Oral prednisolone versus dexamethasone for the treatment of infantile epileptic spasms syndrome: Current status and way forward
Jitendra Kumar Sahu, Sandeep Negi
Pediatric Neurology Unit, Department of Pediatrics, Postgraduate Institute of Medical Education and Research, Chandigarh, India
Correspondence Address:
Jitendra Kumar Sahu
Pediatric Neurology Unit, Department of Pediatrics, Postgraduate Institute of Medical Education and Research, Chandigarh
India
Source of Support: None, Conflict of Interest: None
CheckDOI: 10.4103/aian.aian_751_22
West syndrome (renamed infantile epileptic spasms syndrome [IESS]) is one of the most common causes of infantile-onset developmental and epileptic encephalopathy. It is peculiar for its response with hormonal therapy (intramuscular adrenocorticotropic hormone therapy or oral prednisolone), which forms the first-line treatment choice. In this issue of the journal, Deswal and colleagues[1] attempted to compare the short-term effectiveness of oral dexamethasone versus standard oral prednisolone in controlling epileptic spasms at two weeks. The study was based on the assumption that due to a better blood–brain penetration kinetics of dexamethasone, its efficacy should not be inferior to prednisolone. In this pilot randomized controlled trial (RCT), the authors demonstrated comparable effectiveness and adverse effect profile. The study is remarkable for its RCT study design, and allocation of intervention was concealed. The study's limitations were small sample size, heterogeneity of the studied population, and lack of intermediate or long-term outcomes. Overall, the study provides crucial insights into the effectiveness of oral dexamethasone in the management of IESS.
In view of the limited efficacy of hormonal therapy, there is a quest to identify a better treatment choice.[2] The main challenges with such studies are small sample size, short follow-up, and lack of developmental outcome or quality of life assessment. A recently developed and validated Hindi version of Quality of Life of the Infant (QUALIN) for infants might be adopted for outcome assessment in future studies.[3] Children with IESS in developing countries commonly have a structural etiology and a long treatment lag. These two factors might affect the therapeutic response with hormonal therapy.[4],[5] It often limits the external applicability of the findings of the study. The treatment regime of hormonal therapy is quite variable in terms of dose, preparation, and duration. Each component has its influence on the outcome. All these issues need to be considered while designing a RCT in IESS.
Hormonal therapy is associated with systemic adverse events. There is need to develop novel treatment solutions with selective efficacy and minimum side effect. Ganaxolone is a neuroactive steroid and demonstrated potential benefit refractory epileptic spasms.[6] Future randomized clinical trial should explore safety and effectiveness of ganaxolone in children with IESS.
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