RESUMO
BACKGROUND AND AIMS: Prognosis of hepatoblastoma patients has increased with cisplatin-based chemotherapy and high-quality resection including liver transplant. Consequently current risk-adapted therapeutic strategy aims to reduce long-term side effects in patients with standard risk disease. METHODS: We report long-term mortality and morbidity data concerning 151 2-year hepatoblastoma survivors treated with SIOPEL risk-adapted strategies (sex-ratio M/F = 1.6, median age at diagnosis = 2.6 years [range 0-17.7], median year at diagnosis = 2008 [1994-2017]). Fifty-three patients had loco-regional risk factors VPEFR, 12 were PRETEXT-IV and 30 were metastatic. All received cisplatin and 84 anthracyclines. Twelve had liver transplant. To assess hearing, renal and cardiac functions, audiograms were performed in 116/151 patients (76.8%), glomerular filtration rate in 113/151 (74.8%) and cardiac ultrasound in 65/84 (77.4%) anthracycline-exposed patients. RESULTS: With a median follow-up of 9.4 years (range 2.1-25.8), four late relapses, one second malignancy (Acute Myeloid Leukemia AML-M5) and two deaths (one from hepatoblastoma, one from AML) occurred. The 10-years event free survival and overall survival probabilities were 95.5% (95% CI 91.9-99.1) and 98.7% (95% CI 96.8-100), respectively. Sixty-eight non-oncologic health-events included 57 cases of hearing loss (including 25 Brock 3-4), three liver cirrhosis, three pre-operative portal cavernoma, two focal nodular hyperplasia, two grade-1 chronic kidney diseases and one asymptomatic cardiac dysfunction were reported. Ototoxicity was significantly associated with cisplatin cumulative dose (OR = 2.07, 95% CI 1.32-3.24, p = 0.001) and carboplatin exposure (OR = 3.14, 95% CI 1.30-7.58, p = 0.01) in multivariable analysis adjusted for sex and age at diagnosis. CONCLUSIONS: With current risk-adapted strategies, hepatoblastoma is a highly curable disease, with very rare relapses, and few late effects except hearing loss which remains a serious condition in these very young patients.
Assuntos
Hepatoblastoma , Neoplasias Hepáticas , Adolescente , Protocolos de Quimioterapia Combinada Antineoplásica/efeitos adversos , Carboplatina/uso terapêutico , Criança , Pré-Escolar , Cisplatino/efeitos adversos , Humanos , Lactente , Recém-Nascido , Neoplasias Hepáticas/tratamento farmacológico , Morbidade , SobreviventesRESUMO
Hepatic hydrothorax is a rare complication of portal hypertension. The optimal treatment for this condition is liver transplantation. Liver transplantation is significantly more manageable in children who weigh more than 8 kg. Here, an implantable pleural access device was used in a 5-month-old infant for painless iterative punctures to relieve respiratory symptoms, while waiting for liver transplantation and the patient's growth. The patient underwent successful transplantation 3 months later with a more optimal weight.
Assuntos
Atresia Biliar/complicações , Hidrotórax/terapia , Atresia Biliar/terapia , Gerenciamento Clínico , Humanos , Hidrotórax/fisiopatologia , Lactente , MasculinoRESUMO
We report the results of a study of survival, liver and kidney functions, and growth with a median follow-up of 24 years following liver transplantation in childhood. From 1988 to 1993, 128 children underwent deceased donor liver transplantation (median age: 2.5 years). Twenty-year patient and graft survival rates were 79% and 64%, respectively. Raised serum aminotransferase and/or γ-glutamyl transferase activities were present in 42% of survivors after a single transplantation. Graft histology (35 patients) showed signs of chronic rejection in 11 and biliary obstruction in 5. Mean total fibrosis scores were 4.5/9 and 3/9 in patients with abnormal and normal serum liver tests, respectively. Glomerular filtration rate was <90 mL·min-1 in 35 survivors, including 4 in end-stage renal disease who were undergoing dialysis or had undergone renal transplantation. Median final heights were 159 cm for women and 172 cm for men; final height was below the target height in 37 patients. Twenty-year survival after childhood liver transplantation may be close to 80%, and final height is within the normal range for most patients. However, chronic kidney disease or altered liver biochemistries are present in over one third of patients, which is a matter of concern for the future.
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Rejeição de Enxerto/mortalidade , Sobrevivência de Enxerto , Falência Renal Crônica/mortalidade , Transplante de Fígado/mortalidade , Complicações Pós-Operatórias , Diálise Renal/estatística & dados numéricos , Sobreviventes/estatística & dados numéricos , Adolescente , Adulto , Criança , Pré-Escolar , Feminino , Seguimentos , França/epidemiologia , Taxa de Filtração Glomerular , Rejeição de Enxerto/epidemiologia , Humanos , Incidência , Lactente , Falência Renal Crônica/epidemiologia , Testes de Função Renal , Masculino , Prognóstico , Fatores de Risco , Taxa de Sobrevida , Adulto JovemRESUMO
INTRODUCTION: In France, 26 regional pediatric palliative care teams (ERRSPP) were created between 2008 and 2012. We conducted the first prospective French study to describe the main specifications of the initial contact with an ERRSPP and to analyze the responses given. DESIGN AND METHODS: All the requests for interventions on the part of the ERRSPP were collected between September 2013 and September 2014. We prospectively completed a questionnaire on the patient's clinical and demographic data (age, sex, disease), details regarding the request (type of applicant, unit's specialty, request pattern), and the answers provided (interval between diagnosis and request, duration of care by ERRSPP, death, changes compared to the initial request). The diseases were classified within one of the six groups of palliative pediatric care diseases, based on the standards of the Canadian palliative care association. RESULTS: We gathered 67 requests, 61 relating to patients. The median age at the request was 49.8 months (range: 2.3-145). The original pattern was multiple: multidisciplinary decision-making (42 %), coordination of care (34 %), symptom management (21 %), logistic support for home care (19 %), education (9 %) and case discussion (6 %). Requests concerning multidisciplinary decision-making were predominant within the neonatal period (61 %); coordination of care was significant for children and adolescents (78 % after 4 years of age). The study of the median time from diagnosis to request compared to the groups of diseases revealed a short time in group 5 (neonatology: 0.36 months) and a long time in group 4 (irreversible and nonscalable diseases: 54.6 months) (P<0.001). At the end of the study, the follow-up of 50.8 % of the patients by ERRSPP was still going on (median duration of care by the ERRSPP of Languedoc-Roussillon region [ERRSPP-LR], 3.4 months [range: 0.2-5.5]). No request was formulated by a general practitioner. CONCLUSION: This study shows the heterogeneity of the initial contact made with an ERRSPP, confirming its different assignments and the need for a multidisciplinary team. The ERRSPP's answer was expanded in half of the cases, attesting to the changing needs over time.
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Necessidades e Demandas de Serviços de Saúde , Cuidados Paliativos/organização & administração , Equipe de Assistência ao Paciente/organização & administração , Programas Médicos Regionais/organização & administração , Adolescente , Criança , Pré-Escolar , Feminino , França , Humanos , Lactente , Recém-Nascido , Masculino , Estudos Prospectivos , Inquéritos e QuestionáriosRESUMO
BACKGROUND: Constructive feedback is an essential element of the educational process, helping trainees reach their maximum potential and increasing their skill level. Video-based feedback has been described as highly effective in various educational contexts. The present study aimed to evaluate the feasibility and acceptability of video-based, on-ward supervision for final year students in a clinical context with real patients. METHODS: Nine final year medical students (three male, six female; aged 25.1 ± 0.7 years) and eight patients (five male, three female; aged 59.3 ± 16.8 years) participated in the pilot study. Final year students performed routine medical procedures at bedside on internal medicine wards at the University of Heidelberg Medical Hospital. Students were filmed and were under supervision. After performing the procedures, an oral feedback loop was established including student, patient and supervisor feedback on communicative and procedural aspects of skills performed. Finally, students watched their video, focusing on specific teachable moments mentioned by the supervisor. Written evaluations and semi-structured interviews were conducted that focused on the benefits of video-based, on-ward supervision. Interviews were analysed qualitatively, using open coding to establish recurring themes and overarching categories to describe patients' and students' impressions. Descriptive, quantitative analysis was used for questionnaire data. RESULTS: Supervised, self-chosen skills included history taking (n = 6), physical examination (n = 1), IV cannulation (n = 1), and ECG recording (n = 1). The video-based, on-ward supervision was well accepted by patients and students. Supervisor feedback was rated as highly beneficial, with the video material providing an additional opportunity to focus on crucial aspects and to further validate the supervisor's feedback. Students felt the video material would be less beneficial without the supervisor's feedback. The setting was rated as realistic, with filming not influencing behaviour. CONCLUSION: Video-based, on-ward supervision may be a powerful tool for improving clinical medical education. However, it should be regarded as an additional tool in combination with supervisors' oral feedback. Acceptance was high in both students and patients. Further research should address possibilities of efficiently combining and routinely establishing these forms of feedback in medical education.
Assuntos
Competência Clínica , Educação de Graduação em Medicina/métodos , Feedback Formativo , Medicina Interna/educação , Gravação em Vídeo , Adulto , Idoso , Estudos de Coortes , Avaliação Educacional , Feminino , Alemanha , Hospitais Universitários , Humanos , Masculino , Pessoa de Meia-Idade , Quartos de Pacientes , Relações Médico-Paciente , Projetos Piloto , Estudantes de Medicina/estatística & dados numéricos , Adulto JovemRESUMO
BACKGROUND: Over multiple years, the didactic concept of "peer-assisted learning" (PAL) has proved to be valuable for medical education. Particularly in the field of the nowadays widely established Skills-Labs, the assignment of student tutors is both popular and effective. The aim of the underlying study is to assess the current status of PAL programs within German medical faculties' Skills-Labs regarding their distribution, extent, structure and content based on a nation-wide survey. METHODS: All 36 medical faculties in Germany were contacted and asked for their participation (via telephone or in written form) in the survey encompassing 16 central questions as to the structure of established PAL programs. Data obtained were subject to quantitative and qualitative analysis. RESULTS: 35 of 36 (97.2%) medical faculties participated in the survey. A PAL program was shown to be established at 33 (91.7%) faculties. However, the results show distinct differences between different faculties with respect to extent and content of the PAL programs. CONCLUSIONS: Among German medical Skills-Labs, PAL has been established almost ubiquitously. Further studies on the conception and standardization of training concepts appear to be pivotal for the advancement of PAL in the context of Skills-Labs.
Assuntos
Competência Clínica , Currículo , Educação de Graduação em Medicina/organização & administração , Docentes de Medicina/organização & administração , Grupo Associado , Atitude do Pessoal de Saúde , Alemanha , Humanos , Entrevistas como Assunto , Medicina , Mentores/educação , Inquéritos e QuestionáriosRESUMO
BACKGROUND: Meso-Rex bypass (MRB) and portosystemic surgical shunt (PSS) are both used to treat extrahepatic portal vein obstruction (EHPVO) in children. The aim of this study was to analyse the outcome of MRB and PSS to select patients who could benefit from a prophylactic MRB. METHODS: This single-centre retrospective study of children who underwent either MRB or PSS for EHPVO was conducted between 1996 and 2010. Details of patient demographics and preoperative evaluation were collected. Success rates, defined as shunt patency after a minimum of 6 months and clearance of varices or symptoms, were compared. Determinants of outcomes were explored. RESULTS: Sixty-nine patients underwent a MRB or PSS. Median (interquartile range, i.q.r.) age at surgery was 6.6 (4.0-10.6) years. Twenty-four patients (35 per cent) had had a neonatal umbilical catheter (NUC) placed previously and 47 (68 per cent) had experienced an upper gastrointestinal bleed. Imaging assessment of the intrahepatic left portal vein was considered favourable in 40 patients. Of 43 MRBs attempted, 11 failed during surgery and four patients had persistent thrombosis after a median of 55 (i.q.r. 18-107) months. The success rate of MRB was 60 per cent (26 of 43) compared with 100 per cent (26 of 26) for PSS (P < 0.001). It was lower among patients in whom a NUC had been used (2 of 10 versus 24 of 33; P = 0.004), for procedures undertaken early in the series (6 of 16 versus 20 of 27; P = 0.020) and when the imaging pattern was unfavourable (0 of 5 versus 26 of 38; P = 0.006). On multivariable analysis, only a previous history of NUC predicted failure (P = 0.016). CONCLUSION: Prophylactic MRB seems a good treatment option for EHPVO in children, but should be done only by an experienced team in patients with favourable imaging and without a previous history of NUC.
Assuntos
Implante de Prótese Vascular/métodos , Prótese Vascular , Veia Porta/cirurgia , Trombose Venosa/cirurgia , Anastomose Cirúrgica/métodos , Criança , Pré-Escolar , Feminino , Sobrevivência de Enxerto , Humanos , Masculino , Politetrafluoretileno/uso terapêutico , Derivação Portossistêmica Cirúrgica/métodos , Cuidados Pós-Operatórios/métodos , Estudos RetrospectivosRESUMO
PURPOSE: To analyse the clinical characteristics and outcome of hepatoblastoma (HB) patients who relapsed after enrolment on SIOPEL studies 1-3. PATIENTS AND METHODS: Analysis of clinical data of all 59 patients (pts) registered in SIOPEL 1-3 studies, who relapsed after achieving complete remission (CR). RESULTS: The median time from the initial diagnosis to relapse was 12 months (4-115 m). The site of relapse was lung N=27, liver N=21, both liver and lung N=5 and other N=5 (missing data-MD: 1 patient). All but 9 pts had an alpha-fetoprotein level >10 ng/mL at the time of relapse. Treatment of the relapse included chemotherapy and surgery N=25, chemotherapy alone N=21, surgery alone N=7 and only palliative treatment N=5 (MD: 1 pt). Overall, 31 pts (52%) achieved a second CR. With a median follow-up of 83 months, 23 pts are alive, (18 in 2nd CR, 5 after a second relapse) and 36 pts have died (35 from disease and 1 from complications). Three-year event-free survival and overall survival are 34% and 43% respectively (95% confidence interval [CI] 0.28-0.69). The main factors associated with a good outcome were PRETEXT group I-III at diagnosis, a high AFP level at relapse and relapse treatment including both chemotherapy and surgery. CONCLUSION: Relapses in HB are rare events occurring in less than 12% of pts after CR. Combined treatment with chemotherapy and surgical removal of the tumour is essential for long-term survival.
Assuntos
Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Hepatectomia , Hepatoblastoma/mortalidade , Neoplasias Hepáticas/mortalidade , Recidiva Local de Neoplasia/mortalidade , Terapia de Salvação , Adolescente , Criança , Pré-Escolar , Terapia Combinada , Feminino , Seguimentos , Hepatoblastoma/patologia , Hepatoblastoma/terapia , Humanos , Lactente , Recém-Nascido , Neoplasias Hepáticas/patologia , Neoplasias Hepáticas/terapia , Masculino , Recidiva Local de Neoplasia/patologia , Recidiva Local de Neoplasia/terapia , Estadiamento de Neoplasias , Prognóstico , Indução de Remissão , Estudos Retrospectivos , Taxa de SobrevidaRESUMO
Hepatic artery thrombosis (HAT), one of the most severe complications of pediatric orthotopic liver transplantation (OLT), often compromises graft and/or child survival. Of 590 OLT performed in 516 children over a 20-year period, 45 were complicated by early HAT, during the first 2 weeks after transplantation. Systematic Doppler ultrasonographic detection of HAT allowed successful surgical revascularization in 19 instances, resulting in a 20-year graft survival rate of 77% versus 24% of cases when revascularization was not attempted or failed. A combination of surgical emergency revascularization, biliary interventional radiology, biliary surgery and/or retransplantation resulted in an 80% 20-year patient survival rate, identical to that of transplanted children who did not experience early HAT. The majority of long-term survivors with their initial graft had normal liver tests, no biliary dilation on ultrasonography and minimal or moderate fibrosis on liver histology. A failed attempt at revascularization did not significantly alter patient survival. Despite these encouraging results, for the children and their parents to overcome the entire process in terms of reoperations, repeated radiological interventions, number of hospitalizations and emotional stress, remains an ordeal of such magnitude that it justifies renewed efforts to progress in the prevention of this complication.
Assuntos
Artéria Hepática/patologia , Transplante de Fígado , Trombose/patologia , Resultado do Tratamento , Criança , Humanos , Taxa de SobrevidaRESUMO
Congenital portosystemic shunts are present in one in 30,000 children. Among the associated risks of severe complications are neonatal cholestasis, benign and malignant liver tumors, hepatopulmonary syndrome, portopulmonary hypertension, and encephalopathy. They can be detected on prenatal ultrasonograms, during the investigation of a positive galactosemia screening test in neonates or of a complication, or be found fortuitously on an abdominal ultrasound. Small intrahepatic shunts may resolve spontaneously within one year of age, but other shunts such as extrahepatic, persistent ductus venosus or persisting intrahepatic shunts, must be closed in one or two steps, by interventional radiology techniques or surgically. The plasticity of the intrahepatic portal system allows revascularization of the liver after shunt closure, even when no intrahepatic portal structures can be detected on imaging studies. This leaves little or no place for liver transplantation in the management of these children.
Assuntos
Veia Porta/anormalidades , Malformações Vasculares/diagnóstico , Colestase/complicações , Encefalopatia Hepática/complicações , Síndrome Hepatopulmonar/complicações , Humanos , Hipertensão Pulmonar/complicações , Lactente , Recém-Nascido , Neoplasias Hepáticas/complicações , Diagnóstico Pré-Natal , Malformações Vasculares/complicações , Malformações Vasculares/cirurgiaRESUMO
OBJECTIVES: The aim of the study was to assess the diagnosis and management of solid pancreatic neoplasm in children and the type of surgical treatment, focusing on short- and long-term outcomes. METHODS: We retrospectively reviewed the charts of all children who had undergone pancreatic resection for suspicion of pancreatic tumor in Kremlin Bicêtre Hospital, Paris, between 1986 and 2008. We studied the symptoms at diagnosis, the type of surgery, and the short- and long-term morbidity and mortality. RESULTS: Of 18 patients identified, there were 7 pseudopapillary tumors, 3 neuroblastomas, 2 rhabdomyosarcomas, 1 acinar cell carcinoma, 1 endocrine cell carcinoma, 1 renal angiomyolipoma, and 3 pancreatic cysts. Symptoms at diagnosis were abdominal trauma, abdominal mass, and jaundice. Operative procedures were duodenopancreatectomy (11), mid-pancreatic resections (2), splenopancreatectomy (2), distal pancreatectomy (1), and tumorectomy (2). There were no deaths related to surgery. The postoperative morbidity rate was 45%, including 2 cases of fistula (11%) occurring after a mid-pancreatic resection and a pancreaticoduodenectomy. The median follow-up was 4.2 years (range 2-11). There was no diabetes mellitus, but there was 1 case of fat diet intolerance requiring pancreatic enzyme substitution. All of the children had a growth curve within normal limits. CONCLUSIONS: In this experience, pancreatic resections have proven to be a safe and efficient procedure, with low long-term morbidity, for the treatment of tumoral and selected nontumoral pancreatic masses.
Assuntos
Crescimento , Pâncreas/cirurgia , Cisto Pancreático/cirurgia , Neoplasias Pancreáticas/cirurgia , Complicações Pós-Operatórias/epidemiologia , Adolescente , Criança , Pré-Escolar , Feminino , Humanos , Lactente , Masculino , Pancreatectomia , Neoplasias Pancreáticas/complicações , Pancreaticoduodenectomia , Paris/epidemiologia , Prevalência , Valores de Referência , Estudos Retrospectivos , Resultado do TratamentoRESUMO
The prenatal diagnosis of abdominal mass poses the problem of its origin. Renal tumors are rarer than neuroblastoma but they are most often congenital mesoblastic nephroma. The congenital mesoblastic nephroma has a good forecast in spite of a sonographic impressive aspect. MRI can help to locate tumor but cannot tell difference between the different kinds of renal tumor. Prenatal forecast is especially linked with hydramnios and hydrops fetalis. Histolological study of the tumor is important for the prognosis. Two morphological subtypes are currently distinguished: the classic type with a good forecast and the atypical or cellular type. Distant metastases have been related only to the cellular form but especially in infants aged more than 3 months and never in the newborns. The diagnosis of the tumor does not change the mode of delivery except in case of an important volume. Complications are searched during the first days of life: hypertension, hypercalcemia, vomiting, hyperreninemia. Radical nephrectomy is performed after the end of the first week. In case of a classic form, the healing is always obtained. In case of cellular form, distant metastases are searched. In any rate, the follow-up is recommended until the end of the growth.
Assuntos
Neoplasias Renais/diagnóstico por imagem , Nefroma Mesoblástico/diagnóstico por imagem , Abdome/diagnóstico por imagem , Abdome/embriologia , Diagnóstico Diferencial , Feminino , Humanos , Lactente , Recém-Nascido , Neoplasias Renais/mortalidade , Neoplasias Renais/patologia , Neoplasias Renais/cirurgia , Nefroma Mesoblástico/mortalidade , Nefroma Mesoblástico/patologia , Nefroma Mesoblástico/cirurgia , Gravidez , Terceiro Trimestre da Gravidez , Resultado do Tratamento , UltrassonografiaAssuntos
Carcinoma Hepatocelular/diagnóstico por imagem , Carcinoma Hepatocelular/cirurgia , Neoplasias Hepáticas/diagnóstico por imagem , Neoplasias Hepáticas/cirurgia , Ultrassonografia/métodos , Carcinoma Hepatocelular/patologia , Criança , Feminino , Humanos , Aumento da Imagem , Neoplasias Hepáticas/patologia , Sensibilidade e Especificidade , Resultado do TratamentoRESUMO
BACKGROUND: The purpose of this study was to evaluate the technical difficulties and complications of a transanal pull-through for Hirschsprung's disease. MATERIAL AND METHODS: This report was based on a multicentric retrospective study of 65 cases. Pull-through procedures were transanal Swenson or Soave procedures in 26 and 39 cases, respectively. RESULTS: Evaluation of the aganglionic level, peri-rectal dissection, and anastomosis were the three steps in the procedure where surgeons encountered difficulties. Such difficulties led to serious complications in 3 cases. A patient with a colon biopsy before the pull-through procedure had a postoperative pneumoperitoneum requiring a second laparoscopy for suture and washing. Another patient had peritonitis due to anastomotic leakage. Finally, a difficult rectal dissection in a neonate led to a urethral injury requiring secondary urethral repair. Only 41 of the 65 patients had no abdominal scars (63 %). CONCLUSION: We considered the transanal pull-through for Hirschsprung's disease to be a reliable technique. Nevertheless, it requires an urethral stent, precise dissection, careful anastomosis and selected indications in order to avoid major complications.
Assuntos
Procedimentos Cirúrgicos do Sistema Digestório/efeitos adversos , Doença de Hirschsprung/cirurgia , Complicações Intraoperatórias , Complicações Pós-Operatórias , Anastomose Cirúrgica/efeitos adversos , Criança , Pré-Escolar , Cicatriz , Feminino , Humanos , Lactente , Recém-Nascido , Obstrução Intestinal/etiologia , Masculino , Peritonite/etiologia , Pneumoperitônio/etiologia , Reoperação , Estudos Retrospectivos , Uretra/lesõesRESUMO
BACKGROUND: In order to lower the long-term toxicity of chemotherapy for hepatoblastoma patients, a prospective study was designed based on pre-operative chemotherapy combining carboplatin and epirubicin (CE). PROCEDURES: Patients under 16 years of age with an epithelial hepatic tumor diagnosed by ultrasound or CT scan and a high serum alpha-foetoprotein (AFP) level were eligible. Patients were treated with a pre-operative chemotherapy regimen combining carboplatin 600 mg/m(2) and epirubicin 80 mg/m(2). Tumor resectability was assessed after four courses given at 3-week intervals. After surgery, patients were given two more courses of CE. Response was assessed based on a drop in serum AFP and tumor shrinkage. RESULTS: Between July 1988 and August 1995, 27 patients with a hepatoblastoma were included. The initial PRETEXT group according to the SIOPEL classification was: group 2 (5 pts), group 3 (15 pts), group 4 (5 pts), and 2 pts were not assessed. Six patients had lung metastases. Response was partial response (PR) in 20/27 (74%) patients, disease was stable in 3 and 4 had progressive disease (PD). A complete surgical resection was performed in 21 pts. Five-year overall and disease-free survival (DFS) were respectively 56% (95%CI: 37-72%) and 63% (95%CI: 44-78%). During the same time period, 7 pts with a hepatocellular carcinoma were treated according to this protocol. Only one achieved a PR. Toxicity was mostly hematologic with > or =grade 3 leukopenia in 23% of the courses, > or =grade 3 thrombocytopenia in 29% of the courses and anemia in 22%. CONCLUSION: The CE protocol is feasible and efficient in hepatoblastoma. However, only a randomized study will permit a valid comparison of the efficacy of cisplatin and carboplatin for the treatment of these patients.
Assuntos
Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Hepatoblastoma/tratamento farmacológico , Neoplasias Hepáticas/tratamento farmacológico , Adolescente , Protocolos de Quimioterapia Combinada Antineoplásica/efeitos adversos , Carboplatina/administração & dosagem , Criança , Pré-Escolar , Epirubicina/administração & dosagem , Hepatoblastoma/cirurgia , Humanos , Lactente , Neoplasias Hepáticas/cirurgia , Neoplasias Pulmonares/tratamento farmacológico , Neoplasias Pulmonares/secundário , Neoplasias Pulmonares/cirurgia , Estadiamento de Neoplasias , Neoplasias Epiteliais e Glandulares/tratamento farmacológico , Neoplasias Epiteliais e Glandulares/cirurgia , Estudos Prospectivos , Taxa de Sobrevida , Resultado do Tratamento , alfa-Fetoproteínas/metabolismoRESUMO
CASE REPORT: A two-month-old male child presented a severe heart failure associated with a malignant hypertension. Abdominal ultrasound revealed a mass connected to the left adrenal gland. CT scan showed a tumor of 7 x 6 x 8 cm, forcing back both the left kidney and the aorta. A biopsy allowed the diagnosis of neuroblastoma without MYCN oncogene amplification. Intensive care stabilized the hemodynamic situation. Under chemotherapy the tumor volume decreased significantly and complete surgical excision became possible. Three years after diagnosis, the patient remained in complete remission. CONCLUSION: Clinical presentation of this neuroblastoma was extremely uncommon. The catecholamines produced by the tumoral cells could induce an increase of the myocardiac work following the left ventricule post charge increase. Theses mechanisms could be synergistic for a myocardial exhaustion.
Assuntos
Neoplasias das Glândulas Suprarrenais/complicações , Neoplasias das Glândulas Suprarrenais/diagnóstico , Hipertensão Maligna/etiologia , Neuroblastoma/complicações , Neuroblastoma/diagnóstico , Choque Cardiogênico/etiologia , Neoplasias das Glândulas Suprarrenais/tratamento farmacológico , Neoplasias das Glândulas Suprarrenais/cirurgia , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Biópsia , Catecolaminas/metabolismo , Diagnóstico Diferencial , Hemodinâmica , Humanos , Hipertensão Maligna/complicações , Lactente , Masculino , Neuroblastoma/tratamento farmacológico , Neuroblastoma/cirurgiaRESUMO
Clinical charts of 80 infants younger than 1 year who presented over a 14-year period (1986 to 2000) with acute liver failure (ALF), defined as prolonged prothrombin time greater than 17 seconds and decrease of clotting factor V plasma level below 50% of normal, were reviewed retrospectively. The main causes of ALF were inherited metabolic disorders in 42.5% of cases, including mitochondrial respiratory chain disorders in 17, type I hereditary tyrosinemia in 12, and urea cycle disorders in 2; neonatal hemochromatosis in 16% of cases; and acute viral hepatitis in 15% of cases (hepatitis B in 6, herpes virus type 6 in 4, and herpes simplex virus type 1 in 2). The cause of ALF remained undetermined in 16% of cases. A total of 19 (24%) infants survived without orthotopic liver transplantation; 38 (47%) infants died from sepsis, multiple organ failure, or because the underlying disease contraindicated orthotopic liver transplantation (12 [15%] infants), and 23 (29%) infants underwent orthotopic liver transplantation within 12 months from onset, 12 of whom are alive with a mean follow-up period of 5.2 years from orthotopic liver transplantation. We conclude that ALF during the first year of life is a severe condition with poor prognosis, despite the advent of liver transplantation.
Assuntos
Hospitais Pediátricos , Falência Hepática Aguda/mortalidade , Falência Hepática Aguda/cirurgia , Transplante de Fígado , Humanos , Lactente , Recém-Nascido , Falência Hepática Aguda/fisiopatologia , Prognóstico , Estudos Retrospectivos , Taxa de Sobrevida , Fatores de Tempo , Resultado do TratamentoRESUMO
The surgical treatment of malignant renal tumours in children (nephroblastomas) is part of a multimodal therapeutic strategy, defined by the International Society of Paediatric Oncology protocol. Resection of the primary tumour and determination of the post-operative stage determine subsequent treatment. The modalities of the surgical procedure are described together with the particular cases concerning infants under the age of 6 months, extension to the renal vein and inferior vena cava, emergency surgery, bilateral, tumours or tumour in a solitary kidney, lymph node involvement, metastatic nephroblastomas and complications of surgery.
Assuntos
Neoplasias Renais/cirurgia , Cirurgia Geral , Humanos , Lactente , PediatriaRESUMO
Wnt/beta-catenin signaling is frequently activated in cancer cells by stabilizing mutations of beta-catenin or loss-of-function mutations of the APC tumor suppressor gene. We have analysed the role of beta-catenin in the pathogenesis of hepatoblastoma (HB), an embryonic liver tumor occurring mainly in children under 2 years of age. Sequence analysis of the beta-catenin NH2-terminal domain in 18 epithelial and mixed HBs revealed missense mutations in the GSK3beta phosphorylation motif or interstitial deletions in 12 tumors (67%). In the remaining cases, no truncating mutation of APC could be evidenced. Immunohistochemical analysis of beta-catenin in 11 HBs demonstrated nuclear/cytoplasmic accumulation of the protein in all tumors analysed, with predominant nuclear beta-catenin immunostaining in undifferentiated cells. Membranous beta-catenin localization was preserved only in fetal-type tumoral hepatocytes and was associated with E-cadherin expression. Moreover, we show that beta-catenin is aberrantly overexpressed in a large spectrum of tumor components, including hepatocyte-like cells at various differentiation stages and heterologous elements such as squamous, osteoid and chrondroid tissues, and in occasional other mesenchymally-derived cells. These data strongly suggest that activation of beta-catenin signaling is an obligatory step in HB pathogenesis, and raise the possibility that it interferes with developmental signals that specify different tissue types at early stages of hepatic differentiation.
Assuntos
Proteínas do Citoesqueleto/genética , Hepatoblastoma/genética , Neoplasias Hepáticas/genética , Mutação , Proteínas de Neoplasias/genética , Transativadores , Transporte Biológico , Caderinas/biossíntese , Caderinas/genética , Proteínas Quinases Dependentes de Cálcio-Calmodulina/metabolismo , Diferenciação Celular , Linhagem Celular , Linhagem da Célula , Pré-Escolar , Códon/genética , Proteínas do Citoesqueleto/metabolismo , Análise Mutacional de DNA , DNA de Neoplasias/genética , Células Epiteliais/metabolismo , Feminino , Quinase 3 da Glicogênio Sintase , Hepatoblastoma/embriologia , Hepatoblastoma/metabolismo , Hepatoblastoma/patologia , Humanos , Lactente , Recém-Nascido , Fígado/embriologia , Neoplasias Hepáticas/embriologia , Neoplasias Hepáticas/metabolismo , Neoplasias Hepáticas/patologia , Masculino , Mesoderma/metabolismo , Proteínas de Neoplasias/metabolismo , Fosforilação , Mutação Puntual , Processamento de Proteína Pós-Traducional/genética , Estrutura Terciária de Proteína , Deleção de Sequência , Transdução de Sinais , Transfecção , beta CateninaRESUMO
UNLABELLED: This study evaluates the results of paediatric liver transplantation (PLT) with split liver grafts at Bicêtre hospital. PATIENTS AND METHODS: Between January 1, 1988 and December 31, 1995, 205 PLT were performed in 180 children. One auxiliary PLT was excluded from the study. The graft was a whole (WLG), reduced (RLG) and split liver graft (SLG) in 76, 112 and 16 cases respectively. The SLG consisted of segments II + III in 14 cases, and II + III + IV in 2 cases. Results of PLT with SLT, RLT and WLG were retrospectively compared. Minimal follow-up was 12 months. RESULTS: In elective PLT, actual 1 year patient (graft) survival were 93.3% (84.4%) with WLG (n = 64), 84.1% (76.4%) with RLG (n = 72), 81.8% (81.8%) with SLG (n = 11). In urgent LT, actual patient (graft) survival were 100% (83.3%) with WLG (n = 6), 58.6% (52.5%) with RLG (n = 40), 25% (20%) with SLG (n = 5). Specific complications of the splitting technique were: 2 Budd-Chiari syndromes in 2 early patients, without any new case after modification of the technique of left hepatic vein to inferior vena cava anastomosis; 4 bile leaks (25%) from the left hepatic duct to Roux-en-Y loop anastomosis. DISCUSSION: In our experience, the results of PLT with SLG were satisfactory in the elective situation, but disappointing in urgent cases. In the current context of liver graft shortage, appropriate use of this sophisticated and demanding technique depends on the experience of the team, recipient's condition, and logistic considerations.