General recommendations on treatment of primary immunodeficiency disorders

Table 2.5-1. General recommendations on treatment of primary immunodeficiency disorders
Type of immunodeficiency	IVIG/SCIG	Allogeneic HSCT	Other treatments	Approximate prevalence (selected examples)
Immunodeficiency affecting predominantly antibodies	+	– (+ for selected cases of CVID)	– Avoidance of live vaccines (except SIGAD, IGGSD, THI) – Antibiotic prophylaxis: All immunodeficiencies of this type if infections continue despite IVIG – Immunomodulators: CVID – Pneumococcal vaccination: SIGAD, IGGSD, SAD	SIGAD, 1:200 CVID, 1:20,000 XLA, 1:200,000
Combined immunodeficiency (including well-defined immunodeficiency syndromes)	+	+	– Avoidance of live vaccines: All immunodeficiencies of this type – Avoidance of nonirradiated blood or products: All immunodeficiencies of this type – Avoidance of CMV-positive blood or cells: All immunodeficiencies of this type – Antibiotic prophylaxis: All immunodeficiencies of this type – Antifungal prophylaxis: SCID, HIGM, HIES, ICD4L – Splenectomy: WAS – Glucocorticoids: WAS – Thymus transplant: DGS – G-CSF, GM-CSF: ICD4L, HIGM – Gene therapy for IL2RG, ADA deficiency, WAS	SCID, 1:50,000 WAS, 1:100,000 DGS, 1:5000 HIES, 1:100,000 HIGM, 1:500,000 ICD4L, very rare
Immunodysregulation disorders	+	+	– Avoidance of live vaccines: All immunodeficiencies of this type – Antibiotic prophylaxis: All immunodeficiencies of this type – Chemotherapy (as in lymphoproliferative neoplasms): XLP, GS – Chemotherapy (associated with allogeneic HSCT): CHS	XLP, 1:1,000,000
Phagocyte defects	–	+	– Avoidance of live vaccines: All immunodeficiencies of this type – Antibiotic prophylaxis: All immunodeficiencies of this type – IFN-gamma: CGD, disorders of IL-12/IFN-gamma axis – Surgical or dental debridement: CGD, LAD – Antifungal prophylaxis: CGD, LAD – G-CSF: Neutropenia – Antituberculous agents: Disorders of IL-12/IFN-gamma axis	CGD, 1:200,000 LAD, very rare
Primary immunodeficiencies of the innate immune system	– (+ for WHIM)	+	– Avoidance of live vaccines: All immunodeficiencies of this type – Antibiotic prophylaxis: All immunodeficiencies of this type – G-CSF and GM-CSF: WHIM – Antifungal agents: CMCC – Antiviral agents: NK-cell deficiency	CMCC (diverse group of diseases), up to 1:10,000 WHIM, very rare
Complement deficiencies	–	–	– Antibiotic prophylaxis: All complement deficiencies – Pneumococcal vaccine: Deficiency of C1a, C1r, C2, C3, C4 – Meningococcal vaccine: Deficiency of C5, C6, C7, C8, C9 – Immunomodulators (IFN-gamma): Deficiency of C1a, C2, C4, factors H and I	C2 deficiency, 1:20,00 C4 deficiency, very rare C5-C9 deficiency, variable, up to 1:2000
Adapted from J Allergy Clin Immunol. 2015;136(5):1186-205.
ADA, adenine deaminase; CGD, chronic granulomatous disease; CHS, Chédiak-Higashi syndrome; CMCC, chronic mucocutaneous candidiasis; CMV, cytomegalovirus; CVID, common variable immunodeficiency; DGS, DiGeorge syndrome; G-CSF, granulocyte-colony stimulating factor; GM-CSF, granulocyte-macrophage colony stimulating factor; GS, Griscelli syndrome; HIES, hyper-IgE syndrome; HIGM, hyper-IgM syndrome; HSCT, hematopoietic stem cell transplant; ICD4L, idiopathic CD4 lymphocytopenia; IFN-gamma, interferon gamma; IGGSD, IgG subclass deficiency; IL, interleukin; IL2RG, IL2 receptor gamma; IVIG, intravenous immunoglobulin; LAD, leukocyte adhesion deficiency; SAD, specific antibody deficiency; SCID, severe combined immunodeficiency; SCIG, subcutaneous immunoglobulin; SIGAD, selective IgA deficiency; THI, transient hypogammaglobulinemia of infancy; WAS, Wiskott-Aldrich syndrome; WHIM, warts, hypogammaglobulinemia, immunodeficiency, and myelokathexis; XLA, X-linked agammaglobulinemia; XLP, X-linked lymphoproliferative syndrome.