Introduction: Infantile spasm syndrome is an epileptic and neurodevelopmental encephalopathy whose first-line treatment includes vigabatrin and hormonal therapy. However, there are refractory cases.
Objective: To compare the efficacy of the available treatments for refractory infantile spasm syndrome (RISS).
Methods: We searched PubMed, Embase, Scopus, Web of Science, Cochrane CENTRAL Clinical Trials, International Clinical Trials Registry Platform (ICTRP) and ClinicalTrials.gov until March 2024 for randomized controlled trials (RCTs) comparing the efficacy of two of the following three treatment alternatives: antiepileptics, ketogenic diet and surgery. The primary outcome was electroclinical response. The GRADE methodology was used to appraise the certainty of the evidence. We generated a Summary of Findings table using the GRADEpro software.
Results: We did not find enough RCTs to do a meta-analysis. Only one trial was close to fulfill our inclusion criteria. This study involved 318 patients with RISS receiving antiepileptic drugs and compared the modified Atkins diet (MAD) versus normal diet. However, we found a high risk of bias.
Conclusion: High-quality RCTs comparing treatment modalities for patients with RISS are urgent due to the devastating nature of this condition.
