Childhood severe asthma: relationship among asthma control scores, FeNO, spirometry and impulse oscillometry.

INTRODUCTION: The evaluation of the asthmatic patient is usually based on clinical and functional parameters that do not necessarily evidence the degree of airway inflammation. The aim of this study was to analyze whether clinical scores (CS) correlate with spirometry (S), impulse oscillometry (IO) and FeNO, in severe asthmatic children. MATERIAL AND METHODS: A multicentric, prospective, cross-sectional study was conducted over a 12-month period. All SA patients (6-18 years old) followed-up in the Pulmonology Department were recruited. CS, FeNO measurements, IO and S were consecutively performed on the same day. Asthma control was ascertained using ACT and GINAq. A cut-off value of ≥ 25 parts per billion (ppb) was used to define airway inflammation. RESULTS: Eighty-one patients were included. ACT: 75% (n 61) were controlled; GINAq: 44.5% (n 36) were controlled; 39.5% (n 32) were partly controlled, and 16% (n 13) were uncontrolled. FeNO had a median value of 24 ppb (IQR 14-41); FeNO ≥ 25 ppb was observed in 49% of patients (n 39). ROC AUC for FeNO vs. ACT was 0.71 (95%CI 0.57-0.86), PPV 0.47, NPV 0.87, SE 0.61, SP 0.80; FeNO vs. GINAq was ROC AUC 0.69 (95%CI 0.54-0.85), PPV 0.34, NPV 0.91, SE 0.62, SP 0.77; Youden cut-off FeNO > 39 ppb for both CS. CONCLUSION: In severe asthmatic children, current symptoms control as evidenced by ACT and GINA correlates with low FeNO values. Clinical scores showed good correlation with airway inflammation.

Health checkups during the pandemic and clinical impact of COVID-19 in children with neuromuscular disease. / Controles de salud durante la pandemia e impacto clínico del COVID-19 en niños con enfermedad neuromuscular.

Introduction. Children with neuromuscular disease (NMD) require chronic health care (CHC) and may develop severe COVID-19. Objectives. To describe CHC for children with NMD during the pandemic and the course of COVID-19 in this group. Population and methods. Prospective, single-center cohort. Patients aged 2 to 18 years with ≥ 1 year of follow-up prior to the pandemic were included. Demographic variables in relation to CHC and COVID-19 were collected from medical records and via telephone surveys. Results. A total of 226 patients with a median age of 11.3 years were included; 71% were males. They had muscular dystrophy (55.7%) and spinal muscular atrophy (23%). When comparing the first year of the pandemic with the previous year, 30% did not have a health checkup and 25% did not receive kinesiotherapy. Others did, but with a lower frequency. A total of 52 COVID-19 cases were reported; 82% were symptomatic: 88.4% were mild/moderate and 11.6%, severe. No patient died. Conclusions. The pandemic had a negative impact on CHC, and COVID-19 cases were mostly mild.

Introducción. Los niños con enfermedad neuromuscular (ENM) requieren cuidados crónicos de salud (CCS) y podrían presentar COVID-19 grave. Objetivos. Describir CCS para niños con ENM durante la pandemia y evolución del COVID-19 en este grupo. Población y métodos. Cohorte prospectiva unicéntrica. Se incluyeron pacientes de 2-18 años, con ≥ 1 año de seguimiento previo a la pandemia. Se recolectaron variables demográficas, relativas a los CCS y al COVID-19 mediante historias clínicas y encuestas telefónicas. Resultados. Se incluyeron 226 pacientes; el 71 % varones, mediana de edad 11,3 años. Presentaban distrofias musculares (55,7 %) y atrofia muscular espinal (23 %). Comparando el primer año de pandemia con el previo, el 30 % no realizó controles médicos y el 25 % no realizó kinesioterapia. Otros disminuyeron la frecuencia. Hubo 52 casos de COVID-19. Fueron sintomáticos el 82 %: el 88,4 % leves/moderados y el 11,6 % graves. No hubo fallecidos. Conclusiones. La pandemia impactó negativamente en los CCS y los casos de COVID-19 fueron mayormente leves.

Feasibility of a pediatric long-term Home Ventilation Program in Argentina: 11 years' experience.

BACKGROUND: Pediatric home ventilation (HV) has increased worldwide. A Home Ventilation Program (HVP) was started in the Pulmonary Department of the "Hospital de Pediatría Prof. Dr. J. P. Garrahan," Argentina, in 2007. This is the largest Argentine national pediatric tertiary care referral center. Limited studies on pediatric HV from Latin American countries have been published. OBJECTIVE: This study describes and analyzes the cohort of children admitted to the HVP during an 11 years period. METHODS: Longitudinal study. POPULATION: all patients (pts) admitted to the HVP between 2007 and 2018. We analyzed demographic and clinical variables, sleep study results, ventilation setting, and start manner collected in a prospective data base. RESULTS: A total of 244 pts were admitted. Median age at ventilation start was 9.41 (3.47-14.08) years, 84% of pts had health insurance. The most frequent underlying diseases were neuromuscular disease (43%) and genetic syndromes (23%). Home-hospital distance was 100-500 km in 16% of cases and greater than 500 km in 34%. Seventy percent of pts had sleep studies before ventilation initiation. Ventilation was started in our general pediatric ward in 83.6%. Noninvasive ventilation was used in 86.1%. The actual number of pts still on follow up is 133 of 244 (54.5%), 16.8% dropped out, 16.4% were transitioned to adult care, 5.32% resolved their sleep-disordered breathing, and 5.32% died. CONCLUSIONS: The HVP admitted pts from all the country. Ventilation was started on the basis of clinical and objective sleep measures. This long-term experience underlines the feasibility of a HVP in an emergent country.

Instrument to assess research projects that apply for a fellowship by the Argentine Society of Pediatrics: Validation using the Delphi method. / Instrumento para evaluar proyectos de investigación que concursan a beca en la Sociedad Argentina de Pediatría: Validación mediante el método Delphi.

INTRODUCTION: The objective of this study was to validate the format and contents of an instrument to assess research projects that apply for a fellowship by the Sociedad Argentina de Pediatría using an expert consultation technique, such as the Delphi method. MATERIAL AND METHODS: A coordinating group selected a panel of research experts who were members of the Sociedad Argentina de Pediatría,designed, and analyzed each of the rounds of consultations. Semistructured questionnaires were sent by personalized e-mail. Agreement among experts > 80 % was established as the criterion for consensus. At each round of consultation, non-consensual aspects were reformulated and new aspects suggested by experts were included. A measure of stability to conclude the consultation was determined when more than 70 % of experts sustained their opinion in successive rounds. RESULTS: Thirteen research experts participated in the process. After 3 rounds, the consultation process was concluded. The consensual instrument contains 47 items. In relation to the total score, 10 % corresponds to the general presentation; 40 %, to methodological quality; 20 %, to relevance and applicability; 20 %, to feasibility; and 10 %, to the fellow's and director's background. CONCLUSIONS: The format and contents of the instrument to assess research projects that apply for a fellowship by the Sociedad Argentina de Pediatría were validated based on expert consensus and objective assessment criteria were established.

El objetivo del presente trabajo fue validar la forma y el contenido, mediante un método de consulta a expertos, como es el método Delphi, del instrumento para evaluar proyectos de investigación que concursan a beca en la Sociedad Argentina de Pediatría. Material y métodos. Un grupo coordinador seleccionó el panel de expertos en investigación pertenecientes a la Sociedad, diseñó y analizó cada una de las rondas de consulta. Los cuestionarios semiestructurados fueron enviados por correo electrónico en forma personalizada. Se estableció como criterio de consenso un acuerdo entre los expertos > 80 %. En cada ronda, se reformularon los aspectos no consensuados y se agregaron nuevos aspectos sugeridos por los expertos. Se consideró como medida de estabilidad para concluir la consulta cuando más del 70 % de los expertos no modificaron su opinión en rondas sucesivas. Resultados. Participaron del proceso 13 expertos en investigación. Luego de 3 rondas, finalizó el método de consulta. El instrumento consensuado contiene 47 ítems. El 10 % de la puntuación total corresponde a presentación general; el 40 %, a calidad metodológica; el 20 %, a relevancia-aplicabilidad; el 20 %, a factibilidad; y el 10 %, a antecedentes del becario y del director. Conclusiones. Se validó la forma y el contenido, mediante consenso de expertos, del instrumento de evaluación de proyectos de investigación que concursan a becas de investigación en la Sociedad y se lograron criterios objetivos de evaluación.

Controles de salud durante la pandemia e impacto clínico del COVID-19 en niños con enfermedad neuromuscular / Health checkups during the pandemic and clinical impact of COVID-19 in children with neuromuscular disease

Introducción. Los niños con enfermedad neuromuscular (ENM) requieren cuidados crónicos de salud (CCS) y podrían presentar COVID-19 grave. Objetivos. Describir CCS para niños con ENM durante la pandemia y evolución del COVID-19 en este grupo. Población y métodos. Cohorte prospectiva unicéntrica. Se incluyeron pacientes de 2-18 años, con ≥ 1 año de seguimiento previo a la pandemia. Se recolectaron variables demográficas, relativas a los CCS y al COVID-19 mediante historias clínicas y encuestas telefónicas. Resultados. Se incluyeron 226 pacientes; el 71 % varones, mediana de edad 11,3 años. Presentaban distrofias musculares (55,7 %) y atrofia muscular espinal (23 %). Comparando el primer año de pandemia con el previo, el 30 % no realizó controles médicos y el 25 % no realizó kinesioterapia. Otros disminuyeron la frecuencia. Hubo 52 casos de COVID-19. Fueron sintomáticos el 82 %: el 88,4 % leves/moderados y el 11,6 % graves. No hubo fallecidos. Conclusiones. La pandemia impactó negativamente en los CCS y los casos de COVID-19 fueron mayormente leves.

Introduction. Children with neuromuscular disease (NMD) require chronic health care (CHC) and may develop severe COVID-19. Objectives. To describe CHC for children with NMD during the pandemic and the course of COVID-19 in this group. Population and methods. Prospective, single-center cohort. Patients aged 2 to 18 years with ≥ 1 year of follow-up prior to the pandemic were included. Demographic variables in relation to CHC and COVID-19 were collected from medical records and via telephone surveys. Results. A total of 226 patients with a median age of 11.3 years were included; 71% were males. They had muscular dystrophy (55.7%) and spinal muscular atrophy (23%). When comparing the first year of the pandemic with the previous year, 30% did not have a health checkup and 25% did not receive kinesiotherapy. Others did, but with a lower frequency. A total of 52 COVID-19 cases were reported; 82% were symptomatic: 88.4% were mild/moderate and 11.6%, severe. No patient died. Conclusions. The pandemic had a negative impact on CHC, and COVID-19 cases were mostly mild.

[Transition experience of patients with neuromuscular disease]. / Experiencia en el proceso de transición de pacientes con enfermedad neuromuscular.

Neuromuscular diseases are mostly genetic disorders, with chronic and progressive course. Affected people are at high risk of developing physical and emotional disabilities. In the last decades, the advance in technology and science has increased chronic pediatric patients survival rate, thus requiring an ongoing assistance in adult hospitals, making the transition a necessity and a challenge. This article reports the clinical practice designed between Hospital Garrahan and Hospital Ramos Mejía for the transition of 27 adolescents during 2015, setting achievements, findings and challenges resulting from this experience.

Las enfermedades neuromusculares tienen, generalmente, origen genético, con evolución crónica y progresiva y con posibilidades de generar limitaciones físico-emocionales. En las últimas décadas, los avances en salud aumentaron la sobrevida de niños y adolescentes con estas enfermedades, y convirtieron la transición al sistema de salud de adultos en una necesidad y un desafío. Este artículo relata la práctica asistencial diseñada e implementada por el equipo interdisciplinario que atiende a pacientes con enfermedad neuromuscular del Hospital Garrahan junto con el Hospital Ramos Mejía para la transición de 27 adolescentes durante 2015. Además, plantea logros, aprendizajes y desafíos surgidos de esta experiencia

Instrumento para evaluar proyectos de investigación que concursan a beca en la Sociedad Argentina de Pediatría: Validación mediante el método Delphi / Instrument to assess research projects that apply for a fellowship by the Argentine Society of Pediatrics: Validation using the Delphi method

El objetivo del presente trabajo fue validar la forma y el contenido, mediante un método de consulta a expertos, como es el método Delphi, del instrumento para evaluar proyectos de investigación que concursan a beca en la Sociedad Argentina de Pediatría. Material y métodos. Un grupo coordinador seleccionó el panel de expertos en investigación pertenecientes a la Sociedad, diseñó y analizó cada una de las rondas de consulta. Los cuestionarios semiestructurados fueron enviados por correo electrónico en forma personalizada. Se estableció como criterio de consenso un acuerdo entre los expertos > 80 %. En cada ronda, se reformularon los aspectos no consensuados y se agregaron nuevos aspectos sugeridos por los expertos. Se consideró como medida de estabilidad para concluir la consulta cuando más del 70 % de los expertos no modificaron su opinión en rondas sucesivas. Resultados. Participaron del proceso 13 expertos en investigación. Luego de 3 rondas, finalizó el método de consulta. El instrumento consensuado contiene 47 ítems. El 10 % de la puntuación total corresponde a presentación general; el 40 %, a calidad metodológica; el 20 %, a relevancia-aplicabilidad; el 20 %, a factibilidad; y el 10 %, a antecedentes del becario y del director. Conclusiones. Se validó la forma y el contenido, mediante consenso de expertos, del instrumento de evaluación de proyectos de investigación que concursan a becas de investigación en la Sociedad y se lograron criterios objetivos de evaluación.

The objective of this study was to validate the format and contents of an instrument to assess research projects that apply for a fellowship by the Sociedad Argentina de Pediatría using an expert consultation technique, such as the Delphi method. Material and methods. A coordinating group selected a panel of research experts who were members of the Sociedad Argentina de Pediatría,designed, and analyzed each of the rounds of consultations. Semistructured questionnaires were sent by personalized e-mail. Agreement among experts > 80 % was established as the criterion for consensus. At each round of consultation, non-consensual aspects were reformulated and new aspects suggested by experts were included. A measure of stability to conclude the consultation was determined when more than 70 % of experts sustained their opinion in successive rounds. Results. Thirteen research experts participated in the process. After 3 rounds, the consultation process was concluded. The consensual instrument contains 47 items. In relation to the total score, 10 % corresponds to the general presentation; 40 %, to methodological quality; 20 %, to relevance and applicability; 20 %, to feasibility; and 10 %, to the fellow's and director's background. Conclusions. The format and contents of the instrument to assess research projects that apply for a fellowship by the Sociedad Argentina de Pediatría were validated based on expert consensus and objective assessment criteria were established.

Experiencia en el proceso de transición de pacientes con enfermedad neuromuscular / Transition experience of patients with neuromuscular disease

Las enfermedades neuromusculares tienen, generalmente, origen genético, con evolución crónica y progresiva y con posibilidades de generar limitaciones físico-emocionales. En las últimas décadas, los avances en salud aumentaron la sobrevida de niños y adolescentes con estas enfermedades, y convirtieron la transición al sistema de salud de adultos en una necesidad y un desafío. Este artículo relata la práctica asistencial diseñada e implementada por el equipo interdisciplinario que atiende a pacientes con enfermedad neuromuscular del Hospital Garrahan junto con el Hospital Ramos Mejía para la transición de 27 adolescentes durante 2015. Además, plantea logros, aprendizajes y desafíos surgidos de esta experiencia.

Neuromuscular diseases are mostly genetic disorders, with chronic and progressive course. Affected people are at high risk of developing physical and emotional disabilities. In the last decades, the advance in technology and science has increased chronic pediatric patients survival rate, thus requiring an ongoing assistance in adult hospitals, making the transition a necessity and a challenge. This article reports the clinical practice designed between Hospital Garrahan and Hospital Ramos Mejía for the transition of 27 adolescents during 2015, setting achievements, findings and challenges resulting from this experience.