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TOPLINE:

Pediatric patients receiving a combination of rituximab and cyclophosphamide therapy for rheumatic disease showed clinical improvement with a low risk for adverse effects.

METHODOLOGY:

• While current clinical practice guidelines recommend the use of either rituximab or cyclophosphamide for the treatment of severe manifestations of systemic vasculitis or connective tissue disease, few studies have examined the safety and efficacy of combination therapy with the two.
• The study involved 89 pediatric patients, aged 12-15 years, who received combination rituximab and cyclophosphamide therapy for a rheumatic disease at a single center between January 2020 and February 2023.
• The patients included 77.5% girls, and two thirds had systemic lupus erythematosus (66%), followed by any of anti-neutrophil cytoplasmic antibody–associated vasculitis, Sjögren disease, and 13 other conditions.
• The primary outcomes were death and infection requiring hospitalization within 12 months.
• Secondary outcomes included changes in serologic lupus disease activity markers and corticosteroid dose, disease flare, infusion reactions, and incident hypogammaglobulinemia.

TAKEAWAY:

• A total of 8.9% of patients were hospitalized for infection.
• The average daily corticosteroid dose significantly decreased during follow-up (P < .001), with 62% of patients able to discontinue steroid therapy.
• Patients improved across all serologic disease activity markers (P < .0001).
• Disease flare occurred in 6.7% of patients. Another 12% had infusion reactions, and 31% had incident hypogammaglobulinemia.

IN PRACTICE:

“Combination rituximab and cyclophosphamide can be safely administered to children with severe, life-threatening rheumatic diseases,” the authors wrote. “This regimen allows for effective treatment of rheumatic disease and successful reduction of corticosteroid exposure.”

SOURCE:

This research was led by Eileen Rife, MD, MPH, a pediatric rheumatologist and fellow at the University of Alabama at Birmingham, Birmingham, Alabama, and presented at the CARRA 2025: Childhood Arthritis and Rheumatology Research Alliance Annual Scientific Meeting.

LIMITATIONS:

This study involved a small population at a single center.

DISCLOSURES:

The authors did not report any disclosures. This research was supported by the National Institute of Arthritis and Musculoskeletal and Skin Diseases and the Arthritis Foundation and used data collected in the CARRA Registry.