Long-term Clinical Outcome in Children with very Severe Aplastic Anemia

Lim, Ji Eun; Lim, Chang Hwan; Lee, Jae Wook; Jang, Pil Sang; Chung, Nak Gyun; Cho, Bin; Jeong, Dae Chul; Kim, Hack Ki

Abstract

PURPOSE: We retrospectively investigated the long-term clinical outcome in children with very severe aplastic anemia (vSAA) for choice of management of them.
METHODS
We reviewed and analyzed the medical records of patients diagnosed with vSAA at the Department of Pediatrics, The Catholic University of Korea between Jan. 1995 to Dec. 2007.
RESULTS
Forty three patients were diagnosed with vSAA during the study period, of whom 9 received only supportive treatment, 21 received immunosuppressive therapy (IST) consisting of cyclosporine A and either ATG or ALG, and 15 (including 2 patients who showed no response to initial IST) underwent hematopoietic stem cell transplantation (HSCT). Of the 9 patients who received only supportive measures, 1 patient recovered spontaneously, 6 patients died from either infection or bleeding within 10 months after diagnosis. Of the patients who received IST, 8 patients showed a response (42.1%) and 11 patients failed to respond. Of the 11 patients who did not respond, 8 patients died from infection or bleeding, 2 patients were lost during follow-up. Of the 15 HSCT's, 13 patients are currently surviving disease-free and 2 patients died from infections. The overall survival of the patients who received supportive care, IST, and HSCT was 33.3+/-15.7%, 47.4+/-14.0% and 86.7+/-8.8%, respectively (P=0.009).
CONCLUSION
As with SAA, HSCT is the best treatment option for vSAA. If IST is undertaken, measures should be undertaken to prevent death from early infection. With only supportive care, median survival approximates only 8 months, calling for more intensive treatment strategies such as IST or HSCT in the care of vSAA patients.