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Design Details
Arms
Arm Details
Sample Characteristics
Outcomes
Outcome Details
Risk of Bias Assessment
Results
Print Data

Extraction form for project: Management of Infantile Epilepsies

Design Details

1. Study Design


	RCT Nonrandomized comparative study Pre/post observational study

2. Country


	USA

3. Total sample size


	103

4. Intervention Type


	Pharmacological Dietary Surgery

5. Funding source


	Investigator initiated grant by Eisai Inc (manufacturer of the tested medication)

Arms

Arm Name	Arm Description
Rufinamide	50.5% started at 5 mg/kg/d, and the other 49.5% started at 10 mg/kg/d. Titration schedules varied. Median dosage at the last follow-up was 42 mg/kg/d (IQR 34-56).

Arm Details

1. Sample size for arm

Rufinamide


	103

2. Inclusion Criteria

Rufinamide


	Age 36 months or fewer, received rufinamide for refractory epilepsy (at least 2 prior medications) between June 2010 and June 2018, and had “adequate” clinical information regarding seizure types, frequency, rufinamide dosing, and adverse events. Authors did not define “adequate.”

3. Exclusion Criteria

Rufinamide


	NR

4. Treatment details

Rufinamide


	50.5% started at 5 mg/kg/d, and the other 49.5% started at 10 mg/kg/d. Titration schedules varied. Median dosage at the last followup was 42 mg/kg/d (IQR 34-56).

Sample Characteristics

1. Gender, number of male

Rufinamide


	60

2. Gender, percentage of male

Rufinamide


	58

3. Age at intervention (mean)

Rufinamide


	NR

4. Age at intervention (SD)

Rufinamide


	NR

5. Age at intervention (range)

Rufinamide


	20 months (IQR 13-28)

6. Age at intervention (median)

Rufinamide


	20 months

7. Seizure types and/or etiology

Rufinamide

8. Prior treatment

Rufinamide


	Levetiracetam 69%, Topiramate 39%, Clobazam 33%, Vigabatrin 32%, Clonazepam 20%, Phenobarbital 17%, ketogenic diet 16%, zonizamide 14%, Valproicvalproic acid 10%, oxcarbazepine 7%, steroid 7%, lacosamide 5%, lamotrigine 5%, others 12%.

9. Comments

Rufinamide


	Boston Children's Hospital, USA

Outcomes

Type	Domain	Specific measurement (i.e., tool/definition/specific outcome)	Populations	Timepoints
Categorical	Seizure freedom		All Participants	Median 15 months
Categorical	Seizure reduction/effective (>50% reduction)		All Participants	Median 15 months
Categorical	Discontinuation due to lack of efficacy		All Participants	Median 15 months
Categorical	Adverse Event	Discontinuation due to adverse effects	All Participants	Median 15 months
Categorical	Adverse Event	Somnolence	All Participants	Median 15 months
Categorical	Adverse Event	Insomnia	All Participants	Median 15 months
Categorical	Adverse Event	Nausea	All Participants	Median 15 months
Categorical	Adverse Event	Vomiting	All Participants	Median 15 months
Categorical	Adverse Event	Mood-behavioral change	All Participants	Median 15 months
Categorical	Adverse Event	Irritability	All Participants	Median 15 months
Categorical	Adverse Event	Appetite change	All Participants	Median 15 months
Categorical	Adverse Event	Weight loss	All Participants	Median 15 months
Categorical	Adverse Event	Diplopua	All Participants	Median 15 months
Categorical	Adverse Event	Temporary blood lactic acid level elevation	All Participants	Median 15 months

Outcome Details

1. For RCTs: Generation of randomization sequence

Seizure freedom

Seizure reduction/effective (>50% reduction)

Discontinuation due to lack of efficacy

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

2. For RCTs: Allocation concealment

Seizure freedom

Seizure reduction/effective (>50% reduction)

Discontinuation due to lack of efficacy

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

3. For RCTs: Baseline imbalance

Seizure freedom

Seizure reduction/effective (>50% reduction)

Discontinuation due to lack of efficacy

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

4. For RCTs: Patient blinded

Seizure freedom

Seizure reduction/effective (>50% reduction)

Discontinuation due to lack of efficacy

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

5. For RCTs: Staff blinded

Seizure freedom

Seizure reduction/effective (>50% reduction)

Discontinuation due to lack of efficacy

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

6. For RCTs: Differential ancillary treatments

Seizure freedom

Seizure reduction/effective (>50% reduction)

Discontinuation due to lack of efficacy

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

7. For RCTs: Adherence

Seizure freedom

Seizure reduction/effective (>50% reduction)

Discontinuation due to lack of efficacy

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

8. For RCTs: Analytic approach to address departures from intended intervention

Seizure freedom

Seizure reduction/effective (>50% reduction)

Discontinuation due to lack of efficacy

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

9. For RCTs: Data on at least 80% of those enrolled

Seizure freedom

Seizure reduction/effective (>50% reduction)

Discontinuation due to lack of efficacy

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

10. For RCTs: Differential dropout <=15%

Seizure freedom

Seizure reduction/effective (>50% reduction)

Discontinuation due to lack of efficacy

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

11. For RCTs: Standard way to measure the outcome

Seizure freedom

Seizure reduction/effective (>50% reduction)

Discontinuation due to lack of efficacy

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

12. For RCTs: Blinded outcome assessor

Seizure freedom

Seizure reduction/effective (>50% reduction)

Discontinuation due to lack of efficacy

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

13. For RCTs: Bias in selection of reported results

Seizure freedom

Seizure reduction/effective (>50% reduction)

Discontinuation due to lack of efficacy

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

14. For nonrandomized comparative studies: Confounding

Seizure freedom

Seizure reduction/effective (>50% reduction)

Discontinuation due to lack of efficacy

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

15. For nonrandomized comparative studies: Selection into study

Seizure freedom

Seizure reduction/effective (>50% reduction)

Discontinuation due to lack of efficacy

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

16. For nonrandomized comparative studies: Classification of interventions

Seizure freedom

Seizure reduction/effective (>50% reduction)

Discontinuation due to lack of efficacy

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

17. For nonrandomized comparative studies: Differential ancillary treatments

Seizure freedom

Seizure reduction/effective (>50% reduction)

Discontinuation due to lack of efficacy

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

18. For nonrandomized comparative studies: Adherence

Seizure freedom

Seizure reduction/effective (>50% reduction)

Discontinuation due to lack of efficacy

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

19. For nonrandomized comparative studies: Data on at least 80% of those enrolled

Seizure freedom

Seizure reduction/effective (>50% reduction)

Discontinuation due to lack of efficacy

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

20. For nonrandomized comparative studies: Differential dropout <=15%

Seizure freedom

Seizure reduction/effective (>50% reduction)

Discontinuation due to lack of efficacy

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

21. For nonrandomized comparative studies: Standard way to measure the outcome

Seizure freedom

Seizure reduction/effective (>50% reduction)

Discontinuation due to lack of efficacy

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

22. For nonrandomized comparative studies: Blinded outcome assessor

Seizure freedom

Seizure reduction/effective (>50% reduction)

Discontinuation due to lack of efficacy

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

23. For nonrandomized comparative studies: Bias in selection of reported result

Seizure freedom

Seizure reduction/effective (>50% reduction)

Discontinuation due to lack of efficacy

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

Adverse Event

24. For single arm studies: Does the design or analysis control account for important confounding and modifying variables through matching, stratification, multivariable analysis, or other approaches?

Seizure freedom


	Some concerns

Seizure reduction/effective (>50% reduction)


	Some concerns

Discontinuation due to lack of efficacy

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

25. For single arm studies: Did researchers rule out any impact from a concurrent intervention or an unintended exposure that might bias results?

Seizure freedom


	High

Seizure reduction/effective (>50% reduction)


	High

Discontinuation due to lack of efficacy

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

26. For single arm studies: Did the study maintain fidelity to the intervention protocol?

Seizure freedom


	Low

Seizure reduction/effective (>50% reduction)


	Low

Discontinuation due to lack of efficacy

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

27. For single arm studies: If attrition (overall or differential nonresponse, dropout, loss to follow-up, or exclusion of participants) was a concern, were missing data handled appropriately (e.g., intention-to-treat analysis and imputation)?

Seizure freedom


	Low

Seizure reduction/effective (>50% reduction)


	Low

Discontinuation due to lack of efficacy

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

28. For single arm studies: Were the outcome assessors blinded to the intervention or exposure status of participants?

Seizure freedom


	High

Seizure reduction/effective (>50% reduction)


	High

Discontinuation due to lack of efficacy

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

29. For single arm studies: Were interventions/exposures assessed/defined using valid and reliable measures, implemented consistently across all study participants?

Seizure freedom


	Low

Seizure reduction/effective (>50% reduction)


	Low

Discontinuation due to lack of efficacy

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

30. For single arm studies: Were outcomes assessed/defined using valid and reliable measures, implemented consistently across all study participants?

Seizure freedom


	Low

Seizure reduction/effective (>50% reduction)


	Low

Discontinuation due to lack of efficacy

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

31. For single arm studies: Were confounding variables assessed using valid and reliable measures, implemented consistently across all study participants?

Seizure freedom


	Low

Seizure reduction/effective (>50% reduction)


	Low

Discontinuation due to lack of efficacy

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

Adverse Event


	Low

32. For single arm studies: Were the potential outcomes prespecified by the researchers? Are all prespecified outcomes reported?

Seizure freedom


	High

Seizure reduction/effective (>50% reduction)


	High

Discontinuation due to lack of efficacy

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

Adverse Event


	High

Risk of Bias Assessment

1. For Risk of Bias Assessment results, see Outcome Details section.

Results

Categorical

Seizure freedom

All Participants
		Descriptive Statistics		Between Arm Comparisons
		Rufinamide
Median 15 months
	Total (N analyzed)		Odds Ratio (OR)
	Events		95% CI low (OR)
	Percentage		95% CI high (OR)
			p value
		Within Arm Comparisons		Net Comparisons
		Rufinamide

Seizure reduction/effective (>50% reduction)

All Participants
		Descriptive Statistics		Between Arm Comparisons
		Rufinamide
Median 15 months
	Total (N analyzed)		Odds Ratio (OR)
	Events		95% CI low (OR)
	Percentage		95% CI high (OR)
			p value
		Within Arm Comparisons		Net Comparisons
		Rufinamide

Discontinuation due to lack of efficacy

All Participants
		Descriptive Statistics		Between Arm Comparisons
		Rufinamide
Median 15 months
	Total (N analyzed)		Odds Ratio (OR)
	Events		95% CI low (OR)
	Percentage		95% CI high (OR)
			p value
		Within Arm Comparisons		Net Comparisons
		Rufinamide

Adverse Event (Discontinuation due to adverse effects)

All Participants
		Descriptive Statistics		Between Arm Comparisons
		Rufinamide
Median 15 months
	Total (N analyzed)		Odds Ratio (OR)
	Events		95% CI low (OR)
	Percentage		95% CI high (OR)
			p value
		Within Arm Comparisons		Net Comparisons
		Rufinamide

Adverse Event (Somnolence)

All Participants
		Descriptive Statistics		Between Arm Comparisons
		Rufinamide
Median 15 months
	Total (N analyzed)		Odds Ratio (OR)
	Events		95% CI low (OR)
	Percentage		95% CI high (OR)
			p value
		Within Arm Comparisons		Net Comparisons
		Rufinamide

Adverse Event (Insomnia)

All Participants
		Descriptive Statistics		Between Arm Comparisons
		Rufinamide
Median 15 months
	Total (N analyzed)		Odds Ratio (OR)
	Events		95% CI low (OR)
	Percentage		95% CI high (OR)
			p value
		Within Arm Comparisons		Net Comparisons
		Rufinamide

Adverse Event (Nausea)

All Participants
		Descriptive Statistics		Between Arm Comparisons
		Rufinamide
Median 15 months
	Total (N analyzed)		Odds Ratio (OR)
	Events		95% CI low (OR)
	Percentage		95% CI high (OR)
			p value
		Within Arm Comparisons		Net Comparisons
		Rufinamide

Adverse Event (Vomiting)

All Participants
		Descriptive Statistics		Between Arm Comparisons
		Rufinamide
Median 15 months
	Total (N analyzed)		Odds Ratio (OR)
	Events		95% CI low (OR)
	Percentage		95% CI high (OR)
			p value
		Within Arm Comparisons		Net Comparisons
		Rufinamide

Adverse Event (Mood-behavioral change)

All Participants
		Descriptive Statistics		Between Arm Comparisons
		Rufinamide
Median 15 months
	Total (N analyzed)		Odds Ratio (OR)
	Events		95% CI low (OR)
	Percentage		95% CI high (OR)
			p value
		Within Arm Comparisons		Net Comparisons
		Rufinamide

Adverse Event (Irritability)

All Participants
		Descriptive Statistics		Between Arm Comparisons
		Rufinamide
Median 15 months
	Total (N analyzed)		Odds Ratio (OR)
	Events		95% CI low (OR)
	Percentage		95% CI high (OR)
			p value
		Within Arm Comparisons		Net Comparisons
		Rufinamide

Adverse Event (Appetite change)

All Participants
		Descriptive Statistics		Between Arm Comparisons
		Rufinamide
Median 15 months
	Total (N analyzed)		Odds Ratio (OR)
	Events		95% CI low (OR)
	Percentage		95% CI high (OR)
			p value
		Within Arm Comparisons		Net Comparisons
		Rufinamide

Adverse Event (Weight loss)

All Participants
		Descriptive Statistics		Between Arm Comparisons
		Rufinamide
Median 15 months
	Total (N analyzed)		Odds Ratio (OR)
	Events		95% CI low (OR)
	Percentage		95% CI high (OR)
			p value
		Within Arm Comparisons		Net Comparisons
		Rufinamide

Adverse Event (Diplopua)

All Participants
		Descriptive Statistics		Between Arm Comparisons
		Rufinamide
Median 15 months
	Total (N analyzed)		Odds Ratio (OR)
	Events		95% CI low (OR)
	Percentage		95% CI high (OR)
			p value
		Within Arm Comparisons		Net Comparisons
		Rufinamide

Adverse Event (Temporary blood lactic acid level elevation)

All Participants
		Descriptive Statistics		Between Arm Comparisons
		Rufinamide
Median 15 months
	Total (N analyzed)		Odds Ratio (OR)
	Events		95% CI low (OR)
	Percentage		95% CI high (OR)
			p value
		Within Arm Comparisons		Net Comparisons
		Rufinamide