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The treatment of pediatric chronic myelogenous leukemia in the imatinib era

Childhood chronic myelogenous leukemia (CML) is a rare hematologic disease, with limited literature on the methods of treatment. Previously, allogeneic hematopoietic stem cell transplantation (HSCT) was considered the only curative treatment for this disease. Treatment with imatinib, a selective inh...

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Autores principales: Lee, Jae Wook, Chung, Nack Gyun
Formato: Online Artículo Texto
Lenguaje:English
Publicado: The Korean Pediatric Society 2011
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3120996/
https://www.ncbi.nlm.nih.gov/pubmed/21738540
http://dx.doi.org/10.3345/kjp.2011.54.3.111
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author Lee, Jae Wook
Chung, Nack Gyun
author_facet Lee, Jae Wook
Chung, Nack Gyun
author_sort Lee, Jae Wook
collection PubMed
description Childhood chronic myelogenous leukemia (CML) is a rare hematologic disease, with limited literature on the methods of treatment. Previously, allogeneic hematopoietic stem cell transplantation (HSCT) was considered the only curative treatment for this disease. Treatment with imatinib, a selective inhibitor of the BCR-ABL tyrosine kinase (TKI), has resulted in prolonged molecular response with limited drug toxicity. Imatinib is now implemented in the primary treatment regimen for children, but the paucity of evidence on its ability to result in permanent cure and the potential complications that may arise from long-term treatment with TKIs have prevented imatinib from superseding HSCT as the primary means of curative treatment in children. The results of allogeneic HSCT in children with CML are similar to those observed in adults; HSCT-related complications such as transplant-related mortality and graft-versus-host disease remain significant challenges. An overall consensus has been formed with regards to the need for HSCT in patients with imatinib resistance or those with advanced-phase disease. However, issues such as when to undertake HSCT in chronic-phase CML patients or how best to treat patients who have relapsed after HSCT are still controversial. The imatinib era calls for a reevaluation of the role of HSCT in the treatment of CML. Specific guidelines for the treatment of pediatric CML have not yet been formulated, underscoring the importance of prospective studies on issues such as duration of imatinib treatment, optimal timing of HSCT and the type of conditioning utilized, possible treatment pre- and post-HSCT, and the role of second-generation TKIs.
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spelling pubmed-31209962011-07-07 The treatment of pediatric chronic myelogenous leukemia in the imatinib era Lee, Jae Wook Chung, Nack Gyun Korean J Pediatr Review Article Childhood chronic myelogenous leukemia (CML) is a rare hematologic disease, with limited literature on the methods of treatment. Previously, allogeneic hematopoietic stem cell transplantation (HSCT) was considered the only curative treatment for this disease. Treatment with imatinib, a selective inhibitor of the BCR-ABL tyrosine kinase (TKI), has resulted in prolonged molecular response with limited drug toxicity. Imatinib is now implemented in the primary treatment regimen for children, but the paucity of evidence on its ability to result in permanent cure and the potential complications that may arise from long-term treatment with TKIs have prevented imatinib from superseding HSCT as the primary means of curative treatment in children. The results of allogeneic HSCT in children with CML are similar to those observed in adults; HSCT-related complications such as transplant-related mortality and graft-versus-host disease remain significant challenges. An overall consensus has been formed with regards to the need for HSCT in patients with imatinib resistance or those with advanced-phase disease. However, issues such as when to undertake HSCT in chronic-phase CML patients or how best to treat patients who have relapsed after HSCT are still controversial. The imatinib era calls for a reevaluation of the role of HSCT in the treatment of CML. Specific guidelines for the treatment of pediatric CML have not yet been formulated, underscoring the importance of prospective studies on issues such as duration of imatinib treatment, optimal timing of HSCT and the type of conditioning utilized, possible treatment pre- and post-HSCT, and the role of second-generation TKIs. The Korean Pediatric Society 2011-03 2011-03-31 /pmc/articles/PMC3120996/ /pubmed/21738540 http://dx.doi.org/10.3345/kjp.2011.54.3.111 Text en Copyright © 2011 by The Korean Pediatric Society http://creativecommons.org/licenses/by-nc/3.0/ This is an open-access article distributed under the terms of the Creative Commons Attribution Non-Commercial License (http://creativecommons.org/licenses/by-nc/3.0/) which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited.
spellingShingle Review Article
Lee, Jae Wook
Chung, Nack Gyun
The treatment of pediatric chronic myelogenous leukemia in the imatinib era
title The treatment of pediatric chronic myelogenous leukemia in the imatinib era
title_full The treatment of pediatric chronic myelogenous leukemia in the imatinib era
title_fullStr The treatment of pediatric chronic myelogenous leukemia in the imatinib era
title_full_unstemmed The treatment of pediatric chronic myelogenous leukemia in the imatinib era
title_short The treatment of pediatric chronic myelogenous leukemia in the imatinib era
title_sort treatment of pediatric chronic myelogenous leukemia in the imatinib era
topic Review Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3120996/
https://www.ncbi.nlm.nih.gov/pubmed/21738540
http://dx.doi.org/10.3345/kjp.2011.54.3.111
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