Background/Objectives: The objective of our study was to quantify the annual costs, from a societal perspective, encompassing direct health care costs, direct non-health care costs, and labor productivity losses associated with spinal muscular atrophy (SMA) patients in Bulgaria and their caregivers. Methods: We applied a prevalence-based, bottom-up costing methodology to assess the socio-economic burden of SMA from a societal perspective. We evaluated and summed up all costs for health services (diagnosis, treatment, follow-up, and rehabilitation), educational and social services, and formal and informal care in the community, as well as indirect costs due to the loss of productivity and work capacity of the SMA patients’ caregivers. Results: Nine parents of SMA patients provided consent and completed the study’s questionnaire. Two children had SMA type III, and seven had SMA type II. The median annual socio-economic burden per SMA patient was EUR 254,968.80. The high direct costs, primarily driven by drug expenses, and the substantial indirect costs resulting from the loss of productivity among informal caregivers were the primary causes. We found no utilization of social care and educational services. Conclusions: We emphasize the need for careful consideration of long-term outcomes, real-world data collection, and performance-based reimbursement. An ideal scenario could achieve these objectives in synergy. A second layer of policy actions and measures must address the unmet needs of SMA patients and their families using a holistic approach. The indirect costs associated with SMA, particularly the productivity loss of informal caregivers, underscore the need for comprehensive support programs.

Background: Cardiac tamponade (CT) is a rare but life-threatening medical emergency caused by fluid accumulation in the pericardial sac, impairing cardiac filling and reducing output. More than 20% of CT cases are iatrogenic. CT is a recognized complication of central venous catheter (CVC) placement, with mortality rates in pediatric patients reported to reach 50%. Clinical presentation is often nonspecific, and echocardiography remains the diagnostic gold standard. Case report: We present two pediatric cases of CT due to late CVC migration, managed in the pediatric intensive care unit (PICU). The first case involved a 25-day-old neonate with short bowel syndrome who received prolonged parenteral nutrition via CVC. Four days after catheter insertion, the patient developed sudden cardiocirculatory collapse. The second case featured a 2-year-old child with Leigh syndrome who required mechanical ventilation and multimodal pharmacological therapy. Six days after CVC placement, the patient developed acute hemodynamic deterioration. In both cases, echocardiography confirmed CT, while chest radiography suggested intracardiac positioning of the catheter tip. Management and outcome: Emergency pericardiocentesis and advanced cardiopulmonary resuscitation were performed. Despite transient hemodynamic stabilization, both patients developed multiorgan failure with fatal outcomes. Conclusions: CT is a critical complication in pediatric patients with CVCs. Accurate verification of catheter tip position is essential, and intracardiac placement should be avoided. Any sudden clinical deterioration in a patient with a CVC should raise suspicion of late catheter migration and requires immediate life-saving intervention.