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Published on: March 5, 2018
Bone marrow transplantation for paediatric AML in first remission: a systematic review and meta-analysis
1Oncology Unit, The Children's Hospital at Westmead, Sydney, NSW, Australia.
Insights
Bone marrow transplantation (BMT) from a matched family donor improves survival for children with acute myeloid leukemia (AML) in first remission. More data is needed to confirm benefits for all AML subgroups and compare autologous bone marrow transplantation (ABMT) with chemotherapy.
Area of Science:
- Pediatric Hematology Oncology
- Cancer Treatment Research
- Clinical Trial Analysis
Background:
- Consolidation therapies for pediatric acute myeloid leukemia (AML) in first complete remission (CR1) include bone marrow transplantation (BMT), autologous bone marrow transplantation (ABMT), and chemotherapy.
- The comparative effectiveness of these AML treatment strategies remains a subject of ongoing debate.
Purpose of the Study:
- To systematically review and meta-analyze clinical trials evaluating the effectiveness of BMT and ABMT in pediatric AML patients in CR1.
- To determine if BMT from a histocompatible family donor improves outcomes compared to no BMT.
- To assess the effectiveness of ABMT compared to non-myeloablative chemotherapy in pediatric AML.
Main Methods:
- Systematic review and meta-analysis of eligible studies including patients under 21 years old with AML in CR1, from 1985 to 2000.
- Studies were categorized into two groups: (1) comparison of outcomes with and without a histocompatible family donor, and (2) randomized controlled trials (RCTs) comparing ABMT with chemotherapy.
- Relative risk (RR) was calculated, and results were pooled if heterogeneity was not excessive to determine overall RR and risk difference.
Main Results:
- Allocation to BMT was associated with a reduced risk of relapse and improved disease-free and overall survival in pediatric AML patients in CR1.
- Significant heterogeneity among RCTs for ABMT precluded pooling of results, preventing definitive conclusions on its effectiveness.
- BMT from a histocompatible family donor demonstrated a clear benefit for patient outcomes.
Conclusions:
- BMT utilizing a histocompatible family donor is an effective consolidation therapy for pediatric AML in CR1, improving survival and reducing relapse rates.
- Insufficient data currently exists to ascertain the efficacy of BMT across all AML subgroups or to definitively establish ABMT's superiority over non-myeloablative chemotherapy.
- Further investigation through an individual patient data meta-analysis is warranted to comprehensively evaluate the existing data on these treatment modalities.
Abstract:
For children with AML in CR1, the major consolidation therapies are BMT, ABMT and intensive chemotherapy. The relative effectiveness of these strategies is still debated. We conducted a systematic review and meta-analysis of trials to determine the effectiveness of BMT and ABMT in CR1 in paediatric AML. Eligible studies enrolled patients <21 years from 1985 to 2000 with AML in CR1. Two groups of studies were identified: (1) Those comparing the outcome of patients with and without a histocompatible family donor; and (2) Randomised controlled trials (RCT) comparing ABMT with non-myeloablative chemotherapy. The relative risk statistic was calculated for outcomes of interest in each trial. If there was no excessive heterogeneity between trials the results were pooled, and an overall relative risk and risk difference for treatment effect across trials were calculated. Results of the analysis showed that allocation to BMT reduced risk of relapse and improved disease-free and overall survival. For ABMT, heterogeneity of effect between RCTs prevented pooling of results. In conclusion, BMT from a histocompatible family donor improves patient outcome. Data are insufficient to determine whether this is true for all subgroups of AML, and whether ABMT is superior to non-myeloablative chemotherapy. An individual patient data meta-analysis is required to further evaluate the available data.
