Myelodysplastic/ Myeloproliferative Neoplasms Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Juvenile Myelomonocytic Leukemia
No consistently effective therapy is available for JMML. Historically, more than 90% of patients have died despite the use of chemotherapy. Patients appeared to follow three distinct clinical courses:
- Rapidly progressive disease and early demise.
- Transiently stable disease followed by progression and death.
- Clinical improvement that lasted for as long as 9 years before progression or, rarely, long-term survival.
A recent retrospective review described 60 children with JMML treated with chemotherapy (nonintensive and intensive) and/or bone marrow transplantation (BMT) using sibling or unrelated human leukocyte antigen (HLA)-matched donor marrow or autologous marrow. The median survival was 4.4 years.[Level of evidence: 3iiiA]
BMT seems to offer the best chance of cure for JMML.[4,9,20,21,22,23] A summary of the outcome of 91 patients with JMML treated with BMT in 16 different reports is as follows: 38 patients (41%) were still alive at the time of reporting, including 30 of the 60 (50%) patients who received grafts from HLA-matched or 1-antigen mismatched familial donors, 2 of 12 (17%) with mismatched donors, and 6 of 19 (32%) with matched unrelated donors.
In a retrospective study investigating the role of BMT for chronic myelomonocytic leukemia (CMML), 43 children with CMML and given BMT were evaluated. In 25 cases, the donor was a HLA-identical or a one-antigen-disparate relative, in four cases a mismatched family donor, and in 14 cases a matched unrelated donor. Conditioning regimens consisted of total-body radiation therapy and chemotherapy in 22 patients, whereas busulfan with other cytotoxic drugs were used in the remaining patients. Six of 43 patients (14%), five of whom received transplants from alternative donors, failed to engraft. Probabilities of transplant-related mortality for children transplanted from HLA-identical/one-antigen-disparate relatives or from matched unrelated donors/mismatched relatives were 9% and 46%, respectively. The probability of relapse for the entire group was 58%; the 5-year event-free survival (EFS) rate was 31%. The authors of this study concluded that children with CMML and an HLA-compatible relative should be transplanted as early as possible.[Level of evidence: 3iiiDii]