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. 2008 Dec;153(6):814-9.
doi: 10.1016/j.jpeds.2008.06.004. Epub 2008 Jul 30.

Long-term outcome of pediatric patients with severe aplastic anemia treated with antithymocyte globulin and cyclosporine

Phillip Scheinberg  1 , Colin O Wu, Olga Nunez, Neal S Young
Affiliations

Long-term outcome of pediatric patients with severe aplastic anemia treated with antithymocyte globulin and cyclosporine

Phillip Scheinberg et al. J Pediatr. 2008 Dec.

Abstract

Objective: To determine the long-term outcomes in children with severe aplastic anemia (SAA) treated with antithymocyte globulin (ATG) and cyclosporine (CsA) through a retrospective analysis of the pediatric patients treated at our institution in all protocols that included horse ATG (h-ATG) and CsA.

Study design: Between 1989 and 2006, a total of 406 patients, 20% of whom were children under age 18 years, received an initial course of immunosuppressive therapy (IST) at our institution. Here we report the outcome of 77 children who were treated with an h-ATG plus CsA-based regimen during this period.

Results: The overall response rate at 6 months was 74% (57/77); the cumulative incidence of relapse at 10 years was 33%, and the median time to relapse was 558 days. The cumulative incidence of evolution after IST was 8.5%; all 3 such events occurred in partial responders. Overall, there were 13 deaths (17%), with 4 occurring within the 3 months after IST in patients who had a pretreatment absolute neutrophil count of < 100/microL and the other 9 occurring more than 6 months after initiation of IST. The median time to death was 570 days. The overall 10-year survival for the entire cohort was 80%; long-term survival in the children who responded to IST was 89%.

Conclusions: The long-term survival in pediatric patients who respond to IST is excellent, at about 90%. IST remains a good alternative in pediatric patients who lack an HLA-matched sibling donor and should be offered as initial therapy before possible hematopoietic stem cell transplantation from an unrelated donor.

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Conflict of interest statement

The authors disclose no conflicts of interest.

Figures

Figure 1
Figure 1
Age distribution of pediatrics patients with severe aplastic anemia treated with horse ATG-based immunosuppressive therapy.
Figure 2
Figure 2
Cumulative incidence of relapse (with 95% CI) among pediatric patients after successful initial treatment with h-ATG-based immunosuppressive therapy.
Figure 3
Figure 3
Cumulative incidence of evolution (with 95% CI) following immunosuppressive therapy with a h-ATG-based regimen.
Figure 4
Figure 4
Overall survival (with 95% CI) for the entire cohort including those who underwent HSCT(4A); overall survival with patients who underwent HSCT censored at the time of transplant (4B); overall survival of patients who responded to IST (4C); overall survival of patients who completed 6 months of IST regardless of response status (4D). HSCT, hematopoietic stem cell transplantation; IST, immunosuppressive therapy.
See this image and copyright information in PMC

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