Patients with congenital agammaglobulinemia — a rare immunodeficiency disorder — who are treated with immunoglobulin replacement therapy have a lower risk of premature death than those treated with hematopoietic stem cell transplant, but take on a major financial burden, a Children's Hospital of Philadelphia study found.
The hospital's study used a computational model with a base-case scenario of a 1-year-old child with the disease who receives either lifelong IRT, matched sibling donor HSCT or matched unrelated donor HSCT, a Nov. 15 news release said.
IRT costs patients between $30,000 and $90,000 a year in the U.S., nearly three times the amount it costs in Canada and Europe, according to the study. While it decreased premature deaths by 37 percent compared to HSCT, it did not improve quality of life because of the cost.
"As a treatment, IRT is an especially good option for older patients and those without suitable donor sources, but our study shows the exorbitant cost for patients in the United States prevents it from being a cost-effective option here," said Neil Romberg, MD, an attending physician with the Division of Allergy and Immunology at the hospital and senior author of the paper. "Until additional treatment options become available, such as gene-based therapies, our findings suggest that reducing IRT cost in the U.S. is an immediately actionable intervention that would make IRT the more cost-effective strategy compared with HSCT."