ABSTRACT
IMPORTANCE: Standardization of outcome measurement using a patient-centered approach in pediatric facial palsy may help aid the advancement of clinical care in this population. OBJECTIVE: To develop a standardized outcome measurement set for pediatric patients with facial palsy through an international multidisciplinary group of health care professionals, researchers, and patients and patient representatives. DESIGN, SETTING, AND PARTICIPANTS: A working group of health care experts and patient representatives (n = 21), along with external reviewers, participated in the study. Seven teleconferences were conducted over a 9-month period between December 3, 2016, and September 23, 2017, under the guidance of the International Consortium for Health Outcomes Measurement, each followed with a 2-round Delphi process to develop consensus. This process defined the scope, outcome domains, measurement tools, time points for measurements, and case-mix variables deemed essential to a standardized outcome measurement set. Each teleconference was informed by a comprehensive review of literature and through communication with patient advisory groups. Literature review of PubMed was conducted for research published between January 1, 1981, and November 30, 2016. MAIN OUTCOMES AND MEASURES: The study aim was to develop the outcomes and measures relevant to children with facial palsy as opposed to studying the effect of a particular intervention. RESULTS: The 21 members of the working group included pediatric facial palsy experts from 9 countries. The literature review identified 1628 papers, of which 395 (24.3%) were screened and 83 (5.1%) were included for qualitative evaluation. A standard set of outcome measurements was designed by the working group to allow the recording of outcomes after all forms of surgical and nonsurgical facial palsy treatments among pediatric patients of all ages. Unilateral or bilateral, congenital or acquired, permanent or temporary, and single-territory or multiterritory facial palsy can be evaluated using this standard set. Functional, appearance, psychosocial, and administrative outcomes were selected for inclusion. Clinimetric and psychometric outcome measurement tools (clinician-, patient-, and patient proxy-reported) and time points for measuring patient outcomes were established. Eighty-six independent reviews of the standard set were completed, and 34 (85%) of the 40 patients and patient representatives and 44 (96%) of the 46 health care professionals who participated in the reviews agreed that the standard set would capture the outcomes that matter most to children with facial palsy. CONCLUSIONS AND RELEVANCE: This international collaborative study produced a free standardized set of outcome measures for evaluating the quality of care provided to pediatric patients with facial palsy, allowing benchmarking of clinicians, comparison of treatment pathways, and introduction of value-based reimbursement strategies in the field of pediatric facial palsy. LEVEL OF EVIDENCE: NA.
Subject(s)
Facial Paralysis/surgery , Patient Reported Outcome Measures , Quality of Health Care/standards , Child , Consensus , Delphi Technique , Humans , International Cooperation , PsychometricsABSTRACT
Cholestenoic acids are formed as intermediates in metabolism of cholesterol to bile acids, and the biosynthetic enzymes that generate cholestenoic acids are expressed in the mammalian CNS. Here, we evaluated the cholestenoic acid profile of mammalian cerebrospinal fluid (CSF) and determined that specific cholestenoic acids activate the liver X receptors (LXRs), enhance islet-1 expression in zebrafish, and increase the number of oculomotor neurons in the developing mouse in vitro and in vivo. While 3ß,7α-dihydroxycholest-5-en-26-oic acid (3ß,7α-diHCA) promoted motor neuron survival in an LXR-dependent manner, 3ß-hydroxy-7-oxocholest-5-en-26-oic acid (3ßH,7O-CA) promoted maturation of precursors into islet-1+ cells. Unlike 3ß,7α-diHCA and 3ßH,7O-CA, 3ß-hydroxycholest-5-en-26-oic acid (3ß-HCA) caused motor neuron cell loss in mice. Mutations in CYP7B1 or CYP27A1, which encode enzymes involved in cholestenoic acid metabolism, result in different neurological diseases, hereditary spastic paresis type 5 (SPG5) and cerebrotendinous xanthomatosis (CTX), respectively. SPG5 is characterized by spastic paresis, and similar symptoms may occur in CTX. Analysis of CSF and plasma from patients with SPG5 revealed an excess of the toxic LXR ligand, 3ß-HCA, while patients with CTX and SPG5 exhibited low levels of the survival-promoting LXR ligand 3ß,7α-diHCA. Moreover, 3ß,7α-diHCA prevented the loss of motor neurons induced by 3ß-HCA in the developing mouse midbrain in vivo.Our results indicate that specific cholestenoic acids selectively work on motor neurons, via LXR, to regulate the balance between survival and death.
Subject(s)
Cholestenes/cerebrospinal fluid , Motor Neurons/physiology , Orphan Nuclear Receptors/metabolism , Animals , Cell Survival , Cells, Cultured , Cholestenes/blood , Female , Humans , LIM-Homeodomain Proteins/metabolism , Liver X Receptors , Male , Mice, Inbred C57BL , Mice, Knockout , Paraparesis, Spastic/blood , Paraparesis, Spastic/cerebrospinal fluid , Transcription Factors/metabolism , Xanthomatosis, Cerebrotendinous/blood , Xanthomatosis, Cerebrotendinous/cerebrospinal fluid , ZebrafishABSTRACT
We report on 34 patients with abnormal body movements (AMs; 11 females, 23 males; mean age 10 y 1 mo, range 3 y 6 mo-15 y 11 mo). Twenty-three of the 34 patients had an organic movement disorder (OMD), five patients fulfilled the diagnostic criteria of documented psychogenic movement disorder (PMD), and six patients displayed probable or possible PMD. Diagnosis of children with OMD included essential tremor (n=7), Tourette syndrome (n=5), primary dystonia (n=2), chronic motor tics (n=2), viral cerebellar ataxia (n=2), drug-induced ataxia (n=1), thyrotoxicosis related tremor (n=1), autosomal inherited dystonia (n=1), poststreptococcal chorea (n=1), and benign head tremor (n=1). Consistent findings among patients with PMD included disappearance of AMs when the patients thought they were not being observed and satisfactory recovery from the AMs after psychotherapy or suggestion. Reduction of the movements when the patient was distracted and variability of AMs during full relaxation, sleep, and stress were reported among patients with both PMD and OMD.
Subject(s)
Movement Disorders/diagnosis , Psychophysiologic Disorders/diagnosis , Adolescent , Child , Child, Preschool , Family Conflict , Female , Humans , Male , Movement Disorders/therapy , Neurologic Examination , Prospective Studies , Psychophysiologic Disorders/therapy , Psychotherapy , Remission, Spontaneous , Sick Role , Stress, Psychological/complications , SuggestionABSTRACT
Three normal children with headache occurring only with exertion were advised to try "head cooling" (eg, immersion of the head in cold water, cold water poured over the head, application of a cold, wet towel or ice pack) at the onset of headache. The patients were followed up quarterly as outpatients, and the effectiveness of head cooling in terms of the frequency of headaches, intensity (interference with play), duration, and side effects was assessed over 18 months.
Subject(s)
Body Temperature/physiology , Cold Temperature , Exercise/physiology , Headache Disorders, Primary/etiology , Headache Disorders, Primary/therapy , Hypothermia, Induced/methods , Adolescent , Carotid Artery, External/physiopathology , Child , Head/physiopathology , Headache Disorders, Primary/physiopathology , Humans , Male , Sports , Treatment Outcome , Vasoconstriction/physiologyABSTRACT
Se describen las actividades de la consulta de dolor del Hospital de La Misericordia entre agosto de 1988 y junio de 1990. Se discuten los hallazgos clínicos y se hace énfasis en la necesidad de implementar este tipo de atención para los pacientes con dolor en la edad pediátrica.
Subject(s)
Humans , Child , Pain Clinics/classification , Pain Clinics/statistics & numerical data , Pain Clinics/organization & administration , Pain Clinics , Pain Clinics/trends , Pain ClinicsSubject(s)
Humans , Infant, Newborn , Pain/classification , Pain/diagnosis , Pain/drug therapy , Pain/prevention & controlABSTRACT
Se describen las características clínicas, sociodemográficas y familiares, al igual que el tratamiento y su respuesta al mismo, en siete pacientes con dolor abdominal recurrente quienes fueron remitidos al servicio de Psiquiatría de la Consulta Externa del Hospital Pediátrico de la Misericordia, en Santafé de Bogotá, durante el período comprendido entre mayo de 1989 y septiembre de 1991. Se trataron con psicoterapia. Cinco de estos pacientes mejoraron con el tratamiento y se mantenían sin dolor abdominal recurrente dos años después de terminado el mismo. El grupo tiene características similares a las informadas en la literatura. El paciente prototipo es una adolescente inteligente, de buen rendimiento escolar, preocupada por obtener una buena imagen de las personas significativas que la rodean, quien tiende a expresar sus emociones desagradables a través de síntomas somáticos. Un 45 por ciento de las madres tenía depresión, ansiedad o incapacidad de expresar verbalmente sus afectos.
Subject(s)
Humans , Child , Abdominal Pain/classification , Abdominal Pain/diagnosis , Abdominal Pain/etiologyABSTRACT
Se describe un caso fatal de meningoencefalitis amibiana primaria tratado en el Hospital de San José, Santafé de Bogotá. La paciente presentaba un cuadro clínico impreciso sugestivo de una meningitis bacteriana, sin respuesta al tratamiento con antibióticos de amplio espectro. Se describen las dos formas de infección del SNC por amibas de vida libre y el tratamiento correspondiente.
