Table 7.

Spectrum of inborn errors of immunity treated with medical therapy or hematopoietic cell therapies ([43] adapted from)

Treatment for inborn errors of immunity from medical management to hematopoietic cell therapy
Medical therapies: IgG replacement Antibiotics Biologic therapies Immune modulators Enzyme replacement Small molecule inhibitors	Exclusively medical	Mostly medical	“In between”	Mostly HCT	Exclusively HCT	Hematopoietic cell therapies: allogeneic HCT Gene therapy
	XLA IgAD APECED Complement deficiencies DiGeorge Thymic defects FMF CAPS DIRA	ALPS STAT3 LOF CVID (no genetic dx) Interferonopathies ADA2 deficiency MSMD Partial LAD-1 CARD 11 DN NLRC4 GOF PLCG2	LRBA deficiency CTLA4 haploinsufficiency PI3K GOF disorders STAT1 GOF STAT3 GOF	CD40/CD40L CGD GATA2 C1Q deficiency NEMO XIAP Deficiency DOCK8 deficiency CARMIL2 deficiency STAT1 LOF	SCID WAS IPEX CD25 deficiency XLP IL-10/IL-10R deficiency Primary HLH Severe LAD-1

ADA2 Adenosine deaminase 2, ALPS Autoimmune lymphoproliferative syndrome, APECED Autoimmune polyendocrinopathy candidiasis ectodermal dystrophy, CAPS Cryopyrin-associated periodic syndrome, CARD11 DN Caspase recruitment domain family, member 11 dominant negative, CARMIL2 Capping protein regulator and myosin linker 2, CGD Chronic granulomatous disease, CTLA-4 Cytotoxic T-lymphocyte associated protein 4, CVID Common variable immune deficiency, DGS DiGeorge syndrome, DIRA Deficiency of interleukin-1 receptor antagonist, DOCK8 Dedicator of cytokinesis 8, FMF Familial Mediterranean fever syndrome, GATA2 GATA-binding factor 2, HLH Hemophagocytic lymphohistiocytosis, IgAD IgA deficiency, IPEX Immunodysregulation, polyendocrinopathy, enteropathy X-linked, LRBA Lipopolysaccharide-responsive and beige-like anchor protein, MSMD Mendelian susceptibility to mycobacterial diseases, NEMO NF-kappa-B essential modulator, NLRC4 NLR family CARD domain-containing 4, LAD-1 Leukocyte adhesion deficiency-1, PI3K Phosphatidylinositol-3-kinase, SCID Severe combined immune deficiency, STAT1 Signal transducer and activator of transcription 1, STAT3 Signal transducer and activator of transcription 3, WAS Wiskott Aldrich syndrome, XIAP X-linked inhibitor of apoptosis protein, XLA X-linked agammaglobulinemia, XLP X-linked lymphoproliferative disease