Ampatzidou Maria, Papadhimitriou Stefanos I, Goussetis Evgenios, Panagiotou John P, Papadakis Vassilios, Polychronopoulou Sophia
Department of Pediatric Hematology-Oncology, Aghia Sophia Children's Hospital, Athens, Greece.
Pediatr Hematol Oncol. 2012 Aug;29(5):389-94. doi: 10.3109/08880018.2012.691946. Epub 2012 Jun 12.
Chronic myeloid leukemia (CML) represents a rare myeloproliferative disease among children where allogeneic stem cell transplantation (SCT) remains the curative gold standard. However, the impressive early cytogenetic and molecular responses achieved by tyrosine kinase inhibitors [TKIs (imatinib, nilotinib, and dasatinib)] as first-line or even sole treatment in adults, has led to their increasing use also among children. Due to limited data regarding long-term results of TKIs and especially those of second generation in pediatric cohorts, we would like to add clinical information in this rare series of patients by reporting on four children with CML over a 10-year period, focusing on TKIs, dose escalations and clinical responses.
慢性髓性白血病(CML)在儿童中是一种罕见的骨髓增殖性疾病,异基因干细胞移植(SCT)仍是治愈的金标准。然而,酪氨酸激酶抑制剂[TKIs(伊马替尼、尼洛替尼和达沙替尼)]在成人中作为一线甚至单一治疗所取得的令人瞩目的早期细胞遗传学和分子反应,已导致它们在儿童中的使用也日益增加。由于关于TKIs长期结果的数据有限,尤其是儿科队列中第二代TKIs的数据,我们希望通过报告10年间4例CML患儿的情况,补充这一罕见患者系列的临床信息,重点关注TKIs、剂量递增和临床反应。