Dipartimento di Farmacia Scienze del Farmaco Università degli Studi di Bari "Aldo Moro", via Orabona 4, Bari 70125, Italy.
Int J Mol Sci. 2020 Jun 23;21(12):4469. doi: 10.3390/ijms21124469.
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.
自酪氨酸激酶抑制剂（TKI）伊马替尼问世以来，慢性髓性白血病（CML）的治疗方法发生了变化，随后又出现了第二代 TKI 达沙替尼、尼洛替尼，最后是第三代药物泊那替尼。目前，这些治疗选择是成人的一线治疗方法。根据临床经验，即使针对儿科患者的疗效和安全性研究仍在等待更具体和高质量的数据，但伊马替尼、达沙替尼和尼洛替尼已获准用于儿童。在这种情况下，至关重要的是前瞻性验证从成人研究中推断的数据，以便通过儿科临床试验制定基于适当制剂的标准治疗管理方案，从而可以仔细监测 TKI 引起的不良反应，特别是在长期接受治疗的生长中的儿童。
