伊马替尼治疗幼儿慢性粒细胞白血病一例并文献复习

Imatinib is effective in a 12-month-old boy with chronic myelogenous leukemia： case report and literature review

目的 总结1例幼儿慢性粒细胞白血病（CML）的临床特点及应用伊马替尼治疗的临床效果.方法 回顾性分析2009年6月白求恩国际和平医院儿科收治的1例幼儿CML应用伊马替尼治疗的病例资料.患儿男,1岁,因反复低热、面色苍白1个月入院.体格检查：中度贫血貌,腹膨隆,巨脾.骨髓穿刺涂片示：慢性粒细胞白血病慢性期（CML-CP）;骨髓染色体示：t（9;22）（q34;q11）;融合基因示：BCR-ABL（p210）阳性.诊断为CML-CP.给予伊马替尼100 mg/d治疗并随访观察.以“儿童”“慢性”“白血病”为关键词,在中国知网和万方数据库（建库至2014年8月）中检索,查到相关文献10篇,共报道了100余例儿童CML.以“children＂“chronic myeloid leukemia”“imatinib”为检索词,在PubMed数据库（建库至2014年8月）中检索到相关文献30篇,报道了近400例患儿,其中1篇详细报道了1例幼儿CML.结合文献,探讨伊马替尼在儿童CML中的临床应用.结果 本例患儿应用伊马替尼治疗,无明显不良反应,6周达血液学完全缓解,3个月达细胞遗传学完全缓解,12个月达分子生物学完全缓解,未影响患儿生长发育,随访62个月患儿病情平稳.国内文献共报道了5例6 ～12岁儿童CML应用伊马替尼治疗,均无明显不良反应,2个月内均达到血液学完全缓解.国外文献报道了伊马替尼在儿童CML中应用的剂量、代谢特点以及较大样本的临床观察,认为伊马替尼在儿童中耐受性良好,疗效理想,可作为儿童CML-CP的首选治疗,但长期应用可能影响儿童骨骼生长.结论 伊马替尼可作为儿童CML的一线治疗,但其长期应用仍需更多的经验积累.

Objective To summarize the clinical characteristics of an infant with chronic myelogenous leukemia （CML） and the effects of imatinib on the case.Method The clinical features of an infant with CML,who was treated with imatinib in the Norman Bethune International Peace Hospital at June 2009,were retrospectively analyzed and the reports in literature were reviewed.The 1-year-old boy suffered from recurrent low-degree fever and pallor.He had a moderate anemia,distended abdomen and marked splenomegaly.Bone marrow aspiration revealed CML in chronic phase （CP）.The t （9; 22） （q34; q11） could be detected and BCR-ABL （p210） was positive.The boy was diagnosed as CML-CP and treated with imatinib 100 mg per day.There were 10 related papers and more than 100 child CML patients were reported as retrieved from CNKI （from its establishment to August 2014） and Wanfang Database （from its establishment to August 2014） when ＂Child＂,＂Chronic＂ and ＂ Leukemia＂ were used as keywords.And there were 30 related papers including 400 cases from PubMed Database （from its establishment to August 2014） and one detailed report of an infant with CML was retrieved when ＂childhood＂ and ＂chronic myeloid leukemia imatinib＂ were used as keywords.The clinical effects of imatinib in infant CML cases were analyzed and summarized based on the literature.Result The boy obtained a complete hematologic response （CHR）at the 6th week of diagnosis,a complete cytogenetic response （CCyR） at the 3rd month and a complete molecular response （CMR） at the 12th month without side effect.This boy grows very well and after a 62-month follow-up,his disease was stable.According to the domestic literature,5 children CML cases aged 6-12 years were treated with imatinib without side effects and got complete hematologic response （CHR） after 2-month-therapy.The dose,metabolic characteristics and clinical observation of imatinib can be found in foreign literature and imatinib showed good response with good tolerance in children with CML.Imatinib is regarded as the first line drug for children CML.But it may affect the development of the children.Conclusion The children with CML-CP had a good response to imatinib,but more experience in the treatment of children with CML with imatinib is needed.