High-Dose Methylprednisolone Treatment of Hepatic Veno-Occlusive Disease in a Child with Wilms Tumor

Veno-occlusive disease (VOD) is a rare complication of chemotherapy in children with cancer. In the literature successful treatment of severe VOD has not been well determined. The authors report a 5-year-old boy with Willms tumor whose condition was complicated with VOD while receiving chemotherapy. He was treated successfully with high-dose methylprednisolone. After therapy, the clinical signs of VOD, such as abdominal ascites, oliguria, and jaundice, disappeared without any adverse effects of therapy.     

细胞因子在暴发性肝衰竭发病机制中的作用

暴发性肝功能衰竭是由于肝细胞大量坏死而出现以肝功能严重受损为特征的综合征,预后极差,病死率很高。其发病机制较为复杂,研究认为细胞因子在其发病机制中有十分重要的作用,各种原因诱导细胞因子产生,引起肝细胞凋亡及坏死。随着对细胞因子研究的深入,有望在暴发性肝功能衰竭的治疗策略上有突破性发展。     

肾母细胞瘤治疗和研究新进展

肾母细胞瘤(Wilms‘Tumour，WT)是小儿实体肿瘤中最常见的一种。近几年来，由于对晚期患儿采取综合治疗，提高了生存率。另外，随着国际间的协作，集合资料进行分析研究，定期总结疗效经验，修订治疗方案指导临床实践，不断提高疗效，也使治愈率有了很大的提高，并且也更关注患儿长期存活的生活质量，现综述如下。     

Aetiology of lcteric Hepatitis and Fulminant Hepatic Failure in Children and the Possible Predisposition to Hepatic Failure by Sickle Cell Disease

ABSTRACT. The aetiological agents of acute icteric hepatitis and fulminant hepatic failure were investigated in 47 children less than 12 years of age presenting at our hospital during the period January to December 1987. Hepatitis A virus was the aetiological agent in 72 %, hepatitis B virus in 11 %, cytomegalovirus in 2 % and non A non B in 15 %. These results confirm the endemicity of these viruses in the Saudi population and that they can be contracted in early life and could lead to severe hepatitis. Three of the patients who had hepatitis A virus infection developed fulminant hepatic failure and two of them died. Two of the patients who developed fulminant hepatic failure were also suffering from sickle cell disease. This raises the question of a possible predisposition to hepatic failure in sickle cell disease upon infection with hepatitis A virus.     

Hepatic Mesenchymoma in Childhood: Successful Combined-Modality Treatment

Malignant, undifferentiated sarcoma (mesenchymoma) of the liver is a rare tumor in children with about 100 cases reported in the literature. The tumor is composed of pleomorphic, mesenchymal matrix elements. It behaves in a malignant fashion and has a poor prognosis. Long-term survival after surgery alone has been poor.Treatment with surgery and multiple-agent chemotherapy seems more successful.This report describes successful treatment in a 5-year-old girl.     

Hepatic Mesenchymoma in Childhood: Successful Combined-Modality Treatment

Malignant, undifferentiated sarcoma (mesenchymoma) of the liver is a rare tumor in children with about 100 cases reported in the literature. The tumor is composed of pleomorphic, mesenchymal matrix elements. It behaves in a malignant fashion and has a poor prognosis. Long-term survival after surgery alone has been poor.Treatment with surgery and multiple-agent chemotherapy seems more successful.This report describes successful treatment in a 5-year-old girl.     

小儿Budd-Chiari综合症并肝功能衰竭1例

1病例资料患儿,男,3岁,因“腹胀、少尿17d”入院。体查:神志淡漠,腹膨隆,呈蛙腹,肝脾肋下未触及,叩诊呈浊音,肠鸣音弱,移动性浊音( )。B超显示:肝、脾肿大,大量腹腔积液,左肾轻度积水。MRI检查显示:肝段下腔静脉(HIVC)部分狭窄,肝静脉(HV)显示不清。血管造影显示:HIVC部分狭窄和HV右支完全阻塞,确诊为Budd-Chiari综合症(BCS)。入院后予中心吸氧,低盐低脂、高蛋白、高热量半流饮食,补充白蛋白,利尿及纠正水电解质、酸碱平衡紊乱等,家长放弃治疗,随访2个月后因肝功能衰竭,病情恶化而死亡。2讨论BCS为HV和/或HIVC部分或完全阻塞引…     

Role of Endomyocardial Biopsy for Children Presenting With Acute Systolic Heart Failure

Myocarditis, an inflammatory disease of the heart, frequently results from viral infections, postviral immune-mediated responses, or both. It is a common cause of acute-onset systolic heart failure in children. Endomyocardial biopsy (EMB) remains the gold standard for the diagnosis of myocarditis. However, EMB is not performed for most myocarditis cases involving children in the United States. Clinical scenarios in which EMB results added unique prognostic data and guidance to therapy have been defined recently. This review outlines the role of EMB in the diagnosis and management of myocarditis for children presenting with acute-onset systolic heart failure.     

Considerations in the Diagnosis and Management of Pediatric Patients With Favorable Histology Wilms Tumor Who Present With Only Pulmonary Nodules

More than 70% of children with stage IV, favorable histology (FH) Wilms tumor will be relapse‐free survivors 16 years after diagnosis. Successful treatment generally includes whole lung radiation therapy and doxorubicin. Such therapy is associated with adverse, long‐term effects, including impaired pulmonary function, congestive heart failure, and second malignant neoplasms, especially breast cancer. Cooperative groups have adopted a risk‐based approach to the treatment of these patients. It is important to recall the good overall prognosis for this group before recommendations for intensification are made based on preliminary data and in the absence of histological confirmation of persistent malignant disease.     

Treatment of children with stage IV favorable histology Wilms tumor: A report from the National Wilms Tumor Study Group

The purpose of this study was to evaluate the effect of the sequential addition of doxorubicin and cyclophosphamide to the combination of vincristine and actinomycin D on the relapse-free survival of children with stage IV/favorable histology Wilms tumor. We reviewed the clinical courses of all randomized patients from National Wilms Tumor Study (NWTS)-2 and 3 with stage IV/favorable histology (FH) Wilms tumor. We determined the four-year relapse-free survival percentage for patients treated on NWTS-2 with the combination of vincristine (VCR) and actinomycin D (AMD) with (regimen D) or without (regimen C) doxorubicin (DOX), and for patients treated on NWTS-3 with the combination of VCR + AMD + DOX with (regimen J) or without (regimen DD-RT) cyclophosphamide (CTX). All children received whole lung radiation therapy. The four-year relapse-free survival percentage for children with stage IV/FH Wilms tumor treated with regimen C was 53.3%, compared to 57.7% for those treated with regimen D (P = 0.63). The four-year relapse-free survival percentage for children with stage IV/FH Wilms tumor treated with regimen DD-RT was 79.0%, compared to 80.9% for those treated on regimen J (P = 0.79). The four-year relapse-free survival for children with lung metastases only treated with regimen D on NWTS-2 was significantly lower than that of children treated with the related regimen DD-RT on NWTS-3 (P = 0.03). We conclude that the addition of doxorubicin to the combination of vincristine and actinomycin D and pulmonary irradiation did not clearly improve the four-year relapse-free survival percentage of children with stage IV/FH Wilms tumor, although the benefit may have been masked by the greater frequency of death due to toxicity in NWTS-2. There was no evidence that the addition of CTX to the three-drug treatment regimen improved the four-year relapse-free survival percentage of children with stage IV/FH Wilms tumor. The data with only two drugs derived from NWTS-2 suggest that there is a population of children with stage IV/FH Wilms tumor who can be successfully treated without an anthracycline. The goal of future research will be to identify this subgroup at the time of initial diagnosis. © 1996 Wiley-Liss, Inc.     

Treatment of Wilms tumor relapsing after initial treatment with vincristine and actinomycin D: a report from the National Wilms Tumor Study Group

PURPOSE: NWTS-5 was a multi-institutional clinical trial for patients less than 16 years of age at diagnosis with specific renal neoplasms who were diagnosed between August 1, 1995 and May 31, 2002. A uniform approach to the treatment of patients with relapse was employed. PATIENTS AND METHODS: Seventy-two patients who relapsed after immediate nephrectomy (stages I and II), initial chemotherapy with vincristine (VCR) and actinomycin D and no radiation therapy were registered on stratum B of the NWTS-5 relapse protocol. Four patients were not evaluable: one due to insufficient data and three due to major protocol violations. Among the 68 remaining patients, one who was 19 years of age at initial diagnosis of Wilms tumor, five with bilateral Wilms tumor at diagnosis, three who developed a contralateral relapse, and one with persistent disease were not included in this analysis. Relapse treatment included surgical excision, when feasible, radiation therapy and alternating courses of VCR, doxorubicin and cyclophosphamide and etoposide and cyclophosphamide. RESULTS: The outcomes of 58 patients were analyzed. The lung was the only site of relapse for 31 patients. Event-free survival 4 years after relapse was 71.1% and 4-year overall survival was 81.8% for all patients and were 67.8 and 81.0% for those who relapsed only to their lungs. The most frequent toxicities were hematological. CONCLUSIONS: These results demonstrate that a significant proportion of children with Wilms tumor who relapse after initial treatment with VCR and actinomycin D can be successfully re-treated.     

Successful Chemotherapeutic Treatment of Diencephalic Syndrome with Continued Tumor Presence

A 7-month-old infant with typical features of diencephalic syndrome (DES) associated with a hypothalamic mass, most probably a glioma, was treated with chemotherapy. The tumor showed clear shrinkage, but after more than 2 years regrowth was noted. During the treatment period the child regained normal growth and became free of symptoms. As radiation therapy, especially at a young age, has significant adverse effects and a neurosurgical approach to the diencephalic region also has the potential to cause significant sequelae, a chemolherapeulic option, when it exists, is preferred. Thus, in an infant in whom a glioma is suspected to be the cause of the DES, based on the clinical picture and the neuroimaging appearance, chemotherapy should be considered the primary therapeutic modality. Even if its effect is temporary, its use is well justified. The most appropriate treatment protocol still needs to be determined.     

Hemophagocytic Syndrome Presenting as Acute Hepatic Failure in Two Infants: Clinical Overlap with Neonatal Hemochromatosis

Two patients with hemophagocytic lymphohistiocytosis who presented with acute liver failure are reported. Both presented with fever, hepatosplenomegaly, markedly elevated liver function tests, abnormal coagulation profiles, and an increase in serum ferritin. Both infants were diagnosed with neonatal hemochromatosis based on a clinical picture of hepatic insufficiency with hyperferritinemia and were referred for liver transplantation. The first patient died of liver failure and septicemia before transplantation. Review of autopsy material revealed a hepatitis-like pattern and extensive infiltration of liver and other organs including bone marrow by histiocytes, some of which were hemophagocytic. The second patient underwent liver transplantation but died 44 days thereafter from progressive hemophagocytic lymphohistiocytosis. Examination of the resected liver demonstrated a hepatitis-like pattern, proliferation of histiocytes, and hemophagocytosis, and the bone marrow revealed hemophagocytic histiocytosis. Hemophagocytosis recurred in the allograft. Hepatic manifestations are common in hemophagocytic lymphohistiocytosis and overt hepatic failure may occur, but initial presentation as fulminant hepatic failure is not well recognized. Elevated serum ferritin can make the distinction from neonatal hemochromatosis and other forms of neonatal liver failure difficult. Hemophagocytic lymphohistiocytosis should be considered in the differential diagnosis of neonatal liver disease, especially when it is accompanied by cytopenias. Received May 5, 1998; accepted August 31, 1998.     

Synchronous Wilms Tumor and Fibrolamellar Hepatocellular Carcinoma: Report of a Case

Fibrolamellar hepatocellular carcinoma (FHCC) is a unique histologic variant of HCC that occurs in a younger subset of patients than classical HCC, and is associated with a better prognosis. Wilms tumor (WT) is a malignant embryonal neoplasm of the kidney and is one of the most common solid tumors of childhood, occurring at an estimated frequency of 1 in 8000 to 10,000 births. Although second malignant neoplasms (SMNs) following therapy for WTs have been reported in the liver, the coexistence of HCC and WT is extremely rare. We present the first report of a synchronous anaplastic WT and FHCC in a previously healthy 4-year-old girl. Despite the presence of focal immunohistochemical positivity for p53 in the WT, molecular analysis failed to reveal a germline or somatic p53 mutation, and was inconclusive in establishing a clonal relation between the two tumors. Received May 14, 1999; accepted August 20, 1999.     

Successful Treatment of a Mediastinal Endodermal Sinus Tumor in a 2-Year-Old Child

The article describes a primary mediastinal endodennal sinus tumor in a child. The employment ofmultidrug therapy combined with surgical treatment resulted in a long-term remission in this rare tumor.