Interindividual differences in the clinical effectiveness of liraglutide in Type 2 diabetes: a real-world retrospective study conducted in Spain.

AIMS: To study the response of clinical variables (HbA1c , body weight, lipid profile and blood pressure) over 24 months of liraglutide treatment in a real-world clinical setting, and to describe the evolution of HbA1c and body weight reduction in response to liraglutide treatment by employing generalized additive mixed models (GAMMs). METHODS: We included people aged ≥ 18 years with Type 2 diabetes mellitus that initiated liraglutide treatment between November 2011 and May 2015. Demographic and clinical data were retrieved retrospectively over 24 months from electronic medical records with a median duration of observation of 7.0 (IQR 3.0-12.0) months. RESULTS: Individuals that initiated liraglutide therapy were obese (BMI 39.1 kg/m2 ), with inadequate HbA1c (68 mmol/mol [8.4%]), blood pressure and lipid levels. Upon liraglutide treatment, HbA1c , body weight, mean systolic and diastolic blood pressure, and lipid levels decreased gradually. GAMMs demonstrated that longer treatment with liraglutide was a predictor of improved HbA1c response, whereas higher baseline HbA1c , longer Type 2 diabetes duration and treatment with insulin were predictors of worse HbA1c response. Higher baseline weight, longer treatment with liraglutide and the interaction between metformin and time were predictors of improved weight response. CONCLUSIONS: In this real-world study, we showed the effectiveness of liraglutide in improving body weight, HbA1c , mean systolic and diastolic blood pressure, and lipid levels. GAMMs indicated that baseline HbA1c and weight, time of treatment with liraglutide, diabetes duration and the use of metformin or insulin are predictors of clinical response to liraglutide.

[Cost-effectiveness analysis of the use of atorvastatin in patients with type 2 diabetes mellitus: a pharmacoeconomic model of the CARDS study]. / Análisis coste-efectividad del uso de atorvastatina en pacientes diabéticos de tipo 2: modelo farmacoeconómico del estudio CARDS.

BACKGROUND AND OBJECTIVE: To perform a cost-effectiveness analysis of the use of Atorvastatin 10 mg in the primary prevention of cardiovascular disease in patients with type 2 diabetes (DM2). METHOD: A deterministic and retrospective model by a decision analysis based on CARDS study (Collaborative Atorvastatin Diabetes Study) was performed. In the CARDS study, a significant reduction in cardiovascular morbimortality by the use of Atorvastatin 10 mg versus placebo (5.8 vs. 9.0%, p=0.001) in DM2 patients with an additional condition, had previously been demonstrated. In the present cost-effectiveness analysis, effectiveness units were life years gained (LYG) and quality adjusted life years (QALY), obtained from differences in morbimortality and life expectancy in DM2 patients, with and without previous cardiovascular events. Costs of the evaluated alternatives were obtained from the CARDS results. RESULTS: Incremental cost-effectiveness ratio of using Atorvastin 10 mg versus placebo was 5,886 euro per LYG and 8,046 euro per QALY. Sensitivity analyses confirmed the model stability. CONCLUSIONS: In the primary prevention of the cardiovascular disease in type 2 diabetic patients, the use of Atorvastatin 10 mg is cost-effective, with a cost per LYG and per QALY below that of other alternatives widely used in the Spanish National Health System, and also below a value considered as a reasonable threshold for our country, which might unofficialy be around 30,000 euro/ QALY.

Análisis coste-efectividad del uso de atorvastatina en pacientes diabéticos de tipo 2: modelo farmacoeconómico del estudio CARDS / Cost-effectiveness analysis of the use of atorvastatin in patients with type 2 diabetes mellitus: a pharmacoeconomic model of the CARDS study

Fundamento y objetivo: Efectuar un análisis coste-efectividad del uso de Atorvastatina 10 mg en la prevención primaria de la enfermedad cardiovascular en el paciente con Diabetes Mellitus tipo 2 (DM2). Método: Se elaboró un modelo retrospectivo y determinístico basado en un análisis de decisión a partir del estudio CARDS (Collaborative Atorvastatin Diabetes Study). En el estudio CARDS se había demostrado que Atorvastatina 10 mg versus placebo en prevención primaria en pacientes con DM2 y alguna patología asociada, reduce la morbimortalidad cardiovascular (5,8 vs. 9,0%, p = 0,001). En el presente análisis coste-efectividad, las unidades de efectividad utilizadas fueron años de vida ganados (AVG) y años de vida ajustados por calidad (AVAC), obtenidos a partir de las diferencias de morbimortalidad y de la esperanza de vida de los diabéticos, con y sin eventos previos, obtenidos de la literatura. El consumo de recursos de las alternativas en evaluación se ha extraído del estudio CARDS aplicando costes españoles. Resultados: El cociente coste-efectividad incremental derivado de utilizar Atorvastatina 10 mg versus placebo fue 5.886 € por AVG y de 8.046 € por AVAC. Los análisis de sensibilidad confirmaron la estabilidad del modelo. Conclusiones: En la prevención primaria del riesgo cardiovascular en pacientes diabéticos tipo 2, el uso de Atorvastatina 10 mg es coste-efectivo, con un coste por AVG y por AVAC por debajo de otras alternativas empleadas ampliamente en el Sistema Nacional de Salud español, y también por debajo de un valor que podría considerarse como un umbral razonable para nuestro país, situado oficiosamente en torno a los 30.000 € por AVAC

Background and objective: To perform a cost-effectiveness analysis of the use of Atorvastatin 10 mg in the primary prevention of cardiovascular disease in patients with type 2 diabetes (DM2). Method: A deterministic and retrospective model by a decision analysis based on CARDS study (Collaborative Atorvastatin Diabetes Study) was performed. In the CARDS study, a significant reduction in cardiovascular morbimortality by the use of Atorvastatin 10 mg versus placebo (5.8 vs. 9.0%, p=0.001) in DM2 patients with an additional condition, had previously been demonstrated. In the present cost-effectiveness analysis, effectiveness units were life years gained (LYG) and quality adjusted life years (QALY), obtained from differences in morbimortality and life expectancy in DM2 patients, with and without previous cardiovascular events. Costs of the evaluated alternatives were obtained from the CARDS results. Results: Incremental cost-effectiveness ratio of using Atorvastin 10 mg versus placebo was 5,886 € per LYG and 8,046 € per QALY. Sensitivity analyses confirmed the model stability. Conclusions: In the primary prevention of the cardiovascular disease in type 2 diabetic patients, the use of Atorvastatin 10 mg is cost-effective, with a cost per LYG and per QALY below that of other alternatives widely used in the Spanish National Health System, and also below a value considered as a reasonable threshold for our country, which might unofficialy be around 30,000 €/ QALY

Hiperplasia adrenal congénita: correlación fenotípica-genotípica. A propósito de cinco casos / Congenital adrenal hyperplasia: phenotypic-genotypic correlation. Five patients' report

La hiperplasia suprarrenal congénita, uno de los trastornos más frecuentes del metabolismo, resulta de un bloqueo enzimático en la síntesis de cortisol. El déficit de 21-hidroxilasa es la causa más común de hiperplasia suprarrenal congénita. Este síndrome se caracteriza por signos de hiperandrogenismo y frecuentemente de déficit mineralocorticoide. Desde el aislamiento del gen responsable de la síntesis de 21-hidroxilasa se ha avanzado mucho en el conocimiento de las alteraciones específicas que causan las diferentes formas de hiperplasia suprarrenal congénita. Aunque existe correlación fenotípica-genotípica, se ha descrito variabilidad fenotípica. El estudio genético es útil para la predicción del cuadro clínico en pacientes con deficiencia de la 21-hidroxilasa, para el diagnóstico y tratamiento prenatal y para el consejo genético en pacientes con distinto grado de gravedad clínica. En este trabajo se han estudiado las mutaciones causantes de la enfermedad en 5 mujeres con diferentes formas clínicas de hiperplasia suprarrenal congénita. Dos gemelas con la forma no clásica presentaban mutación V281L en homocigosis; 2 hermanas con forma clásica virilizante y con pérdida parcial de sal presentaban mutación en el intrón 2 (G656) en homocigosis y mutación I172N en heterocigosis. Una mujer diagnosticada de incidentaloma suprarrenal (hiperplasia macronodular bilateral) presentaba mutaciones V281L y P454S, ambas en heterocigosis. Estos casos pueden contribuir al conocimiento de la correlación genotípica-fenotípica (AU)