Is Lifelong Immunoglobulin Replacement Therapy Cost-Effective to Treat Agammaglobulinemia?

The standard treatment for agammaglobulinemia does not appear cost-effective in the United States.

shafere1 [at] chop.edu (By Emily Shafer)

The Findings

Although it is the standard of care, lifelong immunoglobulin replacement therapy (IRT) for the treatment of congenital agammaglobulinemia is not necessarily cost-effective. The treatment prevents more deaths than a one-time hematopoietic stem cell transplant (HSCT), the alternative treatment option. However, IRT is significantly more expensive and does not substantially increase quality of life vs. HSCT. Researchers at Children’s Hospital of Philadelphia concluded that to be cost-effective, the cost of IRT would need to reduce by 51 percent in the United States, which would make the cost similar to that in countries that implement value-based pricing.

Why It Matters

Congenital agammaglobulinemia is a rare disease that causes immune deficiencies due to a low concentration of antibodies in the blood. The disease is characterized by a low number of or a complete lack of B cells, which leads to recurrent infections and potentially life-threatening complications. In the U.S. and other high-income countries, lifelong IRT is the mainstay treatment, and it is given either as a monthly intravenous infusion or a weekly or biweekly subcutaneous infusion. The therapy reduces the number of infections but does not completely prevent all infections. In addition, patients require regular infusions, which affects their quality of life. The financial burden is also significant: The treatment costs between $30,000 and $90,000 in the U.S.

One-time HSCT is also a possible treatment approach, but this comes with its own pitfalls, namely the difficulty in finding a matched donor, and the risks associated with the procedure itself. No one has previously compared the health risks, outcomes, and costs of HSCT and IRT.

Who Conducted the Study

Neil Romberg, MD, attending physician in the Division of Allergy and Immunology at CHOP was the senior author of the paper. The first author of the paper was Di Sun, MD, MPH, also an attending physician in the Division of Allergy and Immunology.

How They Did It

The researchers built a computer model of a base-case patient scenario of a 1-year-old patient with congenital agammaglobulinemia receiving lifelong IRT vs. HSCT. They conducted microsimulations to estimate premature deaths for each treatment strategy.

Quick Thoughts

“Although our study shows that the quality of life is similar between HSCT and IRT, there is a much higher risk of mortality with HSCT,” Dr. Sun said. “The best way to equalize these two treatment options is to reduce the price of IRT in line with what it costs in other developed countries. The other takeaway is that since there is not a financial reason to choose IRT in the United States, HSCT might be worth considering in selected agammaglobulinemia patients, especially if they have a matched-sibling donor.”

Dr. Romberg added: “Regardless of cost, if a family really values close to perfect years of life, even if it means a shorter life, transplant is more likely to offer that outcome. But if a family prioritizes a long lifespan, even if those years aren’t as high quality, then IRT is probably the better therapy.”

What’s Next?

“The other thing that is important to add is that congenital agammaglobulinemia is an extremely rare disease with clinically significant outcomes to consider across a patient’s lifespan,” Dr. Romberg said. “Because of this, the existing patient population is too small and geographically spread out to conduct large clinical trials over many years. Our model allows us to address important, long-term outcomes using a virtual patient cohort. With some modification, we think this approach can also help patients with different rare diseases, and their providers, make the right therapeutic decisions for them.”

Where the Study Was Published

The study appeared in JAMA Pediatrics.

Want to Learn More?

Check out the CHOP press release.