Abstract

Juvenile myelomonocytic leukemia (JMML) is a rare hematological malignancy of early childhood, classified by the World Health Organization as a myelodysplastic/myeloproliferative disease and is associated with a poor prognosis. Allogeneic hematopoietic stem cell transplantation is the only curative treatment. A two-year-old male child was diagnosed with JMML and was given induction chemotherapy. One year after diagnosis, the patient received allogeneic hematopoietic stem cell transplantation from an HLA sibling donor after a myeloablative conditioning regimen. The patient remained free of disease after 5 years of follow-up, healthy, with complete clinical, immunologic and hematologic recovery, without signs of JMML. Transplantation is the only modality to achieve a cure in JMML patients. The most widely practiced approach is the use of bone marrow or peripheral blood stem cells after a myeloablative conditioning regimen. Post-transplant monitoring chimerism can help identify the patients who are at risk of relapse.

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