Korean J Pediatr.  2005 Feb;48(2):178-185.

Allogeneic Hematopoietic Stem Cell Transplantation in Juvenile Myelomonocytic Leukemia

• Yoo KH
• You DK
• Lee SH
• Sung KW
• Cho EJ
• Koo HH

Abstract

PURPOSE
The purpose of this study was to evaluate the outcome of children with juvenile myelomonocytic leukemia(JMML) treated with allogeneic hematopoietic stem cell transplantation(allo- HSCT).
METHODS
Eleven JMML patients aged 8-39 months underwent allo-HSCT. The sources of grafts were unrelated donors(n=7), HLA-matched siblings(n=3) and an HLA 1-antigen mismatched familial donor. All patients had received chemotherapy +/- 13-cis-retinoic acid(CRA) before transplant, and CRA was used, posttransplant, in six patients.
RESULTS
Only three patients were in complete remission(CR) at the time of transplantation. Initial chimeric status revealed complete donor chimerism(CC) in five patients, mixed chimerism(MC) in five and autologous recovery(AR) in one. One patient with MC having persistent splenomegaly eventually turned to CC and CR after rapid tapering of cyclosporine, combined with daily use of CRA. An AR case relapsed shortly after transplant but was rescued with second, unrelated cord blood transplantation. Ultimately, six patients are alive, event-free, with a median follow-up of 15.5 months posttransplant. All three deaths occurred in patients who failed to achieve CC, leading to disease progression.
CONCLUSION
We suggest that graft-versus-leukemia effect play an important role and CRA a possible role in posttransplant leukemic involution in JMML. In patients whose leukemic burden is still high with MC after transplant, early tapering of immunosuppressants and introduction of CRA might provide a chance of a cure for some patients.

Keyword

Juvenile myelomonocytic leukemia; Allogeneic hematopoietic stem cell transplantation; 13-cis-retinoic acid; Graft-versus-leukemia effect

MeSH Terms

Child
Cyclosporine
Disease Progression
Drug Therapy
Fetal Blood
Follow-Up Studies
Hematopoietic Stem Cell Transplantation*
Hematopoietic Stem Cells*
Humans
Immunosuppressive Agents
Isotretinoin
Leukemia*
Leukemia, Myelomonocytic, Juvenile
Splenomegaly
Tissue Donors
Transplants
Cyclosporine
Immunosuppressive Agents
Isotretinoin