Table 3.
Treatment Options in Infantile Spasms.
| Treatment | Dosage | Class | n | Study | Etiology | Efficacy in cessation of clinical spasms | Efficacy in cessation of clinical spasms and abolishing hypsarrythmia | Efficacy in normalizing cognitive outcome |
|---|---|---|---|---|---|---|---|---|
| ACTH (high-dose) | 150 U/m2/d × 2 wk, then taper | I/II | 15 | Baram, 1996 | Combined | – | 87% (at 2 wk) | – |
| 150 U/m2/d × 3 wk, then taper | I/II | 26 | Hrachovy, 1994 | Combined | – | 50% (during 3 mo) | – | |
| Tetracosactide 1 mg (equivalent to ACTH 100 U/d) q48h × 2 wk, then taper | III | 37 | Kivity, 2004 | Cryptogenic | – | 100% (treatment within 1 mo), 87% (treatment after 1 mo), assessed over 1–4 wk | 100% (treatment within 1 mo), 47% (treatment after 1 mo), assessed over 6–21 y | |
| 110 U/m2/d × 3 wk, then taper | III | 128 | Lombrosso, 1983 | Combined | 48% (after 10 mo) | 43% (after 10 mo) | 50% (ACTH 8 wk), 59% (ACTH 8 wk + 14 wk of oral steroids) after 6 y (in cryptogenic cases, n = 73) | |
| Tetracosactide 0.75 mg (equivalent to ACTH 75 U/d) q48h × 2 wk, then taper | I/III | 25 | Lux, 2004 | Combined | 76% (at 2 wk) | – | – | |
| ACTH (low-dose) | 20–30 U/d, then taper | I/II | 24 | Hrachovy, 1994 | Combined | – | 58% (during 6 wk) | – |
| Oral steroids | Prednisone 2 mg/kg/d × 2 wk, then taper | I/II | 14 | Baram, 1996 | Combined | – | 29% (at 2 wk) | – |
| Prednisolone 40–60 mg/d × 2 wk, then taper | I/III | 30 | Lux, 2004 | Combined | 70% (at 2 wk) | – | – | |
| Prednisolone 8 mg/kg/d (max 60 mg/d) × 2 wk | III | 27 | Hussain, 2014 | Combined | – | 63% (at 2 wk) | – | |
| Prednisolone 2 mg/kg/d × 8 wk, then taper | III | 77 | Lombrosso, 1983 | Combined | 38% (after 10 mo) | 35% (after 10 mo) | 12% after 6 y (in cryptogenic cases, n = 17) | |
| Hormonal therapy | Prednisolone 40–60 mg/d, or tetracosactide 0.75 mg q48h × 2 wk, then taper | I/III | 55 | Lux, 2005 | Combined | 75% (at 12–14 mo) | – | In cryptogenic group, VABS 88.2 (better than vigabatrin group of 78.9) at 12–14 mo |
| Vigabatrin | Initial dose 50 mg/kg/d, maximum 150 mg/kg/d | I | 20 | Appleton, 1999 | Combined | 35% (VGB) vs 10% (placebo) at 5 d, 42% after 24 wk with open-label phase | 25% (VGB) vs 5% (placebo) at 5 d | – |
| Initial dose 50 mg/kg/d, maximum 150 mg/kg/d | I/III | 52 | Lux, 2004 | Combined | 52% (at 2 wk) | – | – | |
| Initial dose 50 mg/kg/d, maximum 150 mg/kg/d | I/III | 52 | Lux, 2005 | Combined | 76% (at 12–14 mo) | – | In cryptogenic group, VABS 78.9 (worse than hormonal therapy group of 88.2) at 12–14 mo | |
| Initial dose 50 mg/kg/d, maximum 150 mg/kg/d | IV | 180 | Djuric, 2014 | Combined | – | 56% (at 2 wk) | 44% (mean follow-up, 11 y after treatment) | |
| Valproate | Initial dose 15–20 mg/kg/d, maximum 60 mg/kg/d | IV | 19 | Bachman, 1982 | Combined | 42% (during 2 y) | – | – |
| Topiramate | Initial dose 25 mg/d, maximum 24 mg/kg/d | IV | 11 | Glauser, 1998 | Combined | – | 45% (during 90 d) | – |
| Zonisamide | Initial dose 3–5 mg/kg/d, maximum 10 mg/kg/d | IV | 11 | Suzuki, 1997 | Combined | – | 36% (at 3 wk) | – |
| 4–13 mg/kg/d | IV | 54 | Suzuki, 2002 | Combined | – | 20% (at 3 wk) | 5/7 (71%) responders had normal DQ (mean follow-up, 53 mo after treatment) | |
| Levetiracetam | Initial dose 20 mg/kg/d, maximum 80 mg/kg/d | IV | 7 | Mikati, 2008 | Combined | – | 14% | – |
| Pyridoxine | Pyridoxal phosphate 30–400 mg/d | IV | 118 | Ohtsuka, 1987 | Combined | 13% | 13% | 43% in responder |
| Ketogenic diet | – | III | 13 | Kossoff, 2008 | Combined | 62% (at 1 mo) | 9% (at 1 mo) | – |
| Surgery | – | IV | 65 | Chugani, 2015 | Lesional cases | 71% class I (mean follow-up period at 45 mo) | – | – |
| – | IV | 47 | Shields, 2002 | Lesional cases | 60% class I (at 24–60 mo) | – | – |
Abbreviations: ACTH, adrenocorticotropic hormone; DQ, Developmental quotient; VABS, Vineland Adaptive Behavior Scales; VGB, vigabatrin.