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Open AccessEditorial

Chronic Myeloid Leukemia Prognosis and Therapy: Criticisms and Perspectives

1
Chair of Hematology, Unit of Blood Diseases and Bone Marrow Transplantation, Department of Clinical and Experimental Sciences, University of Brescia, ASST-Spedali Civili, 25100 Brescia, Italy
2
Servicio de Hematología. Hospital Universitario Ramón y Cajal, IRYCIS, 28034 Madrid, Spain
3
Institute of Hematology “Lorenzo e Ariosto Seràgnoli”, University of Bologna, 40138 Bologna, Italy
*
Author to whom correspondence should be addressed.
Website: http://www.ematologiabrescia.it/.
J. Clin. Med. 2020, 9(6), 1709; https://doi.org/10.3390/jcm9061709
Received: 22 May 2020 / Accepted: 27 May 2020 / Published: 2 June 2020
(This article belongs to the Special Issue Prognosis and Therapy of Chronic Myeloid Leukemia)
Ph+ chronic myeloid leukemia (CML) is a clonal myeloproliferative disease whose clinical course is characterized by progression disease from the early chronic phase (CP) to the fatal blastic phase (BP). This programmed course is closely related to the translocation t(9;22)(q22;q11) and the resulting BCR-ABL1 fusion protein (p210) that drives the leukemic transformation of hematopoietic stem cells. Therefore, the cure of CML can only pass through the abrogation of the Ph+ clone. Allogeneic stem cell transplantation (allo-SCT) and interferon-alpha (IFNα) have been proven to reduce the Ph+ clone in a limited proportion of CML population and this translated in a lower rate of progression to BP and in a significant prolongation of survival. Tyrosine-kinase inhibitors (TKIs), lastly introduced in 2000, by preventing the disease blastic transformation and significantly prolonging the survival in up to 90% of the patient population, radically changed the fate of CML. The current therapy with TKIs induces a chronicization of the disease but several criticisms still persist, and the most relevant one is the sustainability of long-term therapy with TKIs in terms of compliance, toxicity and costs. The perspectives concern the optimization of therapy according to the age, the risk of disease, the potency and the safety profiles of the TKIs. The prolongation of survival is the most important end point which should be guaranteed to all patients. The treatment free remission (TFR) is the new goal that we would like to give to an increasing number of patients. The cure remains the main objective of CML therapy. View Full-Text
Keywords: prognosis; therapy guidelines; tyrosine kinase inhibitor; MRD monitoring; quantitative PCR; digital PCR; NGS mutation; treatment free remission; treatment de-escalation prognosis; therapy guidelines; tyrosine kinase inhibitor; MRD monitoring; quantitative PCR; digital PCR; NGS mutation; treatment free remission; treatment de-escalation
MDPI and ACS Style

Russo, D.; Garcia-Gutierrez, J.V.; Soverini, S.; Baccarani, M. Chronic Myeloid Leukemia Prognosis and Therapy: Criticisms and Perspectives. J. Clin. Med. 2020, 9, 1709.

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