Asunto(s)
Cuidados Paliativos , Cuidado Terminal , Humanos , Dolor , Portugal , Cobertura Universal del Seguro de SaludRESUMEN
INTRODUCTION: Due to epidemiological change, interest in complex chronic conditions has been increasing within the pediatric health system. As such, we aim to evaluate hospital inpatient care in the National Health Service (mainland Portugal) by pediatric patients (0 - 17 years) with complex chronic conditions. MATERIAL AND METHODS: Observational longitudinal retrospective epidemiological study using anonymized administrative data. We selected hospitalizations within the pediatric age limit, 2011 - 2015; healthy newborns and radiotherapy outpatients were excluded. A descriptive analysis of the admissions with complex chronic conditions was analysed by number of complex chronic conditions categories and by complex chronic conditions categories. Non-parametric tests were applied to length of stay, expense, and mortality. RESULTS: Out of 419 927 admissions, 64 918 (15.5%) contained at least one complex chronic conditions code. These admissions due to complex chronic conditions represented 29.8% of hospital days, 39.4% of expense and 87.2% of deaths. Compared to those without complex chronic conditions, expense was double (median 1467 vs 745) and mortality 40 times higher (2.4% vs 0.06%). Of these, 46% were planned (no complex chronic conditions 23.2%); 64.8% occurred in group III - IV hospitals (no complex chronic conditions 27.1%). Malignant was the most frequent category (23.0%); neonatal had the highest median length of stay (12 days, 6 - 41), median expense (3568,929 - 24 602), and number of deaths (43.5% of total). DISCUSSION: As in other developed countries where the number of pediatric admissions is decreasing, in mainland Portugal we found an increase in the proportion of complex chronic conditions admissions, which are longer, costlier and deadlier (trends intensified in the presence of two or more complex chronic conditions categories). CONCLUSION: Complex chronic conditions are relevant in the activity and costs regarding pediatric hospitalizations in mainland Portugal. Recognizing this and integrating pediatric palliative care from the moment of diagnosis are essential to promote appropriate hospital use, through the development of effective and sustainable alternatives that meet the needs of children, families, and healthcare professionals.
Introdução: Fruto da mudança epidemiológica, o interesse pelas doenças crónicas complexas no sistema de saúde pediátrico tem vindo a crescer. Assim, pretendemos avaliar a utilização do internamento hospitalar do Serviço Nacional de Saúde (Portugal Continental) por doentes pediátricos (0 17 anos) com doenças crónicas complexas. Material e Métodos: Estudo epidemiológico observacional longitudinal retrospetivo (base de dados de morbilidade hospitalar anonimizada). Selecionámos episódios de internamento de doentes pediátricos, entre 2011 2015; excluímos recém-nascidos saudáveis e radioterapia ambulatória. Análise descritiva dos episódios de internamento de doentes pediátricos com doenças crónicas complexas, caracterizados por número e categoria de doenças crónicas complexas. Foram aplicados testes não paramétricos à duração de internamento, despesa e mortalidade. Resultados: Nos 419 927 episódios, constavam códigos de doenças crónicas complexas em 64 918 (15,5%). Estes episódios com doenças crónicas complexas representaram 29,8% dos dias de internamento, 39,4% da despesa e 87,2% dos óbitos. Custaram o dobro dos episódios sem doenças crónicas complexas (1467 vs 745) e tiveram uma mortalidade 40 vezes superior (2,4% vs 0,06%). Do total, 46,0% foram programados (sem doenças crónicas complexas 23,2%); 64,8% ocorreram em hospitais grupo III IV (sem doenças crónicas complexas 27,1%). Nos episódios com doenças crónicas complexas, a doença maligna foi a categoria mais frequente (23,0%); a maior demora mediana (12 dias, 6 41), despesa mediana (3568,929 24 602) e mortalidade (13,4%) verificaram-se na categoria neonatais. Discussão: Esta análise mostrou que, embora o número absoluto de internamentos de doentes pediátricos esteja a diminuir em Portugal Continental, à semelhança de outros países desenvolvidos, os internamentos com doenças crónicas complexas têm vindo a aumentar proporcionalmente, sendo mais prolongados, onerosos e com maior probabilidade de morte do que os episódios sem doenças crónicas complexas (tendências acentuadas quando constam duas ou mais doenças crónicas complexas). Conclusão: As doenças crónicas complexas são relevantes na atividade e despesa do internamento hospitalar pediátrico em Portugal Continental. Este reconhecimento e a integração de cuidados paliativos pediátricos desde o diagnóstico são essenciais para adequar a utilização do hospital, desenvolvendo alternativas efetivas e sustentáveis que vão ao encontro das necessidades das crianças, famílias e profissionais.
Asunto(s)
Enfermedad Crónica/epidemiología , Hospitalización/estadística & datos numéricos , Pacientes Internos/estadística & datos numéricos , Adolescente , Niño , Preescolar , Enfermedad Crónica/clasificación , Enfermedad Crónica/economía , Enfermedad Crónica/mortalidad , Costos de la Atención en Salud/estadística & datos numéricos , Hospitalización/economía , Hospitales/clasificación , Humanos , Lactante , Recién Nacido , Tiempo de Internación/economía , Tiempo de Internación/estadística & datos numéricos , Evaluación de Necesidades , Cuidados Paliativos , Portugal/epidemiología , Estudios Retrospectivos , Medicina Estatal/economía , Medicina Estatal/estadística & datos numéricos , Estadísticas no ParamétricasRESUMEN
BACKGROUND: High-dose chemotherapy with haemopoietic stem-cell rescue improves event-free survival in patients with high-risk neuroblastoma; however, which regimen has the greatest patient benefit has not been established. We aimed to assess event-free survival after high-dose chemotherapy with busulfan and melphalan compared with carboplatin, etoposide, and melphalan. METHODS: We did an international, randomised, multi-arm, open-label, phase 3 cooperative group clinical trial of patients with high-risk neuroblastoma at 128 institutions in 18 countries that included an open-label randomised arm in which high-dose chemotherapy regimens were compared. Patients (age 1-20 years) with neuroblastoma were eligible to be randomly assigned if they had completed a multidrug induction regimen (cisplatin, carboplatin, cyclophosphamide, vincristine, and etoposide with or without topotecan, vincristine, and doxorubicin) and achieved an adequate disease response. Patients were randomly assigned (1:1) to busulfan and melphalan or to carboplatin, etoposide, and melphalan by minimisation, balancing age at diagnosis, stage, MYCN amplification, and national cooperative clinical group between groups. The busulfan and melphalan regimen comprised oral busulfan (150 mg/m2 given on 4 days consecutively in four equal doses); after Nov 8, 2007, intravenous busulfan was given (0·8-1·2 mg/kg per dose for 16 doses according to patient weight). After 24 h, an intravenous melphalan dose (140 mg/m2) was given. Doses of busulfan and melphalan were modified according to bodyweight. The carboplatin, etoposide, and melphalan regimen consisted of carboplatin continuous infusion of area under the plasma concentration-time curve 4·1 mg/mL per min per day for 4 days, etoposide continuous infusion of 338 mg/m2 per day for 4 days, and melphalan 70 mg/m2 per day for 3 days, with doses for all three drugs modified according to bodyweight and glomerular filtration rate. Stem-cell rescue was given after the last dose of high-dose chemotherapy, at least 24 h after melphalan in patients who received busulfan and melphalan and at least 72 h after carboplatin etoposide, and melphalan. All patients received subsequent local radiotherapy to the primary tumour site followed by maintenance therapy. The primary endpoint was 3-year event-free survival, analysed by intention to treat. This trial is registered with ClinicalTrials.gov, number NCT01704716, and EudraCT, number 2006-001489-17. FINDINGS: Between June 24, 2002, and Oct 8, 2010, 1347 patients were enrolled and 676 were eligible for random allocation, 598 (88%) of whom were randomly assigned: 296 to busulfan and melphalan and 302 to carboplatin, etoposide, and melphalan. Median follow-up was 7·2 years (IQR 5·3-9·2). At 3 years, 146 of 296 patients in the busulfan and melphalan group and 188 of 302 in the carboplatin, etoposide, and melphalan group had an event; 3-year event-free survival was 50% (95% CI 45-56) versus 38% (32-43; p=0·0005). Nine patients in the busulfan and melphalan group and 11 in the carboplatin, etoposide, and melphalan group had died without relapse by 5 years. Severe life-threatening toxicities occurred in 13 (4%) patients who received busulfan and melphalan and 29 (10%) who received carboplatin, etoposide, and melphalan. The most frequent grade 3-4 adverse events were general condition (74 [26%] of 281 in the busulfan and melphalan group vs 103 [38%] of 270 in the carboplatin, etoposide, and melphalan group), infection (55 [19%] of 283 vs 74 [27%] of 271), and stomatitis (138 [49%] of 284 vs 162 [59%] of 273); 60 (22%) of 267 patients in the busulfan and melphalan group had Bearman grades 1-3 veno-occlusive disease versus 21 (9%) of 239 in the carboplatin, etoposide, and melphalan group. INTERPRETATION: Busulfan and melphalan improved event-free survival in children with high-risk neuroblastoma with an adequate response to induction treatment and caused fewer severe adverse events than did carboplatin, etoposide, and melphalan. Busulfan and melphalan should thus be considered standard high-dose chemotherapy and ongoing randomised studies will continue to aim to optimise treatment for high-risk neuroblastoma. FUNDING: European Commission 5th Framework Grant and the St Anna Kinderkrebsforschung.