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 0.435×10 9 /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.

Severe aplastic anaemia in children: Impact of histopathology profile and treatment on very long‐term outcomes

“…Overall survival in IST studies for SAA in children is usually reported after relatively limited follow‐up, with the notable exception of National Institutes of Health and EBMT series 13‐20 . Here, we report favourable very long outcomes with a median follow‐up of more than 10 years.…”

“…In this study, we found that only female gender and SAA vs VSAA were additional favourable predictive factors for response to IST, considering limited numbers of patients and the retrospective nature of the study. Other predictive factors of response in children include interval between diagnosis and treatment, telomere length, baseline lymphocyte counts and lymphocyte depletion early after IST 1,13‐15 …”

“…Other predictive factors of response in children include interval between diagnosis and treatment, telomere length, baseline lymphocyte counts and lymphocyte depletion early after IST. 1,[13][14][15] Overall survival in IST studies for SAA in children is usually reported after relatively limited follow-up, with the notable exception of National Institutes of Health and EBMT series. [13][14][15][16][17][18][19][20] Here, we report favourable very long outcomes with a median follow-up of more than 10 years.…”

Clinical Outcome of Acquired Post-Immunosuppressive-Therapy Aplastic Anemia in Pediatric Patients: A 13-Year Experience in Two Southern China Tertiary Care Centers

“…26 It is reasonable to hypothesize that the LY change during IST can predict the IST response rate. Gu et al 43 emphasized the role of the decreased LY rate in the first week after IST in IST efficacy prediction. However, the results of the present study found no significant difference in the decreased LY rate in the first week after IST between the response group and the non-response group.…”

Not all thrombocytopenic bleeding is the same

“…The number of platelet transfusions has an inverse correlation with outcomes after an immunosuppressive therapy or stem cell transplantation. [ 6 ] The use of topical antifibrinolytic agents such as tranexamic acid is particularly effective and can be administered in the form of soaked gauzes, mouthwash or crushed tablets applied locally. [ 7 ] Up to 80% of patients with aplastic anemia in India can present with minor bleeding, and avoiding unnecessary platelet transfusions can reduce the cost and complications of treatment.…”