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AIM: To assess the efficacy and safety of repeat abobotulinumtoxinA injections in reducing upper limb spasticity in children with cerebral palsy (CP). METHOD: This was a double-blind, repeat-cycle study (NCT02106351) in children with CP (2-17y). Children were randomized to receive 2U/kg (control), 8U/kg, or 16U/kg abobotulinumtoxinA injections into the target muscle group (wrist or elbow flexors) and additional muscles alongside occupational therapy via a home-exercise therapy program (HETP; minimum five 15min sessions/wk). Children received 8U/kg or 16U/kg plus HETP in cycles 2 to 4. RESULTS: During cycle 1, 210 children (126 males, 84 females; mean age [SD] 9y [4y 5mo], range 2-17y; n=70/group) had at least one upper limb abobotulinumtoxinA injection and 209 complied with the HETP. At week 6 of cycle 1, children in the 8U/kg or 16U/kg groups had significantly lower Modified Ashworth scale scores versus the 2U/kg group (primary outcome: treatment differences of -0.4 [p=0.012] and -0.7 [p<0.001] respectively). All groups improved on Physician Global Assessment and children in all groups achieved their treatment goals at least as expected. Therapeutic benefits were sustained during cycles 2 to 4; muscular weakness was the only treatment-related adverse event reported in at least one child/group (4.3% and 5.7% vs 1.4% respectively). INTERPRETATION: Treatment with 8U/kg or 16U/kg abobotulinumtoxinA significantly reduced upper limb spasticity versus the 2U/kg control dose. Therapeutic benefits of abobotulinumtoxinA plus HETP were sustained with repeat treatment cycles. WHAT THIS PAPER ADDS: AbobotulinumtoxinA injections significantly reduced upper limb spasticity in children with cerebral palsy. Children treated with abobotulinumtoxinA and targeted home exercises showed global improvement and goal attainment. Benefits were sustained over 1 year with repeat cycles of abobotulinumtoxinA and home exercises. AbobotulinumtoxinA injections into the upper limb were well tolerated over 1 year.
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Toxinas Botulínicas Tipo A/uso terapêutico , Paralisia Cerebral/tratamento farmacológico , Espasticidade Muscular/tratamento farmacológico , Fármacos Neuromusculares/uso terapêutico , Extremidade Superior/fisiopatologia , Adolescente , Toxinas Botulínicas Tipo A/efeitos adversos , Paralisia Cerebral/fisiopatologia , Criança , Pré-Escolar , Método Duplo-Cego , Feminino , Humanos , Injeções Intramusculares , Masculino , Espasticidade Muscular/fisiopatologia , Fármacos Neuromusculares/efeitos adversos , Resultado do TratamentoRESUMO
PURPOSE OF THE REVIEW: The goal of the article is to describe a systematic approach through core principles and steps for the transition of the patient with a neurological disorder to the adult model of care, to provide steps and principles to help receiving providers successfully integrate the patient into their practice, and to discuss cultural, systemic, and discipline-based barriers to transition. RECENT FINDING: The literature has expanded rapidly. The recent publications help define the barriers to the process and are currently exploring the best methods to evaluate readiness, needs, barriers, and develop solutions for best practices. There is a consensus that there is a need for a systematic approach to transition and integration of the patient with a neurological disorder. The transition of the child and youth with special health care needs (CYSHCN) is complex with multiple barriers. An important concept is that these patients, their families, and medical care providers all benefit from a coordinated and collaborative methodology.
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Doenças do Sistema Nervoso , Cuidado Transicional , Adolescente , Criança , HumanosAssuntos
Comitês Consultivos , Liderança , Neurologia , Inquéritos e Questionários , Criança , HumanosRESUMO
OBJECTIVE: To characterize the population pharmacokinetics (PK) of oral baclofen and assess impact of patient-specific covariates in children with cerebral palsy (CP) in order to support its clinical use. SUBJECTS DESIGN: Children (2-17 years of age) with CP received a dose of titrated oral baclofen from 2.5 mg 3 times a day to a maximum tolerated dose of up to 20 mg 4 times a day. PK sampling followed titration of 10-12 weeks. Serial R- and S-baclofen plasma concentrations were measured for up to 16 hours in 49 subjects. Population PK modeling was performed using NONMEM 7.1 (ICON PLC; Ellicott City, Maryland). RESULTS: R- and S-baclofen showed identical concentration-time profiles. Both baclofen enantiomers exhibited linear and dose/kg-proportional PK, and no sex differences were observed. Average baclofen terminal half-life was 4.5 hours. A 2-compartment PK model with linear elimination and transit absorption steps adequately described concentration-time profiles of both baclofen enantiomers. The mean population estimate of apparent clearance/F was 0.273 L/h/kg with 33.4% inter-individual variability (IIV), and the apparent volume of distribution (Vss/F) was 1.16 L/kg with 43.9% IIV. Delayed absorption was expressed by a mean transit time of 0.389 hours with 83.7% IIV. Body weight, a possible genetic factor, and age were determinants of apparent clearance in these children. CONCLUSION: The PK of oral baclofen exhibited dose-proportionality and were adequately described by a 2-compartment model. Our population PK findings suggest that baclofen dosage can be based on body weight (2 mg/kg per day) and the current baclofen dose escalation strategy is appropriate in the treatment of children with CP older than 2 years of age.
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Baclofeno/farmacocinética , Paralisia Cerebral/tratamento farmacológico , Relaxantes Musculares Centrais/farmacocinética , Absorção , Administração Oral , Adolescente , Baclofeno/sangue , Baclofeno/uso terapêutico , Peso Corporal , Paralisia Cerebral/sangue , Criança , Pré-Escolar , Relação Dose-Resposta a Droga , Esquema de Medicação , Feminino , Meia-Vida , Humanos , Masculino , Taxa de Depuração Metabólica , Modelos Estatísticos , Análise Multivariada , Relaxantes Musculares Centrais/sangue , Relaxantes Musculares Centrais/uso terapêuticoRESUMO
BACKGROUND AND OBJECTIVES: Gender disparities have been demonstrated across several medical specialties, including neurology. Although women have comprised most of the child neurology trainees since 2007, it is not apparent whether this demographic shift is reflected in the Child Neurology Society (CNS) awards and leadership. This study aimed to evaluate the differences in gender representation among leadership positions and award recipients within the CNS. The primary outcome measure was the total number of board of director (BOD) positions or awards given by gender each year. METHODS: A retrospective review of publicly available data was conducted on CNS members, post-training award recipients, and BOD positions, including nomination records, from 1972 to 2023. Data abstracted were restricted to gender to preserve member and nominee anonymity. Gender identification and consensus were determined through a combination of strategies and study members. Data analysis included descriptive statistics, Pearson χ2 test, and the exact binomial test to compare gender proportions and the probability of being underrepresented in awards, leadership, and nominations over time. Data are presented according to the Strengthening the Reporting of Observational Studies in Epidemiology guidelines. RESULTS: From 1972 to 2023, women represented 29% (44/152) of the BOD positions and 26% (61/236) of post-training award recipients presented by the CNS. Despite the increase in the proportion of women in child neurology, the overall gap in gender representation in leadership positions remains broadly stable. Only 13% (4/32) of CNS presidents have been women, a significant underrepresentation (95% CI 2.3%-52%, p < 0.004), although the representation of women in nonpresidential positions increased from 2003 to 2023. Women are also underrepresented as overall awardees (95% CI 12%-38%, p < 0.00001) except for the Philip R. Dodge Young Investigator Award, which is an investigator-initiated application. DISCUSSION: Women remain underrepresented at the highest levels of recognition in child neurology despite representing most of the field. Reasons for disparities are known to be multifactorial and likely include gender bias and structural sexism. We present several discussion topics that seek to rationalize this disparity and provide suggestions for improving diversity, equity, and inclusion for leadership roles and awards.
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Distinções e Prêmios , Liderança , Neurologia , Médicas , Sociedades Médicas , Humanos , Feminino , Masculino , Estudos Retrospectivos , Médicas/estatística & dados numéricos , Sexismo , PediatriaRESUMO
Background and Objectives: Cerebral palsy (CP), the most common motor disability of childhood, is variably diagnosed. We hypothesized that child neurologists and neurodevelopmentalists, often on the frontlines of CP diagnosis in North America, harbor uncertainties regarding the practical application of the most recent CP consensus definition from 2006. Methods: We conducted a cross-sectional survey of child neurologists and neurodevelopmentalists at the 2022 Child Neurology Society Annual Meeting. Attendees were provided the 2006 CP consensus definition and asked whether they had any uncertainties about the practical application of the definition across four hypothetical clinical vignettes. Results: Of 230 attendees, 164 responded to the closing survey questions (71%). 145/164 (88%) expressed at least one uncertainty regarding the clinical application of the 2006 definition. Overwhelmingly, these areas of uncertainty focused on: 1) Age, both with regards to the minimum age of diagnosis and the maximum age of brain disturbance or motor symptom onset, (67/164, 41%), and 2) Interpretation of the term "non-progressive" (48/164, 29%). The vast majority of respondents (157/164, 96%) answered 'Yes' to the question: Do you think we should revise the 2006 consensus definition of CP? Discussion: We propose that the uncertainties we identified could be addressed by operationalizing the 2006 consensus definition to support a more uniform CP diagnosis. To address the most common CP diagnostic uncertainties we identified, we propose 3 points of clarification based on the available literature: 1) Motor symptoms/signs should be present by 2 years old; 2) CP can and should be diagnosed as early as possible, even if activity limitation is not yet present, if motor symptoms/signs can be reasonably predicted to yield activity limitation (e.g. by using standardized examination instruments, Brain MRI, and a suggestive clinical history); and 3) The clinical motor disability phenotype should be non-progressive through 5 years old. We anticipate that operationalizing the 2006 definition of CP in this manner could clarify the uncertainties we identified among child neurologists and neurodevelopmentalists and reduce the diagnostic variability that currently exists.
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OBJECTIVE: This exploratory analysis of a large, randomized, double-blind study (NCT02106351) describes the effect of treatment with abobotulinumtoxinA followed by a tailored home exercises therapy programme in enabling children with upper limb spasticity due to cerebral palsy to achieve their functional goals using goal attainment scaling (GAS). METHODS: Children with cerebral palsy and spasticity in ≥ 1 upper limb received up to 4 injection cycles of abobotulinumtoxinA (2 U/kg (cycle 1 only), 8U/kg and 16U/kg) into the elbow and wrist flexors and other upper limb muscles selected to support individual treatment goals. Children followed a home exercises therapy programme, which included stretches and exercises specifically chosen to facilitate goal achievement and engagement in activities. RESULTS: For cycle 1, most children had active function goals set as their primary goal (69.7% vs 19.2% passive function goals). GAS T- scores and goal responder rates at week 16 indicated that most types of primary goal were achieved at least as expected during cycle 1 (all groups). Primary goal GAS T-scores were generally maintained for the first 3 abobotulinumtoxinA treatment cycles. CONCLUSION: Most children with upper limb spasticity treated with repeat cycles of abobotulinumtoxinA supported by an individualized home exercises therapy programme achieved their functional goals.
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Toxinas Botulínicas Tipo A , Paralisia Cerebral , Fármacos Neuromusculares , Criança , Humanos , Fármacos Neuromusculares/uso terapêutico , Paralisia Cerebral/tratamento farmacológico , Resultado do Tratamento , Toxinas Botulínicas Tipo A/uso terapêutico , Espasticidade Muscular/tratamento farmacológico , Extremidade SuperiorRESUMO
BACKGROUND: Cerebral palsy (CP) is the most common childhood motor disability. The emergence of genetic CP etiologies, variable inclusion of hypotonic CP in international registries, and involvement of different medical disciplines in CP diagnosis can promote diagnostic variability. This variability could adversely affect patients' understanding of their symptoms and access to care. Therefore, we sought to determine the presence and extent of practice variability in CP diagnosis. METHODS: We surveyed physicians in the United States and Canada interested in CP on the basis of membership in the American Academy of Cerebral Palsy and Developmental Medicine or the Child Neurology Society Neonatal Neurology, Movement Disorders, or Neurodevelopmental Disabilities Special Interest Groups. The survey included the 2007 consensus definition of CP and 4 hypothetical case scenarios. RESULTS: Of 695 contacted physicians, 330 (47%) completed the survey. Two scenarios yielded consensus: (1) nonprogressive spastic diplegia after premature birth with periventricular leukomalacia on brain MRI (96% would diagnose CP) and (2) progressive spastic diplegia (92% would not diagnose CP). Scenarios featuring genetic etiologies or hypotonia as the cause of nonprogressive motor disability yielded variability: only 46% to 67% of practitioners would diagnose CP in these settings. CONCLUSIONS: There is practice variability in whether a child with a nonprogressive motor disability due to a genetic etiology or generalized hypotonia will be diagnosed with CP. This variability occurred despite anchoring questions with the 2007 consensus definition of CP. On the basis of these results, we have suggested ways to reduce diagnostic variability, including clarification of the consensus definition.
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Paralisia Cerebral/diagnóstico , Paralisia Cerebral/epidemiologia , Médicos/normas , Inquéritos e Questionários/normas , Adolescente , Canadá/epidemiologia , Criança , Pré-Escolar , Estudos Transversais , Feminino , Humanos , Masculino , Estados Unidos/epidemiologia , Adulto JovemRESUMO
Background: Guidelines recommend botulinum toxin-A in pediatric upper limb spasticity as part of routine practice. Appropriate dosing is a prerequisite for treatment success and it is important that injectors have an understanding on how to tailor dosing within a safe and effective range. We report upper limb dosing data from a phase 3 study of abobotulinumtoxinA injections in children with cerebral palsy. Methods: This was a double-blind, repeat-treatment study (NCT02106351). In Cycle 1, children were randomized to abobotulinumtoxinA at 2 U/kg control dose or clinically relevant 8 U/kg or 16 U/kg doses. Doses were divided between the primary target muscle group (PTMG, wrist or elbow flexors) and additional muscles tailored to clinical presentation. During Cycles 2-4, children received doses of 8 U/kg or 16 U/kg and investigators could change the PTMG and other muscles to be injected. Injection of muscles in the other upper limb and lower limbs was also permitted in cycles 2-4, with the total body dose not to exceed 30 U/kg or 1,000 U (whichever was lower) in the case of upper and lower limb treatment. Results: 212 children were randomized, of which 210 received ≥1 abobotulinumtoxinA injection. Per protocol, the elbow and wrist flexors were the most commonly injected upper limb muscles. Across all 4 cycles, the brachialis was injected in 89.5% of children (dose range 0.8-6 U/kg), the brachioradialis in 83.8% (0.4-3 U/kg), the flexor carpi ulnaris in 82.4% (0.5-3 U/kg) and the flexor carpi radialis in 79.5% (0.5-4 U/kg). Other frequently injected upper limb muscles were the pronator teres(70.0%, 0.3-3 U/kg). adductor pollicis (54.3%, 0.3-1 U/kg), pronator quadratus (44.8%, 0.1-2 U/kg), flexor digitorum superficialis (39.0%, 0.5-4 U/kg), flexor digitorum profundus (28.6%, 0.5-2 U), flexor pollicis brevis/opponens pollicis (27.6%, 0.3-1 U/kg) and biceps (27.1%, 0.5-6 U/kg). AbobotulinumtoxinA was well-tolerated at these doses; muscular weakness was reported in 4.3% of children in the 8 U/kg group and 5.7% in the 16 U/kg group. Conclusions: These data provide information on the pattern of injected muscles and dose ranges used in this study, which were well-tolerated. Per protocol, most children received injections into the elbow and wrist flexors. However, there was a wide variety of other upper limb muscles injected as physicians tailored injection patterns to clinical need.
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OBJECTIVE: To measure the Tardieu Scale's reliability in children with cerebral palsy (CP) when used by raters with and without experience in using the scale, before and after training. DESIGN: Single-center, intrarater and interrater reliability study. SETTING: Institutional ambulatory care. PARTICIPANTS: Referred children with CP in the pretraining phase (n=5), during training (n=3), and in the posttraining phase (n=15). INTERVENTIONS: The Tardieu Scale involves performing passive muscle stretch at 2 velocities, slow and fast. The rater derives 2 parameters; the Spasticity Angle X is the difference between the angles of arrest at slow speed and of catch-and-release or clonus at fast speed; the Spasticity Grade Y is an ordinal variable that grades the intensity (gain) of the muscle reaction to fast stretch. In phase 1, experienced raters without formalized training in the scale graded elbow, knee, and ankle plantar flexors bilaterally, without and with a goniometer. In phase 2, after training, the experienced and nonexperienced raters graded the same muscles unilaterally. MAIN OUTCOME MEASURES: Intrarater and interrater reliability of the Tardieu Scale. RESULTS: After training, nonexperienced raters had mean +/- SD intrarater and interrater agreement rates across all joints and parameters of 80%+/-14% and 74%+/-16%, respectively. For experienced raters, intrarater and interrater agreement rates before training were 77%+/-13% and 66%+/-15%, respectively, versus 90%+/-8% and 81%+/-13%, respectively, after training (P<.001 for both). Specific angle measurements at the knee were less reliable for the angles of catch measured at fast speed. Across all joints, agreement rates were similar using visual or goniometric measurements. CONCLUSIONS: Both parameters of the Tardieu Scale have excellent intrarater and interrater reliability when assessed at the elbow and ankle joints of children with CP, with no difference noted between visual and goniometric measurements. Angle measurements were less reliable at the knee joints. Training was associated with a highly significant improvement in reliability.
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Paralisia Cerebral/complicações , Paralisia Cerebral/reabilitação , Técnicas de Exercício e de Movimento/instrumentação , Espasticidade Muscular/classificação , Adolescente , Artrometria Articular , Criança , Técnicas de Exercício e de Movimento/educação , Feminino , Humanos , Joelho/fisiopatologia , Masculino , Espasticidade Muscular/etiologia , Espasticidade Muscular/fisiopatologia , Músculo Esquelético/fisiopatologia , Postura , Amplitude de Movimento Articular , Reflexo de Estiramento , Reprodutibilidade dos Testes , EnsinoRESUMO
Pediatric movement disorders (PMDs) consist of a heterogeneous group of signs and symptoms caused by numerous neurological diseases. Different neurological disorders in children also share overlapping movement disorders making a diagnosis of the underlying cause of the movement disorder challenging. The similarity of the symptoms across multiple disease types suggests that there may be a final common motor pathway causing the overlapping movement disorders. There are numerous disorders in children associated with disturbances in tone and involuntary movements. This chapter will focus primarily on those disorders that involve abnormalities of tone and other important considerations of pediatric movement disorders. This chapter will address rating scales and goals for treatment and will include a review of symptomatic treatment and, where possible, the treatment of the underlying disease processes. The chapter will review representative disorders, including an inborn error of metabolism, an autoimmune disorder, and a group of neurodegenerative disorders. These examples demonstrate how the disorder's underlying pathophysiology results in a specific approach to the underlying disease and the associated conditions of tone and involuntary movements. Finally, the multiple treatment options for cerebral palsy and considerations of cerebral palsy mimics will be discussed.
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Transtornos dos Movimentos/diagnóstico , Transtornos dos Movimentos/terapia , Tono Muscular/fisiologia , Modalidades de Fisioterapia , Doenças Autoimunes/diagnóstico , Doenças Autoimunes/genética , Doenças Autoimunes/terapia , Paralisia Cerebral/diagnóstico , Paralisia Cerebral/genética , Paralisia Cerebral/terapia , Criança , Discinesias/diagnóstico , Discinesias/genética , Discinesias/terapia , Humanos , Levodopa/farmacologia , Levodopa/uso terapêutico , Erros Inatos do Metabolismo/diagnóstico , Erros Inatos do Metabolismo/genética , Erros Inatos do Metabolismo/terapia , Transtornos dos Movimentos/genética , Tono Muscular/efeitos dos fármacos , Doenças Neurodegenerativas/diagnóstico , Doenças Neurodegenerativas/genética , Doenças Neurodegenerativas/terapia , Resultado do TratamentoRESUMO
In September 2017, the Child Neurology Society (CNS) convened a special task force to review the practice of child neurology in the United States. This was deemed a necessity by our membership, as our colleagues expressed discouragement and burnout by the increase in workload without additional resources; reliance on work relative value units (wRVUs) as the sole basis of compensation; a push by administrators for providers to see more patients with less allotted time; and lack of administrative, educational, and research support. The CNS Task Force designed and distributed a survey to multiple academic divisions of various sizes, as well as to private practices. Our findings were strikingly similar across different practices, demonstrating high workloads, lack of resources, poor electronic medical record support, and high provider symptoms of fatigue and burnout. From the results, the CNS Task Force has concluded that wRVUs cannot be the sole basis of compensation for child neurology. We have also made several specific recommendations for alleviating the current situation, including innovative ways to fund child neurology as well as ways to enhance job satisfaction.
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Neurologia/economia , Pediatria/economia , Escalas de Valor Relativo , HumanosRESUMO
OBJECTIVE: To contextualize the role of child neurologists and neurodevelopmentalists (CNs/NDDs) in cerebral palsy (CP) care, we review the changing landscape of CP diagnosis and survey stakeholder CNs/NDDs regarding their roles in CP care. METHODS: The optimal roles of the multiple specialties involved in CP care are currently unclear, particularly regarding CP diagnosis. We developed recommendations regarding the role of CNs/NDDs noting (1) increasing complexity of CP diagnosis given a growing number of genetic etiologies and treatable motor disorders that can be misdiagnosed as CP and (2) the views of a group of physician stakeholders (CNs/NDDs from the Child Neurology Society Cerebral Palsy Special Interest Group). RESULTS: CNs/NDDs felt that they were optimally suited to diagnose CP. Many (76%) felt that CNs/NDDs should always be involved in CP diagnosis. However, 42% said that their patients with CP were typically not diagnosed by CNs/NDDs, and 18% did not receive referrals to establish the diagnosis of CP at all. CNs/NDDs identified areas of their expertise critical for CP diagnosis including knowledge of the neurologic examination across development and early identification of features atypical for CP. This contrasts with their views on CP management, where CNs/NDDs felt that they could contribute to the medical team, but were necessary primarily when neurologic coexisting conditions were present. DISCUSSION: Given its increasing complexity, we recommend early referral for CP diagnosis to a CN/NDD or specialist with comparable expertise. This contrasts with current consensus guidelines, which either do not address or do not recommend specific specialist referral for CP diagnosis.
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Paralisia Cerebral/diagnóstico , Paralisia Cerebral/fisiopatologia , Transtornos do Neurodesenvolvimento/diagnóstico , Inquéritos e Questionários , Humanos , Transtornos do Neurodesenvolvimento/fisiopatologia , Exame Neurológico/métodos , Medição de Risco , Papel (figurativo)RESUMO
Purpose: This secondary analysis of a randomized, double-blind study plus open-label extension (NCT01249417/NCT01251380) evaluated the efficacy of abobotulinumtoxinA versus placebo in improving gait pattern in children with dynamic equinus due to cerebral palsy (CP) as assessed by the observational gait scale (OGS). Methods: Ambulatory children with CP (N = 241, aged 2-17) and dynamic equinus were randomized to treatment with abobotulinumtoxinA (10 or 15U/kg/leg) or placebo injected into the gastrocsoleus. All children received abobotulinumtoxinA in the open-label phase. Results: In the double-blind phase, abobotulinumtoxinA significantly improved OGS total scores versus placebo at Week 4 (treatment effect vs. placebo: 10U/kg/leg: 1.5 [0.7, 2.3], p = .0003; 15U/kg/leg: 1.1 [0.3, 1.9], p = .01). In the open-label phase, treatment with abobotulinumtoxinA continued to improve the OGS score at the same magnitude as seen in the double-blind study. Conclusion: Repeat treatment with abobotulinumtoxinA improved gait in children with dynamic equinus.
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Toxinas Botulínicas Tipo A/uso terapêutico , Paralisia Cerebral/tratamento farmacológico , Marcha , Fármacos Neuromusculares/uso terapêutico , Adolescente , Toxinas Botulínicas Tipo A/administração & dosagem , Toxinas Botulínicas Tipo A/efeitos adversos , Paralisia Cerebral/reabilitação , Criança , Pré-Escolar , Método Duplo-Cego , Esquema de Medicação , Feminino , Humanos , Injeções Intramusculares , Masculino , Fármacos Neuromusculares/administração & dosagem , Fármacos Neuromusculares/efeitos adversosRESUMO
PURPOSE OF REVIEW: With advances in medical care, the number of youths surviving with medically complex conditions has been steadily increasing. Inadequate transition planning and execution can lead to gaps in care, unexpected emergency department visits, and an increase in health care costs and patient/caregiver anxiety. Many barriers that prevent adequate transition have been identified, including insufficient time or staff to provide transition services, inadequate reimbursement, resistance from patients and caregivers, and a dearth of accepting adult providers. RECENT FINDINGS: Transition is distinct from transfer of care. Transition is a planned multistage process, while transfer refers to a point in time where responsibility of care shifts from one provider to another. Key differences exist between the pediatric and adult models of care. A successful transition should empower the patient to understand and take responsibility in managing his or her condition; foster independent functioning to the extent that is possible; integrate educational, legal, and community resources in the care plan; and identify appropriate adult health care providers at the time of transfer. Different models have been proposed to streamline the transition process, with improvement in patients' knowledge of their condition, self-efficacy, and confidence. SUMMARY: Neurologists have a key role in supporting their patients in the transition to adulthood. This article reviews basic tenets and provides tools to assist in navigating the complex transition process. These tenets are intended to improve quality of care and decrease clinician burden and remain an active area of research.
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Doenças do Sistema Nervoso , Transição para Assistência do Adulto , Adolescente , Criança , Humanos , Masculino , Adulto JovemRESUMO
This was a prospective, repeat-treatment, open-label study (NCT01251380) of abobotulinumtoxinA for the management of lower limb spasticity in children who had completed a double-blind study. Children (2-17 years) received injections into the gastrocnemius-soleus complex, and other distal and proximal muscles as required (maximum total dose per injection cycle: 30 U/kg or 1000U). A total of 216 of the 241 double-blind patients entered the extension study and 207 received ≥1 open label injection into the gastrocnemius-soleus; 17-24% of patients also had injections into the hamstrings. The most frequent adverse events were related to common childhood infections and the most frequent treatment-related adverse event was injection site pain (n = 10). There was no evidence of a cumulative effect on adverse events. Sustained significant clinical improvements in muscle tone (Modified Ashworth Scale), spasticity (Tardieu Scale), overall clinical benefit (Physicians Global Assessment), and goal attainment (Goal Attainment Scale) were also observed across treatment cycles.
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Toxinas Botulínicas Tipo A/uso terapêutico , Paralisia Cerebral/tratamento farmacológico , Espasticidade Muscular/tratamento farmacológico , Fármacos Neuromusculares/uso terapêutico , Adolescente , Toxinas Botulínicas Tipo A/efeitos adversos , Paralisia Cerebral/fisiopatologia , Criança , Pré-Escolar , Feminino , Humanos , Extremidade Inferior/fisiopatologia , Masculino , Espasticidade Muscular/fisiopatologia , Músculo Esquelético/efeitos dos fármacos , Músculo Esquelético/fisiopatologia , Fármacos Neuromusculares/efeitos adversos , Paresia/tratamento farmacológico , Paresia/fisiopatologia , Índice de Gravidade de Doença , Resultado do TratamentoRESUMO
This secondary analysis of a large (n = 241), randomized, double-blind study evaluated the efficacy of 2 doses of abobotulinumtoxinA + standard of care (SOC) versus placebo + SOC in enabling children with dynamic equinus due to cerebral palsy to achieve their functional goals using Goal Attainment Scaling. Most parents/caregivers selected goals targeting aspects of gait improvement as most relevant. Mean (95% confidence interval) Goal Attainment Scaling T scores at week 4 were higher for both abobotulinumtoxinA groups versus placebo (treatment difference vs placebo: 10 U/kg/leg: 5.32 [2.31, 8.32], P = .0006, and 15 U/kg/leg 4.65 [1.59, 7.71], P = .0031). Superiority of both abobotulinumtoxinA doses versus placebo was maintained at week 12. Best goal attainment T scores were higher in the abobotulinumtoxinA groups versus placebo for the common goals of improved walking pattern, decreased falling, decreased tripping, and improved endurance. These findings demonstrate that single injections of abobotulinumtoxinA (10 and 15 U/kg/leg) significantly improved the ability of pediatric cerebral palsy patients to achieve their functional goals.
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Toxinas Botulínicas Tipo A/uso terapêutico , Paralisia Cerebral/tratamento farmacológico , Pé Equino/tratamento farmacológico , Marcha/efeitos dos fármacos , Fármacos Neuromusculares/uso terapêutico , Adolescente , Toxinas Botulínicas Tipo A/farmacologia , Paralisia Cerebral/complicações , Criança , Pré-Escolar , Método Duplo-Cego , Pé Equino/etiologia , Feminino , Objetivos , Humanos , Masculino , Fármacos Neuromusculares/farmacologia , Resultado do TratamentoRESUMO
Congenital insensitivity to pain and anhidrosis (CIPA) is one of the hereditary autonomic and sensory neuropathies. Typically presenting in infancy, it manifests as hyperpyrexia from defects in sweating (autonomic) and self-mutilating injuries from pain insensitivity (sensory). CIPA being rare in North America, diagnosis is often missed due to variable presentation. Subsequent management of its complications is therefore delayed. We report an unusual presentation in a 2-year-old girl with preexisting diagnosis of CIPA who was evaluated for bilateral upper extremity paresis of insidious onset. MRI revealed a mass compressing her cervical spine as the cause, and work up suggested immune dysfunction as possible etiology. To our knowledge, this complication has not been reported before in association with the disease. We introduce the disease by explaining the molecular pathology behind its presenting features. The neurological findings, documented in association with CIPA, are summarized and serve as a reference for the various presentations of this rare disorder. Since this disease is known to affect the immune system, immune defects in CIPA are discussed with recommendations for surveillance of patient's immune status.