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Allogeneic bone marrow transplantation for infants with acute leukemia or myelodysplastic syndrome
1The Department of Hematology-Oncology, St Jude Children's Research Hospital, 332 N. Lauderdale Street, Memphis, TN 38105, USA.
Insights
Allogeneic bone marrow transplantation (BMT) offers a 5-year survival of 45.5% for infants with leukemia or myelodysplasia. Disease remission before BMT significantly improves survival outcomes for these young patients.
Area of Science:
- Pediatric Hematology Oncology
- Stem Cell Transplantation
Background:
- Allogeneic bone marrow transplantation (BMT) is a critical treatment for pediatric acute leukemia and myelodysplasia.
- Outcomes for infants (≤24 months) undergoing BMT are less understood compared to older children.
- Assessing long-term sequelae and immune reconstitution is vital for this vulnerable population.
Purpose of the Study:
- To evaluate the survival rates and prognostic factors in infants undergoing allogeneic BMT for acute leukemia or myelodysplasia.
- To analyze the incidence of late sequelae and immune reconstitution in infant survivors post-BMT.
- To compare long-term outcomes of infant BMT survivors with matched older controls.
Main Methods:
- Retrospective analysis of 22 infants who received allogeneic BMT for acute leukemia or myelodysplasia.
- Evaluation of survival rates, prognostic factors, late sequelae, and immune reconstitution.
- Comparison of 10 infant survivors with 10 matched older controls.
Main Results:
- The 5-year event-free survival was 45.5%.
- Disease remission status at BMT was the most significant prognostic factor (P = 0.005); 5-year survival was 56% for those in remission versus 0% for those not in remission.
- Late sequelae and immune function at 1 year post-transplant were similar between infant survivors and older controls.
Conclusions:
- Allogeneic BMT can be effective for infants with acute leukemia or myelodysplasia, with remission status being a critical determinant of success.
- Long-term outcomes, including immune reconstitution and late sequelae, appear comparable between infants and older children post-BMT.
- Further research into optimizing BMT strategies for infants is warranted to improve survival and long-term health.
Abstract:
The objective of this study is to investigate the outcome of children 24 months of age or younger (infants) at the time of allogeneic bone marrow transplantation (BMT) for acute leukemia or myelodysplasia. We analyzed the survival rate, prognostic factors, incidences of late sequelae, and immune reconstitution in 22 infants who underwent allogeneic BMT. The 5-year event-free survival estimate was 45.5% (95% confidence interval (CI), 24.4% to 63.3%). Six patients died of transplant-related complications and six died of disease relapse. Remission status at the time of BMT was the most important prognostic factor (P = 0.005): no patient who received a transplant while their disease was not in remission survived, whereas the 5-year survival estimate for infants who underwent BMT during remission was 56% (95% CI, 31% to 75%). Long-term outcomes in the 10 infant survivors were compared with those of 10 older controls matched for diagnosis, disease status at the time of BMT, calendar year at the time of BMT, and source of stem cells. Immune function 1 year after transplantation and the incidences and spectra of late sequelae were similar for both groups during a median of 3.5 years (range, 1.5 to 7.2 years) of follow-up.