Bortezomib for refractory autoimmunity in pediatrics.

Area of Science:

• Immunology
• Hematology
• Pediatrics

Background:

• Refractory autoimmune diseases in children present significant therapeutic challenges.
• Standard treatments often prove insufficient for managing severe autoimmunity.
• Targeting plasma cells offers a potential strategy for autoimmune disease management.

Purpose of the Study:

• To evaluate the efficacy and safety of bortezomib in pediatric patients with refractory autoimmunity.
• To assess bortezomib's impact on plasma cells and associated cytopenias.
• To explore bortezomib as a salvage therapy for treatment-resistant autoimmune conditions.

Main Methods:

• A cohort of 7 pediatric patients with refractory autoimmunity received bortezomib (1.3 mg/m(2)) intravenously or subcutaneously every 72 hours.
• Patients received rituximab if B cells were present and plasmapheresis prior to bortezomib.
• Treatment was initiated a median of 120 days post-autoantibody confirmation, following failure of ≥2 standard therapies.

Main Results:

• Six out of seven patients achieved resolution of cytopenias.
• Two patients experienced recurrence of cytopenias after an initial positive response.
• Adverse events included nausea, thrombocytopenia, C. difficile colitis, febrile neutropenia, and injection site cellulitis.

Conclusions:

• Bortezomib demonstrates potential as a beneficial therapeutic option for pediatric refractory autoimmunity.
• Targeting plasma cells with bortezomib may effectively manage autoimmune-induced cytopenias.
• Further investigation is warranted to confirm bortezomib's role in pediatric autoimmune disease treatment.