Abstract
Haploidentical hematopoietic stem cell transplant (haplo-HSCT) provides an important alternative for children and adolescents with acquired severe aplastic anemia (SAA) lacking of matched donors. To test whether pre-transplant serum ferritin (SF) represents a candidate predictor for survival and a potential biomarker for graft-versus-host disease (GvHD) in pediatric haplo-HSCT, we retrospectively evaluated 147 eligible patients with SAA who underwent haplo-HSCT. The patients were divided into the low-SF group (< 1000 ng/ml) and the high-SF group (≥ 1000 ng/ml). We found that SF ≥1000 ng/ml independently increased the risk of grade II-IV aGvHD (HR = 2.596, 95% CI 1.304-5.167, p = .007) and grade III-IV aGvHD (HR = 3.350, 95% CI 1.162-9.658, p = .025). Similar probabilities of transplant-related mortality at 100 days were observed in the two groups (6.19 ± 2.45% vs 8.00 ± 3.84%, p = .168). The 2-year overall survival (85.29 ± 3.89% vs 92.00 ±3.84%, p = .746) and fai lure-free survival (83.23% ± 4.08% vs 83.37 ± 6.27%, p = .915) were comparable. GvHD-/failure-free survival were 60.06 ± 5.10% and 75.56 ± 6.87%, respectively (p = .056). In conclusion, the elevated pre-transplant SF level is associated with higher incidences of grade II-IV aGvHD and grade III-IV aGvHD. However, it is not associated with worse survival after haplo-HSCT for children and adolescent patients with SAA.
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