Table 2.
Summary of ASMs used for CDD-related epilepsy based on data from four large observational studies: Clobazam, lamotrigine, valproic acid, and vigabatrin were more commonly in the top-five most effective ASMs, but response rate declines over time.
| Study Type (Level of Evidence) |
Seizure Reduction Criteria |
ACTH / Cortico- steroid % (N) * |
CBZ / OXC % (N) |
CLB % (N) |
LTG % (N) |
LVT % (N) |
PB % (N) |
RFM % (N) |
TPM % (N) |
VGB % (N) |
VPA % (N) |
ZNS %(N) |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Retros Obs (4) [27] | >50% at 3 months | 19 (5/26) | 10 (3/29) | 24 (4/17) | 22 (5/23) | 16 (5/31) | 8 (2/26) | 8 (1/13) | 16 (5/31) | 32 (8/25) | 21 (7/34) | 18 (2/11) |
| >50% at 12 months | 0 (0/26) | 7 (2/29) | 0 (0/17) | 9 (2/23) | 0 (0/31) | 8 (2/26) | 0 (0/13) | 3 (1/31) | 4 (1/25) | 9 (3/34) | 0 (0/18) | |
| Caregiver Survey (4) [33] | “Effective” not defined | 11 (1/9) | 0 (0/7) | 43 (6/14) | 15 (2/13) | 11 (3/27) | 0 (0/21) | 0 (0/1) | 7 (1/15) | 52 (12/23) | 19 (5/27) | 29 (2/7) |
| Retros Obs (4) [14] | ≥50% | 41 (9/22)** | 0 (0/21) | 0 (0/22) | 5 (1/21) | 0 (0/21) | 0 (0/19) | 0 (0/5) | 0 (0/20) | 50 (1/2) | 7 (2/28) | 0 (0/23) |
| Retros Obs (4) [15] | ≥50% for 2 weeks | 38 (15/40) | 19 (5/26) | 48 (12/25) | 40 (6/15) | 14 (8/56) | 37 (13/35) | 47 (7/15) | 26 (10/38) | 56 (15/27) | 36 (9/25) | 0 (0/13) |
| ≥50% for 3 months | 0 (0/40) | 8 (2/26) | 36 (9/25) | 13 (2/15) | 5 (3/56) | 9 (3/35) | 27 (4/15) | 13 (5/38) | 33 (9/27) | 28 (7/25) | 0 (0/13) |
Levetiracetam was commonly used, but clobazam, lamotrigine, vigabatrin, and valproic acid were most consistently effective for seizure reduction. Response rate to ASMs declined over time when reported longitudinally. Bold text indicates top-five most effective medications, excluding ACTH and corticosteroid, each with minimum ten or more sample size, from each study. Level of evidence is sourced from Oxford Centre for Evidence-Based Medicine (http://www.cebm.net/index.aspx?o=5653).
(Legend: ACTH = adrenocorticotropic hormone. ASMs = Anti-seizure medications. CBZ/OXC = carbamazepine or oxcarbazepine. CDD = CDKL5 deficiency disorder. CLB = clobazam. LTG = lamotrigine. LVT = levetiracetam. PB = phenobarbital. Retros Obs = retrospective observational study. RFN = rufinamide. TPM = topiramate. VGB = vigabatrin. VPA = valproic acid. ZNS = zonisamide.
= Response was not differentiated by seizure types (spasms versus other seizure types).
= ACTH effects were reported to be short-lived.)