TABLE 2.
Clinical Characteristics and Treatment Response in 41 Individuals With SAS
| SATB2 ID# | Genomic Alteration | EEG | Clinical Seizures | Age at Seizure Onset, Years | Epilepsy | Brain MRI | Current Medications | Other Prior Medications | Seizure Outcome |
|---|---|---|---|---|---|---|---|---|---|
| 168 | 17.8Mb deletion at 2q32.1q33.1 | ED | No | N/A | None | N/A | None | None | No clinical seizures |
| 30 | 15Mb deletion at 2q33.1q34 | SB | Possible | 1 | ? Generalized tonic-clonic | Abnormal CC, small pons | None | LEV | Well controlled |
| 19 | 8.4Mb deletion at 2q32.3q33.2 | ED | Yes | 3 | Focal epilepsy | N/A | None | None | Well controlled |
| 77 | 8.1Mb deletion at 2q33.1q33.3 | ED, SB | Yes | NVA | Focal epilepsy | Normal | OXC | LEV | Well controlled |
| 12 | 3.6Mb deletion at 2q33.1 | ED | Possible | 7 | ? Focal epilepsy (laughing fits with disorientation) | At, HM, T2H | LTG, OXC | CBD | Somewhat controlled |
| 11 | 1.77Mb deletion at 2q33.1 | ED | Possible | 6 | ? Focal epilepsy | HM | None | None | Somewhat controlled |
| 143 | Intragenic deletion exons 5–11 | ED | Yes | 2 | Mixed epilepsy (benign rolandic epilepsy, atonic, tonic-clonic) | Normal | GBP, TPM | LEV, OXC | Somewhat controlled |
| 80 | Intragenic deletion exons 4–8 | Excess BA | No | N/A | None | N/A | None | None | No clinical seizures |
| 2 | Intragenic deletion exons 4–8 | ED | Yes | 4 | Focal epilepsy | N/A | OXC | LEV, ESC | Somewhat controlled |
| 92 | Intragenic deletion exon 7 | ED | Yes | 2 | Febrile seizures and generalized tonic-clonic epilepsy | HM, T2H | None | LEV, VPA, LTG | Well controlled |
| 78 | Intragenic deletion exon 4 | ED | Possible | 5 | ? Focal epilepsy (staring episodes) | Abnormal CC, At, HM, large Ven | OXC | - | Well controlled |
| 159 | c.163delG (p.Val55Trpfs*4) | ED, SB | No | N/A | None | N/A | None | None | No clinical seizures |
| 136 | c.257T>G (p.Leu86Arg) | ED, SB | No | N/A | Nonepileptic events | N/A | CBD | - | No clinical seizures |
| 39 | c.346G>T (p.Gly116*) | ED | No | N/A | Nonepileptic events | HM | CLZ | - | No clinical seizures |
| 11184 | c.580G>T (p.Glu194*) | ED | No | N/A | None | Normal | None | None | No clinical seizures |
| 16 | c.583dupT (p.Cys195Leufs*14) | ED, SB | Possible | NVA | ? Focal epilepsy (staring episodes) | T2H | None | None | |
| 114 | c.715C>T (p.Arg239*) | ED | No | N/A | None | HM, T2H | CBD | LEV, ESC, LCM | No clinical seizures |
| 121 | c.715C>T (p.Arg239*) | ED, ESES like | No | N/A | Nonepileptic events | SC | VPA, LTG | - | No clinical seizures |
| 68 | c.760C>T (p.His254Tyr) | ED | Yes | 0.3 | Mixed epilepsy (infantile spasms, intractable focal epilepsy, generalized convulsive epilepsy, epilepsy with continuous spikewave during slowwave sleep, Lennox-Gastaut syndrome) | Abnormal CC, At, HM | CLB, ZNS, BRI, VNS | VGB, LEV, CS, KD | Well controlled |
| 46 | c.1131_1132delGT (p.Ser378Profs*18) | ED, SB | Yes | 5 | Mixed epilepsy (both focal and generalized epilepsy : benign rolandic epilepsy, generalized tonic-clonic, absence, atonic | Normal | CLB, LTG | - | Well controlled |
| 41 | c.1165C>T (p.Arg389Cys) | ED | Yes | 6 | Focal epilepsy | T2H | LTG | LEV | Well controlled |
| 153 | c.1165C>T (p.Arg389Cys) | ED | Yes | 3 | Mixed epilepsy (focal epilepsy of childhood, absence) | N/A | VPA | - | Somewhat controlled |
| 34 | c.1174–2A>G | ED, ESES like, SB | Yes | 2 | Focal epilepsy (staring episodes) | N/A | None | CLB | Somewhat controlled |
| 112 | c.1175G>A (p.Gly392Glu) | ED, ESES, SB | Yes | 2 | Focal epilepsy | Normal | LEV, GBP, CLB | - | Somewhat controlled |
| 53 | c.1196G>A (p.Arg399His) | ED | No | N/A | None | Abnormal CC, HM | OXC | LEV | No clinical seizures |
| 94 | c. 1196G>A (p.Arg399His) | ED | No | N/A | Non epileptic events | Normal | None | VPA, ESM, PHT | No clinical seizures |
| 134 | c.1196G>A (p.Arg399His) | ED | Yes | 2 | Generalized epilepsy (absence) | N/A | OXC | - | Well controlled |
| 98 | c.1196delG (p.Arg399Leufs*14) | ED | Yes | 5 | Mixed epilepsy (intractable atonic seizures and benign rolandic epilepsy) | N/A | VPA | - | Well controlled |
| 29 | c.1285C>T (p.Arg429*) | ED | No | N/A | Nonepileptic events | Normal | LEV, B6 | - | No clinical seizures |
| 14 | c.1286G>A (p.Arg429Gln) | SB | Possible | 5 | ? Focal epilepsy (one tonic-clonic episode; staring episodes) | T2H | VPA | - | Somewhat controlled |
| 23 | c.1286G>A (p.Arg429Gln) | ED | Possible | 4 | ? Focal epilepsy (Staring episodes) | T2H | None | Keppra | Somewhat controlled |
| 70 | c.1286G>A (p.Arg429Gln) | ED | No | N/A | None | Normal | None | None | No clinical seizures |
| 105 | c.1286G>A (p.Arg429Gln) | ED | Possible | 7 | ? Focal epilepsy (single convulsive episode, staring spells) | HM | None | None | Well controlled |
| 164 | c.1307A>T (p.Glu436Val) | ED, SB | Yes | 5 | Mixed epilepsy (focal, generalized atonic) | Abnormal CC, HM, large Ven | ZNS | - | Somewhat controlled |
| 124 | c.1375C>T (p.Arg459*) | ED | No | N/A | None | Normal | None | None | No clinical seizures |
| 1093 | c.1425_1426dupGG (p.Val476Glyfs*21) | ED | No | N/A | None | UF asymmetry, T2H | None | None | No clinical seizures |
| 38 | c.1495A>T (p.Lys499*) | ED | Possible | 7 | ? Focal epilepsy (mouth twitching) | Large Ven, T2H | None | None | |
| 109 | c.1554T>G (p.Cys518Trp) | ED, SB | Yes | 2 | Focal versus generalized epilepsy (atypical absence) | Normal | VPA, LTG | LEV, TPM | Somewhat controlled |
| 75 | c.1592dupA (p.Asn531Lysfs*21) | ED | Yes | 4.5 | Mixed epilepsy (both focal and intractable generalized nonconvulsive epilepsy, generalized convulsive epilepsy) | HM | None | VPA, LEV, B6, KD | Well controlled |
| 7 | c.1728delT (p.Glu577Serfs*47) | ED | No | N/A | Nonepileptic events | HM, T2H | CBZ | LEV, CBZ | No clinical seizures |
| 116 | c.1903G>T (p.Asp635Tyr) | ED, ESES | Yes | 3 | Focal epilepsy | HM | CLB, OXC | - | Well controlled |
Abbreviations:
At = Atrophy
B6 = vitamin B6;
BA = Beta activity
BRI = Brivaracetam
CBD = Cannabidiol oil
CBZ = Carbamazepine
CC = Corpus callosum
CLB = Clobazam
CLZ = Clonazepam
CS = Corticosteroids
ED = Epileptiform discharges
EEG = Electroencephalography
ESC = Eslicarbazepine
ESES = Electrical status epilepticus in sleep
ESM = Ethosuximide
GBP = Gabapentin
HM = Hypomyelination
KD = Ketogenic diet
LCM = Lacosamide
LEV = Levetiracetam
LTG = Lamotrigine
MRI = Magnetic resonance imaging
N/A = Not applicable
NAV = Not available
OXC = Oxcarbazepine
PHT = Phenytoin
SAS = SATB2-associated syndrome
SB = Slow background
T2H = T2 hyperintensities
TPM = Topiramate
UF = Uncinated fascicle
Ven = Ventricles
VGB = Vigabatrin
VNS = Vagus nerve stimulation
VPA = Valproate
ZNS = Zonisamide