Your browser doesn't support javascript.
loading
Mostrar: 20 | 50 | 100
Resultados 1 - 20 de 39
Filtrar
1.
N Engl J Med ; 378(25): 2376-2385, 2018 Jun 21.
Artigo em Inglês | MEDLINE | ID: mdl-29924955

RESUMO

BACKGROUND: Cisplatin chemotherapy and surgery are effective treatments for children with standard-risk hepatoblastoma but may cause considerable and irreversible hearing loss. This trial compared cisplatin with cisplatin plus delayed administration of sodium thiosulfate, aiming to reduce the incidence and severity of cisplatin-related ototoxic effects without jeopardizing overall and event-free survival. METHODS: We randomly assigned children older than 1 month and younger than 18 years of age who had standard-risk hepatoblastoma (≤3 involved liver sectors, no metastatic disease, and an alpha-fetoprotein level of >100 ng per milliliter) to receive cisplatin alone (at a dose of 80 mg per square meter of body-surface area, administered over a period of 6 hours) or cisplatin plus sodium thiosulfate (at a dose of 20 g per square meter, administered intravenously over a 15-minute period, 6 hours after the discontinuation of cisplatin) for four preoperative and two postoperative courses. The primary end point was the absolute hearing threshold, as measured by pure-tone audiometry, at a minimum age of 3.5 years. Hearing loss was assessed according to the Brock grade (on a scale from 0 to 4, with higher grades indicating greater hearing loss). The main secondary end points were overall survival and event-free survival at 3 years. RESULTS: A total of 109 children were randomly assigned to receive cisplatin plus sodium thiosulfate (57 children) or cisplatin alone (52) and could be evaluated. Sodium thiosulfate was associated with few high-grade toxic effects. The absolute hearing threshold was assessed in 101 children. Hearing loss of grade 1 or higher occurred in 18 of 55 children (33%) in the cisplatin-sodium thiosulfate group, as compared with 29 of 46 (63%) in the cisplatin-alone group, indicating a 48% lower incidence of hearing loss in the cisplatin-sodium thiosulfate group (relative risk, 0.52; 95% confidence interval [CI], 0.33 to 0.81; P=0.002). At a median of 52 months of follow-up, the 3-year rates of event-free survival were 82% (95% CI, 69 to 90) in the cisplatin-sodium thiosulfate group and 79% (95% CI, 65 to 88) in the cisplatin-alone group, and the 3-year rates of overall survival were 98% (95% CI, 88 to 100) and 92% (95% CI, 81 to 97), respectively. CONCLUSIONS: The addition of sodium thiosulfate, administered 6 hours after cisplatin chemotherapy, resulted in a lower incidence of cisplatin-induced hearing loss among children with standard-risk hepatoblastoma, without jeopardizing overall or event-free survival. (Funded by Cancer Research UK and others; SIOPEL 6 ClinicalTrials.gov number, NCT00652132 ; EudraCT number, 2007-002402-21 .).


Assuntos
Cisplatino/efeitos adversos , Perda Auditiva/prevenção & controle , Hepatoblastoma/tratamento farmacológico , Neoplasias Hepáticas/tratamento farmacológico , Tiossulfatos/uso terapêutico , Adolescente , Criança , Pré-Escolar , Cisplatino/administração & dosagem , Doxorrubicina/administração & dosagem , Quimioterapia Combinada , Feminino , Seguimentos , Taxa de Filtração Glomerular , Perda Auditiva/induzido quimicamente , Hepatoblastoma/mortalidade , Humanos , Incidência , Lactente , Neoplasias Hepáticas/mortalidade , Masculino , Método Simples-Cego , Análise de Sobrevida , Tiossulfatos/administração & dosagem , Tiossulfatos/efeitos adversos
2.
Childs Nerv Syst ; 37(5): 1547-1561, 2021 05.
Artigo em Inglês | MEDLINE | ID: mdl-33665678

RESUMO

PURPOSE: Endoscopic extended transsphenoidal surgery (EETSS) has gained popularity for treatment of craniopharyngiomas. The aim of this study is to assess the outcome of endoscopic extended transsphenoidal surgery (EETSS) for newly diagnosed paediatric craniopharyngiomas. METHODS: Patient details were obtained from a prospective database of all endoscopic transnasal operations performed by a single surgeon. Outcomes including visual function, pituitary function, body mass index (BMI), postoperative neurological deficit, extent of resection and recurrence on follow-up were obtained. Obesity was defined as BMI percentile of equal to or greater than 95%. RESULTS: Between January 2011 and January 2020, 15 of 16 children (5-18 years old) with newly diagnosed craniopharyngiomas underwent EETSS. Four patients had a conchal-type sphenoid sinus. Gross total resection (GTR) was achieved in 4 patients and near total resection (NTR) in 5 patients. The remaining 6 had subtotal resection (STR). Postoperative radiotherapy was used in 6 patients (4 with STR, 2 with NTR). There were no postoperative deaths, strokes or CSF leaks. Normalisation of visual fields (VF) occurred in 9/13 patients with preoperative VF defects. One patient developed a new visual field defect. During a median follow-up period of 74 (8-104) months, 2 patients have required further surgery for tumour progression following initial STR, where a tumour remnant was left in situ to preserve the pituitary stalk. 6/11 patients developed new anterior pituitary dysfunction as a result of surgery and 9/12 developed new diabetes insipidus (DI). At the time of last follow-up, 14/15 children had anterior panhypopituitarism, 13/15 had DI and 1 patient developed new onset obesity. Two patients, who were obese preoperatively, were no longer obese at last follow-up. CONCLUSIONS: EETSS can be performed as the first option in the majority of children with newly diagnosed craniopharyngioma, despite factors such as small nose, non-pneumatised sphenoid sinus, small sella or purely suprasellar tumour location. Preservation of the pituitary stalk at the expense of leaving residual tumour may not be in the best interests of the patient.


Assuntos
Craniofaringioma , Neuroendoscopia , Neoplasias Hipofisárias , Adolescente , Criança , Pré-Escolar , Craniofaringioma/diagnóstico por imagem , Craniofaringioma/cirurgia , Humanos , Recidiva Local de Neoplasia/cirurgia , Neoplasias Hipofisárias/diagnóstico por imagem , Neoplasias Hipofisárias/cirurgia , Complicações Pós-Operatórias , Estudos Retrospectivos , Resultado do Tratamento
3.
Skeletal Radiol ; 48(6): 995-998, 2019 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-30374635

RESUMO

Scurvy is a disease that is rarely encountered in modern medicine. A condition that was classically associated with sailors, its incidence has decreased dramatically since the discovery of its association with vitamin C deficiency. We present the case of a 2-year-old boy, whose treatment for neuroblastoma was complicated by gastrointestinal disease, which necessitated enteral feeding. While still undergoing treatment, he started to complain about increasing pain in his lower limbs, which appeared to be markedly tender on palpation. Radiographic findings suggested a diagnosis of scurvy, which was subsequently confirmed on serum biochemistry. This was an unexpected finding, as the child had been receiving adequate vitamin C in his enteral feeds. However, his absorption had become severely impaired due to pseudomembranous gastritis and enteritis, leading to his deficient state. He significantly improved after intravenous ascorbic acid replacement and demonstrated a full recovery, both clinically and radiologically. This case highlights the importance of considering scurvy in the differential diagnosis for at-risk patients. Early recognition can facilitate the simple treatment of this potentially serious condition.


Assuntos
Perna (Membro) , Escorbuto/diagnóstico por imagem , Escorbuto/etiologia , Ácido Ascórbico/uso terapêutico , Diagnóstico Diferencial , Nutrição Enteral , Enterite/complicações , Gastrite/complicações , Humanos , Lactente , Masculino , Neuroblastoma/tratamento farmacológico , Escorbuto/tratamento farmacológico
4.
Pediatr Blood Cancer ; 65(12): e27386, 2018 12.
Artigo em Inglês | MEDLINE | ID: mdl-30230225

RESUMO

BACKGROUND: High-risk neuroblastoma (HR NBL) treatment requires intensive induction chemotherapy. The profoundly emetogenic agents used can compromise nutritional status. Our institution introduced a new antiemetic guideline in 2010 incorporating regular dexamethasone, in addition to ondansetron, for all highly emetogenic protocols. PROCEDURE: A retrospective comparative review of pediatric patients diagnosed with HR NBL who received rapid COJEC induction chemotherapy as per HR-SIOPEN NBL trial. Prophylactic antiemetics were prescribed regardless of chemotherapy emetogenicity in group A (2004-2010) but for defined time periods considering chemotherapy emetogenicity in group B (2010-2017). RESULTS: Sixty-three children were eligible for inclusion (median age, 31 months; range, 1-88 months). Group A had more episodes of emesis than group B (189 vs. 116, P < 0.0001). There was a significant difference in weight-for-age Z score change between the groups by induction end (P = 0.0027). Four children (13%) in group A lost >10% body weight versus none in group B. Nutrition support (NS) was utilized by 29 children (94%) in group A and 22 children (69%) in group B. Group A had a median of 3 (range, 1-7) admissions for febrile neutropenia (FN) versus a median of 1.5 (range, 0-4) for group B (P = 0.003) during induction. CONCLUSION: The review of our guidelines led to reduced emesis frequency for group B. They also required less NS, followed expected growth trajectories more closely and had fewer FN admissions. We propose that this may have occurred due to better emesis control resulting in improved nutritional status and associated enhanced immune function.


Assuntos
Antieméticos/uso terapêutico , Quimioterapia de Indução/efeitos adversos , Neuroblastoma/tratamento farmacológico , Estado Nutricional/efeitos dos fármacos , Vômito/prevenção & controle , Protocolos de Quimioterapia Combinada Antineoplásica/efeitos adversos , Criança , Pré-Escolar , Dexametasona/uso terapêutico , Feminino , Humanos , Lactente , Recém-Nascido , Masculino , Náusea/induzido quimicamente , Náusea/prevenção & controle , Ondansetron/uso terapêutico , Guias de Prática Clínica como Assunto , Estudos Retrospectivos , Vômito/induzido quimicamente
6.
J Pediatr Hematol Oncol ; 38(6): e191-2, 2016 08.
Artigo em Inglês | MEDLINE | ID: mdl-27322716

RESUMO

A male preterm infant was born with dysmorphic features consistent with Rubinstein-Taybi syndrome (RTS). An undescended right testicle was noted on examination. At 5 months of age he developed a palpable right-sided abdominal mass and an elevated alpha-fetoprotein. Histology revealed a malignant germ cell neoplasm arising within the undescended testis. This is the first reported case of a germ cell tumor occurring in a pediatric patient with RTS. Urologic abnormalities occur in approximately 52% of RTS patients, of which cryptorchidism is the commonest. Given the frequency of undescended testes in this population, closer screening may be warranted.


Assuntos
Criptorquidismo/complicações , Neoplasias Embrionárias de Células Germinativas/patologia , Síndrome de Rubinstein-Taybi/complicações , Neoplasias Testiculares/patologia , Humanos , Lactente , Masculino , Neoplasias Embrionárias de Células Germinativas/cirurgia , Neoplasias Testiculares/cirurgia
7.
Clin Cancer Res ; 30(16): 3395-3406, 2024 Aug 15.
Artigo em Inglês | MEDLINE | ID: mdl-38869831

RESUMO

Osteosarcoma and Ewing sarcoma are bone tumors mostly diagnosed in children, adolescents, and young adults. Despite multimodal therapy, morbidity is high and survival rates remain low, especially in the metastatic disease setting. Trials investigating targeted therapies and immunotherapies have not been groundbreaking. Better understanding of biological subgroups, the role of the tumor immune microenvironment, factors that promote metastasis, and clinical biomarkers of prognosis and drug response are required to make progress. A prerequisite to achieve desired success is a thorough, systematic, and clinically linked biological analysis of patient samples, but disease rarity and tissue processing challenges such as logistics and infrastructure have contributed to a lack of relevant samples for clinical care and research. There is a need for a Europe-wide framework to be implemented for the adequate and minimal sampling, processing, storage, and analysis of patient samples. Two international panels of scientists, clinicians, and patient and parent advocates have formed the Fight Osteosarcoma Through European Research consortium and the Euro Ewing Consortium. The consortia shared their expertise and institutional practices to formulate new guidelines. We report new reference standards for adequate and minimally required sampling (time points, diagnostic samples, and liquid biopsy tubes), handling, and biobanking to enable advanced biological studies in bone sarcoma. We describe standards for analysis and annotation to drive collaboration and data harmonization with practical, legal, and ethical considerations. This position paper provides comprehensive guidelines that should become the new standards of care that will accelerate scientific progress, promote collaboration, and improve outcomes.


Assuntos
Neoplasias Ósseas , Osteossarcoma , Sarcoma de Ewing , Manejo de Espécimes , Humanos , Osteossarcoma/terapia , Osteossarcoma/patologia , Osteossarcoma/diagnóstico , Sarcoma de Ewing/terapia , Sarcoma de Ewing/patologia , Sarcoma de Ewing/diagnóstico , Europa (Continente) , Neoplasias Ósseas/terapia , Neoplasias Ósseas/patologia , Manejo de Espécimes/métodos , Manejo de Espécimes/normas , Biomarcadores Tumorais , Bancos de Espécimes Biológicos
8.
Childs Nerv Syst ; 29(3): 367-74, 2013 Mar.
Artigo em Inglês | MEDLINE | ID: mdl-23319103

RESUMO

INTRODUCTION: Although uncommon, there is significant morbidity and mortality associated with paediatric spinal glioblastoma. The paucity of cases makes treatment options difficult. The current recommended standard of care is biopsy followed by adjuvant chemo-radiotherapy, with emerging data supporting the role of safe gross total resection. OBJECTIVE: The purpose of this paper is to provide a single-institution case study and to discuss current and future therapeutic treatment strategies. CASE PRESENTATION: A 14-year-old boy presented with a 2-year history of intermittent back pain with recent progressively worsening motor and sensory deficits of the right side. Pre-operative MRI revealed an enhancing intra-medullary tumour extending from C2 to C7. During the operative case, no tumour-cord margin could be identified, and the patient underwent a subtotal excision. Histopathology confirmed glioblastoma. In the subsequent weeks, the patient's clinical condition deteriorated. Adjuvant therapy was declined by the family, and the patient died 9 weeks after initial presentation. CONCLUSION: Despite major advances in surgical techniques, peri-operative neuro-imaging as well as chemo-radiotherapy, the prognosis of a paediatric intra-medullary high-grade spinal tumour remains poor. Detailed analysis of our understanding of tumour dynamics in this patient group is important in establishing future therapeutic strategies.


Assuntos
Glioblastoma/terapia , Neoplasias da Medula Espinal/terapia , Adolescente , Vértebras Cervicais , Evolução Fatal , Glioblastoma/diagnóstico por imagem , Glioblastoma/patologia , Humanos , Masculino , Radiografia , Neoplasias da Medula Espinal/diagnóstico por imagem , Neoplasias da Medula Espinal/patologia
9.
Neuro Oncol ; 24(6): 997-1007, 2022 06 01.
Artigo em Inglês | MEDLINE | ID: mdl-34850167

RESUMO

BACKGROUND: Larotrectinib is a first-in-class, highly selective tropomyosin receptor kinase (TRK) inhibitor approved to treat adult and pediatric patients with TRK fusion-positive cancer. The aim of this study was to evaluate the efficacy and safety of larotrectinib in patients with TRK fusion-positive primary central nervous system (CNS) tumors. METHODS: Patients with TRK fusion-positive primary CNS tumors from two clinical trials (NCT02637687, NCT02576431) were identified. The primary endpoint was investigator-assessed objective response rate (ORR). RESULTS: As of July 2020, 33 patients with TRK fusion-positive CNS tumors were identified (median age: 8.9 years; range: 1.3-79.0). The most common histologies were high-grade glioma (HGG; n = 19) and low-grade glioma (LGG; n = 8). ORR was 30% (95% confidence interval [CI]: 16-49) for all patients. The 24-week disease control rate was 73% (95% CI: 54-87). Twenty-three of 28 patients (82%) with measurable disease had tumor shrinkage. The 12-month rates for duration of response, progression-free survival, and overall survival were 75% (95% CI: 45-100), 56% (95% CI: 38-74), and 85% (95% CI: 71-99), respectively. Median time to response was 1.9 months (range 1.0-3.8 months). Duration of treatment ranged from 1.2-31.3+ months. Treatment-related adverse events were reported for 20 patients, with grade 3-4 in 3 patients. No new safety signals were identified. CONCLUSIONS: In patients with TRK fusion-positive CNS tumors, larotrectinib demonstrated rapid and durable responses, high disease control rate, and a favorable safety profile.


Assuntos
Antineoplásicos , Glioma , Neoplasias , Adulto , Antineoplásicos/uso terapêutico , Criança , Glioma/tratamento farmacológico , Humanos , Neoplasias/patologia , Proteínas de Fusão Oncogênica/genética , Inibidores de Proteínas Quinases/uso terapêutico , Pirazóis/uso terapêutico , Pirimidinas/farmacologia , Pirimidinas/uso terapêutico
10.
J Pediatr Hematol Oncol ; 33(6): e226-30, 2011 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-21792028

RESUMO

Despite high survival rates, many survivors of hepatoblastoma develop late effects including ototoxicity and cardiomyopathy. With the goal of minimizing long-term toxicities, our institution treated hepatoblastoma with continuous infusion of doxorubicin and cisplatinum (PLADO), rather than short infusion or bolus dosing as used in other treatment protocols. This retrospective cohort study includes consecutive patients diagnosed between 1985 and 2007. Patients were scheduled for treatment with 6 cycles of continuous infusion of PLADO with resection after the third or fourth cycle. Audiograms and echocardiograms were obtained at baseline, after every 2 chemotherapy cycles and yearly after the completion of therapy. Fifty-five patients were treated (34 localized; 21 metastatic). Fifty-one patients received at least 1 cycle of PLADO. Median follow-up was 7.0 years (range, 0.11 to 17.8 y). Event-free and overall survival for these 51 patients were 72.2% (standard error 6.3%) and 75.6% (standard error 6.2%) respectively. Of the 38 survivors treated with cisplatin who had an audiogram during follow-up, 4 (11%) demonstrated severe (Brock grade 3/4) and 13 (34%) mild (Brock grade 1/2) hearing loss. At a median of 10.0 years (range, 5.0 to 13.0 y) after therapy, 2 of 41 (5%) patients who were still alive had evidence of cardiac dysfunction. Overall, continuous infusion of PLADO therapy resulted in survival rates consistent with those observed in intergroup studies, but rates of chronic cardiac and ototoxicity did not differ sufficiently from those observed after shorter infusion of PLADO therapy to warrant the use of continuous infusions.


Assuntos
Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Hepatoblastoma/tratamento farmacológico , Neoplasias Hepáticas/tratamento farmacológico , Criança , Pré-Escolar , Cisplatino/administração & dosagem , Estudos de Coortes , Doxorrubicina/administração & dosagem , Feminino , Seguimentos , Hepatoblastoma/patologia , Humanos , Lactente , Recém-Nascido , Infusões Intravenosas , Neoplasias Hepáticas/patologia , Masculino , Estudos Retrospectivos , Taxa de Sobrevida , Resultado do Tratamento
11.
Fetal Pediatr Pathol ; 30(3): 156-60, 2011.
Artigo em Inglês | MEDLINE | ID: mdl-21355680

RESUMO

Congenital fibrosarcoma is a rare, soft tissue malignancy of infancy, most commonly involving the distal extremities. We report a case of congenital fibrosarcoma of the ileum in a 5-day-old boy who presented with an acute abdomen due to ileal perforation. Partial ileal resection was carried out with successful anastomosis. Grossly, the resected small bowel showed focal luminal stenosis with a thickened, indurated wall. Histology showed a transmural primitive spindle cell proliferation with a morphology consistent with congenital fibrosarcoma. The associated hallmark chromosomal translocation t(12;15)(q13;q25) was demonstrated by reverse transcriptase polymerase chain reaction.


Assuntos
Fibrossarcoma/congênito , Neoplasias do Íleo/congênito , Cromossomos Humanos Par 12/genética , Cromossomos Humanos Par 15/genética , Fibrossarcoma/genética , Fibrossarcoma/patologia , Fibrossarcoma/cirurgia , Humanos , Neoplasias do Íleo/genética , Neoplasias do Íleo/patologia , Neoplasias do Íleo/cirurgia , Recém-Nascido , Perfuração Intestinal/etiologia , Perfuração Intestinal/cirurgia , Masculino , Fusão Oncogênica , Proteínas de Fusão Oncogênica/genética , Translocação Genética
12.
Ir J Med Sci ; 189(4): 1223-1236, 2020 Nov.
Artigo em Inglês | MEDLINE | ID: mdl-32424602

RESUMO

BACKGROUND: Some studies indicate that survival of adolescents and young adults (AYA) with cancer may be inferior to that of younger children with similar cancers, possibly related (in part) to differences in access to centralized or standardized treatment. AIMS: This study aims to evaluate differences in survival for AYA patients when compared with paediatric patients treated in Ireland over a 20-year time period. METHODS: This study compares relative survival for patients diagnosed in Ireland at ages 0-15 (paediatric group) and 16-24 (AYA group) during 1994-2013, followed to the end of 2014, for cancers defined by the International Classification of Childhood Cancer (ICCC) (Third Edition) group or subgroup. Five-year relative survival estimates, and excess hazard ratios (EHR) comparing excess mortality associated with a cancer diagnosis among AYA with that in the paediatric group, are presented. This research did not receive any specific grant from funding agencies in the public, commercial, or not-for-profit sectors. RESULTS: Significantly higher excess mortality was found for AYA with leukaemias, lymphomas, astrocytomas, malignant bone tumours, and Ewing and related bone sarcomas, soft tissue sarcomas and 'other/unspecified' epithelial cancers, rhabdomyosarcomas, and 'other and unspecified' carcinomas. In contrast, lower excess mortality was found in the AYA group for all cancers and intracranial/intraspinal tumours, and for gliomas other than astrocytomas or ependymomas. Comparing 1994-2003 and 2004-2013 cohorts, age-related survival differences narrowed for lymphoid leukaemias, but widened for all cancers combined and intracranial/intraspinal tumours combined. Centralization of services varied depending upon cancer subtype, with leukaemias, CNS tumours and bone sarcomas most centralized. Within these, improvements in survival for leukaemias and CNS tumours have been seen for the AYA population. CONCLUSIONS: Reasons for age-related survival differences, and differences in time-trend by age group, are not clear. The significant narrowing of survival differences by age in more recent years for lymphoid leukaemias reflects a more marked recent increase in survival among AYA. More work is required to explain and improve other age-related survival differences.


Assuntos
Neoplasias/mortalidade , Adolescente , Fatores Etários , Criança , Pré-Escolar , Feminino , História do Século XX , História do Século XXI , Humanos , Lactente , Recém-Nascido , Irlanda , Masculino , Análise de Sobrevida , Fatores de Tempo , Adulto Jovem
13.
Childs Nerv Syst ; 25(10): 1293-301, 2009 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-19360417

RESUMO

INTRODUCTION: Chemotherapy has limited role in the up-front management of ependymoma. At the time of recurrence, the role of chemotherapy is also ill defined and the choice of chemotherapeutic agents is often arbitrary, based on anecdotal data and personal experience. METHODS: The purpose of this review is to describe and critically analyze the published literature on chemotherapy in patients with recurrent and metastatic ependymoma. DISCUSSION: The disappointing response rate with single agents (12.9%) and combinations (17.4%) emphasizes the need to re-evaluate the current chemotherapeutic approach of intracranial ependymoma, and biological studies are needed to identify targets that may be considered for clinical trials.


Assuntos
Ependimoma/tratamento farmacológico , Metástase Neoplásica/tratamento farmacológico , Recidiva Local de Neoplasia/tratamento farmacológico , Terapia de Salvação/métodos , Neoplasias Encefálicas/tratamento farmacológico , Criança , Humanos , Neoplasias da Medula Espinal/tratamento farmacológico
14.
J Plast Reconstr Aesthet Surg ; 72(8): 1388-1395, 2019 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-31101426

RESUMO

INTRODUCTION: Malignant melanoma is increasing in frequency worldwide; however, this disease is rare in children. As large-scale studies on paediatric melanoma are lacking, management is currently often based upon the understanding of the disease process in adults. The aim of this study was to characterise cases of paediatric melanoma diagnosed in the Republic of Ireland over a 21-year period. METHODS: This was a retrospective, multicentre study using national data provided by the National Cancer Registry of Ireland and individual practitioners. RESULTS: Twenty-four cases of melanoma treated in 11 different centres were included in the study. The median patient age at diagnosis was 15 years. The majority of cases arose on the limbs. The median Breslow thickness in patients of the pre-pubertal age group was 8.25 mm, while in children more than 13 years, it was 1.65 mm. Eight patients had disease recurrence and five patients died. CONCLUSION: The diagnosis of melanoma remains rare in children. This study contributes to our current understanding of malignant melanoma in paediatric patients; however, further investigation of the disease characteristics in this group is necessary to achieve optimal management of these cases and therefore improve outcomes.


Assuntos
Melanoma/epidemiologia , Neoplasias Cutâneas/epidemiologia , Adolescente , Criança , Pré-Escolar , Feminino , Humanos , Incidência , Lactente , Irlanda/epidemiologia , Metástase Linfática , Masculino , Margens de Excisão , Melanoma/patologia , Melanoma/cirurgia , Metástase Neoplásica , Estadiamento de Neoplasias , Recidiva , Sistema de Registros , Estudos Retrospectivos , Fatores de Risco , Biópsia de Linfonodo Sentinela , Neoplasias Cutâneas/patologia , Neoplasias Cutâneas/cirurgia , Resultado do Tratamento , Melanoma Maligno Cutâneo
15.
Eur J Cancer ; 109: 36-50, 2019 03.
Artigo em Inglês | MEDLINE | ID: mdl-30685685

RESUMO

BACKGROUND: High-grade osteosarcoma is a primary malignant bone tumour mainly affecting children and young adults. The European and American Osteosarcoma Study (EURAMOS)-1 is a collaboration of four study groups aiming to improve outcomes of this rare disease by facilitating randomised controlled trials. METHODS: Patients eligible for EURAMOS-1 were aged ≤40 years with M0 or M1 skeletal high-grade osteosarcoma in which case complete surgical resection at all sites was deemed to be possible. A three-drug combination with methotrexate, doxorubicin and cisplatin was defined as standard chemotherapy, and between April 2005 and June 2011, 2260 patients were registered. We report survival outcomes and prognostic factors in the full cohort of registered patients. RESULTS: For all registered patients at a median follow-up of 54 months (interquartile range: 38-73) from biopsy, 3-year and 5-year event-free survival were 59% (95% confidence interval [CI]: 57-61%) and 54% (95% CI: 52-56%), respectively. Multivariate analyses showed that the most adverse factors at diagnosis were pulmonary metastases (hazard ratio [HR] = 2.34, 95% CI: 1.95-2.81), non-pulmonary metastases (HR = 1.94, 95% CI: 1.38-2.73) or an axial skeleton tumour site (HR = 1.53, 95% CI: 1.10-2.13). The histological subtypes telangiectatic (HR = 0.52, 95% CI: 0.33-0.80) and unspecified conventional (HR = 0.67, 95% CI: 0.52-0.88) were associated with a favourable prognosis compared with chondroblastic subtype. The 3-year and 5-year overall survival from biopsy were 79% (95% CI: 77-81%) and 71% (95% CI: 68-73%), respectively. For patients with localised disease at presentation and in complete remission after surgery, having a poor histological response was associated with worse outcome after surgery (HR = 2.13, 95% CI: 1.76-2.58). In radically operated patients, there was no good evidence that axial tumour site was associated with worse outcome. CONCLUSIONS: In conclusion, data from >2000 patients registered to EURAMOS-1 demonstrated survival rates in concordance with institution- or group-level osteosarcoma trials. Further efforts are required to drive improvements for patients who can be identified to be at higher risk of adverse outcome. This trial reaffirms known prognostic factors, and owing to the large numbers of patients registered, it sheds light on some additional factors to consider.


Assuntos
Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Neoplasias Ósseas/mortalidade , Osteossarcoma/mortalidade , Adolescente , Adulto , Neoplasias Ósseas/tratamento farmacológico , Neoplasias Ósseas/secundário , Criança , Cisplatino/administração & dosagem , Estudos de Coortes , Doxorrubicina/administração & dosagem , Feminino , Seguimentos , Humanos , Masculino , Metotrexato/administração & dosagem , Metástase Neoplásica , Osteossarcoma/tratamento farmacológico , Osteossarcoma/patologia , Prognóstico , Taxa de Sobrevida
16.
Eur J Cancer ; 43(7): 1171-9, 2007 May.
Artigo em Inglês | MEDLINE | ID: mdl-17379506

RESUMO

BACKGROUND: The aim of United Kingdom Children's Cancer Study Group (UKCCSG) HD82 was to establish the efficacy of chlorambucil/vinblastine/procarbazine/prednisolone (ChlVPP) in the treatment of childhood Hodgkin's lymphoma stages II-IV and radiotherapy (RT) alone in stage I patients. We report on the status of these patients to a follow-up of 20 years. METHODS: Treatment consisted of 35Gy involved-field RT for stage I and ChlVPP alone for stages II-IV. Adjuvant RT (35Gy) was administered to those with bulky mediastinal disease. RESULTS: Of the 358 patients, the 10-year EFS/OS per stage is I (65.4%/92.6%), II (80.0%/93.3%), III (68.8%/85.0%), IV (45.5%/72.7%). The corresponding 20-year OS rates are similar with a combined (all stage) rate dropping from 89.3% to 89.0% over the decade. The cumulative 20-year malignancy rate is 7.29%. CONCLUSION: Single modality treatment provided relatively low EFS at 10-years but comparable long-term OS, relative to contemporary published combined modality regimens, for stages I-III but not for stage IV patients.


Assuntos
Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Doença de Hodgkin/tratamento farmacológico , Adolescente , Protocolos de Quimioterapia Combinada Antineoplásica/administração & dosagem , Criança , Pré-Escolar , Clorambucila/administração & dosagem , Intervalo Livre de Doença , Seguimentos , Doença de Hodgkin/mortalidade , Doença de Hodgkin/radioterapia , Humanos , Lactente , Recém-Nascido , Recidiva Local de Neoplasia/mortalidade , Segunda Neoplasia Primária/mortalidade , Prednisolona/administração & dosagem , Procarbazina/administração & dosagem , Radioterapia Adjuvante , Resultado do Tratamento , Vimblastina/administração & dosagem
18.
Neuro Oncol ; 19(10): 1398-1407, 2017 Oct 01.
Artigo em Inglês | MEDLINE | ID: mdl-28499018

RESUMO

BACKGROUND: Craniopharyngiomas are frequent hypothalamo-pituitary tumors in children, presenting predominantly as cystic lesions. Morbidity from conventional treatment has focused attention on intracystic drug delivery, hypothesized to cause fewer clinical consequences. However, the efficacy of intracystic therapy remains unclear. We report the retrospective experiences of several global centers using intracystic interferon-alpha. METHODS: European Société Internationale d'Oncologie Pédiatrique and International Society for Pediatric Neurosurgery centers were contacted to submit a datasheet capturing pediatric patients with cystic craniopharyngiomas who had received intracystic interferon-alpha. Patient demographics, administration schedules, adverse events, and outcomes were obtained. Progression was clinical or radiological (cyst reaccumulation, novel cysts, or solid growth). RESULTS: Fifty-six children (median age, 6.3 y) from 21 international centers were identified. Median follow-up from diagnosis was 5.1 years (0.3-17.7 y). Lesions were cystic (n = 22; 39%) or cystic/solid (n = 34; 61%). Previous progression was treated in 43 (77%) patients before interferon use. In such cases, further progression was delayed by intracystic interferon compared with the preceding therapy for cystic lesions (P = 0.0005). Few significant attributable side effects were reported. Progression post interferon occurred in 42 patients (median 14 mo; 0-8 y), while the estimated median time to definitive therapy post interferon was 5.8 (1.8-9.7) years. CONCLUSIONS: Intracystic interferon-alpha can delay disease progression and potentially offer a protracted time to definitive surgery or radiotherapy in pediatric cystic craniopharyngioma, yet demonstrates a favorable toxicity profile compared with other therapeutic modalities-important factors for this developing age group. A prospective, randomized international clinical trial assessment is warranted.


Assuntos
Craniofaringioma/radioterapia , Interferon-alfa/metabolismo , Neoplasias Hipofisárias/radioterapia , Adolescente , Criança , Pré-Escolar , Craniofaringioma/metabolismo , Feminino , Humanos , Injeções Intralesionais/métodos , Masculino , Estudos Retrospectivos
19.
Eur J Oncol Nurs ; 10(3): 198-206, 2006 Jul.
Artigo em Inglês | MEDLINE | ID: mdl-16384746

RESUMO

The ultimate aim of our research program is to provide strategies that facilitate parental decision-making for parents of children with cancer receiving end-of-life care. As a first step to develop this program, we needed insight into parents' reactions and opinions about the research methods planned for a larger study. In particular, we needed their opinions about the general experience of making the decision between palliative cytotoxic chemotherapy and supportive care alone and the factors that parents regard as important when making this decision. In addition, we wished to know whether the methodology proposed for the future study was easy to understand and whether it might cause unnecessary emotional trauma. Finally, we asked their opinions regarding the appropriate target sample of parents to include in the future study. Qualitative data about these issues were collected using focus group methodology involving seven participants. The comments made during the focus group discussions were content-analyzed for common themes. The results from the focus group discussion led to particular modifications in the proposed design and interview strategies planned for the future larger study. We found it was extremely beneficial to include a focus group pre-phase in a study that will interview parents in a high sensitivity area.


Assuntos
Atitude Frente a Saúde , Tomada de Decisões , Grupos Focais/métodos , Neoplasias/terapia , Pais/psicologia , Seleção de Pacientes , Assistência Terminal/psicologia , Adaptação Psicológica , Criança , Grupos Focais/estatística & dados numéricos , Comportamento de Ajuda , Humanos , Moral , Avaliação das Necessidades/organização & administração , Neoplasias/psicologia , Papel do Profissional de Enfermagem/psicologia , Pesquisa Metodológica em Enfermagem , Enfermagem Oncológica , Assistência Centrada no Paciente , Enfermagem Pediátrica , Relações Profissional-Família , Desenvolvimento de Programas , Pesquisa Qualitativa , Qualidade de Vida/psicologia , Apoio Social , Inquéritos e Questionários , Assistência Terminal/métodos
SELEÇÃO DE REFERÊNCIAS
Detalhe da pesquisa