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17-01-2025 | Juvenile Arthritis | Editor's Choice | News

Allogeneic HSCT ‘offers real hope’ for treating lung disease in refractory juvenile arthritis

Author: Sara Freeman

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medwireNews: Allogeneic hematopoietic stem cell transplantation (HSCT) may be a “valuable treatment option” for lung disease (LD) in some patients with treatment-refractory systemic juvenile idiopathic arthritis (sJIA), according to a team of international researchers.

“With a lack of available and effective medical therapies for the treatment of sJIA-LD, allogeneic HSCT should be considered for children with worsening pulmonary function or severe treatment-related morbidity,” say Grant Schulert of Cincinnati Children’s Hospital Medical Center in Ohio, USA, and co-authors in The Lancet Rheumatology.

“Our findings suggest that allogeneic HSCT in these children can induce remission of the inflammatory features of sJIA and lead to improvements in lung function.”

The study included 10 girls and three boys with sJIA-LD who had received allogeneic HSCT at one of nine hospitals in the USA and Europe. At diagnosis, the median age of the study participants was 4.8 years, and the median age at transplantation was 9.0 years.

After a median follow-up of 16 months, nine (69%) of the 13 patients were alive and all had experienced a complete response to treatment, defined as having no active signs of sJIA, such as fever or arthritis, no use of systemic immunomodulation therapy, and the discontinuation of supplemental oxygen therapy if used. The researchers note that five of the patients required supplemental oxygen before receiving allogeneic HSCT.

Schulert et al note that among patients with pre- and post-transplantation pulmonary function results, there were “some improvements” in forced vital capacity and forced expiratory volume in 1 second but these were not statistically significant. Three of the four patients with pre- and post-transplantation diffusing capacity for carbon monoxide findings showed improvements, as did four of the six patients with digital clubbing.

Three of the six patients who underwent post-transplantation chest computed tomography had “notable improvement” in sJIA-LD and two had stable disease, but one patient showed worsening underlying LD and died.

Pierre Quartier (Université Paris-Cité, France) comments in a related editorial that “[s]ince sJIA-LD in non-transplanted patients is often fatal, it is encouraging that the rates of remission and pulmonary scans improved after allogenic HSCT.”

He continues that the “high survival rate illustrates the progress made in the field of HSCT.”

However, four participants died – two from post-transplantation infection with cytomegalovirus (CMV) resulting in pneumonitis, one from progressive LD, and one from an intracranial hemorrhage during asymptomatic COVID-19 infection. These patients “were at high risk of post-HSCT complication and death due to progression of lung disease, severe or opportunistic infections in the context of long-lasting immunosuppressive therapy, and inflammatory alloimmune complications,” the commentator writes.

Post-transplantation complications included acute graft-versus-host disease in five participants, bacteremia in eight participants, CMV reactivation in six participants, and macrophage activation syndrome in three participants.

Quartier suggests that research now needs to focus on how to identify patients who could benefit from HSCT sooner, avoiding other treatments that “are less effective and possibly  deleterious." Targeted bridging therapy before HSCT and prophylactic antiviral treatment to prevent CMV infection may also be of benefit, he observes.

Quartier concludes: “Allogeneic HSCT is a high-risk treatment for immunocompromised patients. However, there are some ways to improve patient’s prognosis, and this pioneer study offers real hope.”

medwireNews is an independent medical news service provided by Springer Healthcare Ltd. © 2025 Springer Healthcare Ltd, part of the Springer Nature Group

Lancet Rheumatol 2025; doi:10.1016/S2665-9913(24)00275-3
Lancet Rheumatol 2025; doi:10.1016/S2665-9913(24)00299-6

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