Long-term outcomes of patients with moderate-severe pediatric systemic lupus erythematosus treated with combination rituximab and cyclophosphamide therapy: A single center cohort experience and review of literature

Objective: To determine the long-term outcomes of patients with moderate-severe pediatric systemic lupus erythematosus (pSLE) treated with systematic administration of combination rituximab (RTX) and cyclophosphamide (CYC) at our institution. Methods: Patients with moderate-severe pSLE treated with the combination RTX/CYC therapy between 2013 and 2023 at a single center were included. Steroid exposure, Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) scores, and Systemic Lupus International Collaborating Clinics/American College of Rheumatology (SLICC/ACR) damage indices were collected as primary outcomes. We also reviewed the literature of the use of RTX/CYC therapy in pSLE. Results: Eleven patients received RTX/CYC therapy in our cohort. Six of 11 patients (55%) were Black. All patients achieved sustained low disease activity as defined by SLEDAI score less than 4 and prednisone dose less than 8 mg/day. There was no statistically significant change in the SLICC/ACR damage index over time. Positive clinical outcomes were observed in all racial groups. Two of 11 patients (18%) required hemodialysis, with one requiring a transplant. Other adverse effects included anaphylaxis and cytopenias. Several reports of use of RTX/CYC therapy were identified in our literature search. Our cohort had a more racially diverse cohort compared to other studies identified in our literature review. Conclusions: Our study supports the use of rituximab and cyclophosphamide use in patients with pSLE and demonstrates its efficacy among a racially diverse cohort over the long-term follow-up period. Our study has important limitations, and larger, prospective randomized clinical trials would allow for improved protocol evaluation and comparison.