Al-Ghemlas Ibrahim, Al-Daama Saad, Aqueel Hawazin, Siddiqui Khawar, El-Solh Hassan, Omer Hala, AlRajeh Loloah, Al-Seraihy Amal, Alahmari Ali, AlSaedi Hawazen, AlAnazi Awatif, Ayas Mouhab
Department of Pediatric Hematology/Oncology, King Faisal Specialist Hospital and Research Center, Riyadh, 11211, Saudi Arabia.
Department of Pediatric Hematology/Oncology, King Fahad Specialist Hospital, Dammam, Saudi Arabia.
Int J Pediatr Adolesc Med. 2022 Sep;9(3):160-164. doi: 10.1016/j.ijpam.2022.04.001. Epub 2022 Jun 2.
Clinical, laboratory and outcome data were reviewed for pediatric patients who were diagnosed with chronic myeloid leukemia (CML) and managed at two tertiary care hospitals in Saudi Arabia, between January 2011 and December 2017 to assess the response to tyrosine kinase inhibitors (TKI) focusing on the monitoring of BCR-ABL fusion gene transcript levels and to look at the overall outcome.
CML patients were identified based on the cytogenetic and molecular results.
Twelve pediatric patients diagnosed with CML at a median age of 8.4 year; treated with TKI as first-line therapy, 11 (91.7%) patients were started with imatinib (first-generation TKI), while one received dasatinib (second-generation TKI) due to his three-way Philadelphia chromosome sensitivity. Eight patients (72.7%) starting on imatinib were switched to dasatinib (six patients due to drug resistance, and two patients due to intolerance of Imatinib) and two patients (25%) of whom had already achieved major molecular response (MMR) on Imatinib. Response rate to imatinib in terms of achieving MMR as first-line therapy was achieved in five out of 11 patients (45.5%) and only three of them continued to maintain their MMR. Six out of eight patients who were switched to dasatinib achieved MMR. Two patients underwent hematopoietic stem cell transplant (SCT): one due to blast crisis and one due to the side effects of TKI. With a median follow-up time of 78 months (range, 40.5-108), all of our patients were alive at last update.
We report an excellent outcome with an overall survival (OS) of 100% at 5-year and disease-free survival (DFS) of 91.7% (8.0%). All our patients achieved MMR and only one patient had loss of MMR on follow-up. Eight patients (66.7%) achieved complete molecular response (CMR).
回顾了2011年1月至2017年12月期间在沙特阿拉伯两家三级医疗中心确诊并接受治疗的慢性髓性白血病（CML）儿科患者的临床、实验室及预后数据，以评估酪氨酸激酶抑制剂（TKI）的疗效，重点监测BCR-ABL融合基因转录水平，并观察总体预后情况。
根据细胞遗传学和分子学结果确定CML患者。
12例儿科CML患者，中位年龄8.4岁；接受TKI一线治疗，11例（91.7%）患者起始使用伊马替尼（第一代TKI），1例因对费城染色体三体敏感而接受达沙替尼（第二代TKI）治疗。起始使用伊马替尼的8例患者（72.7%）换用达沙替尼（6例因耐药，2例因不耐受伊马替尼），其中2例患者在伊马替尼治疗时已获得主要分子学缓解（MMR）。作为一线治疗，11例患者中有5例（45.5%）达到MMR，其中仅3例继续维持MMR。换用达沙替尼的8例患者中有6例达到MMR。2例患者接受了造血干细胞移植（SCT）：1例因急变期，1例因TKI的副作用。中位随访时间78个月（范围40.5 - 108个月），最后一次随访时所有患者均存活。
我们报告了出色的预后，5年总生存率（OS）为100%，无病生存率（DFS）为91.7%（8.0%）。所有患者均达到MMR，随访期间仅1例患者MMR丢失。8例患者（66.7%）达到完全分子学缓解（CMR）。
