Department of Pediatric Hematology/Oncology, King Faisal Specialist Hospital & Research Center, PO Box 3354, MBC 53 Riyadh 11211, Saudi Arabia.
Haematologica. 2010 Jul;95(7):1211-5. doi: 10.3324/haematol.2009.015180. Epub 2010 Apr 21.
As chronic myeloid leukemia is rare in children, most data on imatinib mesylate therapy is derived from adult studies. We retrospectively evaluated pediatric (<14 years) patients with Ph+ chronic myeloid leukemia treated with imatinib mesylate, from January 2003 through June 2008. Of the 12 chronic myeloid leukemia patients (2% of all leukemias) 11 were in chronic phase while one had myeloid blast crisis. Six subsequently underwent stem cell transplantation. Five patients had grade 3-4 arthralgia requiring therapy alteration. None achieved complete molecular remission (MR) with imatinib mesylate alone. In contrast 3/6 patients post stem cell transplantation have undetectable BCR-ABL. Three patients relapsed to chronic phase (1 imatinib mesylate; 2 stem cell transplantation). Relapse free survival is 65.6% at four years and all are alive. Imatinib mesylate is effective therapy for children with chronic myeloid leukemia. However, cure probably requires stem cell transplantation. Acute toxicity of imatinib mesylate is tolerable, but long-term effects on growing children are unknown. Pediatric patients with chronic myeloid leukemia should undergo stem cell transplantation when appropriate related donors are available.
由于儿童慢性髓性白血病较为罕见，关于甲磺酸伊马替尼治疗的大多数数据均来源于成人研究。我们回顾性评估了 2003 年 1 月至 2008 年 6 月期间接受甲磺酸伊马替尼治疗的费城染色体阳性慢性髓性白血病患儿（<14 岁）。在 12 例慢性髓性白血病患者（占所有白血病的 2%）中，11 例处于慢性期，1 例处于髓性原始细胞危象期。其中 6 例随后进行了干细胞移植。5 例出现 3-4 级关节痛，需要改变治疗方案。没有 1 例单用甲磺酸伊马替尼达到完全分子缓解（MR）。相比之下，干细胞移植后 3/6 例患者 BCR-ABL 不可检测。3 例患者复发至慢性期（1 例甲磺酸伊马替尼；2 例干细胞移植）。4 年无病生存率为 65.6%，所有患者均存活。甲磺酸伊马替尼是治疗儿童慢性髓性白血病的有效方法。然而，治愈可能需要干细胞移植。甲磺酸伊马替尼的急性毒性可耐受，但对生长中儿童的长期影响尚不清楚。当有合适的相关供体时，患有慢性髓性白血病的儿科患者应进行干细胞移植。
