Development and validation of a nomogram for early prognostic prediction in childhood aplastic anemia receiving cyclosporine monotherapy.

Abstract

The prognosis for aplastic anemia (AA) patients undergoing cyclosporine is variable; therefore, a prognostic model is needed to optimize stratified treatment strategies. This observational retrospective cohort study included patients with AA who received immunosuppressive therapy with cyclosporine. A total of 437 patients were randomly assigned to the training cohort (n = 262) or the validation cohort (n = 175) at a ratio of 6:4. Independent prognostic factors were identified through univariate regression and collinearity screening, and the Cox regression model was used to construct a nomogram for prognosis prediction. ROC curves, calibration curves and DCA curves were used to evaluate the discrimination, calibration performance and clinical practicality of the models. A nomogram for predicting overall survival was constructed using 13 identified independent predictors. ROC curve analysis revealed that the AUC values for both the training and validation sets exceeded or approach 0.80, indicating good model discrimination. The calibration curve showed a high degree of consistency between the model prediction results and the actual observed values, and DCA confirmed the model's relevance in clinical decision-making. In this study, a new nomogram model for predicting the prognosis of children with AA was developed, providing a tool to support doctors in predicting early-stage patient prognosis and developing reasonable treatment plans. Cyclosporine is the cornerstone drug for immunosuppressive therapy in acquired aplastic anemia. Pediatric patients with acquired aplastic anemia exhibit variable outcomes following single-agent cyclosporine therapy. This study developed a prognostic prediction model for childhood aplastic anemia patients treated with cyclosporine monotherapy, providing a basis for the stratified management of aplastic anemia, with strong clinical applicability. This nomogram model has not been reported in studies on early prognosis prediction of childhood aplastic anemia.