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

Abstract

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.