Sun D, Heimall JR, Greenhawt MJ, et al. - Immunoglobulin replacement therapy (IRT) is more costly than hematopoietic stem cell transplant (HSCT) for agammaglobulinemia treatment in the US. IRT averted more premature deaths but failed to substantially increase quality of life vs HSCT. IRT may become a more cost-effective strategy if US IRT cost is decreased by 51% to a value similar to IRT costs in countries implementing value-based pricing.

• This is an economic analysis with Markov analysis to model the base-case scenario of a patient aged 12 months with congenital agammaglobulinemia undergoing lifelong IRT vs matched sibling donor (MSD) or matched unrelated donor (MUD) HSCT.

• Among patients with congenital agammaglobulinemia, lifelong IRT was more expensive than HSCT ($1,512,946 vs $563,776 [MSD] and $637,036 [MUD]) and provided similar quality-adjusted life-years (20.61 vs 17.25 [MSD] and 17.18 [MUD]).

• Lifelong IRT was not cost-effective than HSCT but reduced premature deaths 37% relative to HSCT without a commensurate increase in quality of life.

• Reduction in IRT annual price from $60 ,145 to less than $29, 469 may render it the more cost-effective strategy.