Effect of new modalities of treatment on physicians management plan for patients with spinal muscular atrophy.

OBJECTIVE: To determine physicians` attitudes and stated practice in the management of patients with spinal muscular atrophy (SMA). We also aimed to explore their knowledge about consensus statement for standard of care in SMA and the role of new treatment modalities in changing the method of practice in the management of these cases. METHODS: This is a quantitative observational cross-sectional study, conducted from February to May 2017 among physicians who manage SMA patients in Kingdom of Saudi Arabia. The study cohort included pediatric neurologists, adult neurologists, and physicians of other sub-specialties who manage SMA patients. We used online and paper-based questionnaires. RESULTS: Half of the 169 participants were aware of the consensus guidelines for the care of SMA patients. With regard to the newly released Nursinersen treatment protocol for SMA-diagnosed patients, half of the participants were uncertain, and the other half were hesitant about its outcomes. Junior physicians tended to be significantly more inclined to reverse the do-not-resuscitate (DNR) status of an SMA-diagnosed child than more senior physicians. CONCLUSION: Our results indicate the existence of wide differences in physician practice with children of SMA disease. Our data demonstrate a need for increased awareness of consensus guidelines and further awareness about the physician`s role in the variability of care for children with SMA.

Respiratory support attitudes among pediatric intensive care staff for spinal muscular atrophy patients in Saudi Arabia.

OBJECTIVE: To explore therapeutic attitude of healthcare providers practicing in pediatric critical care in Saudi Arabia toward patients with Spinal Muscular Atroph (SMA) Type I, and to explore their awareness about the International Consensus statement for SMA care. METHODS: A cross-sectional survey was conducted in April 2015 during 6th Saudi Critical Care Conference, targeting physicians and respiratory therapists practicing in Pediatric Critical Care. RESULTS: Sixty participants accepted to participate in this survey. Out of those who answered the questionnaire, 44 were included in the analysis. Majority (66%) of participants were unaware of the International Consensus guidelines for SMA. Endotracheal intubation was reported as an acceptable intervention in SMA patients with acute respiratory failure by 43% of participants. Similarly, chronic home ventilation was agreed by 41% of participants. CONCLUSION: A nationwide adaptation of the International SMA Consensus guidelines for children with SMA I is recommended, aiming to decrease variability and standardize their management across various healthcare facilities in Saudi Arabia.

Long-stay patients in pediatric intensive care units. Five-years, 2-points, cross-sectional study.

OBJECTIVES: To explore the changing patterns of long-stay patients (LSP) to improve the utilization of pediatric intensive care units (PICUs) resources. METHODS: This is a 2-points cross-sectional study (5 years apart; 2014-2019) conducted among PICUs and SCICUs in Riyadh, Saudi Arabia. Children who have stayed in PICU for more than 21 days were included. RESULTS: Out of the 11 units approached, 10 (90%) agreed to participate. The prevalence of LSP in all these hospitals decreased from 32% (48/150) in 2014 to 23.4% (35/149) in 2019. The length of stay ranged from 22 days to 13.5 years. The majority of LSP had a neuromuscular or cardiac disease and were admitted with respiratory compromise. Ventilator-associated pneumonia was the most prevalent complication (37.5%). The most commonly used resources were mechanical ventilation (93.8%), antibiotics (60.4%), and blood-products transfusions (35.4%). The most common reason for the extended stay was medical reasons (51.1%), followed by a lack of family resources (26.5%) or lack of referral to long-term care facilities (22.4%). CONCLUSION: A long-stay is associated with significant critical care bed occupancy, complications, and utilization of resources that could be otherwise utilized as surge capacity for critical care services. Decreasing occupancy in this multicenter study deserves further engagement of the healthcare leaders and families to maximize the utilization of resources.