Stem-Cell Therapy Restores Lung Health in Severe Arthritis
Research By: Grant Schulert, MD, PhD | Rebecca Marsh, MD
Post Date: April 2, 2025 | Publish Date: April 2025
Rheumatology | Top Scientific Achievement
An international research team led by Grant Schulert, MD, PhD, and Rebecca Marsh, MD, at Cincinnati Children’s has found that allogeneic hematopoietic stem-cell transplantation (HSCT) can restore immune and lung function in children with treatment-refractory systemic juvenile idiopathic arthritis (sJIA) and associated lung disease (sJIA-LD).
The multicenter study—the first of its kind—included 13 children across the United States and Europe who had not responded to an average of six different medications. Results were published in April 2025 in The Lancet Rheumatology.
After HSCT, all nine surviving patients achieved complete remission: they no longer required oxygen, corticosteroids, or biologic therapies. Many also showed improved lung imaging and pulmonary function test results. However, the procedure was not without risk—four children died, primarily from infection-related complications such as cytomegalovirus pneumonitis.
“These findings show that stem-cell transplantation can completely reset the immune system and offer a path to remission for children who otherwise face progressive lung failure,” says Schulert. “While the risks are significant, the outcomes highlight the need to consider HSCT earlier for children with worsening oxygen dependence or severe therapy-related toxicity.”
The research highlights the immune-driven nature of sJIA-LD and supports the use of HSCT as a potentially curative option. Investigators now aim to identify which patients could benefit most and determine optimal timing for referral to transplantation, before severe lung damage develops.
Longer-term follow-up studies will be conducted to track pulmonary and immune recovery, refine transplant protocols, and reduce transplant-related complications.
About the Study
Cincinnati Children’s co-authors also included Michael Matt, MD; Daniel Drozdov, MD, MSc; Sharat Chandra, MD; Christopher Towe, MD; Andrew Schapiro, MD; and Alexei Grom, MD. Co-authors also included experts with Nationwide Children’s Hospital, Memorial Sloan Kettering Cancer Center, University Children’s Hospital Zurich, and other centers across the U.S. and Europe.
This study was supported by the National Institute of Arthritis and Musculoskeletal and Skin Diseases, National Institutes of Health (R01-AR079525), and The Marcus Foundation.
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| Original title: | Allogeneic haematopoietic stem-cell transplantation for children with refractory systemic juvenile idiopathic arthritis and associated lung disease: outcomes from an international, retrospective cohort study |
| Published in: | The Lancet Rheumatology |
| Publish date: | April 2025 |
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