Which children do benefit from bone marrow transplant? The EBMT Paediatric Diseases Working Party
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Published on: March 17, 2020
D Niethammer1, T Klingebiel, W Ebell
1Dept. of Paediatrics, Univ. of Tübingen, Germany.
Insights
Chemotherapy cures most childhood acute lymphoblastic leukemia (ALL) cases. For the 20-25% with refractory disease, bone marrow transplantation (BMT) offers a chance for cure, especially in relapsed ALL.
Area of Science:
- Pediatric Oncology
- Hematology
- Hematopoietic Stem Cell Transplantation
Background:
- Childhood acute lymphoblastic leukemia (ALL) treatment has seen significant advances with chemotherapy.
- While cure rates are high, 20-25% of children do not achieve a cure with chemotherapy alone.
- Bone marrow transplantation (BMT) is an established alternative for refractory ALL cases.
Purpose of the Study:
- To summarize current knowledge on the role of BMT in treating childhood ALL.
- To define patient groups that may benefit from BMT, particularly in first and second complete remission (CR).
- To compare the efficacy of BMT versus chemotherapy in different relapse scenarios.
Main Methods:
- Review and synthesis of existing data on chemotherapy and BMT for childhood ALL.
- Analysis of treatment outcomes based on remission status and relapse timing.
- Comparison of BMT efficacy against conventional chemotherapy strategies.
Main Results:
- Autologous BMT (ABMT) in first CR may benefit a select group of ALL patients.
- In second CR, chemotherapy effectiveness is highly dependent on the duration of the first remission.
- Allogeneic BMT is crucial for cure in very early relapses and superior to chemotherapy in early and late relapses.
Conclusions:
- BMT is a vital therapeutic option for a significant subset of children with ALL.
- Allogeneic BMT offers the best chance for cure in patients with early or very early relapses.
- Further consensus is needed on optimal BMT indications, especially in first CR and very late relapses.
Abstract:
The development of chemotherapy in childhood ALL has been the leader of the success story of paediatric oncology. At least 2/3 of the children can be cured nowadays at the first attempt of treatment. From the remaining again 1/3 can be treated successfully for the relapse of their disease with conventional therapeutic strategies. This means, however, that there is no chance for cure with chemotherapy alone for 20 to 25% of the children. BMT has been shown for a long time to be an alternative therapy especially in those cases in which conventional chemotherapy fails. In spite of the fact that many children with ALL have been transplanted during recent years there is still no general agreement on the question which children need BMT. However a few statements can be made: The value of ABMT in ALL is probably not better than that of chemotherapy alone. In 1st CR a group of children can be defined, which might benefit from BMT. In 2nd CR the value of chemotherapy depends very much from the duration of 1st remission. Allogeneic BMT is the only chance for cure in very early relapses, superior to chemotherapy in early and late relapses and possibly equal to chemotherapy in very late relapses. The paper tries to summarise our current knowledge about the situation.
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