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Competitive Transplants to Evaluate Hematopoietic Stem Cell Fitness
Published on: August 31, 2016
The Pretransplant EASIX Score Predicts Transplant-Related Mortality in Children Undergoing Hematopoietic Stem Cell
Vedat Uygun1, Volkan Hazar2, Koray Yalçın3
1Department Of Pediatric Bone Marrow Transplantation Unit, İstinye University School of Medicine, MedicalPark Antalya Hospital, Antalya, Turkey.
Insights
The Endothelial Activation and Stress Index (EASIX) score effectively predicts transplant-related mortality in pediatric patients undergoing hematopoietic stem cell transplantation (HSCT). A high pretransplant EASIX score is linked to increased mortality and lower survival rates.
Area of Science:
- Pediatric Hematology
- Transplantation Immunology
- Biomarker Discovery
Background:
- Hematopoietic stem cell transplantation (HSCT) carries significant risks for pediatric patients.
- The Endothelial Activation and Stress Index (EASIX) is a validated adult risk assessment tool.
- Limited data exists on EASIX utility in pediatric HSCT for non-malignant diseases.
Purpose of the Study:
- To evaluate the pretransplant EASIX score (EASIXpre) as a predictor of transplant-related mortality (TRM) in pediatric patients.
- To determine an optimal EASIXpre cut-off for risk stratification in this population.
- To assess the association between EASIXpre and overall survival post-HSCT.
Main Methods:
- Retrospective analysis of 475 pediatric patients with non-malignant diseases undergoing allogeneic HSCT.
- Receiver Operating Characteristic (ROC) curve analysis to identify the optimal EASIXpre cut-off value.
- Kaplan-Meier survival analysis and multivariate Cox regression to assess TRM and overall survival.
Main Results:
- An EASIXpre cut-off of 2 was identified as optimal via ROC analysis.
- Patients with high EASIXpre scores (≥2) had significantly higher TRM (24.6%) compared to low scores (<2) (7.9%) within two years (p < 0.001).
- Two-year overall survival was significantly lower in the high EASIXpre group (73.6%) versus the low EASIXpre group (92.3%) (p < 0.001).
Conclusions:
- The pretransplant EASIX score is a valuable, cost-effective biomarker for predicting transplant outcomes in pediatric HSCT for non-malignant conditions.
- The identified EASIXpre cut-off of 2 demonstrates internal consistency for TRM prediction in this cohort.
- Further external validation in independent pediatric cohorts is required to confirm these findings.
Abstract:
The Endothelial Activation and Stress Index (EASIX) represents a significant advancement in the assessment of risk for pediatric patients undergoing hematopoietic stem cell transplantation (HSCT). While EASIX has been extensively studied in adults, emerging research indicates its potential utility in children. We conducted a retrospective analysis involving 475 pediatric patients with non-malignant diseases who underwent allogeneic HSCT. The primary objective was to explore the correlation between the pretransplant EASIX score (EASIXpre) and transplant-related mortality (TRM) following HSCT. Our study utilized ROC curve analysis to determine an optimal cut-off point at EASIX-pre = 2. Within two years, TRM was 24.6% (95% CI 15.6-38.6) in patients with high EASIX scores, compared to just 7.9% (95% CI 5.7-11.0) in those with low scores (p < 0.001). Additionally, overall survival rates at two years were lower for the high EASIX group: 73.6% (95% CI 62.1-85.1) versus 92.3% (95% CI 89.8-94.9) (p < 0.001). In multivariate analyses, the ROC-optimized cut-off of 2 demonstrated internal consistency in our cohort, confirming its association with TRM. However, this does not represent external validation, which requires independent pediatric cohorts. In conclusion, the EASIX score stands out as a valuable and cost-effective biomarker for predicting transplant outcomes in pediatric patients undergoing allogeneic HSCT for non-malignant diseases.
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