Table 2.

Genetic diagnoses and clinical reclassification before and after genetic testing

DiseaseGeneTotal, nPre-genetic testing classification (n = ID, AID, non-ID/AID)IUIS 2024 group
Cryopyrin-associated periodic syndrome NLRP3 4 0, 4, 0 Autoinflammatory diseases 
CTLA4 haploinsufficiency CTLA4 2 1, 0, 1 Diseases of immune dysregulation 
NFKB1 deficiency NFKB1 1 1, 0, 0 Predominantly antibody deficiencies 
NFKB2 deficiency NFKB2 1 1, 0, 0 Predominantly antibody deficiencies 
VEXAS syndrome UBA1 2 0, 0, 2 Somatic-associated autoinflammatory diseases 
Blau syndrome NOD2 1 0, 0, 1 Autoinflammatory diseases 
Perforin deficiency PRF1 1 1, 0, 0 Diseases of immune dysregulation 
GATA2 deficiency GATA2 1 1, 0, 0 Combined immune deficiency 
ROSAH syndrome ALPK1 1 0, 1, 0 Autoinflammatory diseases 
ICOS deficiency ICOS 1 1, 0, 0 Predominantly antibody deficiencies 

This table summarizes the genetic diagnoses identified in the cohort, including associated genes, IUIS disease categories, and corresponding clinical classification before and after genetic testing. The pre-genetic classification was defined prior to genetic testing based on the dominant phenotype: ID, AID, or non-ID/AID. The post-genetic classification reflects the final clinical categorization reassigned after integrating genetic findings and the corresponding IUIS disease group. ROSAH, retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis, headache.

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