Cost utility of lifelong immunoglobulin replacement therapy vs hematopoietic stem cell transplant to treat agammaglobulinemia
D Sun, JR Heimall, MJ Greenhawt, NJ Bunin… - JAMA …, 2022 - jamanetwork.com
Importance Lifelong immunoglobulin replacement therapy (IRT) is standard-of-care
treatment for congenital agammaglobulinemia but accrues high annual costs ($30 000-$90
000 per year) and decrements to quality of life over patients' life spans. Hematopoietic stem
cell transplant (HSCT) offers an alternative 1-time therapy, but has high morbidity and
mortality. Objective To evaluate the cost utility of IRT vs matched sibling donor (MSD) and
matched unrelated donor (MUD) HSCT to treat patients with agammaglobulinemia in the …
treatment for congenital agammaglobulinemia but accrues high annual costs ($30 000-$90
000 per year) and decrements to quality of life over patients' life spans. Hematopoietic stem
cell transplant (HSCT) offers an alternative 1-time therapy, but has high morbidity and
mortality. Objective To evaluate the cost utility of IRT vs matched sibling donor (MSD) and
matched unrelated donor (MUD) HSCT to treat patients with agammaglobulinemia in the …