Everolimus for cardiac rhabdomyomas in children with tuberous sclerosis. The ORACLE study protocol (everOlimus for caRdiac rhAbdomyomas in tuberous sCLErosis): a randomised, multicentre, placebo-controlled, double-blind phase II trial.

INTRODUCTION: Tuberous sclerosis complex is a rare genetic disorder leading to the growth of hamartomas in multiple organs, including cardiac rhabdomyomas. Children with symptomatic cardiac rhabdomyoma require frequent admissions to intensive care units, have major complications, namely, arrhythmias, cardiac outflow tract obstruction and heart failure, affecting the quality of life and taking on high healthcare cost. Currently, there is no standard pharmacological treatment for this condition, and the management includes a conservative approach and supportive care. Everolimus has shown positive effects on subependymal giant cell astrocytomas, renal angiomyolipoma and refractory seizures associated with tuberous sclerosis complex. However, evidence supporting efficacy in symptomatic cardiac rhabdomyoma is limited to case reports. The ORACLE trial is the first randomised clinical trial assessing the efficacy of everolimus as a specific therapy for symptomatic cardiac rhabdomyoma. METHODS: ORACLE is a phase II, prospective, randomised, placebo-controlled, double-blind, multicentre protocol trial. A total of 40 children with symptomatic cardiac rhabdomyoma secondary to tuberous sclerosis complex will be randomised to receive oral everolimus or placebo for 3 months. The primary outcome is 50% or more reduction in the tumour size related to baseline. As secondary outcomes we include the presence of arrhythmias, pericardial effusion, intracardiac obstruction, adverse events, progression of tumour reduction and effect on heart failure. CONCLUSIONS: ORACLE protocol addresses a relevant unmet need in children with tuberous sclerosis complex and cardiac rhabdomyoma. The results of the trial will potentially support the first evidence-based therapy for this condition.

Partial-Cost Analysis and Economic Impact of Ambulatory Coronary Angioplasty in a Private Hospital in the Caribbean: Análisis Parcial de Costos e Impacto Económico de la Angioplastia Coronaria Ambulatoria en un Hospital Privado del Caribe.

OBJECTIVES: This study aimed to assess direct costs of percutaneous coronary intervention (PCI) without hospital admission versus PCI with hospital admission longer than 24 hours in a private hospital-institutional perspective in the Dominican Republic in 2022. METHODS: This study has a comparative approach based on a prospective cross-sectional partial-cost analysis. We evaluated the direct costs of 10 patients from PCI without hospital admission approach and 10 patients from a hospital admission longer than 24 hours as a control group. We used a "first-come-first-served" approach from December 2021 to March 2022. The analysis used the electronic invoice generated for each patient. RESULTS: PCI without hospital admission approach represents $472.56 in patient savings, equivalent to a cost reduction of 12.5%. The subcosts analysis showed the pharmacy section as the main driver of the overall cost difference. CONCLUSIONS: PCI without hospital admission was economically cost-saving compared with the control approach in direct costs in the Dominican perspective. The economic benefit is substantial and compliments the ease of use. This analysis may lead to improvements in institutional management of resources and can potentially be adapted to other health systems in the region.