 months. Four tumors had multifocal disease at the time of diagnosis. Six patients received multiagent chemotherapy including 3 patients with autologous bone marrow transplantation, and 6 patients received radiation therapy. Median survival was 9 months from the time of diagnosis. CONCLUSIONS: In spite of aggressive therapy, the prognosis for ATT/RT remains dismal. The search for effective treatment strategies will require a better understanding of the biology and molecular genetics of this tumor.     

经导管动脉化疗栓塞在肝母细胞瘤综合治疗中的应用评价及文献回顾

目的：探讨经导管动脉化疗栓塞术（TACE ）在肝母细胞瘤综合治疗中的作用及疗效。方法回顾性总结2005年-2013年收治的10例经TACE 治疗的肝母细胞瘤患儿的临床资料。随访治疗后患儿的全身情况、血AFP值及瘤体体积大小的变化及生存情况。结果患儿10例,男女比例4∶1,发病中位年龄9.5个月（1个月～10岁）,10例患儿首诊后均予以1～4次TACE 治疗,其中6例TACE＋手术＋化疗,1例TACE＋化疗,3例在TACE 治疗后放弃继续治疗。介入治疗后肿瘤体积较前均有明显缩减（26.2％～10.00％,平均70.0％）,血清AFP值下降明显（28.5％～99.7％,平均83.6％）。所有患儿在接受TACE 治疗后均出现不同程度的发热、呕吐、一过性肝功能损害（Ⅰ度3例,Ⅱ度2例,Ⅲ度1例）、轻度骨髓抑制、贫血等不适,未见明显心脏毒性及肾毒性损害。随访时间2～114个月,平均随访时间为36.1个月,1年存活率100％（7／7）,2年存活率86％（6／7）,3年存活率71％（5／7）。6例患儿在接受TACE治疗后成功行手术切除,术后均接受全身化疗,1例仅经过4次TACE治疗及化疗后肿瘤消失,未行手术治疗,均无瘤存活至今。3例放弃治疗患儿分别于2～8个月后死亡。结论 TACE治疗可作为肝母细胞瘤术前重要的辅助治疗方式,能够使肿瘤体积明显缩小,血供减少,促进肿瘤包膜增厚,为尽可能完整切除创造了条件,能够改善肝母细胞瘤患儿的生活质量,提高长期存活率。     

儿童侵袭性纤维瘤的临床特征及预后分析（附27例报告）

目的　分析儿童侵袭性纤维瘤的临床特征及预后情况,以提高临床认识及诊疗水平。方法　回顾性分析2007年1月至2018年1月首都医科大学附属北京儿童医院收治的27例原发性侵袭性纤维瘤患儿临床资料,并进行随访。结果　27例患儿中,男16例、女11例,中位发病年龄65（35,96）个月,原发肿瘤部位分型中,腹壁型3例、腹内型5例、腹外型19例。术前影像学检查提示原发肿瘤最大径中位数为9.8（5.3,12.1） cm。所有患儿初诊时均接受手术治疗,10例接受化疗（甲氨喋呤+长春碱类）。27例患儿中,无病生存9例,带瘤生存15例,死亡3例。5年总体存活率85.8%,5年无事件存活率32.6%。生存分析结果显示,手术切缘阳性与肿瘤复发相关。结论　儿童侵袭性纤维瘤是一种罕见的中间性肿瘤,手术切除效果不佳者极易复发,化疗对控制肿瘤的增长有一定作用。多数患儿预后良好,但部分患儿可因肿瘤持续增长而致残甚至死亡。     

Opsoclonus-myoclonus syndrome associated with non-metastatic neuroblastoma. Long-term survival. Study of the French Society of Pediatric Oncologists]

Opsoclonus-myoclonus is a rare syndrome characterized by multidirectional chaotic eye movements, myoclonus and ataxia. In children, it could be a paraneoplastic syndrome in association with neuroblastoma, usually with a high survival rate, but having a high frequency of neurologic and psychologic sequelae. OBJECTIVES: The aim of this study was to describe oncologic outcome (prospectively) and neurologic outcome (retrospectively) in children with non-metastatic neuroblastoma, and to determine its best treatment. PATIENTS AND METHODS: Data were collected on 21 children diagnosed with localized neuroblastoma and opsoclonus-myoclonus between 1990-1999 from the French Society of Pediatric Oncology institutions. RESULTS: Median age at diagnosis was 18 months. Location of the tumor was abdominal in 14 cases, thoracic in three cases, pelvic in three cases, and cervical in the last case. There was a majority of small tumors with a maximal diameter < 5 cm in 13 cases. Only four tumors were initially considered as unresectable tumors and received first-line chemotherapy. Complete macroscopic resection was performed in 20 cases (four after primary chemotherapy). Nine children received chemotherapy. Twenty children remained in first complete remission, and one relapsed and died (the unique NMYC amplified case). Treatment for opsoclonus-myoclonus varied widely. Only one child received no medical treatment for opsoclonus-myoclonus, because of complete resolution of neurologic symptoms after exclusive surgery. The following agents were used: corticosteroids in 18 cases, intravenously immune globulin in five cases, and antiepileptic drugs in seven cases. Ten patients experienced relapses of opsoclonus-myoclonus symptoms, mainly related to the decrease of steroid therapy (5/10). Ten of 16 assessable children had persistent neurologic deficits including speech delay or cognitive deficits (8/16), ataxia (6/16), motor delay (2/16), and behavioral problems (2/16). There is no correlation between neurologic outcome, and either age at diagnosis or duration of neurologic symptoms, or type of treatment of the tumor, particularly chemotherapy. CONCLUSION: Persistent neurologic deficits are characteristic for children with neuroblastoma and opsoclonus-myoclonus. Neurologic outcome seems unrelated to the treatment of neuroblastoma, which should exclusively be conducted according to oncological criteria. The treatment of opsoclonus-myoclonus should be standardized, mainly based on high-dose hydrocortisone, with a very low decreasing dosage, associated to intravenously immune globulin in severe cases. A biological immunologic work-up of the disease and cautious neurologic and psychologic standardized follow-up should be performed.     

Eosinophilic granuloma of the skull: a retrospective analysis

BACKGROUND: The authors describe 9 cases of children with eosinophilic granuloma (EG) of the skull and report on their clinical manifestations, treatment, and prognosis. METHOD: Nine consecutive patients were diagnosed as EG of the skull and confirmed pathologically between 1996 and 2005. In the present study, multi- and single-system Langerhans' cell histiocytosis without skull involvement were excluded. Patients with EG of the skull were divided into two groups: (1) those with only a single bone lesion and those with (2) multiple bone lesions. Surgical removal was performed between 2 and 10 years of age (mean, 4.2 years). RESULTS: Eight (88.9%) of the study subjects were found to have a single bone lesion at diagnosis, and 1 had multiple bone lesions. Seven patients had a painless skull mass and 2 patients had a painful skull mass. Total removal was performed in all 9 patients. Eight patients received postoperative chemotherapy or indomethacin as adjuvant therapy. Of the 8 patients who received adjuvant therapy, 4 were treated with indomethacin and the remaining 4 received methotrexate-based chemotherapy. Eight patients did not experience EG recurrence, however, 1 patient developed additional lesions 2 years after surgical excision. CONCLUSIONS: EG of the skull is a clinicopathological entity with a good outcome. However, therapies and prognoses are dependent on age at diagnosis and the number of bony involvements.     

Intraventricular rhabdoid tumor

Malignant rhabdoid tumor (MRT) most commonly occurs in kidney. In the central nervous system, cerebellum is the most common site of occurrence. CNS rhabdoid tumors typically occur in small children, do not respond favorably to treatment and are usually fatal within 1-year. Here is reported a 4-year-old child who presented with features of raised intracranial pressure. Apart from papillodema, there were no neurological signs. Imaging revealed a left lateral ventricular heterogeneous mass abutting the foramen of monro, with mild irregular contrast enhancement and hydrocephalus. the child underwent right ventriculo-peritoneal shunt followed by craniotomy and gross total tumor resection. He was discharged 10-days after surgery without any neurological deficits. Histopathology revealed features compatible with rhabdoid tumor. Despite radiotherapy and chemotherapy, the child died of progressive disease 10-months after surgery. the highly malignant nature of this tumor makes early diagnosis essential for aggressive management and prognostication.     

Bilateral Wilms' tumor--surgical aspects.

Four to eight percent of all Wilms' tumors are bilateral. Achieving curative resection of such tumors by partial nephrectomy or tumor enucleation while maintaining sufficient renal function represents a surgical challenge. Effective preoperative chemotherapy facilitates this aim considerably. Seven patients who were diagnosed to have bilateral synchronous Wilms' tumors between 1990 and 1994 are being reviewed. At the time of initial diagnosis, their mean age was 24.4 months, range 7 to 45 months. In 4 cases, pre-operative imaging did not reveal the full extent of the lesions, and in one of these, involvement of the second kidney was only detected by surgical exploration. Five of the children received at least 4 weeks of neo-adjuvant chemotherapy without primary biopsy, followed by bilateral tumor resection. One child had to be operated on as an emergency without any preliminary treatment, and in one, chemotherapy was interrupted after 3 weeks because of veno-occlusive disease. After a follow-up period of 66 months on the average (range 50 to 81 months), five of the patients are free of recurrence and clinically well--one with a kidney graft. The remaining two patients have died. Discussion is focussed on different management strategies of this rather rare pathology considering SIOP und NWTS protocols.     

Brain tumors in children. II. Advances in neurosurgery and radiation oncology

In this article, the contributions of neurosurgery and radiation oncology to the management of childhood brain tumors are described. Progress in a particular discipline rarely occurs in an isolated fashion, and it is clear that neurosurgical advances owe much to similar advances in anesthesiology, neuroradiology, and intensive care management. These advances in various disciplines have all permitted bolder yet safer attempts at radical resection by the neurosurgeon. The goal of radical surgical resection is cure in the case of low-grade brain tumors. However, even in those situations where surgery alone cannot be curative, the reduction of tumor bulk facilitates the task of both radiation therapy and chemotherapy, with improvement in progression-free survival as well as overall survival. For those tumors that cannot be cured by surgical resection alone, the standard of therapy thereafter still remains irradiation of the residual tumor. It is clear that the single major advance in the treatment of childhood medulloblastoma has been Cushing's recognition of the value of craniospinal irradiation. Refinements in radiation machines and treatment planning have permitted more accurate delivery of radiation therapy with a slight reduction in toxicity. Newer approaches, such as hyperfractionated irradiation and interstitial irradiation, attempt to improve therapeutic efficacy while minimizing toxicity. Nevertheless, as more children are surviving their brain tumors following surgery and radiation therapy, the price of the successful therapy is being increasingly realized in terms of developmental deficits, particularly in the very young child. It is the desire of all those involved in the management of children with primary brain tumors to seek alternative approaches to wide-field irradiation of the brain in children with high-grade tumors.     

Pancreatoblastoma in childhood: clinical course and therapeutic management of seven patients

BACKGROUND: To analyze the clinical course of pancreatoblastoma in children and to propose management and therapy. PROCEDURE: Retrospective review of seven cases of pancreatoblastoma treated in France over a 20-year period and literature review. RESULTS: Median age of patients was 6 years. The tumor was well encapsulated in one patient and had direct extension to adjacent tissues in two others. Three patients had regional lymph node involvement and one had liver metastases. The tumor arose in the head of the pancreas in three children, in the tail and body in two and in the tail and in the body, one each. Elevation of AFP serum levels was noted in four out of 6 cases. Five tumor resections were performed, one initially and 4 after neoadjuvant chemotherapy, and cisplatin plus doxorubicin seemed effective. Two children received post-operative irradiation because of incomplete resection. In all, four children are disease free with a median follow-up of 50 months ( range, 5--120 months) : one had a complete removal of tumor at diagnosis and no further treatment, three had unresectable tumor at diagnosis and received neoadjuvant chemotherapy with cisplatin and doxorubicin. One of them also received post-operative irradiation. CONCLUSIONS: Pancreatoblastoma is a curable tumor. Examination of serum AFP levels may be useful for diagnosis and to follow the course of the disease. Complete resection is the treatment of choice. However, tumor is often unresectable at diagnosis and preoperative chemotherapy is needed to reduce tumor volume. We suggest a regimen that include cisplatin and doxorubicin. In patients with incompletely resected disease, postoperative radiation may be indicated.     

Sacral Chordoma in Early Childhood: Clinicopathological and Immunohistochemical Study

Two cases of sacral chordoma in a 7-year, 9-month-old boy and a 3-year, 4-month-old boy are presented. In addition to the typical histology of conventional chordoma, both tumors showed the less differentiated sarcomatoid appearance of atypical chordoma in the major portion. Immunohistochemically, in both cases neoplastic cells in areas of conventional as well as atypical chordoma were positive for keratins (CAM 5.2, AE1 and AE3), epithelial membrane antigen, vimentin, S-100 protein, carcinoembryonic antigen, and glial fibrillary acidic protein. Both patients underwent resection of the tumor and chemotherapy. In comparison with conventional chordomas in adults, however, these two tumors showed more aggressive clinical course and were less amenable to therapeutic control. The older boy died of multiple metastasis 1 year after initial diagnosis. At the last follow-up, 15 months after initial diagnosis, the younger boy was alive, but with recurrent and metastatic disease of the left parasacral area and chest wall. Our studies of these two cases and the reported cases suggest that sacral chordoma in children has distinctive clinicopathologic features denoting a highly aggressive tumor and that it should be treated as such. Received January 9, 1997; accepted August 15, 1997.     

Askin's tumor in children: report of two cases

Two cases of Askin's tumor of the thoracopulmonary region in children (a 6-year-old female and a 5-year-old male) are presented. Primary surgery was unfeasible and despite aggressive chemotherapy both died within 304 and 300 days, respectively, from the onset of the disease. Although rare, Askin's tumor should be considered in the differential diagnosis of any thoracopulmonary mass in childhood and adolescence through appropriate histology and immunohistochemistry of incisional biopsies. Its prognosis is poor unless radical surgery is performed as soon as possible: unresectable tumors can be treated with chemotherapy, but prolonged chemotherapy yields potentially lethal complications. We conclude that only early diagnosis and cytogenetic recognition can give children with Askin's tumor a chance of curative surgery.     

Experience with aggressive therapy in three children with unresectable malignant liver tumors

BACKGROUND: Children with malignant liver tumors often present with unresectable disease but need not be considered incurable. The advent of effective chemotherapy makes aggressive management feasible, as our experience with three such patients demonstrates. Procedure and Results One child with an unresectable undifferentiated sarcoma of the liver and two others with unresectable primary hepatoblastoma and lung metastases were treated with initial chemotherapy, followed by aggressive surgical management. Treatment with chemotherapy followed by hepatectomy and liver transplantation (cadaveric or live donor) in two children has resulted in disease-free survivals of 79 and 38 months. The third patient is alive and well 24 months following chemotherapy and aggressive resection of the primary and 12 metastatic lesions. CONCLUSIONS: Initial chemotherapy for unresectable liver tumors with or without metastases is supported by the review of the literature. Consideration of orthotopic liver transplantation (OLT) from cadaveric or living related donor is warranted when the malignancy is demonstrably chemosensitive, independent of initial staging. Aggressive resection of primary and metastatic disease may be called for in selected cases.     

The role of biopsy in the diagnosis of renal tumors of childhood: Results of the UKCCSG Wilms tumor study 3

BACKGROUND: The United Kingdom Children's Cancer Study Group (UKCCSG) Wilms Tumor Study 3 has adopted preoperative chemotherapy for Wilms tumors (WT), but required prechemotherapy biopsy for histologic diagnosis. The aims of this review were to assess the usefulness and safety of prechemotherapy biopsy and to compare histologic features of WT before and after chemotherapy. PROCEDURE: There were 286 eligible patients but only 241 biopsies and 226 nephrectomy case slides were submitted for panel review. The presence of different histologic components of WT before and after chemo therapy was retrospectively assessed. RESULTS: Among the 241 cases, the biopsy material in 9 (4%) was not diagnostic, in 28 (12%) that were clinically and radiologically consistent with WT, the biopsy revealed tumors other than WT, and in the remaining 204 (85%) WT was confirmed. Of 188 WT suitable cases, blastema was found in 89% of tumors at biopsy, but in only 50% at nephrectomy; the remainder were either completely necrotic (17%) or showed only epithelial and/or stromal elements (33%). Of 182 children who had percutaneous cutting needle biopsy (PCNB), a fall in haemoglobin (20% of cases) and local pain (19%) were the most common complications. One child required emergency nephrectomy due to massive intratumoral bleeding, another had tumor rupture and subsequently died, and a third developed a needle track recurrence 8 months after the biopsy. CONCLUSIONS: A number of renal tumors (12%) can have the correct histologic diagnosis made by PCNB. Preoperative chemo therapy markedly decrease in the number of samples with preserved blastema. The morbidity associated with PCNB is small. Needle biopsy of any renal mass prior to initiation of chemotherapy is recommended.