Pediatric Therapy-Related Myelodysplastic Syndrome/Acute Myeloid Leukemia: The M. D. Anderson Cancer Center Experience

Background: Therapy-related myelodysplastic syndrome/acute myeloid leukemia (t-MDS/AML) is a long-term complication of pediatric cancer and it carries a poor prognosis.

Patients and Methods: We retrospectively studied pediatric t-MDS/AML patients treated at M. D. Anderson from 1975 to 2007. We also compared these patients to pediatric patients with de novo MDS/AML during this time interval.

Results: Among 2589 children with cancer treated at M. D. Anderson, we identified 22 (0.85%) patients with t-MDS/AML and 141 (5.4%) patients with de novo MDS/AML. Patients with t-MDS/AML had a median age of 14 years (range, 3–20). There was a male and Hispanic predominance. The most common primary malignancies were osteosarcoma and Hodgkin lymphoma. The median latency period was 4.1 years. Fourteen patients received AML-type chemotherapy, 5 underwent allogeneic stem cell transplantation (SCT) as induction therapy, and 3 received supportive care. Fourteen patients underwent SCT as induction (n=5), post-remission (n=5), or salvage therapy (n=4). Their respective 2-year survival rates were 20%, 40%, and 25% (p= 0.85). Patients with de novo AML were younger (p=0.001), and had higher rates of CR (p=0.03), and survival (p<0.0001). Independent factors predicting shorter survival were poor/intermediate-risk cytogenetics (p=0.01), lower hemoglobin level (p=0.0001), and t-MDS/AML (vs. de novo) (p=0.003).

Conclusion: Childhood t-MDS/AML has a poor prognosis. Although patients benefited from AML-type induction chemotherapy followed by SCT as post-remission therapy, effective therapies are needed.