Article type
Abstract
Background:
Myelin oligodendrocyte glycoprotein antibody–associated disease (MOGAD) is a newly identified inflammatory disorder of the central nervous system. To improve the evidence-based care of children with MOGAD, we explored developing a clinical practice guideline (CPG) for pediatric MOGAD in 2023.
Objective:
We aim to offer valuable insights into the formation of CPGs for rare diseases featured by limited evidence by sharing our experience.
Methods:
The GIN‐McMaster Guideline Development checklist and GRADE methodology were followed throughout the guideline process. A guideline panel was formulated first consisting of relevant stakeholders of MOGAD. A preliminary literature search was conducted to define the guideline scope. A systematic search was then conducted in both English and Chinese databases to identify relevant articles published from January 2010 to May 2022. Systematic reviews were performed for the key questions. Two parents of children with MOGAD were invited to vote in moving from evidence to decision.
Results:
After a panel discussion based on the priority assessment results, 12 out of 16 original clinical questions were retained, covering 6 topics about MOGAD. A total of 185 studies were finally included in the evidence synthesis across these categories comprising 110 case series, 70 case reports, and 5 cohort studies. Notably, case series and case reports are very low-quality evidence, and 5 cohort studies were assessed as very low quality by using the tool Risk of Bias In Non-randomised Studies of Interventions (ROBINS-I). Expert evidence and data from adults with MOGAD were used as indirect evidence. We formulated 5 strong recommendations and 2 conditional recommendations guided by GRADE evidence to decision frameworks.
Conclusion:
Although the evidence predominantly comprises case reports and case series, the evidence summary on MOGAD not only enhances the understanding of MOGAD diagnosis and treatment but also provides detailed insights into long-term follow-up in pediatric patients, elucidating disease course, phenotypic variances, and long-term prognosis discrepancies between pediatric and adult cohorts. Furthermore, it illuminates the dynamic shifts in MOG antibodies and their implications for disease progression. Additionally, it outlines future research directions, emphasizing the need for prospective comparative clinical studies to clarify preventive and treatment strategies targeting MOGAD mechanisms.
Myelin oligodendrocyte glycoprotein antibody–associated disease (MOGAD) is a newly identified inflammatory disorder of the central nervous system. To improve the evidence-based care of children with MOGAD, we explored developing a clinical practice guideline (CPG) for pediatric MOGAD in 2023.
Objective:
We aim to offer valuable insights into the formation of CPGs for rare diseases featured by limited evidence by sharing our experience.
Methods:
The GIN‐McMaster Guideline Development checklist and GRADE methodology were followed throughout the guideline process. A guideline panel was formulated first consisting of relevant stakeholders of MOGAD. A preliminary literature search was conducted to define the guideline scope. A systematic search was then conducted in both English and Chinese databases to identify relevant articles published from January 2010 to May 2022. Systematic reviews were performed for the key questions. Two parents of children with MOGAD were invited to vote in moving from evidence to decision.
Results:
After a panel discussion based on the priority assessment results, 12 out of 16 original clinical questions were retained, covering 6 topics about MOGAD. A total of 185 studies were finally included in the evidence synthesis across these categories comprising 110 case series, 70 case reports, and 5 cohort studies. Notably, case series and case reports are very low-quality evidence, and 5 cohort studies were assessed as very low quality by using the tool Risk of Bias In Non-randomised Studies of Interventions (ROBINS-I). Expert evidence and data from adults with MOGAD were used as indirect evidence. We formulated 5 strong recommendations and 2 conditional recommendations guided by GRADE evidence to decision frameworks.
Conclusion:
Although the evidence predominantly comprises case reports and case series, the evidence summary on MOGAD not only enhances the understanding of MOGAD diagnosis and treatment but also provides detailed insights into long-term follow-up in pediatric patients, elucidating disease course, phenotypic variances, and long-term prognosis discrepancies between pediatric and adult cohorts. Furthermore, it illuminates the dynamic shifts in MOG antibodies and their implications for disease progression. Additionally, it outlines future research directions, emphasizing the need for prospective comparative clinical studies to clarify preventive and treatment strategies targeting MOGAD mechanisms.