Evaluación de la efectividad del tratamiento Elexacaftor / Tezacaftor / Ivacaftor (ETI) en pacientes con fibrosis quística avanzada desde un enfoque bayesiano
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2024
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Abstract
Objetivos: Este estudio buscó evaluar la efectividad del tratamiento con ETI, a partir de la recuperación de la función pulmonar (FP), en pacientes con fibrosis quística (FQ) avanzada, específicamente en comparación entre poblaciones homocigotas y heterocigotas para la proteína reguladora de la conductancia transmembrana de la fibrosis quística (CFTR), desde un enfoque bayesiano.
Materiales y Métodos: Se trata de un estudio multicéntrico, ambispectivo y observacional en el que se reclutaron 114 pacientes en seguimiento en 16 unidades nacionales de FQ. Se evaluó la FP medida como el porcentaje de volumen espiratorio forzado en el primer segundo (FEV1%), comparando el valor obtenido cada visita del tratamiento frente a los valores de la visita basal y frente a la visita anterior, además de la diferencia en la mejora, obtenida en cada visita, entre grupos genéticos homocigotos y heterocigotos para la proteína CFTR. Se emplearon enfoques frecuentistas y bayesianos. Se utilizaron diferentes estrategias de conjuntos de datos (total de pacientes, pacientes que acabaron el estudio, pacientes con datos imputados) debido a la alta tasa de deserción obtenida.
Resultados: A partir de la visita realizada a los tres meses tras iniciar el tratamiento se observó un incremento significativo en la proporción de pacientes con una FP leve (FEV1% ≥ 60%) frente a los observados antes de empezar el tratamiento, persistiendo esta mejoría durante los 12 meses de seguimiento. Esta mejora fue particularmente notable en la primera visita. Al comparar la respuesta al tratamiento entre los grupos genéticos, no se encontró una diferencia significativa a la respuesta del tratamiento. Este hallazgo sugiere que ambos grupos genéticos responden igual al tratamiento.
Conclusión: La evaluación de la efectividad del tratamiento con ETI en pacientes con FQ avanzada mostró una mejoría rápida de la FEV1% inicial, que se mantuvo estable en visitas posteriores sin diferencias significativas entre grupos genéticos
Abstract Objectives: This study aimed to evaluate the effectiveness of ETI treatment based on lung function (LF) recovery in patients with advanced cystic fibrosis (CF), specifically comparing homozygous and heterozygous populations for the cystic fibrosis transmembrane conductance regulator (CFTR) protein from a Bayesian perspective. Materials and Methods: This is a multicentre, ambispective, observational study in which 114 patients were recruited from 16 national CF units. Lung function was assessed, measured as the percentage of forced expiratory volume in the first second (FEV1%), comparing the value obtained at each treatment visit against the baseline visit values and the previous visit values. Additionally, the difference in improvement, obtained at each visit, between homozygous and heterozygous genetic groups for the CFTR protein was analysed. Frequentist and Bayesian approaches were employed. Different data set strategies (total patients, patients who completed the study, patients with imputed data) were used due to the high dropout rate. Results: A significant increase in the proportion of patients with mild LF was observed from the three-month visit after starting treatment (FEV1% ≥ 60%) compared to those observed before starting treatment, and this improvement persisted over 12 months of follow-up. This improvement was particularly noticeable at the first visit. When comparing treatment response between the genetic groups, no significant difference to treatment response was found. This finding suggests that both genetic groups respond equally to treatment. Conclusion: Evaluation of the effectiveness of ETI treatment in patients with advanced CF showed a rapid improvement in baseline FEV1%, which remained stable at subsequent visits with no significant differences between genetic groups.
Abstract Objectives: This study aimed to evaluate the effectiveness of ETI treatment based on lung function (LF) recovery in patients with advanced cystic fibrosis (CF), specifically comparing homozygous and heterozygous populations for the cystic fibrosis transmembrane conductance regulator (CFTR) protein from a Bayesian perspective. Materials and Methods: This is a multicentre, ambispective, observational study in which 114 patients were recruited from 16 national CF units. Lung function was assessed, measured as the percentage of forced expiratory volume in the first second (FEV1%), comparing the value obtained at each treatment visit against the baseline visit values and the previous visit values. Additionally, the difference in improvement, obtained at each visit, between homozygous and heterozygous genetic groups for the CFTR protein was analysed. Frequentist and Bayesian approaches were employed. Different data set strategies (total patients, patients who completed the study, patients with imputed data) were used due to the high dropout rate. Results: A significant increase in the proportion of patients with mild LF was observed from the three-month visit after starting treatment (FEV1% ≥ 60%) compared to those observed before starting treatment, and this improvement persisted over 12 months of follow-up. This improvement was particularly noticeable at the first visit. When comparing treatment response between the genetic groups, no significant difference to treatment response was found. This finding suggests that both genetic groups respond equally to treatment. Conclusion: Evaluation of the effectiveness of ETI treatment in patients with advanced CF showed a rapid improvement in baseline FEV1%, which remained stable at subsequent visits with no significant differences between genetic groups.