Cost Utility of Lifelong Immunoglobulin Replacement Therapy vs Hematopoietic Stem Cell Transplant to Treat Agammaglobulinemia
Authors:
Open Access
- 15 November 2021
- journal article
- research article
- Published by American Medical Association (AMA) in JAMA Pediatrics
- Vol. 176 (2) , 176-184
Abstract
Congenital agammaglobulinemia comprises a group of primary antibody deficiencies that share early-stage B cell developmental arrest, absent peripheral B cells, and critically low serum immunoglobulin concentrations.1 X-linked agammaglobulinemia, caused by mutation of the sex-linked gene BTK, accounts for 85% of congenital agammaglobulinemia.2 The other 15% is associated with defects in genes essential to B-cell development, such as biallelic variations of IGHM, IGLL1, CD79A, CD79B, BLNK, and PI3KR1, and heterozygous mutations of LRRC8A, TCF3, and SPI1.1,3-7Keywords
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