Multivariate logistic analysis of predictors of response to immunosuppressive therapy in children with aplastic anemia: a double-center study

ABSTRACT

Background

Immunosuppressive therapy (IST) composed of antithymocyte globulin (ATG) and cyclosporine A (CSA) is one of the standard therapies in pediatric patients with acquired aplastic anemia (AA), but predictors of IST are lack of consensus.

Procedures

Ninety-four patients from two pediatric medical centers in China were included between January 2005 and March 2018. Clinical factors associated with the efficacy were analyzed according to multivariate logistic regression model previously established.

Results

We discovered that overall responsiveness was 77.66%. Five out of 35 factors were statistically significant in univariate analysis. Based on the cutoff point chosen by receiver operating characteristic (ROC) curve, 5 continuous variables were made categorical, among which 3 variables with significance were employed to establish the logistic regression equation. Based on these 3 variables, we found that starting IST within 126 days of the first appearance of symptoms (X1, p = .003), absolute neutrophil count (ANC) higher than 0.435×10⁹/L (X2, p = .012), and rate of decreased actual lymphocyte count (ALC) higher than 59.2% within the 1st week after IST (X3, p = .001) were three independent risk factors for response to IST. The rate of decreased ALC higher than 59.2% after IST was the most significant variable (OR = 9.355, Log (P) = −2.161 + 2.149X1 + 1.662X2 + 2.236X3). The accuracy, sensitivity, and specificity of the model were 86.2%, 94.5% and 57.1%, respectively.

Conclusion

Duration of AA, ANC and decreased ALC rate after IST might predict the response to IST, among which the rate of decreased ALC after IST is the most important predictive factor.

KEYWORDS:

• Aplastic anemia
• immunosuppressive therapy
• antithymocyte globulin
• logistic analysis
• predictive factor
• children
• lymphocyte
• responsiveness

Acknowledgements

The authors would like to thank Dr. Qian Zhang for the assistance and suggestion of this article. We also thank all of the patients, families, nursing and medical staff for their collaboration.

Disclosure statement

No potential conflict of interest was reported by the authors.

ORCID

Changjuan Gu http://orcid.org/0000-0002-3173-8169