Syndromic autism

Syndromic autism (or syndromic autism spectrum disorder) denotes cases of autism that are associated with a broader medical condition, generally a syndrome. Cases without such association, which account for the majority of total autism cases, are known as non-syndromic autism (or non-syndromic autism spectrum disorder).

Studying the differences and similarities (e.g., common pathways) between syndromic and non-syndromic cases can provide insights about the pathophysiology of autism and pave the way to new autism therapies.

Syndromic autism represents about 25% of the total ASD cases. In most cases, its etiology is known. Monogenic disorders are one of the causes of syndromic autism, which in this case are also known as monogenic autism spectrum disorders. They account for about 5% of the total ASD cases.[citation needed] SCN2A is the leading monogenic cause of autism.

Classification

A 2017 study proposed to replace the classification syndromic/non-syndromic ASD into one based on the genetic etiology of the condition, specifying if the syndromic condition occurs in the context of a "phenotype first" clinically defined syndrome or from a "genotype first" molecularly defined syndrome.[clarification needed]

Following the proposal, ASD would be divided into genetic categories, including:

Clinically defined

Syndromes recognized by clinicians (depending on their experience), typically confirmed by a targeted genetic testing.

  • Chromosomal (e.g., Down syndrome)
  • Syndromes caused by mutations in single genes (e.g., SCN2A-related autism, NF1 mutation, tuberous sclerosis, PTEN-associated macrocephaly syndrome, some males with fragile X syndrome)
  • Syndromes caused by CNVs (e.g., DiGeorge syndrome)
  • Teratogens (e.g., fetal valproate spectrum disorder)

Molecularly defined

Syndromes recognized by genome-wide testing, not by hypothesis-driven testing (since clinical recognition is difficult).

  • Chromosomal (e.g., isodicentric 15q)
  • Autism-associated genes (e.g., ADNP, ARIDB1B, ANK2, SCN2A, KCNH1)
  • Autism-associated CNVs (e.g., 16p11.2 deletion/duplication, exonic NRXN1 deletions)
Characteristics of syndromic ASD conditions
Condition Cause Chromosome(s) involved (if a mutation) ASD prevalence (95% CI) Clinically/Molecularly defined Other characteristics Ref.
Fragile X syndrome Monogenic disorder:
FMR1 (encodes FMRP)
X  30% (20.0–31.0) [male individuals only]
 22% (15.0–30.0) [mixed sex]
14% (13–18) [female individuals only]
Clinically defined [in some males] Long/narrow face, macroorchidism, long ears and philtrum, hyperactivity, mild to moderate intellectual disability (ID), seizures
Rett syndrome Monogenic disorder:
MECP2
X 61.0% (46.0–74.0) [female individuals only] Clinically defined Microcephaly, breathing irregularities, language deficits, repetitive/stereotyped hand movements, epilepsy, ID
MECP2 duplication syndrome Monogenic disorder:
MECP2
X 100% [in a single study composed by 9 male participants] Clinically defined Brachycephaly, spasticity, recurrent respiratory infections, gastrointestinal hypermotility, genitourinary abnormalities, epilepsy, ID
Tuberous sclerosis complex Monogenic disorder:
TSC1
TSC2
9
16
 36.0% (33.0–40.0) Clinically defined Benign tumours in multiple organs, epilepsy
Angelman's syndrome Monogenic disorder:
UBE3A
15  34.0% (24.0–37.0) Cheerful demeanour, microcephaly, speech deficits, sleep disturbance, epilepsy, ID
Phelan-McDermid syndrome Monogenic disorder:
SHANK3
22  84% [in a single study composed by 32 participants] Molecularly defined
KCNH1-related disorders Monogenic disorder: KCNH1 1 Molecularly defined, formerly clinically defined as Temple–Baraitser Syndrome or Zimmermann–Laband Syndrome Mild to severe developmental delay, profound intellectual disability, neonatal hypotonia, myopathic facial appearance, and infantile-onset seizures
Timothy syndrome Monogenic disorder:
CACNA1C
12  80% [in a single study composed by 17 participants] Clinically defined
Smith-Lemli-Opitz syndrome Monogenic disorder:
DHCR7
11 55% [in a single study composed by 33 participants]
Neurofibromatosis type I Monogenic disorder:
NF1
17  18% (9.0–29.0) Clinically defined
PTEN hamartoma tumor syndrome Monogenic disorder:
PTEN
10  17% (8–27) Clinically defined
Down syndrome Chromosomal disorder:
trisomy 21
21 16% (8.0–24.0) Clinically defined
Cohen's syndrome Monogenic disorder:
VPS13B
8  54% (44.0–64.0) Clinically defined
Cornelia de Lange syndrome Polygenic disorder  43% (32.0–53.0) Clinically defined
CHARGE syndrome Monogenic disorder:
CHD7
8  28% (16–41) Clinically defined
Noonan's syndrome Polygenic disorder  15% (7.0–26.0)
Williams syndrome Microdeletion syndrome:
7q11.23
7  12% (6.0–20.0)
22q11.2 deletion syndrome Microdeletion syndrome:
22q11.2
22 11% (5.0–19.0) Clinically defined
Fetal valproate spectrum disorder Teratogen:
valproate
 8–15% [in VPA exposed children] Clinically defined

See also

  • Causes of autism
  • Conditions comorbid to autism

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