J Korean Pediatr Soc.  2002 Mar;45(3):370-375.

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

• Lee J
• Kim SY
• Cho B
• Jang PS
• Chung NG
• Kim HK

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.

Keyword

Myelodysplastic syndrome(MDS); Hematopoietic stem cell transplantation(HSCT)

MeSH Terms

Adult
Anemia, Refractory, with Excess of Blasts
Child
Fetal Blood
Follow-Up Studies
Hematopoietic Stem Cell Transplantation*
Hematopoietic Stem Cells*
Humans
Leukemia, Myeloid, Acute
Myelodysplastic Syndromes*
Prognosis
Recurrence
Siblings
Stem Cells
Survival Rate