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Management
Section 12. Immune Reconstitution Therapy for Inborn Errors
of Immunity
Recommendation 12.1
➤ We suggest that allogeneic hematopoietic stem cell transplant (HSCT)
for patients with IEI be performed at a center with experience in HSCT
for IEI. (C-M)
Recommendation 12.2
➤ We recommend that patients with typical SCID or leaky/atypical SCID
receive definitive therapy with allogeneic HSCT or gene therapy. (S-H)
Recommendation 12.3
➤ We recommend that patients with congenital athymia disorders be
treated with cultured thymus tissue implantation (CTTI). (S-M)
Recommendation 12.4
➤ We recommend that patients with CID disorders who have severe
cellular immune defects or who manifest severe or refractory disease
complications be considered for allogeneic HSCT. (S-M)
Recommendation 12.5
➤ We recommend that patients with primary HLH disorders and patients
with X-linked lymphoproliferative disease type 1 be evaluated for
allogeneic HSCT. (S-M)
Recommendation 12.6
➤ We suggest that patients with immune dysregulation who manifest
severe or refractory disease complications be evaluated for allogeneic
HSCT. (C-M)
Recommendation 12.7
➤ We recommend HSCT for patients with defects in neutrophil number or
function associated with severe clinical phenotypes. (S-H)
