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Normal neurodevelopment in four young children treated with bone marrow transplantation for acute leukemia or
T A Kaleita1, W D Shields, A Tesler
1Department of Pediatrics, UCLA School of Medicine 90024.
Insights
Bone marrow transplants in young children with leukemia or aplastic anemia show remarkable neurodevelopmental outcomes. This study indicates that such treatments are not contraindicated by risks of developmental issues.
Area of Science:
- Pediatric Oncology
- Neurodevelopmental Biology
- Hematology
Background:
- Bone marrow transplantation (BMT) is a critical treatment for pediatric acute leukemia and aplastic anemia.
- Concerns exist regarding potential neurodevelopmental sequelae in young children undergoing BMT, particularly with conditioning regimens including cyclophosphamide and total body irradiation.
- This study focuses on a unique cohort of very young children (<2 years) receiving BMT.
Observation:
- Longitudinal neurodevelopmental assessments were conducted on four children who underwent BMT before 24 months of age.
- The conditioning regimen included high-dose cyclophosphamide, with three patients also receiving total body irradiation.
- Follow-up ranged from 28 to 71 months post-transplantation.
Findings:
- All four children exhibited normal somatic growth.
- Neurodevelopmental outcomes, including intelligence, language, perception, and motor coordination, were within normal ranges.
- No debilitating neurodevelopmental sequelae were observed in this cohort.
Implications:
- The findings suggest that BMT with cyclophosphamide and total body irradiation is a viable therapeutic option for infants and young children with acute leukemia or aplastic anemia.
- These results may alleviate concerns about long-term neurodevelopmental risks, encouraging further investigation and treatment in this age group.
- Future therapeutic strategies for pediatric BMT can proceed without prohibitive concerns regarding neurodevelopmental deficits.
Abstract:
Longitudinal neurodevelopmental studies of four consecutive young children treated by bone marrow transplantation for acute leukemia or aplastic anemia are presented. The children, the only four children less than 2 years of age who have received bone marrow transplants for these diseases at UCLA Medical Center, ranged in age from 36 weeks to 24 months at the time of transplantation. Conditioning involved high-dose cyclophosphamide treatment; three also had total body irradiation prior to bone marrow transplantation. Their respective outcomes after follow-up times of 28 months to 71 months posttransplantation are remarkable for normal somatic growth and normal development of intelligence, language, perception, and motor coordination. These findings indicate that future therapeutic studies of infants and young children with acute leukemia or aplastic anemia using total body irradiation, cyclophosphamide, and bone marrow transplantation are not contraindicated by risks of debilitating neurodevelopmental sequelae.