Nusinersen in spinal muscular atrophy: Respiratory outcomes at tertiary care centers

Fahad Alsohime; Mohamad-Hani Temsah; Muneera Al-Jelaify; Hind Ali Bafaqih; Kamal El Masri; Ahmed Abusibah; Ayman Al-Eyadhy; Gamal Hasan; Ali Alhaboob; Majed Alabdulhafid; Mohammed Abdulrahman Almazyad; Abdulaziz Alghamdi; Fahad A Bashiri

doi:10.4103/JNSM.JNSM_68_19

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ORIGINAL ARTICLE

Year : 2020 \| Volume : 3 \| Issue : 3 \| Page : 165-169

Nusinersen in spinal muscular atrophy: Respiratory outcomes at tertiary care centers Fahad Alsohime¹, Mohamad-Hani Temsah¹, Muneera Al-Jelaify¹, Hind Ali Bafaqih², Kamal El Masri³, Ahmed Abusibah⁴, Ayman Al-Eyadhy¹, Gamal Hasan⁵, Ali Alhaboob¹, Majed Alabdulhafid¹, Mohammed Abdulrahman Almazyad¹, Abdulaziz Alghamdi⁶, Fahad A Bashiri⁷ ¹ Department of Pediatric, College of Medicine, King Saud University; Department of Pediatric, Pediatric Intensive Care Unit, King Saud University Medical City, Riyadh, Saudi Arabia ² Department of Pediatrics, Division of Pediatric Intensive Care, Prince Sultan Military Medical City, Riyadh, Saudi Arabia ³ Department of Pediatric, Pediatric Intensive Care Unit, King Fahad Specialist Hospital, Dammam, Saudi Arabia ⁴ Department of Pediatric, Pediatric Intensive Care Unit, Arryan Hospital, Dr. Suliman Al Habib Medical Group, Riyadh, Saudi Arabia ⁵ Department of Pediatric, Pediatric Intensive Care Unit, King Saud University Medical City, Riyadh, Saudi Arabia; Department of Pediatrics, Faculty of Medicine, Assiut University, Asyut, Egypt ⁶ Department of Pediatrics, Division of Pediatric Neurology, Prince Sultan Military Medical City, Riyadh, Saudi Arabia ⁷ Department of Pediatric, College of Medicine, King Saud University; Department of Pediatric, Division of Neurology, King Saud University Medical City, Riyadh, Saudi Arabia Correspondence Address: Fahad Alsohime Department of Pediatrics, College of Medicine, King Saud University, P. O. 14135, Riyadh 11424 Saudi Arabia Source of Support: None, Conflict of Interest: None DOI: 10.4103/JNSM.JNSM_68_19

Objectives: The objective of the study wasto describe the effect of nusinersen therapy and its 1^st-year associated outcomes, weaning of ventilator support, and length of stay in pediatric intensive care among ventilator-dependent spinal muscular atrophy (SMA) patients. Methods: This is a multicenter, retrospective case series study that was conducted in four tertiary care centers in Saudi Arabia. We report all the genetically diagnosed SMA ventilator-dependent patients who were admitted to the pediatric intensive care unit (PICU) and received a course of nusinersen therapy from (January 2017 to March 2018). The study endpoints were success to wean or extubate the patient and discharge from the PICU. Results: Five patients from four tertiary PICUs were included in this report. All five patients received a minimum of four doses of nusinersen intrathecally as per the manufacturer. At 12 months postinitiation of therapy, only one patient showed clinical improvement, while the other four patients did not experience improvement in their respiratory status following the administration of nusinersen. These four patients are still admitted in the PICU due to the inability to wean them from respiratory support. The remaining patient was discharged to home successfully and neither require any home ventilatory support nor oxygen therapy. Conclusion: In conclusion, our clinical experience and data suggest that the response to nusinersen therapy is very likely but mostly dependent on the time of initiation of nusinersen therapy and the cumulative number of doses among other variables related to disease severity. However, further studies targeting a larger group of patients with better standardized respiratory care are needed to investigate the effect of nusinersen on particularly the long-term outcomes.



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