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Successful Bone Marrow Transplantation With Intensive Post-transplant Intrathecal Chemotherapy for CNS Relapsed AML
Jeffrey R Andolina1, Carol Fries1, Renee Boulware1
1Department of Pediatrics, Golisano Children's Hospital, University of Rochester Medical Center, Rochester.
Insights
Infant acute myeloid leukemia (AML) is rare. A non-radiation approach using myeloablative conditioning and intrathecal chemotherapy showed promise for treating central nervous system relapse in two infant AML cases.
Area of Science:
- Pediatric Oncology
- Hematology
- Leukemia Research
Background:
- Infant acute myeloid leukemia (AML) represents a rare and aggressive hematologic malignancy.
- Early diagnosis and effective treatment strategies are critical for improving outcomes in affected infants.
Observation:
- This report details two pediatric cases of infant AML presenting with hyperleukocytosis.
- Both patients experienced isolated central nervous system (CNS) relapse despite ongoing chemotherapy.
- Successful bone marrow transplantation (BMT) was performed using myeloablative conditioning (thiotepa, busulfan, cyclophosphamide) without radiation.
Findings:
- Post-BMT, both infants received 12 empiric lumbar punctures with intrathecal cytarabine for CNS-directed therapy.
- Patients tolerated the conditioning regimen and intrathecal chemotherapy well.
- No post-transplant complications such as infections or chronic graft-versus-host disease were observed.
Implications:
- Nonradiation-based myeloablative conditioning combined with empiric intrathecal chemotherapy may be a viable strategy for high-risk infant AML with CNS involvement.
- This approach warrants further investigation as a potential treatment paradigm to reduce long-term sequelae in pediatric leukemia survivors.
Background:
Infant acute myeloid leukemia is a rare but aggressive form of leukemia.
Observation:
We report 2 children who presented with hyperleukocytosis, subsequently diagnosed with infant acute myeloid leukemia, and both developed isolated central nervous system relapse while on chemotherapy. Both infants underwent successful bone marrow transplantation with myeloablative conditioning (thiotepa, busulfan, and cyclophosphamide) without radiation, followed by 12 empiric post-transplant lumbar punctures with intrathecal cytarabine. Both patients tolerated these therapies well, and are without infections, chronic graft-versus-host disease, or any post-transplant sequelae.
Conclusion:
Nonradiation-based conditioning followed by empiric central nervous system-directed intrathecal chemotherapy may be considered for high-risk infants with leukemia.
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