Using the model developed by CHOP, researchers found that the high cost of treatment most likely to prevent premature death in patients with agammaglobulinemia diminished its benefits

Philadelphia Children's Hospital

Philadelphia, November 15, 2021-Patients with rare immunodeficiency diseases treated with lifetime immunoglobulin replacement therapy (IRT) have a lower risk of premature death than patients treated with hematopoietic stem cell transplantation (HSCT), but their premature death The risk of death is also reduced. According to a new study led by the Children's Hospital of Philadelphia (CHOP), the quality of life must bear a huge financial burden. Researchers used computational models to calculate the costs and benefits of IRT and HSCT for patients with agammaglobulinemia, and concluded that the high cost of IRT in the United States weakened its cost utility, especially when compared with Canada and European countries. The cost of IRT in Canada and European countries is almost one-third of that in the United States

"The model we developed allows us to answer questions about cost-effectiveness and outcomes. These patient populations are often too small to power the robust clinical trials needed to answer these questions," lead author, MD, Public Health Master, Doctor of Medicine, Master of Public Health said. Doctor of CHOP Department of Allergy and Immunology. "Given our modeling results, we believe that IRT should still be used to treat gammaglobulinemia patients in the United States, but regulatory efforts should focus on reducing its price."

Congenital agammaglobulinemia includes a group of primary antibody deficiencies characterized by a lack or complete lack of B cells, leading to recurrent infections and potentially life-threatening complications. In high-income countries such as the United States, the main treatment is lifetime IRT, intravenous infusion (IVIG) once a month, or subcutaneous infusion (SCIG) once a week or every two weeks. Although this therapy can effectively reduce many infections, it cannot completely prevent all infections, and the requirement for regular infusions has reduced the quality of life of many patients. IRT also brings a huge financial burden, spending 30,000 to 90,000 US dollars per year in the United States

Hematopoietic stem cell transplantation is another way to treat gammaglobulinemia, but finding a matching donor can be challenging, and surgery itself has its own risks. However, so far, no one has compared the health risks, outcomes, and costs of HSCT and IRT.

To this end, the researchers used the basic situation of a one-year-old child with congenital agammaglobulinemia in the United States who received a lifetime IRT, a matched sibling donor HSCT, or a matched unrelated donor HSCT to establish a computational model. Using cost, probability, and quality of life indicators derived from the literature, the researchers used their model to simulate more than 10,000 patients over a 100-year period. 

Using this method, the researchers found that lifetime IRT reduced premature deaths by 37% compared with HSCT, but due to its high cost, it is not cost-effective compared with HSCT. The life extension caused by IRT has not been accompanied by a corresponding improvement in the quality of life. The researchers determined that if the annual price of IRT was reduced by 51%, it would become a more cost-effective strategy.

"As a treatment method, IRT is a particularly good option for elderly patients and those who do not have a suitable source of donors, but our research shows that the high cost of patients in the United States prevents it from becoming cost-effective here. Selection," Neal D. Romberg, MD, attending physician in the Department of Allergy and Immunology at CHOP, and the senior author of the paper. "Before other treatment options are available, such as gene-based therapies, our findings indicate that reducing the cost of IRT in the United States is an immediately actionable intervention that will make IRT a more cost-effective intervention compared to HSCT. Strategy."

Sun D. et al. "The cost-effectiveness of lifelong immunoglobulin replacement therapy and hematopoietic stem cell transplantation in the treatment of agammaglobulinemia", JAMA Pediatrics, published online on November 15, 2021, DOI: 10.1001/jamapediatrics.2021.4583

About the Children's Hospital of Philadelphia: Founded in 1855, the Children's Hospital of Philadelphia was the first pediatric hospital in the United States. Through a long-term commitment to providing excellent patient care, training a new generation of pediatric healthcare professionals, and groundbreaking major research projects, Children's Hospital has made many discoveries that benefit children around the world. Its pediatric research project is one of the largest in the country. In addition, its unique family-centered care and public service programs make the 595-bed hospital a major advocate for children and adolescents. For more information, please visit http://www.chop.edu

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Camillia Travia Philadelphia Children's Hospital traviac@chop.edu

Philadelphia Children's Hospital

Copyright © 2021 American Association for the Advancement of Science (AAAS)

Copyright © 2021 American Association for the Advancement of Science (AAAS)