Peer-reviewed article
Risk factors analysis and validation of early treatment non-response in children with primary ITP: a multicenter retrospective cohort study
Mingwei Li, Mei Yan ·
Summary
Written by AI, not yet AICS's view · claude-sonnet-5-5This multicenter retrospective study looked at 335 children with newly diagnosed primary immune thrombocytopenia (ITP), a blood platelet disorder. It asked which factors are linked to not responding to first-line treatment within 14 days. About one in four children (23.9%) did not respond. Older age, a higher platelet-to-lymphocyte ratio and a positive antinuclear antibody test were each independently linked to non-response. A model combining these three factors was tested in a separate group of 78 children and performed less well there than in the original group.
Key points
- 255 of 335 children (76.1%) responded within 14 days of first-line therapy; 80 (23.9%) did not.
- Older age was independently associated with non-response (OR = 1.769, 95% CI 1.629–1.920).
- Higher platelet-to-lymphocyte ratio (OR = 1.622, 95% CI 1.112–2.367) and ANA positivity (OR = 3.142, 95% CI 1.626–6.071) were also independently associated with non-response.
- The combined model had an AUC of 0.831 in the derivation cohort and 0.718 in the external validation cohort of 78 children.
- Calibration was acceptable in both cohorts (Hosmer–Lemeshow P = 0.609 and 0.220; Brier scores 0.129 and 0.137).
Limitations
The study is retrospective and observational, so it shows associations rather than causes. The external validation group was small and discrimination was weaker there (AUC 0.718, wide confidence interval). The abstract does not describe the specific treatments given, longer-term outcomes, or whether using the model improves care.
Abstract
Objective To identify factors associated with early non-response to first-line treatment in children with primary immune thrombocytopenia (ITP), and to develop and externally validate a prediction model. Methods This multicenter retrospective cohort study included 335 children with newly diagnosed primary ITP as the derivation cohort, including 255 responders and 80 non-responders. Clinical data obtained after admission were collected. Candidate variables were screened using correlation analysis and least absolute shrinkage and selection operator (LASSO) regression, followed by multivariable logistic regression to identify factors associated with early treatment non-response. Model discrimination was evaluated using the area under the receiver operating characteristic curve (AUC). An independent external validation cohort of 78 children was additionally included to assess model discrimination. Results Among the 335 children, 255 (76.1%) achieved a treatment response within 14 days after first-line therapy, whereas 80 (23.9%) did not. Multivariable analysis showed that older age (OR = 1.769, 95% CI: 1.629–1.920, P < 0.001), higher platelet-to-lymphocyte ratio (PLR) (OR = 1.622, 95% CI: 1.112–2.367, P = 0.012), and antinuclear antibody (ANA) positivity (OR = 3.142, 95% CI: 1.626–6.071, P < 0.001) were independently associated with early treatment non-response. The combined model incorporating age, PLR, and ANA achieved an AUC of 0.831 (95% CI: 0.778–0.885) in the derivation cohort and 0.718 (95% CI: 0.591–0.845) in the independent external validation cohort. Calibration was acceptable in both cohorts, with Hosmer–Lemeshow P values of 0.609 and 0.220 and Brier scores of 0.129 and 0.137, respectively. Conclusion Older age, higher PLR, and ANA positivity were independently associated with early non-response to first-line treatment within 14 days in children with newly diagnosed primary ITP. These findings may help identify children at high risk of treatment non-response and support individualized monitoring and treatment strategies.