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Short-term Results of Hematopoietic Stem Cell Transplantation for Children with Myelodysplastic Syndrome

Journal of the Korean Pediatric Society 2002;45(3):370-375.
Published online March 15, 2002.
Short-term Results of Hematopoietic Stem Cell Transplantation for Children with Myelodysplastic Syndrome
Jin Lee, Soh Yeon Kim, Bin Cho, Pil Sang Jang, Nak Gyun Chung, Hack Ki Kim
Department of Pediatrics, College of Medicine, The Catholic University of Korea, Seoul, Korea
소아 골수이형성 증후군에서 조혈모세포이식의단기간 결과 분석
이진, 김소연, 조빈, 장필상, 정낙균, 김학기
가톨릭대학교 의과대학 소아과학교실
Correspondence: 
Bin Cho, Email: chobinkr@cmc.cuk.ac.kr
Abstract
Purpose
: In most cases, myelodysplastic syndrome(MDS) transforms into a more aggressive state or acute myelogenous leukemia; it's prognosis is very poor. It is believed that hematopoietic stem cell transplantation(HSCT) is the only curative treatment of MDS, but available data in children are very sparse. In this report, the short term outcome of HSCT in childhood MDS was analyzed.
Methods
: Ten children with MDS(CMMoL 5, RAEB 3, RAEBt 2) underwent HSCT(HLA- matched sibling transplantation 4, HLA-matched unrelated transplantation 4, cord blood transplantation 1, HLA-mismatched familial transplantation 1) between November 1995 and January 2001 at St. Mary's Hospital. Median follow-up duration was 11 months.
Results
: Engraftment was successful in all cases and 8 patients are alive without disease. Three cases of VOD were observed and improved without complication. Four cases of grade II and 1 case of grade III acute GVHD were observed and well controlled with treatment. Three patients relapsed after transplantation. One patient is alive without disease after cytoreduction with allogenic stem cell rescue and 2 patients died of relapse.
Conclusion
: HSCT is a curative strategy of MDS and the survival rate is relatively higher than that of adults. But there is an obvious need for more studies because of the small number of patients and the short duration of the follow-up.
Key Words: Myelodysplastic syndrome(MDS), Hematopoietic stem cell transplantation(HSCT)


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