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Three decades of transplantation for chronic myeloid leukemia: what have we learned?
Jirí Pavlu1, Richard M Szydlo, John M Goldman
1Department of Haematology, Imperial College at Hammersmith Hospital, London, UK. jiri.pavlu@imperial.nhs.uk
Insights
Hematopoietic stem cell transplantation has been a curative treatment for chronic myeloid leukemia (CML) for 30 years. While tyrosine kinase inhibitors are effective, transplantation remains crucial for resistant cases and offers new therapeutic strategies.
Area of Science:
- Hematology
- Oncology
- Transplantation Immunology
Background:
- Hematopoietic stem cell transplantation (HSCT) has been a cornerstone in treating chronic myeloid leukemia (CML) for three decades.
- Evolution of HSCT techniques progressed from identical twins to HLA-identical siblings and unrelated donors, making CML a leading indication for allogeneic transplantation.
- The biological characteristics of CML, including its homogeneity, graft-versus-leukemia effect, and monitoring capabilities, positioned it at the forefront of transplantation research.
Purpose of the Study:
- To review the historical development and current role of hematopoietic stem cell transplantation in managing chronic myeloid leukemia.
- To highlight the significance of HSCT as a curative option, particularly for patients refractory to or intolerant of tyrosine kinase inhibitors.
- To explore emerging strategies combining tyrosine kinase inhibitors with transplantation for CML and other malignancies.
Main Methods:
- Review of historical data and clinical outcomes of HSCT for CML.
- Analysis of the impact of advancements in transplantation techniques and donor selection.
- Evaluation of the integration of BCR-ABL1 tyrosine kinase inhibitors with HSCT.
Main Results:
- HSCT has demonstrated long-term curative potential for chronic myeloid leukemia over 30 years.
- CML became the most frequent indication for allogeneic HSCT globally during the 1990s.
- Tyrosine kinase inhibitors provide long-term disease control for most CML patients, but HSCT remains vital for non-responders or intolerant individuals.
Conclusions:
- Hematopoietic stem cell transplantation remains an effective therapeutic option for chronic myeloid leukemia, especially when tyrosine kinase inhibitors fail.
- The combination of tyrosine kinase inhibitors and HSCT represents a promising new strategy for CML management.
- This combined approach offers potential inspiration for treating other hematologic malignancies.
Abstract:
Last year marked 30 years of hematopoietic stem cell transplantation as a curative treatment of chronic myeloid leukemia (CML). Initially studies used stem cells from identical twins but techniques rapidly developed to use cells first from HLA-identical siblings and later unrelated donors. During the 1990s CML became the most frequent indication for allogeneic transplantation worldwide. This, together with the relative biologic homogeneity of CML in chronic phase, its responsiveness to graft-versus-leukemia effect and the ability to monitor low level residual disease placed CML at the forefront of research into different strategies of stem cell transplantation. The introduction of BCR-ABL1 tyrosine kinase inhibitors during the last decade resulted in long-term disease control in the majority of patients with CML. In those who fail to respond and/or develop intolerance to these agents, transplantation remains an effective therapeutic solution. The combination of tyrosine kinase inhibitors with transplantation is an exciting new strategy and it provides inspiration for similar approaches in other malignancies.
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