RESUMEN
OBJECTIVE: To compare efficacy and side effect profile data on conservative, behavioral, pharmacological, and surgical treatments used for pediatric saliva control. STUDY DESIGN: A cohort study of children (n = 483) referred to a specialty Saliva Control service between May 2014 and November 2019 was performed, using quantitative data from pretreatment and post-treatment questionnaires (the Drooling Impact Scale [DIS], Drooling Rating Scale [DRS]) and recording of side effects. Overall, 483 children were included; treatment choices were based on published international guidelines. RESULTS: The greatest improvement was seen after intraglandular botulinum toxin A (BTX-A) injections (n = 207; 551 courses; mean DIS change, 34.7; 95% CI = 29.2-35.7) or duct transpositional surgery (n = 31; mean change in DIS, 29.0; 95% CI, 22.3-35.7). Oral anticholinergics were associated with good outcomes, with no significant statistical difference between glycopyrronium bromide (n = 150; mean DIS change, 21.5; 95% CI, 19.1-24.0) or trihexyphenidyl (n = 87; mean DIS change, 22.4; 95% CI, 18.9-25.8). Inhaled ipratropium bromide was not as efficacious (n = 80; mean DIS change, 11.1; 95% CI, 8.9-13.3). Oromotor programs were used in a selected group with reliable outcomes (n = 9; mean DIS change, 13.0). Side effects were consistent with previous studies. Overall, in cases of milder severity, enterally administered therapies provided a good first-line option. With more severe problems, BTX-A injections or saliva duct transpositional surgery were more effective and well tolerated. CONCLUSIONS: We describe a large, single-center pediatric saliva control cohort, providing direct comparison of the efficacy and side effect profiles for all available interventions and inform clinical practice for specialists when considering different options. BTX-A injections or saliva duct transpositional surgery seem to be more effective for saliva control that is more severe.
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Toxinas Botulínicas Tipo A , Parálisis Cerebral , Sialorrea , Niño , Humanos , Saliva , Sialorrea/tratamiento farmacológico , Sialorrea/etiología , Estudios de Cohortes , Toxinas Botulínicas Tipo A/uso terapéutico , Conductos Salivales , Resultado del Tratamiento , Parálisis Cerebral/complicacionesRESUMEN
BACKGROUND: The COVID-19 pandemic resulted in an unprecedented societal and healthcare global crisis. Associated changes in regular healthcare provision and lifestyle through societal lockdown are likely to have affected clinical management and well-being of children/young people with neurodisability, who often require complex packages of multidisciplinary care. METHODS: We surveyed 108 families of children/young people with severe physical neurodisability and multiple comorbidities to understand how the pandemic had affected acute clinical status, routine healthcare provision, schooling and family mental and social well-being. RESULTS: A significant proportion of families reported missing hospital appointments and routine therapy, with subsequent worsening of symptoms and function. Families additionally described worsening stress and anxiety during the pandemic, regardless of their baseline level of socio-economic deprivation. CONCLUSION: This highlights the profound effect of the COVID-19 pandemic on health and function in young people with severe neurodisabilities and emphasizes the clear need to better understand how to support this vulnerable population moving forwards.
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COVID-19 , Personas con Discapacidad , Adolescente , COVID-19/epidemiología , Niño , Control de Enfermedades Transmisibles , Humanos , Pandemias , Encuestas y CuestionariosRESUMEN
AIM: To evaluate clinicians' perspectives on the impact of 'lockdown' during the COVID-19 pandemic for children and young people with severe physical neurodisability and their families. METHOD: Framework analysis of comments from families during a recent service review was used to code the themes discussed according to the World Health Organization International Classification of Functioning, Disability and Health (ICF) and interpreted into emergent themes to summarize the impact of lockdown (Stage 1). They were presented to a clinician focus group for discussion (consultants and physiotherapists working in a specialist motor disorders service, [Stage 2]). RESULTS: Three overarching themes 'Uncertainty and Anxiety', 'Exacerbation of Existing Inequalities' and 'Care Provision: Reaction, Adaptation, and Innovation' summed up the impact of the COVID-19 pandemic on health and well-being in children and young people with neurodisability and their families. All themes were influenced by time. INTERPRETATION: This study reflects clinician's perceptions of family experiences of the pandemic and lockdown. Significant impact is apparent in the entire U.K. population, but the complexity of care needs for children with physical neurodisability exacerbates this. Lobbying for government policy is vital to ensure that all children, and in particular those with significant health and social care needs, are protected and continue to access services. During the restoration and recovery phase of the pandemic, there is a need for service reconfiguration that utilizes what we have learned and is adaptive to individual family circumstances.
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COVID-19 , Personas con Discapacidad , Adolescente , COVID-19/epidemiología , Niño , Control de Enfermedades Transmisibles , Grupos Focales , Humanos , PandemiasRESUMEN
AIM: To explore factors predicting acquisition and loss of best walking ability in young people with bilateral cerebral palsy (CP). METHOD: In our population cohort (Study of Hips And Physical Experience) of 338 children (201 males, 137 females) with bilateral CP, age at achieving walking was recorded and walking ability predicted from early motor milestones. Walking was assessed at 5 to 8 years (mean 7y) and in 228 of 278 survivors at 13 to 19 years (mean 16y). Parent carers reported their view of any loss of best achieved walking. Factors potentially associated with loss of best achieved walking were explored: severity and type of motor disorder; intellect and communication; manipulative skill; general health and comorbidity; pain; orthopaedic surgery; musculoskeletal spine and lower limb deformity; weight; fatigue; mood; and presence of regular exercise regime. RESULTS: The ability to walk independently was reliably predicted by the motor milestone 'getting to sit and maintain sitting' by the age of 36 months (without aids) and 55 months (with aids). Forty-five per cent of the cohort never walked 10 steps independently. Not all who achieved walking without aids were still doing so by a mean age of 16 years, which was associated with later age at achieving walking and the degree of musculoskeletal deformity, as was the parent carers' report of loss of best walking. INTERPRETATION: In this study, development of musculoskeletal deformity was a significant factor in not maintaining best achieved walking by mean age 16 years, which is most likely to occur in young people whose walking ability is with aids over short distances or in therapy only. Prediction of future walking ability in a child with bilateral CP can be made from early motor milestones.
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Parálisis Cerebral/fisiopatología , Desarrollo Infantil/fisiología , Trastornos Neurológicos de la Marcha/fisiopatología , Caminata/fisiología , Adolescente , Adulto , Parálisis Cerebral/complicaciones , Parálisis Cerebral/diagnóstico , Niño , Preescolar , Estudios de Cohortes , Femenino , Trastornos Neurológicos de la Marcha/diagnóstico , Trastornos Neurológicos de la Marcha/etiología , Humanos , Masculino , Adulto JovenRESUMEN
AIM: To assess the efficacy, safety, and tolerability of oromucosal nabiximols cannabinoid medicine as adjunct therapy for children with spasticity due to cerebral palsy/traumatic central nervous system injury with inadequate response to existing treatment. METHOD: Overall, 72 patients (mean [SD] age 12y 4mo [3y 1mo], range 8-18y) were randomized at a ratio of 2:1 to receive nabiximols (n=47; 29 males, 18 females) or placebo (n=25; 15 males, 10 females) for 12 weeks (12 sprays/day max. based on clinical response/tolerability). The primary outcome was change from baseline in level of spasticity on a 0 to 10 Numerical Rating Scale (NRS), assessed by the primary caregiver at 12 weeks. Secondary outcomes included additional measures for spasticity, sleep quality, pain, health-related quality of life, comfort, depression, and safety. RESULTS: There was no significant difference in the spasticity 0 to 10 NRS between nabiximols versus placebo groups after 12 weeks. No statistically significant differences were observed for any secondary endpoint. Adverse events were predominantly mild or moderate in severity; however, three cases of hallucinations were reported. INTERPRETATION: Nabiximols was generally well tolerated; however, neuropsychiatric adverse events were observed. No significant reduction in spasticity with nabiximols treatment versus placebo was observed. WHAT THIS PAPER ADDS: Oromucosal nabiximols is generally well tolerated by paediatric patients. However, three cases of hallucinations were observed, one of which involved auditory hallucinations and a suicide attempt. Oromucosal nabiximols versus placebo did not reduce cerebral palsy/central nervous system injury-related spasticity.
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Lesiones Traumáticas del Encéfalo/complicaciones , Cannabidiol/uso terapéutico , Parálisis Cerebral/complicaciones , Parálisis Cerebral/tratamiento farmacológico , Dronabinol/uso terapéutico , Espasticidad Muscular/tratamiento farmacológico , Administración Oral , Adolescente , Niño , Método Doble Ciego , Combinación de Medicamentos , Femenino , Humanos , Masculino , Espasticidad Muscular/complicaciones , Resultado del TratamientoAsunto(s)
Parálisis Cerebral/rehabilitación , Adulto , Enfermedades Óseas/etiología , Parálisis Cerebral/complicaciones , Parálisis Cerebral/psicología , Atención a la Salud/organización & administración , Atención a la Salud/normas , Evaluación de la Discapacidad , Accesibilidad a los Servicios de Salud , Humanos , Trastornos Mentales/etiología , Dolor/etiología , Trastornos Respiratorios/etiología , Índice de Severidad de la EnfermedadRESUMEN
AIM: To assess whether preoperative botulinum neurotoxin A (BoNT-A) affects pain after major hip surgery for children with bilateral cerebral palsy (CP). METHOD: This was a randomized, parallel arms, placebo-contolled trial. Children with hypertonic CP aged 2 to 15 years awaiting bony hip surgery at a tertiary hospital were randomized to receive either BoNT-A or placebo injections into the muscles of the hip on a single occasion immediately before surgery. The primary outcome was the paediatric pain profile (PPP), which was assessed at baseline and weekly for 6 weeks. Treatment allocation was by minimization. Participants, clinicians, and outcome assessors were masked to group assignment. RESULTS: Twenty-seven participants (17 males, 10 females; mean 8y 8mo [SD 3y 9mo], range 3y 4mo-15y 10mo) were allocated to BoNT-A and 27 participants (14 males, 13 females; mean 8y 11mo [SD 3y 5mo], range 4y 1mo-15y 2mo) to placebo. Mean (SD) PPP at 6 weeks for the BoNT-A group (n=24 followed up) was 10.96 (7.22) and for the placebo group (n=26) was 10.04 (8.54) (p=0.69; 95% confidence interval [CI] -4.82, 3.18). There were 16 serious adverse events in total during 6 months of follow-up (n=6 in BoNT-A group). INTERPRETATION: Use of BoNT-A immediately before bony hip surgery for reducing postoperative pain for children with CP was not supported. WHAT THIS PAPER ADDS: Botulinum neurotoxin A (BoNT-A) does not reduce postoperative pain following bony hip surgery. BoNT-A also does not affect postoperative quality of life.
NEUROTÓXINA A BOTULÍNICA PREOPERATORIA PARA NIÑOS CON PARÁLISIS CEREBRAL BILATERAL QUE VAN A SER SOMETIDOS A UNA CIRUGÍA MAYOR DE CADERA: UN ENSAYO ALEATORIO, DOBLE CIEGO, CONTROLADO CON PLACEBO: OBJETIVE: Evaluar si la neurotóxina A botulínica preoperatoria (BoNT-A) afecta el dolor después de una cirugía mayor de cadera en niños con parálisis cerebral bilateral (PC). MÉTODO: Este fue un ensayo aleatorio, con brazos paralelos, controlado con placebo. Los niños con PC hipertónica de 2 a 15 años de edad que esperaban una cirugía de cadera en un hospital terciario se escogieron al azar para recibir inyecciones de BoNT-A o de placebo en los músculos de la cadera una sola administración previa a la cirugía. El resultado primario fue el perfil de dolor pediátrico (PPP, siglas en ingles), que se evaluó al inicio del estudio y semanalmente durante 6 semanas. La asignación del tratamiento fue por minimización. Tanto los participantes, como los clínicos y evaluadores de resultados, eran desconocidos para la asignación de grupo. RESULTADOS: Veintisiete participantes (17 varones y 10 mujeres; medios 8 años 8 meses) [Desviación Estandar SD 3 años 9 meses], rango 3 años 4 meses-15 años 10 meses) se les administro BoNT-A y 27 participantes (14 varones y 13 mujeres; media 8 años 11 meses [SD 3 años 5 meses], rango 4 años 1 mes - 15 años 2 meses) a placebo. La PPP media (SD) a las 6 semanas para el grupo de BoNT-A (n = 24 seguidas) fue de 10,96 (7,22) y para el grupo de placebo (n = 26) fue de 10,04 (8,54) (p = 0,69; intervalo de confianza del 95% [CI, siglas en ingles] -4,82, 3,18). Hubo 16 eventos adversos graves en total durante 6 meses de seguimiento (n = 6 en el grupo BoNT-A). INTERPRETACIÓN: El uso de BoNT-A inmediatamente antes de la cirugía de cadera con el fin de reducir el dolor postoperatorio en niños con PC no fue consistente.
NEUROTOXINA BOTULÍNICA A PRÉ-OPERATÓRIA PARA CRIANÇAS COM PARALISIA CEREBRAL BILATERAL SUBMETIDAS A GRANDE CIRURGIA DE QUADRIL: UM ESTUDO RANDOMIZADO, DUPLO-CEGO, CONTROLADO POR PLACEBO: OBJETIVO: Avaliar se a neurotoxina botulínica tipo A (BTA) pré-operatória A afeta a dor após grande cirurgia de quadril em crianças com paralisia cerebral bilateral (PC). MÉTODO: Este foi um estudo randomizado, com braços paralelos e controlado por placebo. Crianças com PC espástica com idade entre 2 a 15 anos aguardando cirurgia óssea de quadril em um hospital terciário foram randomizadas para receber ou BTA ou injeções de placebo nos músculos do quadril em uma única ocasião imediatamente antes da cirurgia. O desfecho primário foi o perfil de dor pediátrica (PDP), que foi avaliado na linha de base e semanalmente por 6 semanas. A alocação de tratamento foi por minimização. Os participantes, clínicos e avaliadores de resultados foram cegados quanto a atribuição de grupo. RESULTADOS: Vinte e sete participantes (17 homens, 10 mulheres; média de 8 anos e 8 meses [DP 3 anos e 9 meses], com idade entre 3 anos e 4 meses à 15 anos e 10 meses) foram alocados para o grupo BTA e 27 participantes (14 homens, 13 mulheres; média de 8 anos e 11 meses [DP 3 anos e 5 meses], com idade entre 4anos e 1mês à 15anos e 2 meses) foram alocados no grupo placebo. A média (DP) do PDP às 6 semanas para o grupo BTA (n = 24) foi de 10,96 (7,22) e para o grupo placebo (n = 26) foi de 10,04 (8,54) (p = 0,69; intervalo de confiança de 95% [IC] -4,82, 3,18). Houve 16 eventos adversos sérios no total durante 6 meses de acompanhamento (n = 6 no grupo BTA). INTERPRETAÇÃO: O uso da BTA imediatamente antes da cirurgia óssea do quadril para reduzir a dor pós-operatória em crianças com PC não foi apoiado.
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Toxinas Botulínicas Tipo A/uso terapéutico , Parálisis Cerebral/cirugía , Articulación de la Cadera/cirugía , Fármacos Neuromusculares/uso terapéutico , Procedimientos Ortopédicos/métodos , Dolor Postoperatorio/prevención & control , Adolescente , Niño , Preescolar , Método Doble Ciego , Femenino , Humanos , Masculino , Procedimientos Ortopédicos/efectos adversos , Calidad de Vida , Resultado del TratamientoRESUMEN
While there remains limited intervention to address the damage to the developing brain, current multidisciplinary management of cerebral palsy (CP) needs to minimise the impact of secondary musculoskeletal complications. A focus on comorbidities to maximise function for activity and participation by supporting the child and family in their environment is required. Comprehensive clinical guidance was published by National Institute for Health and Care Excellence (NICE) earlier this year. This article aims to provide a practical clinical approach to the child and family based on:(1) art: empathy, listening and weighing up the clinical picture of the child and family in context; diagnosis, the need for support and space; and care coordination at the right time; and (2) science: the current science in CP care is rapidly expanding in terms of plasticity, pathophysiology, functional assessments and treatments.
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Parálisis Cerebral/terapia , Toxinas Botulínicas/uso terapéutico , Parálisis Cerebral/diagnóstico , Parálisis Cerebral/fisiopatología , Niño , Comunicación , Comorbilidad , Evaluación de la Discapacidad , Humanos , Clasificación Internacional del Funcionamiento, de la Discapacidad y de la Salud , Procedimientos Neuroquirúrgicos , Terapia Ocupacional , Procedimientos Ortopédicos , Padres , Modalidades de Fisioterapia , Relaciones Profesional-Familia , Apoyo Social , LogopediaRESUMEN
AIM: We explored factors associated with pain and its severity in a population cohort of young people with bilateral cerebral palsy, comparing parent/carer and young people self-reports. METHOD: Of 278 survivors (mean age 16y 8mo, SD 1y 4mo, range 13y 8mo-19y 3mo) from the South Thames in the Study of Hips and Physical Experience cohort of 338 young people with bilateral cerebral palsy, 212 parents/carers and 153 young people completed questionnaires on the presence, severity, timing, site, associated factors, impact, and treatment of pain. RESULTS: Seventy per cent of parents/carers reported pain within 3 months, 59% the previous week, and 50% the previous day with 56% reporting 'regularly experienced'. Of young people able to do so, 63% reported pain within 3 months, 50% the previous week, and 42% the previous day, with 48% reporting regular pain. There was strong agreement between the parent/carer and young people, reporting pain severity over the previous 3 months. Pain severity was associated with increased motor impairment and comorbidity, particularly constipation, spasticity, equipment use, and higher emotional score, but not sex, intellectual disability, speech, or maternal education. Multiple sites of musculoskeletal pain were reported in two-thirds of individuals. Pain was associated with voluntary movement in individuals with less motor impairment and with being moved in those with severe motor impairment. Greater pain severity had a negative effect on both physical and psychological quality of life. INTERPRETATION: Increasing awareness of the comorbidities in cerebral palsy may aid effective treatment, reducing pain experienced by young people with cerebral palsy. WHAT THIS STUDY ADDS: Regular moderate or severe pain is reported in young people with bilateral cerebral palsy (CP) in all Gross Motor Function Classification System levels. Pain is reported more frequently in young people who are non-ambulant. General ill health is strongly associated with severity of pain after controlling for severity of CP, especially constipation. Pain occurs most often in ambulant young people during voluntary activity and in those who are non-ambulant when being moved. There is strong agreement between parents/carers and young people about pain presence and severity.
FACTORES ASOCIADOS AL DOLOR EN ADOLESCENTES CON PARÁLISIS CEREBRAL BILATERAL: OBJETIVO: Exploramos los factores asociados con el dolor y su gravedad en una cohorte poblacional de jóvenes con parálisis cerebral bilateral, comparando los autoinformes de padres/cuidadores y jóvenes. MÉTODO: De 278 sobrevivientes (edad media 16 años 8 meses, DS 1 año 4 meses, rango 13 años 8 meses - 19 años 3 meses) del Támesis Meridional, en el Estudio de caderas y experiencia física de 338 jóvenes con parálisis cerebral bilateral, 212 padres/cuidadores y 153 los jóvenes completaron cuestionarios sobre la presencia, la gravedad, el momento, el sitio, los factores asociados, el impacto y el tratamiento del dolor. RESULTADOS: El 70% de los padres/cuidadores reportaron dolor dentro de los 3 meses, el 59% en la semana anterior y el 50% en el día anterior, y el 56% informaron tener dolor "como experiencia regular". De los jóvenes que pueden hacerlo, 63% reportaron dolor dentro de los 3 meses, 50% la semana anterior y 42% el día anterior, con 48% reportando dolor regular. Hubo un fuerte acuerdo entre los padres/cuidadores y los jóvenes, que informaron sobre la gravedad del dolor durante los 3 meses anteriores. La gravedad del dolor se asoció con un aumento del deterioro motor y la comorbilidad, en particular el estreñimiento, la espasticidad, el uso de equipos y una mayor puntuación emocional, pero no el sexo, la discapacidad intelectual, el habla o la educación materna. Se reportaron múltiples sitios de dolor musculoesquelético en dos tercios de los individuos. El dolor se asoció con el movimiento voluntario en individuos con menos discapacidad motora y con el traslado en personas con insuficiencia motora grave. Una mayor gravedad del dolor tuvo un efecto negativo en la calidad de vida tanto física como psicológica. INTERPRETACIÓN: Un mayor conocimiento de las comorbilidades en la parálisis cerebral puede ayudar a un tratamiento eficaz, reduciendo el dolor que experimentan los jóvenes con parálisis cerebral.
FATORES ASSOCIADOS COM DOR EM ADOLESCENTES COM PARALISIA CEREBRAL BILATERAL: OBJETIVO: Exploramos os fatores associados com dor e sua severidade em uma coorte de jovens com paralisia cerebral bilateral, comparando auto-relatos dos jovens e relatos dos pais/cuidadores. MÉTODO: Dos 278 sobreviventes (média de idade 16a 8m, DP 1a 4m, variação 13a 8m-19a 3m) de South Thames, da coorte de 228 jovens com paralisia cerebral bilateral do Estudo dos Quadris e Experiência Física, 212 pais/cuidadores e 153 jovens completaram os questionários de presença, severidade, momento, local, fatores associados, impacto, e tratamento da dor. RESULTADOS: Setenta por cento dos pais/cuidadores relataram dor em 3 meses, 59% na semana anterior, e 50% no dia anterior, com 56% indicando "sente regularmente". Dos jovens capazes de fazê-lo, 63% reportaram dor em 3 meses, 50% na última semana, e 42% no dia anterior, com 48% reportando dor regular. Houve forte concordância entre pais/cuidadores e jovens, relatando severidade da dor nos 3 meses anteriores. A severidade da dor se associou com maior comprometimento motor e comorbidades, particularmente constipação, espasticidade, uso de equipamentos, e maior pontuação emocional, mas não com o sexo, deficiência intelectual, fala, ou educação materna. Múltiplos locais de dor músculo-esquelética foram relatados em dois terços dos indivíduos. A dor foi associada com movimento voluntário em indivíduos com menor comprometimento motor, e com ser movido naqueles com comprometimento severo. Maior severidade da dor teve efeito negativo tanto na qualidade de vida física e psicológica. INTERPRETAÇÃO: Aumentar a conscientização sobre as comorbidades em paralisia cerebral pode ajudar tratamentos efetivos, reduzindo a dor sentida por jovens com paralisia cerebral.
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Parálisis Cerebral/complicaciones , Espasticidad Muscular/complicaciones , Dolor/etiología , Calidad de Vida , Adolescente , Femenino , Humanos , Masculino , Dolor/diagnóstico , Dimensión del Dolor , Factores de Riesgo , Índice de Severidad de la Enfermedad , Adulto JovenRESUMEN
AIM: Evaluation of topical Glyceryl trinitrate as a therapeutic option to improve peripheral circulation in 16 children with severe complex neurodisability, poor capillary return and signs of vascular insufficiency, including discomfort. METHODS: If insufficient improvement in capillary return was obtained using conservative measures, a 5 mg GTN patch was placed each day on the front of the tibia of each leg. Children were reviewed clinically for up to 12 months of treatment. Distal limb comfort was measured using a Likert scale, either patient or carer reported. Standardised capillary refill time was assessed at each clinical review. RESULTS: Use of the patches led to improved capillary refill time and parent/patient reported comfort in all children. Healing of skin ulcers in lower limbs after application of the patch was also noted. There was universal parent/patient satisfaction with use of the patch. One child with Aicardi Goutieres syndrome had a skin reaction with prolonged use and minor adverse effects were reported in 4 others but this did not result in discontinuation of treatment. SUMMARY: GTN patches were relatively well tolerated in this group of children. We suggest that use of GTN patches be considered for children with severe neurodisability and poor peripheral circulation causing discomfort. GTN patches may also have a role in healing of persistent skin ulcers for these children.
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Circulación Sanguínea/efectos de los fármacos , Enfermedades del Sistema Nervioso Central/tratamiento farmacológico , Nitroglicerina/uso terapéutico , Vasodilatadores/uso terapéutico , Adolescente , Niño , Femenino , Humanos , Masculino , Proyectos de InvestigaciónRESUMEN
CLN2 disease (neuronal ceroid lipofuscinosis type 2) is a rare, autosomal recessive, pediatric-onset, rapidly progressive neurodegenerative lysosomal storage disorder caused by tripeptidyl peptidase 1 (TPP1) enzyme deficiency, and is characterized by language delay, seizures, rapid cognitive and motor decline, blindness, and early death. No management guidelines exist and there is a paucity of published disease-specific evidence to inform clinical practice, which currently draws upon experience from the field of childhood neurodisability. Twenty-four disease experts were surveyed on CLN2 disease management and a subset met to discuss current practice. Management goals and strategies are consistent among experts globally and are guided by the principles of pediatric palliative care. Goals and interventions evolve as the disease progresses, with a shift in focus from maintenance of function early in the disease to maintenance of quality of life. A multidisciplinary approach is critical for optimal patient care. This work represents an initial step toward the development of consensus-based management guidelines for CLN2 disease.
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Lipofuscinosis Ceroideas Neuronales/terapia , Manejo de la Enfermedad , Humanos , Tripeptidil Peptidasa 1RESUMEN
OBJECTIVE: To investigate longer-term physical and neurodevelopmental outcomes of patients with hypoplastic left heart syndrome (HLHS) compared with other patients with functionally single-ventricle circulation surviving beyond the age of 10â years. DESIGN: A retrospective, observational study from a UK tertiary centre for paediatric cardiology. RESULTS: 58 patients with HLHS and 44 non-HLHS patients with single-ventricle physiology were included. Subjective reduction in exercise tolerance was reported in 72% (95% CI 61% to 84%) of patients with HLHS and 45% (31% to 60%) non-HLHS patients. Compared with non-HLHS patients, educational concerns were reported more frequently in patients with HLHS, 41% (29% to 54%) vs 23% (10% to 35%), as was a diagnosis of a behaviour disorder (autism or attention deficit hyperactivity disorder) 12% (4% to 21%) vs 0%, and referral to other specialist services 67% (55% to 79%) vs 48% (33% to 63%). CONCLUSIONS: Within a group of young people with complex congenital heart disease, those with HLHS are likely to have worse physical, psychological and educational outcomes.
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Trastornos de la Conducta Infantil/fisiopatología , Desarrollo Infantil , Ventrículos Cardíacos/anomalías , Síndrome del Corazón Izquierdo Hipoplásico/fisiopatología , Desempeño Psicomotor/fisiología , Adolescente , Niño , Trastornos de la Conducta Infantil/psicología , Femenino , Humanos , Síndrome del Corazón Izquierdo Hipoplásico/psicología , Masculino , Derivación y Consulta , Estudios Retrospectivos , Centros de Atención Terciaria , Reino UnidoRESUMEN
BACKGROUND: Drooling saliva is a common problem in children with neurodevelopmental disorders. The negative consequences of drooling include skin breakdown, dehydration, and damage to clothing and equipment. Children and families often suffer social embarrassment due to drooling. There is no evidence about the relative effectiveness, side effect profiles or patient acceptability of the two medications most commonly used to reduce drooling - glycopyrronium and hyoscine. Consequently, there is no consensus or guideline to aid clinical decisions about which drug to use, and at what dose. METHODS/DESIGN: A multi-centre, randomised trial of treatment with glycopyrronium or hyoscine in children with problematic drooling and non-progressive neurodisability. Ninety children aged between 3 and 15 years who have never received medication for drooling will be stratified by severity of drooling and care centre. Randomisation to receive treatment with glycopyrronium or hyoscine will be computer generated from the trial randomisation website. Dose adjustment and side effect monitoring will occur via telephone consultation. Medication arm will be known to participants and clinicians but not the Trial Outcome Assessor.The primary outcome measure is the Drooling Impact Scale score at four weeks, at which time all children will be on the maximum tolerated dose of their medication. Secondary outcome measures include change in Drooling Impact Scale score between baseline, 4, 12 and 52 weeks, change in Drooling Severity and Frequency Scale score and difference between groups in the Treatment Satisfaction Questionnaire for Medication score. A structured interview with children and young people of sufficient age, cognitive and communication ability will explore their perceptions of drooling and the effectiveness and acceptability of the medications. DISCUSSION: The primary objective of the study is to identify whether glycopyrronium or hyoscine is more effective in treating drooling in children with non-progressive neurodisability. The study will also determine which medications at what doses are most acceptable and have fewest side effects. This information will be used to develop evidence based guidance to inform the medical treatment of drooling. DRI TRIAL REGISTRATION: Current Controlled Trials: ISRCTN75287237.EUDRACT: 2013-000863-94.Medicines and Healthcare products Regulatory Agency (MHRA): 17136/0264/001-0003.
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Discapacidades del Desarrollo/complicaciones , Glicopirrolato/uso terapéutico , Antagonistas Muscarínicos/uso terapéutico , Enfermedades del Sistema Nervioso/complicaciones , Proyectos de Investigación , Salivación/efectos de los fármacos , Escopolamina/uso terapéutico , Sialorrea/tratamiento farmacológico , Adolescente , Niño , Preescolar , Protocolos Clínicos , Inglaterra , Glicopirrolato/efectos adversos , Humanos , Antagonistas Muscarínicos/efectos adversos , Satisfacción del Paciente , Escopolamina/efectos adversos , Sialorrea/diagnóstico , Sialorrea/etiología , Sialorrea/fisiopatología , Método Simple Ciego , Encuestas y Cuestionarios , Factores de Tiempo , Resultado del TratamientoRESUMEN
The descriptive term of cerebral palsy encompasses the largest group of childhood movement disorders. Severity and pattern of clinical involvement varies widely dependent on the area of the central nervous system compromised. A multidisciplinary team approach is vital for all the aspects of management to improve function and minimise disability. From a medical viewpoint, there are two pronged approaches. First a focus on developmental and clinical comorbidities such as communication, behaviour, epilepsy, feeding problems, gastro-oesophageal reflux and infections; and second on specifics of muscle tone, motor control and posture. With regards to the latter, there is an increasing number of available treatments including oral antispasticity and antidystonic medications, injectable botulinum toxin, multilevel orthopaedic and neurosurgical options and a variety of complementary and alternative therapies.
Asunto(s)
Parálisis Cerebral/fisiopatología , Parálisis Cerebral/terapia , Toxinas Botulínicas Tipo A/uso terapéutico , Encéfalo/embriología , Encéfalo/crecimiento & desarrollo , Parálisis Cerebral/epidemiología , Niño , Terapias Complementarias , Estimulación Encefálica Profunda , Evaluación de la Discapacidad , Dopaminérgicos/uso terapéutico , Trastornos Distónicos/fisiopatología , Trastornos Distónicos/terapia , Humanos , Bombas de Infusión , Inyecciones Intramusculares , Relajantes Musculares Centrales/uso terapéutico , Espasticidad Muscular/fisiopatología , Espasticidad Muscular/terapia , Fármacos Neuromusculares/uso terapéutico , Procedimientos Ortopédicos , Grupo de Atención al Paciente , Células Madre Pluripotentes/trasplante , Rizotomía , Índice de Severidad de la EnfermedadRESUMEN
An interdisciplinary European group of clinical experts in the field of movement disorders and experienced Botulinum toxin users has updated the consensus for the use of Botulinum toxin in the treatment of children with cerebral palsy (CP). A problem-orientated approach was used focussing on both published and practice-based evidence. In part I of the consensus the authors have tabulated the supporting evidence to produce a concise but comprehensive information base, pooling data and experience from 36 institutions in 9 European countries which involves more than 10,000 patients and over 45,000 treatment sessions during a period of more than 280 treatment years. In part II of the consensus the Gross Motor Function Measure (GMFM) and Gross Motor Function Classification System (GMFCS) based Motor Development Curves have been expanded to provide a graphical framework on how to treat the motor disorders in children with CP. This graph is named "CP(Graph) Treatment Modalities - Gross Motor Function" and is intended to facilitate communication between parents, therapists and medical doctors concerning (1) achievable motor function, (2) realistic goal-setting and (3) treatment perspectives for children with CP. The updated European consensus 2009 summarises the current understanding regarding an integrated, multidisciplinary treatment approach using Botulinum toxin for the treatment of children with CP.