Treat-to-target achievement and predictive analysis in childhood-onset systemic lupus erythematosus

Abstract Objective To validate the achievement of treat-to-target (T2T) in childhood-onset systemic lupus erythematosus (cSLE) at Wuhan Children’s Hospital. Method This study involved a retrospective application of the childhood lupus low disease activity state (cLLDAS) and remission definitions to prospectively gathered data from a real-world cSLE cohort. Disease activity was evaluated using the SLE Disease Activity Index 2000 (SLEDAI-2K) and Physician Global Assessment (PGA). Result Ninety-seven untreated patients were included in the study, with a median follow-up of 2.6 years. Among them , 75(77.32%),70/97(72.16%), 64/90(65.98%) and 41/97（42.27%）patients achieved LLDAS, cLLDAS, Definitions of Remission in SLE (DORIS) and childhood-onset clinical remission (cCR). The median times to achieve these targets were 1.22, 1.44, 1.49 and 1.52 years, respectively. The proportions of patients who maintained stability in LLDAS, cLLDAS, DORIS, and cCR for over half of the follow-up period (LLDAS/cLLDAS/DORIS/cCR-50) were 40.21%, 30.93%, 23.71%, and 19.59%, respectively. Male patients achieved cLLDAS and DORIS earlier. A longer disease duration at initial diagnosis was a positive factor for achieving cLLDAS and cCR. In contrast, glucocorticoid (GC) pulse therapy was a negative predictor for time to cLLDAS and DORIS. Earlier achievement of the first cLLDAS or cCR was associated with a higher probability of reaching cLLDAS-50 and cCR-50. Conclusion This single-center study demonstrates that cLLDAS and cCR are achievable targets in real-life clinical practice. However, maintaining stability in both states remains highly challenging. These findings highlight the need for optimized treatment strategies to improve long-term disease control and outcomes in cSLE especially for lighter or younger cSLE patients.