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Controversies in the treatment of CML in children and adolescents: TKIs versus BMT?
Biol Blood Marrow Transplant 2011; 17(1 Suppl):S115-22BB

Abstract

Chronic myeloid leukemia (CML) is a relatively rare hematopoietic malignancy in the pediatric and adolescent population. This makes it difficult to perform clinic trials that can define the best therapeutic option when considering the impact of tyrosine kinase inhibitors (TKIs) versus the established approach of allogeneic hematopoietic cell transplantation (HCT). With the relatively low toxicity of TKIs, there are little data regarding when HCT or long-term TKI therapy is a better option. There are even less data regarding the duration of TKI treatment in the pediatric CML in chronic phase (CML-CP) patients who may receive over 60 years of therapy. As children and adolescent are treated for longer times with TKIs, it has become clear that toxicities may make long-term TKI therapy less attractive compared to allogeneic HCT. HCT has the long-term complications of growth failure, infertility, chronic graft-versus-host disease (GVHD), metabolic syndrome, and secondary malignancies, whereas prolonged TKIs may cause growth failure, hepatic, and cardiac complications. Moreover, HCT is a potentially curative intervention, whereas TKI is not curative, requiring prolonged exposure. In this article, we discuss the relative merit of the 2 therapeutic approaches and recommend that all children and adolescents with CML-CP should initially be treated with imatinib and maintained with TKI therapy indefinitely if there is a good response. We recommend that allogeneic HCT with an HLA-identical sibling donor or closely matched unrelated donor be considered for patients with treatment failure or recurrence after receiving salvage second-generation TKI treatment. We also conclude that randomized international trials are urgently needed to evaluate the best therapies for pediatric CML.

Authors+Show Affiliations

Division of Pediatric Hematology-Oncology-Blood and Marrow Transplantation, University Hospital Carl Gustav Carus, Fetscherstr. 74, Dresden, Germany.No affiliation info availableNo affiliation info available

Pub Type(s)

Comparative Study
Journal Article

Language

eng

PubMed ID

21195300

Citation

Suttorp, Meinolf, et al. "Controversies in the Treatment of CML in Children and Adolescents: TKIs Versus BMT?" Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation, vol. 17, no. 1 Suppl, 2011, pp. S115-22.
Suttorp M, Yaniv I, Schultz KR. Controversies in the treatment of CML in children and adolescents: TKIs versus BMT? Biol Blood Marrow Transplant. 2011;17(1 Suppl):S115-22.
Suttorp, M., Yaniv, I., & Schultz, K. R. (2011). Controversies in the treatment of CML in children and adolescents: TKIs versus BMT? Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation, 17(1 Suppl), pp. S115-22. doi:10.1016/j.bbmt.2010.09.003.
Suttorp M, Yaniv I, Schultz KR. Controversies in the Treatment of CML in Children and Adolescents: TKIs Versus BMT. Biol Blood Marrow Transplant. 2011;17(1 Suppl):S115-22. PubMed PMID: 21195300.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Controversies in the treatment of CML in children and adolescents: TKIs versus BMT? AU - Suttorp,Meinolf, AU - Yaniv,Isaac, AU - Schultz,Kirk R, PY - 2010/09/01/received PY - 2010/09/08/accepted PY - 2011/1/4/entrez PY - 2011/1/14/pubmed PY - 2011/5/28/medline SP - S115 EP - 22 JF - Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation JO - Biol. Blood Marrow Transplant. VL - 17 IS - 1 Suppl N2 - Chronic myeloid leukemia (CML) is a relatively rare hematopoietic malignancy in the pediatric and adolescent population. This makes it difficult to perform clinic trials that can define the best therapeutic option when considering the impact of tyrosine kinase inhibitors (TKIs) versus the established approach of allogeneic hematopoietic cell transplantation (HCT). With the relatively low toxicity of TKIs, there are little data regarding when HCT or long-term TKI therapy is a better option. There are even less data regarding the duration of TKI treatment in the pediatric CML in chronic phase (CML-CP) patients who may receive over 60 years of therapy. As children and adolescent are treated for longer times with TKIs, it has become clear that toxicities may make long-term TKI therapy less attractive compared to allogeneic HCT. HCT has the long-term complications of growth failure, infertility, chronic graft-versus-host disease (GVHD), metabolic syndrome, and secondary malignancies, whereas prolonged TKIs may cause growth failure, hepatic, and cardiac complications. Moreover, HCT is a potentially curative intervention, whereas TKI is not curative, requiring prolonged exposure. In this article, we discuss the relative merit of the 2 therapeutic approaches and recommend that all children and adolescents with CML-CP should initially be treated with imatinib and maintained with TKI therapy indefinitely if there is a good response. We recommend that allogeneic HCT with an HLA-identical sibling donor or closely matched unrelated donor be considered for patients with treatment failure or recurrence after receiving salvage second-generation TKI treatment. We also conclude that randomized international trials are urgently needed to evaluate the best therapies for pediatric CML. SN - 1523-6536 UR - https://www.unboundmedicine.com/medline/citation/21195300/full_citation/Controversies_in_the_treatment_of_CML_in_children_and_adolescents:_TKIs_versus_BMT L2 - https://linkinghub.elsevier.com/retrieve/pii/S1083-8791(10)00383-6 DB - PRIME DP - Unbound Medicine ER -