CD19 CAR-T cell therapy in a pediatric patient with MDA5+ dermatomyositis and rapidly progressive interstitial lung disease by Andre´ s Parı´s-Mun˜ oz,Rosa M. Alcobendas-Rueda,Cristina Verdu´-Sa´ nchez, ...,Gonzalo Ruiz-Zurita,Miguel A. Ferna´ ndez-Garcı´a,´rez-Martı´nezAntonio Pe ISBN 101016/JMEDJ2025100676 instant download

CD19 CAR-T cell therapy in a pediatric patient with MDA5+ dermatomyositis and rapidly progressive interstitial lung disease by Andre´ s Parı´s-Mun˜ oz,Rosa M. Alcobendas-Rueda,Cristina Verdu´-Sa´ nchez, ...,Gonzalo Ruiz-Zurita,Miguel A. Ferna´ ndez-Garcı´a,´rez-Martı´nezAntonio Pe ISBN 101016/JMEDJ2025100676 instant download

CONTEXT AND SIGNIFICANCE MDA5+ dermatomyositis with interstitial lung disease (MDA5+DM-RPILD) is arare autoimmune disease that lacks a well-defined treatment. Because B cells are key players in autoimmunity, CD19 CAR-T cell therapies have been proposed as a strategy to deeply deplete this cell population. Inthis report, researchers from Hospital Universitario La Paz describe the use of CD19 CAR-T cell therapy in apediatric patient with MDA5+DM-RPILD after a complex immunosuppressive regimen. For 11 months in animmunosuppressant-free context, the subject progressively exhibited motor and lung improvements andrestoration of the B cell compartment in the absence of autoimmune episodes. This work joins the list of autoimmune cases successfully treated with CAR-T cell therapies and demonstrates their efficacy and safetyeven in dire clinical conditions.SUMMARYBackground: Anti-melanoma differentiation-associated protein 5 dermatomyositis (MDA5+DM) is a potentially fatal subtype of dermatomyositis. The most severe cases are characterized by rapidly progressiveinterstitial lung disease (RPILD), the leading cause of death in these patients. There is currently no curativ