Your browser doesn't support javascript.
loading
Mostrar: 20 | 50 | 100
Resultados 1 - 20 de 139
Filtrar
1.
Front Rehabil Sci ; 5: 1294999, 2024.
Artigo em Inglês | MEDLINE | ID: mdl-38370854

RESUMO

Introduction: Optimizing care for young adults with cerebral palsy is crucial for their physical and psychological well-being. The inadequacy of proximal environment may play a role in the provision of health services. The aim of this study is to explore the association between unmet environmental needs in the physical, social and attitudinal domains and unmet healthcare needs in four interventions: physiotherapy, occupational therapy, speech therapy and psychological counselling. Methods: Young adults with cerebral palsy were recruited in the SPARCLE3 European multicenter cross-sectional study. Healthcare needs and coverages were assessed using the Youth Health Care, Satisfaction, Utilization and Needs questionnaire. The need and availability of environmental factors in physical, social and attitudinal domains were collected using the European Adult Environment Questionnaire. Logistic regressions were conducted separately for each intervention to measure associations between unmet environmental needs and unmet healthcare needs. Results: We studied 310 young adults with cerebral palsy, with a mean age of 24.3 years; 37.4% could not walk independently, 51.5% had an IQ below 70, 34.2% had severe communication difficulties. The most commonly expressed need was physiotherapy (81.6% of participants). Unmet healthcare needs were reported by 20.9%, 32.4%, 40.3% and 49.0% of participants requiring physiotherapy, occupational therapy, psychological counselling and speech therapy, respectively. The physical environment was never significantly associated with unmet healthcare needs. In contrast, the social environment was significantly associated with unmet healthcare needs across all interventions, with odds ratios over 2.5, depending on the number of unmet needs and the nature of intervention needed. With regard to the attitudinal environment, when at least one unmet attitudinal environmental need was reported, the odds of also reporting an unmet healthcare need were of 3.68 for speech therapy and 3.77 for physiotherapy. The latter association was significant only for individuals with severe motor impairment. Discussion: Our results highlight the importance of the social and attitudinal environment in meeting healthcare needs in young adults with cerebral palsy. The lack of correlation between unmet healthcare needs and the physical environment suggests that it can be partly compensated for by social support.

2.
Front Med (Lausanne) ; 11: 1306504, 2024.
Artigo em Inglês | MEDLINE | ID: mdl-38352143

RESUMO

Background: Young people with chronic health conditions and disabilities rely on the healthcare system to maintain their best possible health. The appropriate delivery and utilization of healthcare services are key to improve their autonomy, self-efficacy and employment outcomes. The research question of our study is directed toward investigating if poor availability and accessibility of healthcare services in general, as identified by unmet needs in healthcare, are associated with dissatisfaction with healthcare. Methods: Within a European multicenter observational study, 357 young adults with cerebral palsy aged 19-28 were included. We assessed special healthcare needs, utilization of healthcare services, and satisfaction with healthcare applying the short-form of the YHC-SUN-SF, environmental and social variables (EAEQ) as well as indicators for severity of condition and functionality (e.g., GMFCS) of these participants based on a self-, assisted self- or proxy-reports. We used correlation analyses to explore associations between satisfaction with healthcare and respective indicators related to availability and accessibility of healthcare services as well as severity of the condition. In addition, we included reference values for satisfaction with heath care from young adults with various chronic conditions assessed within population-based surveys from some of the European countries included in the study. Results: We identified several unmet healthcare needs, especially for widely used and established services (e.g., physical therapy). Satisfaction with healthcare (YHC-SUN-SF general and subscale scores) was moderate to high and almost consistently better for the sample of young adults with cerebral palsy as compared to reference values for young adults with various chronic conditions assessed within general population surveys). Correlation coefficients between satisfaction with healthcare and utilization of services and (unmet) healthcare needs were low, also with different indicators for severity of the condition or functionality. Conclusion: Young adults with cerebral palsy reports of unmet healthcare needs varied largely but showed substantial deficits in some aspects. This seems to have no impact on the satisfaction with healthcare those patients currently receive. We conclude that these are two different constructs and somewhat independent indicators to evaluate the quality of healthcare. Clinicians and other practitioners should consider this distinction when monitoring patient needs in their daily practice.

3.
Arch Phys Med Rehabil ; 105(5): 906-920, 2024 May.
Artigo em Inglês | MEDLINE | ID: mdl-38206241

RESUMO

OBJECTIVES: To present the development of the European Adult Environment Questionnaire (EAEQ), to assess to what extent it covers the International Classification of Functioning, Disability and Health (ICF), and to describe the adequacy of the physical, social, and attitudinal environment to the specific needs of young adults with cerebral palsy (CP). DESIGN: Cross-sectional. SETTING: Administrative regions in France, Germany, Italy, Portugal, and Sweden. PARTICIPANTS: Young adults with CP (N=357), with varying severity profiles, aged 19-28 years at time of interview (2018-20). INTERVENTIONS: Not applicable. MAIN OUTCOME MEASURE(S): Physical, social, and attitudinal environment unmet needs. RESULTS: Relevant environmental factors (EFs) for young adults with CP were identified during focus groups in England and Portugal. EFs were mapped to the ICF environmental classification and the EAEQ analytical structure resulted from this linking procedure. It comprised 61 items, linked to 31 ICF environmental classification categories, and covered 4 of its 5 chapters. Content validity assessed with the bandwidth index (percentage coverage of ICF Core Sets for adults with CP) was satisfactory (79.3%). A descriptive analysis was carried out. Participants had a mean age of 24 years, 56% were men, 38% had severely limited mobility. Less than 16% reported unmet needs for EFs relating to home, college/work/day placement, and communication in the Products and technology chapter. Unmet needs were higher (>20%) for the other items in the Public use and Land development categories. Social support, attitudes, and understanding of relatives were often adequate to the participants' needs. The proportion of unmet needs varied by sex (women were more often concerned) and raised with increasing gross motor impairment. CONCLUSION: The EAEQ describes in detail the adequacy of the environment to the specific needs of young adults with CP. Its ICF-based structure opens up possibilities for use in a universal conceptual framework.


Assuntos
Paralisia Cerebral , Humanos , Paralisia Cerebral/reabilitação , Masculino , Feminino , Adulto Jovem , Adulto , Estudos Transversais , Inquéritos e Questionários , Classificação Internacional de Funcionalidade, Incapacidade e Saúde , Avaliação da Deficiência , Europa (Continente) , Meio Social , Avaliação das Necessidades , Pessoas com Deficiência/reabilitação , Pessoas com Deficiência/psicologia , Grupos Focais , Meio Ambiente
4.
Dev Med Child Neurol ; 66(2): 176-186, 2024 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-37501281

RESUMO

AIM: To study long-term sequelae in children with Guillain-Barré syndrome (GBS). METHOD: This was a prospective observational study with children from two French tertiary centres. Data were from clinical and several standardized scales or questionnaires. RESULTS: Fifty-one patients were included with a median follow-up of 6 years 4 months (range 3-20 years) after the acute phase. The sequelae rate was 67% (95% confidence interval [CI] 53-78) and did not vary with time. Most children had minor sequelae (Guillain-Barré Syndrome Disability Score [GBSDS] = 1); only one was unable to run (GBSDS = 2). The most frequent complaints were paraesthesia (43%), pain (35%), and fatigue (31%). The neurological examination was abnormal in 18% of children, autonomy was compromised in 14%, and symptoms of depression occurred in 34%. The factors associated with late-onset sequelae were correlated with severity during the initial phase (i.e. initial GBSDS >4, odds ratio 6.6, 95% CI 1.8-33; p = 0.009). The predictive factors of more severe late-onset conditions were initial severity (p = 0.002) and sex (female patients; p = 0.01). INTERPRETATION: Two-thirds of children with GBS had late-onset sequelae following an episode, often minor, but sometimes with continuing effects on their everyday lives. Particularly affected were those who had severe GBS during the acute phase and who lost the ability to walk. WHAT THIS PAPER ADDS: Two-thirds of children with Guillain-Barré syndrome (GBS) had persistent sequelae. Sequelae were often minor, but daily repercussions of them were sometimes serious. Sequelae were significantly associated with severe GBS during the acute phase.


Assuntos
Síndrome de Guillain-Barré , Humanos , Criança , Feminino , Síndrome de Guillain-Barré/complicações , Síndrome de Guillain-Barré/diagnóstico , Estudos Prospectivos , Progressão da Doença , Inquéritos e Questionários , Fadiga/complicações
5.
Dev Med Child Neurol ; 66(2): 250-257, 2024 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-37488719

RESUMO

AIM: This paper introduces the Surveillance of Cerebral Palsy in Europe (SCPE) classification of events contributing to postneonatally acquired cerebral palsy, presents its interrater reliability, and describes the cases identified in the SCPE database. METHOD: The development of the classification, based on literature review and expert discussions, resulted in six main categories and 19 subcategories. The first chronological event designated as the primary event was mainly reported. Interrater reliability was assessed through online exercise providing 24 clinical vignettes representing single/complex pathways. Percent agreement and Gwet's AC1 index of reliability were estimated. Primary events were described using data of 221 children born between 2008 and 2012. RESULTS: Thirty-nine professionals (21 registries) participated in the reliability exercise. Substantial overall agreement was reached (0.75), with some contrast between complex (0.48, moderate agreement) and single events involved (0.89, almost perfect). The distribution of primary events showed that 32.1% were infections (category A), 23.1% head injuries (B), 15.4% related to surgery or medical interventions (C), 13.1% cerebrovascular accidents (D), 9.1% hypoxic brain damaging events of other origins (E), and 7.2% miscellaneous (F). INTERPRETATION: This classification allows all the events involved to be recorded while consistently reporting the primary event, and may be used in different settings. WHAT THIS PAPER ADDS: A standardized classification enables the description of the events contributing to postneonatal cerebral palsy (CP). The first chronological event in complex pathway leading to CP is coded. Category choice and coding of the primary event identify preventable situations. The detailed 2-level classification is easy to use in various settings. Substantial overall interrater reliability shows that main categories can be consistently differentiated.


Assuntos
Paralisia Cerebral , Acidente Vascular Cerebral , Criança , Humanos , Paralisia Cerebral/epidemiologia , Paralisia Cerebral/etiologia , Reprodutibilidade dos Testes , Encéfalo , Sistema de Registros
6.
Disabil Health J ; 17(2): 101554, 2024 Apr.
Artigo em Inglês | MEDLINE | ID: mdl-38129262

RESUMO

BACKGROUND: Children and adolescents with disabilities are known to participate less in most areas of life than their non-disabled peers. OBJECTIVES: (1) To estimate differences in participation between young adults with cerebral palsy (CP) and their non-disabled peers; (2) to test the mediating role of financial difficulties and student status; (3) to test the moderating role of personal factors on participation difference; and (4) to test the moderating role of impairment. METHODS: A cross-sectional study was conducted in young adults [19-28 years] with CP (n = 228) and non-disabled peers (n = 2861) in France, Germany and Sweden. Participation was assessed using the Questionnaire of Young People's Participation adapted for young adults (QYPP-YA). Differences in five domains of participation were estimated using structural equation modeling with WLSMV method and bias-corrected bootstrap confidence intervals. RESULTS: Young adults with CP showed lower participation than others in all domains, with the largest difference in the "intimate relationships" domain (ß = 1.71 bcCI95[1.46; 1.95]). Student status mediated the difference in "intimate relationships", "interpersonal relationships" and "independence". Women showed greater differences than men on "independence". Impairments moderated difference in participation. The less severely impaired young adults showed no difference with their non-CP peers in "interpersonal relationships", "social life" and "independence", but made autonomous everyday decisions more often than their peers. CONCLUSIONS: Young adults with CP do not have the same opportunities to attain the participation level of non-CP people of the same age. Continuance of education could help to reduce participation difference in "interpersonal relationships" and "independence".


Assuntos
Paralisia Cerebral , Pessoas com Deficiência , Feminino , Humanos , Masculino , Adulto Jovem , Estudos Transversais , Relações Interpessoais , Inquéritos e Questionários , Adulto
7.
BMC Pediatr ; 23(1): 563, 2023 11 15.
Artigo em Inglês | MEDLINE | ID: mdl-37968589

RESUMO

Duchenne Muscular Dystrophy (DMD) is a neuromuscular disease that inevitably leads to total loss of autonomy. The new therapeutic strategies aim to both improve survival and optimise quality of life. Evaluating quality of life is nevertheless a major challenge. No DMD-specific quality of life scale to exists in French. We therefore produced a French translation of the English Duchenne Muscular Dystrophy module of the Pediatric Quality of Life Inventory (PedsQLTMDMD) following international recommendations. The study objective was to carry out a confirmatory validation of the French version of the PedsQLTMDMD for paediatric patients with DMD, using French multicentre descriptive cross-sectional data. The sample consisted of 107 patients. Internal consistency was acceptable for proxy-assessments, with Cronbach's alpha coefficients above 0.70, except for the Treatment dimension. For self-assessments, internal consistency was acceptable only for the Daily Activities dimension. Our results showed poor metric qualities for the French version of the PedsQLTMDMD based on a sample of about 100 children, but these results remained consistent with those of the original validation. This confirms the interest of its use in clinical practice.


Assuntos
Distrofia Muscular de Duchenne , Qualidade de Vida , Criança , Humanos , Inquéritos e Questionários , Distrofia Muscular de Duchenne/diagnóstico , Estudos Transversais , Objetivos , Psicometria , Reprodutibilidade dos Testes , Pais
8.
Neurology ; 101(24): e2509-e2521, 2023 Dec 12.
Artigo em Inglês | MEDLINE | ID: mdl-37857495

RESUMO

BACKGROUND AND OBJECTIVES: To report on prevalence, associated impairments, severity, and neuroimaging findings in children with ataxic cerebral palsy (CP). METHODS: In children coded as having ataxic CP in the Central database of Joint Research Center-Surveillance of Cerebral Palsy in Europe (JRC-SCPE) and born during 1980-2010, birth characteristics, severity profiles including associated impairments, neuroimaging patterns, and the presence of syndromes were analyzed. Definitions were according to validated SCPE guidelines. Prevalence over time was estimated using Poisson regression. RESULTS: In total, 679 children with ataxic CP were identified in 20 European CP registers. The proportion with ataxic CP was 3.8% and varied from 0% to 12.9%. Prevalence over time showed no significant trend. Approximately 70% of children with ataxic CP were able to walk, and 40% had severe intellectual impairment and a high impairment index. Children with ataxic CP were mostly born at term (79%) and with normal birth weight (77%). Neuroimaging patterns revealed normal findings in 29%, brain maldevelopments in 28.5%, miscellaneous findings in 23.5%, and brain injuries in 19%, according to the SCPE classification. Genetic syndromes were described in 9%. DISCUSSION: This register-based multicenter study on children with ataxic CP provides a large sample size for the analysis of prevalence, severity, and origin of this rare CP subtype. Even with strict inclusion and classification criteria, there is variation between registers on how to deal with this subtype, and diagnosis of ataxic CP remains a challenge. Ataxic cerebral palsy differs from other CP subtypes: children with ataxic CP have a disability profile that is more pronounced in terms of cognitive than gross motor dysfunction. They are mostly term born and the origin rarely suggests acquired injuries. In addition to neuroimaging, a comprehensive genetic workup is particularly recommended for children with this CP type.


Assuntos
Paralisia Cerebral , Criança , Humanos , Paralisia Cerebral/diagnóstico por imagem , Paralisia Cerebral/epidemiologia , Paralisia Cerebral/genética , Prevalência , Europa (Continente)/epidemiologia , Neuroimagem , Sistema de Registros
9.
Eur J Obstet Gynecol Reprod Biol ; 291: 16-21, 2023 Dec.
Artigo em Inglês | MEDLINE | ID: mdl-37806026

RESUMO

OBJECTIVE: To compare cesarean rates and maternal and neonatal morbidity according to the cervical ripening method used among obese pregnant women requiring induction of labor at or after 41 weeks of gestation. DESIGN: A secondary analysis of two multicenter randomized controlled trials conducted in French maternity units between 2015 and 2018. PARTICIPANTS: 336 women with a body mass index ≥30 kg/m2, a pregnancy ≥41 weeks, and an induction of labor requiring cervical ripening. INTERVENTIONS: Cervical ripening with a PGE2 dinoprostone pessary (Propess®), or low-dose vaginal PGE1 (misoprostol) or a double-balloon catheter. MEASUREMENTS AND FINDINGS: The rates of cesarean delivery did not differ significantly according to the cervical ripening method (PGE2 pessary vs PGE1, RR: 1.18, 95% CI: 0.80-1.75; PGE2 pessary vs double balloon catheter: RR, 0.88, 95% CI: 0.60-1.29), p = 0.52; double balloon catheter vs PGE1, RR: 1.34, 95% CI: 0.77-2.32, p = 0.29). More oxytocin was required for women from the double-balloon group compared to those from both the PGE1 and PGE2 pessary groups (respectively, RR: 1.31, 95% CI: 1.08-1.58, p = 0.005; RR: 1.17, 95% CI: 1.03-1.32, p = 0.01). The risk of perineal tears or episiotomy was significantly lower for women induced with the PGE2 pessary than with PGE1 (0.85; 95% CI: 0.74-0.99), p = 0.03). KEY CONCLUSIONS AND IMPLICATIONS FOR PRACTICE: No cervical ripening method was associated with a lower cesarean rate in obese women who required cervical ripening from 41 weeks. Further trials are required among obese women to determine the cervical ripening method most efficacious for reducing the cesarean rate.


Assuntos
Dinoprostona , Ocitócicos , Recém-Nascido , Feminino , Gravidez , Humanos , Alprostadil , Trabalho de Parto Induzido/métodos , Maturidade Cervical , Ensaios Clínicos Controlados Aleatórios como Assunto , Obesidade/complicações
10.
JAMA Netw Open ; 6(9): e2331988, 2023 09 05.
Artigo em Inglês | MEDLINE | ID: mdl-37676661

RESUMO

Importance: Compared with term-born peers, children born very preterm generally perform poorly in executive functions, particularly in working memory and inhibition. By taking advantage of neuroplasticity, computerized cognitive training of working memory in those children could improve visuospatial processing by boosting visual inhibition via working memory. Objective: To evaluate the long-term effect of cognitive working memory training on visuospatial processing in children aged 5½ to 6 years born very preterm who have working memory impairment. Design, Setting, and Participants: This multicenter (18 French university hospitals), open-label randomized clinical trial with 2 parallel groups (EPIREMED) was conducted from November 2016 to April 2018, with the last follow-up during August 2019. Eligible children from the EPIPAGE 2 cohort were aged 5½ to 6 years, were born between 24 and 34 weeks' gestation, and had a global intelligence quotient greater than 70 and a working memory index less than 85. Data were analyzed from February to December 2020. Intervention: Children were randomized 1:1 to standard care management and a working memory cognitive training program (Cogmed software) for 8 weeks (25 sessions) (intervention) or to standard management (control). Main Outcomes and Measures: The primary outcome was the visuospatial index score from the Wechsler Preschool and Primary Scale of Intelligence, 4th Edition. Secondary outcomes were working memory, intellectual functioning, executive and attention processes, language skills, behavior, quality of life, and schooling. Neurobehavioral assessments were performed at inclusion and after finishing training at 6 months (intermeditate assessment; secondary outcomes) and at 16 months (final assessment; primary outcome). Results: There were 169 children randomized, with a mean (SD) age of 5 years 11 months (2 months); 91 (54%) were female. Of the participants, 84 were in the intervention group (57 of whom [68%] completed at least 15 cognitive training sessions) and 85 were in the control group. The posttraining visuospatial index score was not different between groups at a mean (SD) of 3.0 (1.8) months (difference, -0.6 points; 95% CI, -4.7 to 3.5 points) or 12.9 (2.6) months (difference, 0.1 points; 95% CI, -5.4 to 5.1 points). The working memory index score in the intervention group significantly improved from baseline at the intermediate time point (difference, 4.7 points; 95% CI, 1.2-8.1 points), but this improvement was not maintained at the final assessment. Conclusions and Relevance: This randomized clinical trial found no lasting effect of a cognitive training program on visuospatial processing in children aged 5½ to 6 years with working memory disorders who were born very preterm. The findings suggest that this training has limited long-term benefits for improving executive function. Transient benefits seemed to be associated with the developmental state of executive functions. Trial Registration: ClinicalTrials.gov Identifier: NCT02757794.


Assuntos
Memória de Curto Prazo , Transtornos Mentais , Pré-Escolar , Recém-Nascido , Criança , Feminino , Humanos , Masculino , Treino Cognitivo , Lactente Extremamente Prematuro , Qualidade de Vida , Transtornos da Memória
11.
Early Hum Dev ; 184: 105837, 2023 Sep.
Artigo em Inglês | MEDLINE | ID: mdl-37595540

RESUMO

OBJECTIVE: To determine the prevalence, short-term prognosis and pharmacologic management of pulmonary hypertension (PH) among very preterm infants born before 32 weeks gestation (WG). STUDY DESIGN: In the EPIPAGE-2 French national prospective population-based cohort of preterm infants born in 2011, those presenting with PH were identified and prevalence was estimated using multiple imputation. The primary outcome was survival without severe morbidity at discharge and was compared between infants with or without PH after adjusting for confounders, using generalized estimating equations models. Subgroup analysis was performed according to gestational age (GA) groups. RESULTS: Among 3383 eligible infants, 3222 were analyzed. The prevalence of PH was 6.0 % (95 % CI, 5.2-6.9), 14.5 % in infants born at 22-27+6 WG vs 2.7 % in infants born at 28-31+6 WG (P < .001). The primary outcome (survival without severe morbidity at discharge) occurred in 30.2 % of infants with PH vs 80.2 % of infants without PH (P < .001). Adjusted incidence rate ratios for survival without severe morbidity among infants with PH were 0.42 (0.32-0.57) and 0.52 (0.39-0.69) in infants born at 22-27+6 weeks gestation and those born at 28-31+6 weeks, respectively. Among infants with PH, 92.2 % (95 % CI, 87.7-95.2) received sedation and/or analgesia, 63.5 % (95 % CI, 56.6-69.9) received inhaled NO and 57.6 % (95 % CI, 50.9-64.0) received hemodynamic treatments. CONCLUSION: In this population-based cohort of very preterm infants, the prevalence of PH was 6 %. PH was associated with a significant decrease of survival without severe morbidity in this population.

12.
Am J Perinatol ; 2023 Jul 10.
Artigo em Inglês | MEDLINE | ID: mdl-37429323

RESUMO

OBJECTIVE: Preterm birth, defined as birth before 37 weeks of gestation, is a leading cause of perinatal and infant mortality throughout the world. Preterm birth is also associated with long-term neurological disabilities and other significant health issues in children. A short cervix in the second trimester has been noted to be one of the strongest predictors of subsequent spontaneous preterm birth in both singleton and multiple pregnancies. Some studies have shown that cervical support in the form of an Arabin pessary lowers the risk of preterm birth in women with a singleton gestation and short cervical length; however, other studies have conflicting results. Our objective was to form an international collaborative of planned or ongoing randomized trials of pessary in singleton and twin gestations with a short cervix. STUDY DESIGN: In November 2014, an international group of investigators, who had initiated or were planning randomized trials of pessary for pregnant people with a short cervix and singleton or twin gestation to prevent preterm birth, formed a collaboration to plan a prospective individual patient data (IPD) meta-analysis of randomized trials (PROspective Meta-analysis of Pessary Trials [PROMPT]). The PROMPT investigators agreed on meta-analysis IPD hypotheses for singletons and twins, eligibility criteria, and a set of core baseline and outcome measures. The primary outcome is a composite of fetal death or preterm delivery before 32 weeks' gestation. Secondary outcomes include maternal and neonatal morbidities. The PROMPT protocol may be viewed as a written agreement among the study investigators who make up the PROMPT consortium (PROSPERO ID# CRD42018067740). RESULTS: Results will be published in phases as the individual participating studies are concluded and published. Results of the first phase of singleton and twin pessary trials are expected to be available in late 2022. Updates are planned as participating trials are completed and published. KEY POINTS: · Short cervical length predicts preterm birth.. · Results of prior cervical pessary trials are mixed.. · Meta-analysis of pessary trials protocol..

13.
J Am Soc Nephrol ; 34(9): 1561-1573, 2023 09 01.
Artigo em Inglês | MEDLINE | ID: mdl-37303085

RESUMO

SIGNIFICANCE STATEMENT: Shiga toxin-related hemolytic uremic syndrome (STEC-HUS) is a serious condition, characterized by multiorgan thrombotic microangiopathy, mainly affecting children. Renal involvement is severe, with approximately half of patients requiring dialysis. So far, no specific treatment has been proven efficient in STEC-HUS. The use of eculizumab, a monoclonal antibody inhibiting terminal complement complex, has demonstrated remarkable success in atypical hemolytic uremic syndrome, but its use in uncontrolled studies to treat STEC-HUS has yielded inconsistent results. In this Phase 3 randomized, placebo-controlled trial in 100 pediatric patients with STEC-HUS, the findings did not show efficacy of eculizumab during the acute phase of the disease. However, the results indicated a reduction of renal sequelae in eculizumab-treated patients at 1-year follow-up. Larger prospective studies would be needed to further explore eculizumab as a potential treatment. BACKGROUND: Shiga toxin-related hemolytic uremic syndrome (STEC-HUS) in children is a severe condition, resulting in approximately 50% of patients requiring RRT. Furthermore, at least 30% of survivors experience kidney sequelae. Recently, activation of the complement alternative pathway has been postulated as a factor in STEC-HUS pathophysiology, leading to compassionate use of eculizumab, a monoclonal antibody inhibiting the terminal complement complex, in affected patients. Given the lack of therapy for STEC-HUS, a controlled study of eculizumab efficacy in treating this condition is a priority. METHODS: We conducted a Phase 3 randomized trial of eculizumab in children with STEC-HUS. Patients were randomly assigned in a 1:1 ratio to receive either eculizumab or placebo during 4 weeks. Follow-up lasted for 1 year. The primary end point was RRT duration <48 hours after randomization. Secondary endpoints included hematologic and extrarenal involvement. RESULTS: Baseline characteristics were similar among the 100 patients who underwent randomization. The rate of RRT <48 hours did not differ significantly between the two groups (48% in the placebo versus 38% in the eculizumab group; P = 0.31) or in the course of ARF. The two groups also exhibited similar hematologic evolution and extrarenal manifestations of STEC-HUS. The proportion of patients experiencing renal sequelae at 1 year was lower in the eculizumab group than in the placebo group (43.48% and 64.44%, respectively, P = 0.04). No safety concern was reported. CONCLUSIONS: In pediatric patients with STEC-HUS, eculizumab treatment does not appear to be associated with improved renal outcome during acute phase of the disease but may reduce long-term kidney sequelae. CLINICAL TRIALS REGISTRATIONS: EUDRACT (2014-001169-28) ClinicalTrials.gov ( NCT02205541 ).


Assuntos
Síndrome Hemolítico-Urêmica Atípica , Infecções por Escherichia coli , Criança , Humanos , Estudos Prospectivos , Complexo de Ataque à Membrana do Sistema Complemento , Toxina Shiga/uso terapêutico , Anticorpos Monoclonais Humanizados/efeitos adversos , Síndrome Hemolítico-Urêmica Atípica/tratamento farmacológico , Síndrome Hemolítico-Urêmica Atípica/complicações
14.
Acta Paediatr ; 112(9): 1916-1925, 2023 09.
Artigo em Inglês | MEDLINE | ID: mdl-37191836

RESUMO

AIM: To assess the efficacy of post-hospital psychomotor therapy in the development of very preterm infants at nine and 24 months. METHODS: We conducted a randomised controlled study at Toulouse Children's Hospital between 2008 and 2014 among preterm infants aged under 30 weeks. All infants in both groups could benefitt from physiotherapy to prevent motor disorders. The intervention group received 20 early post-hospital psychomotor therapy sessions. The development was assessed by the Bayley Scale Infant Development at nine and 24 months. RESULTS: The intervention and control group contained 77 and 84 infants, respectively, with 57 infants in each group undergoing assessment at 24 months. Boys accounted for 56% of the population. Median gestational age was 28 weeks, range 25-29. The development scores at 24 months did not significantly differ between the randomisation groups. At 9 months, we observed improvements in global motricity (mean difference 0.9 point, p = 0.04) and fine motricity for the subgroup containing educationally underserved mothers (mean difference 1.6 point, p = 0.008). There was no significant difference in neuromotor functioning between the two groups. CONCLUSION: The benefits of psychomotor therapy were short-lived and did not persist post-intervention. Our results and this organisational model encouraged us to persevere towards similar multi-professional care.


Assuntos
Doenças do Prematuro , Recém-Nascido Prematuro , Lactente , Masculino , Feminino , Criança , Recém-Nascido , Humanos , Recém-Nascido de muito Baixo Peso , Desenvolvimento Infantil , Idade Gestacional , Doenças do Prematuro/prevenção & controle
15.
Acta Obstet Gynecol Scand ; 102(6): 669-680, 2023 06.
Artigo em Inglês | MEDLINE | ID: mdl-37101411

RESUMO

INTRODUCTION: Periodontal diseases (gingivitis and periodontitis) are chronic non-communicable inflammatory diseases. The risk of developing gingivitis and periodontitis increases during pregnancy. Also, periodontitis increases the risk of developing adverse pregnancy outcomes such as preterm birth and preeclampsia. Early diagnosis of adverse pregnancy outcomes is essential and periodontitis could be an early sign to take into consideration. MATERIAL AND METHODS: We conducted a longitudinal observational study (PERISCOPE study: CNIL, no. 1 967 084 v 0; CER, no. 01-0416) on 121 pregnant women in the first trimester to determine their oral and periodontal health status. We explored the relations between oral and periodontal health status and sociodemographic and behavior characteristics, as well as their course and outcome of pregnancy. RESULTS: A total of 47.1% of the women had periodontitis, of which only 66.7% presented clinical manifestations associated with the disease such as gingival bleeding. These women had a poorer oral and periodontal health, and a higher body mass index, and more of them developed gestational diabetes during the course of pregnancy. The remaining 33.3% showed only discreet and isolated inflammatory signs and, unless thoroughly examined, would have gone undiagnosed for periodontitis. Interestingly these women were more often primiparous, still active professionally and had had a recent oral examination. CONCLUSIONS: The PERISCOPE study is one of the few studies that reports the oral and periodontal health status of pregnant women in the first trimester. Furthermore, the results highlight the need for early oral and periodontal assessment and treatment, even in the absence of exterior clinical signs, in order to prevent periodontal disease aggravation and also, by reducing low grade systemic inflammation, possibly adverse pregnancy outcomes.


Assuntos
Gengivite , Periodontite , Nascimento Prematuro , Feminino , Gravidez , Recém-Nascido , Humanos , Primeiro Trimestre da Gravidez , Estudos Longitudinais , Periodontite/complicações , Periodontite/diagnóstico , Periodontite/epidemiologia , Gengivite/diagnóstico , Gengivite/complicações , Resultado da Gravidez
16.
Paediatr Perinat Epidemiol ; 37(5): 404-412, 2023 Jul.
Artigo em Inglês | MEDLINE | ID: mdl-36722642

RESUMO

BACKGROUND: Public health indicators (PHIs) play an increasingly important role in health policy decision-making. Although cerebral palsy (CP) is the commonest physical disability in children, its impact at population level has not been systematically measured so far. OBJECTIVES: We aimed to propose six PHIs for CP designed to annually document the extent of CP and effectiveness of perinatal organisation, the burden of this condition, access to health services and preventive health strategies in the post-neonatal period and to report on the latest updated estimations using population-based data routinely collected by European CP registries. METHODS: The study included children with CP born between 2002 and 2011. Harmonised data (number of cases, functional profile, imaging) were extracted from the Surveillance of Cerebral Palsy in Europe (SCPE) database. Eligibility criteria for analyses were applied separately for each indicator by selecting registries, birth years and CP cases. Current estimates were based on the last 3 birth years, while trends were reported over a 10-year period. All analyses were descriptive. Sensitivity analyses were carried out to examine the stability of the results using various thresholds of percentages of missing values. RESULTS: Analyses were performed on a total of 8621 children with CP from 12 to 17 SCPE registries. A decreasing prevalence of pre/perinatal CP overall, as well as in preterm and full-term-born children, was observed. The burden of the condition was strongly dependent on CP subtype and the presence of associated impairments. Access to brain imaging ranged from 80% to 100% depending on registries. The overall prevalence of post-neonatally acquired CP was approximately 0.8 per 10,000 live births over the study period. CONCLUSIONS: Population-based CP registries can provide data that are relevant for generating key outcomes of interest at the population level, thus potentially contributing to improving public health policies for children with disabilities.


Assuntos
Paralisia Cerebral , Recém-Nascido , Criança , Gravidez , Feminino , Humanos , Paralisia Cerebral/diagnóstico , Paralisia Cerebral/epidemiologia , Saúde Pública , Europa (Continente)/epidemiologia , Sistema de Registros , Prevalência
17.
Arch Dis Child Fetal Neonatal Ed ; 108(2): 149-155, 2023 Mar.
Artigo em Inglês | MEDLINE | ID: mdl-36008103

RESUMO

OBJECTIVE: To identify the characteristics of early life growth associated with later overweight or obesity (OWO) in very preterm population. DESIGN: Length, weight and body mass index (BMI) were prospectively recorded from three prospective, population-based cohorts with 5 years (Loire Infant Follow-up Team (LIFT), EPIPAGE2 (Etude EPIdémiologique sur les Petits Ages GEstationnels 2)) and 15 years (EPIPAGEADO, Etude EPIdémiologique sur les Petits Ages GEstationnels-Adolescents) of follow-up. Missing data were imputed. SETTING: Regional (LIFT), national (EPIPAGE2) and multiregional (EPIPAGEADO) cohorts in France. PATIENTS: Eligible infants born before 33 weeks of gestation in 1997 (EPIPAGEADO), between 2003 and 2014 (LIFT), and in 2011 (EPIPAGE2). MAIN OUTCOME MEASURES: OWO was determined as BMI Z-score >85th percentile of the WHO reference curves at 5 years (LIFT, EPIPAGE2) and 15 years (EPIPAGEADO). RESULTS: In EPIPAGEADO, LIFT and EPIPAGE2, BMI Z-scores were known for 302 adolescents, 1016 children and 2022 children, respectively. In EPIPAGEADO, OWO was observed in 42 (13.9%, 95% CI 10.5 to 18.3) adolescents. In multivariable models, birthweight Z-score, increase in weight Z-score during neonatal hospital stay and increase in BMI between discharge and at 2 years of corrected age were positively associated with OWO at 15 years (adjusted OR (aOR)=3.65, 95% CI 1.36 to 9.76; aOR=3.82, 95% CI 1.42 to 10.3; and aOR=2.55, 95% CI 1.72 to 3.78, respectively, by Z-score), but change in length Z-score during neonatal hospital stay was negatively associated (aOR=0.41, 95% CI 0.21 to 0.78, p=0.007). These four associations with OWO assessed at 5 years were confirmed in the LIFT and EPIPAGE2 cohorts. CONCLUSIONS: Change in length Z-score during hospitalisation, a putative proxy of quality of neonatal growth, was negatively associated with risk of later OWO when change in BMI between discharge and at 2 years was included in the multivariable model.


Assuntos
Recém-Nascido Prematuro , Sobrepeso , Lactente , Criança , Feminino , Adolescente , Recém-Nascido , Humanos , Sobrepeso/epidemiologia , Estudos Prospectivos , Recém-Nascido de muito Baixo Peso , Retardo do Crescimento Fetal , Obesidade/epidemiologia , Índice de Massa Corporal
19.
Dev Med Child Neurol ; 64(12): 1494-1506, 2022 12.
Artigo em Inglês | MEDLINE | ID: mdl-35952356

RESUMO

AIM: To determine trends and current estimates in regional and global prevalence of cerebral palsy (CP). METHOD: A systematic analysis of data from participating CP registers/surveillance systems and population-based prevalence studies (from birth year 1995) was performed. Quality and risk of bias were assessed for both data sources. Analyses were conducted for pre-/perinatal, postnatal, neonatal, and overall CP. For each region, trends were statistically classified as increasing, decreasing, heterogeneous, or no change, and most recent prevalence estimates with 95% confidence intervals (CI) were calculated. Meta-analyses were conducted to determine current birth prevalence estimates (from birth year 2010). RESULTS: Forty-one regions from 27 countries across five continents were represented. Pre-/perinatal birth prevalence declined significantly across Europe and Australia (11 out of 14 regions), with no change in postneonatal CP. From the limited but increasing data available from regions in low- and middle-income countries (LMICs), birth prevalence for pre-/perinatal CP was as high as 3.4 per 1000 (95% CI 3.0-3.9) live births. Following meta-analyses, birth prevalence for pre-/perinatal CP in regions from high-income countries (HICs) was 1.5 per 1000 (95% CI 1.4-1.6) live births, and 1.6 per 1000 (95% CI 1.5-1.7) live births when postneonatal CP was included. INTERPRETATION: The birth prevalence estimate of CP in HICs declined to 1.6 per 1000 live births. Data available from LMICs indicated markedly higher birth prevalence. WHAT THIS PAPER ADDS: • Birth prevalence of pre-/perinatal cerebral palsy (CP) in high-income countries (HICs) is decreasing. • Current overall CP birth prevalence for HICs is 1.6 per 1000 live births. • Trends in low- and middle-income countries (LMICs) cannot currently be measured. • Current birth prevalence in LMICs is markedly higher than in HICs. • Active surveillance of CP helps to assess the impact of medical advancements and social/economic development. • Population-based data on prevalence and trends of CP are critical to inform policy.


Assuntos
Paralisia Cerebral , Feminino , Humanos , Recém-Nascido , Gravidez , Austrália/epidemiologia , Paralisia Cerebral/epidemiologia , Europa (Continente)/epidemiologia , Pobreza , Prevalência
20.
PLoS One ; 17(5): e0268108, 2022.
Artigo em Inglês | MEDLINE | ID: mdl-35588131

RESUMO

AIM: To study the association between the socioeconomic environment of area of residence and prevalence and characteristics of children with cerebral palsy (CP). METHOD: Data on 8-year-old children with CP born in 2000-2011 (n = 252) were extracted from a regional population-based register in France. The European Deprivation Index (EDI), available at census block level, characterised socioeconomic deprivation in the child's area of residence at age of registration. The prevalence of CP was estimated in each group of census units defined by EDI distribution tertiles in the general population. The association between deprivation level and CP severity was assessed according to term/preterm status. RESULTS: CP prevalence differed between deprivation risk groups showing a J-shaped form with the prevalence in the most deprived tertile (T3) being the highest but not significantly different of the prevalence in the least deprived one (T1). However, the prevalence in the medium deprivation tertile (T2) was significantly lower than that in the most deprived one with a prevalence risk ratio (PRR) of: PRRT2/T3 = 0.63 95% CI [0.44-0.89]). Prevalences of CP with associated intellectual disability (ID) and CP with inability to walk were significantly higher in the most deprived tertile compared to the least deprived one (respectively PRRT3/T1 = 1.86 95% CI [1.19-2.92] and PRRT3/T1 = 1.90 95% CI [1.07-3.37]). Compared to children living in the least deprived areas, children with CP born preterm living in the most deprived areas had more severe forms of motor impairment, such as an inability to walk or a combination of an inability to walk and moderate to severe impairment of bimanual function. They also had more associated intellectual disability. No associations were observed among term-born children. INTERPRETATION: A significant association between area deprivation group and CP severity was observed among preterm children but not among term-born children.


Assuntos
Paralisia Cerebral , Deficiência Intelectual , Adulto , Paralisia Cerebral/complicações , Paralisia Cerebral/epidemiologia , Criança , Humanos , Recém-Nascido , Deficiência Intelectual/complicações , Prevalência , Fatores de Risco , Classe Social , Adulto Jovem
SELEÇÃO DE REFERÊNCIAS
DETALHE DA PESQUISA