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Bcr-Abl Tyrosine Kinase Inhibitors in the Treatment of Pediatric CML

Dipartimento di Farmacia Scienze del Farmaco Università degli Studi di Bari “Aldo Moro”, via Orabona 4, Bari 70125, Italy
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Int. J. Mol. Sci. 2020, 21(12), 4469; https://doi.org/10.3390/ijms21124469
Received: 14 May 2020 / Revised: 10 June 2020 / Accepted: 18 June 2020 / Published: 23 June 2020
(This article belongs to the Special Issue Paediatric Formulation: Design and Development)
The therapeutic approach to Chronic Myeloid Leukemia (CML) has changed since the advent of the tyrosine kinase inhibitor (TKI) imatinib, which was then followed by the second generation TKIs dasatinib, nilotinib, and, finally, by ponatinib, a third-generation drug. At present, these therapeutic options represent the first-line treatment for adults. Based on clinical experience, imatinb, dasatinib, and nilotinib have been approved for children even though the studies that were concerned with efficacy and safety toward pediatric patients are still awaiting more specific and high-quality data. In this scenario, it is of utmost importance to prospectively validate data extrapolated from adult studies to set a standard therapeutic management for pediatric CML by employing appropriate formulations on the basis of pediatric clinical trials, which allow a careful monitoring of TKI-induced adverse effects especially in growing children exposed to long-term therapy. View Full-Text
Keywords: chronic myeloid leukemia; tyrosine kinase inhibitors; pediatric age; imatinib; dasatinib; nilotinb; ponatinib; formulation chronic myeloid leukemia; tyrosine kinase inhibitors; pediatric age; imatinib; dasatinib; nilotinb; ponatinib; formulation
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Carofiglio, F.; Lopalco, A.; Lopedota, A.; Cutrignelli, A.; Nicolotti, O.; Denora, N.; Stefanachi, A.; Leonetti, F. Bcr-Abl Tyrosine Kinase Inhibitors in the Treatment of Pediatric CML. Int. J. Mol. Sci. 2020, 21, 4469.

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