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Immunosuppressive therapy for pediatric aplastic anemia
Additional information is provided in the Online Supplementary Table S1.
For the 80 subjects who underwent second-line HSCT therapy, donors included 12 matched sibling donors, 61 matched unrelated donors (MUD), and 7 haplo-identical donors. Stem cell sources within the MUD cohort consist- ed of 42 from BM, five from peripheral blood, 12 from cord blood, and two without available data. Transplant
preparative regimens varied widely both within and between institutions.
Overall survival for patients receiving a second treat- ment is shown in Figure 4A. Among all subjects (n=110) and the hATG/CyA group (n=92) who received second- line treatment, 20 (18.2%) and 15 (16.3%) died, respec- tively. In a Cox proportional hazards model, there was no significant effect of time from IST to second treatment on
A
B
C
Figure 4. Outcomes after second-line therapy for relapsed/refractory disease. Kaplan-Meier analysis of (A) overall survival and (B) event-free survival for all subjects or subjects treated with horse anti-thymocyte globulin (rATG)/cyclosporine (CyA). (C) Log-rank test was used to compare event-free survival after second-line treat- ment with hematopoietic stem cell transplantation (HSCT) versus immunosuppressive therapy (IST) for all subjects or subjects treated with hATG/CyA.
haematologica | 2019; 104(10)
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