PN016 Outcome of allogeneic stem cell transplantation in children with juvenile myelomonocytic leukemia

Document Type





Purpose: Juvenile myelomonocytic Leukemia (JMML) is a rare childhood Leukemia for which allogeneic stem cell transplantation (ASCT) is the only curative therapy. Early relapse remains the single most important obstacle to successful use of this treatment modality. We report here the outcome of ASCT in 18 pediatric JMML patients treated in our institution.
Method: Eighteen consecutive patients (12 boys, 6 girls) with JMML underwent ASCT at our institution between Jan 1995 and Nov 2010. Prior to transplant, all patients but one were treated with chemotherapy. Splenectomy had been performed in 3 patients. At transplant, the disease was progressive, stable or in complete remission in 1, 13 and 4 patients, respectively. The median time to transplantation was 5.5 months (range: 2.5–15.3). The grafts were related marrows in 11 patients (HLA-identical relative in 10 patients and one-antigen mismatched in one) or partially matched unrelated cord in 7 patients (one-antigen mismatched in 2 and two-antigen mismatched in 5 patients). The conditioning regimen consisted of busulfan, cyclophosphamide and Etoposide in 12 and of busulfan and cyclophosphamide in 6 patients.
Results: Four patients relapsed at a median time of 2.6 months (range: 1.4–7.8) post-transplantation, and subsequently died of progressive disease. All relapses occurred in children older than one year at presentation. One patient died of severe veno-occlusive disease and 2 patients died of septic shock at 14, 8 and 33 days post transplantation, respectively. The 5-year overall and event-free survivals of the whole group were 60% with a median follow up of the surviving patients being 74.5 months (range: 8.6–165).
Conclusion: ASCT has dramatically improved survival of children with JMML. Age older than one year at presentation may predict an increased risk of disease recurrence however this merits a prospective evaluation in a larger cohort of patients.


This work was published before the author joined Aga Khan University


Pediatric Blood & Cancer