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“儿童白血病之王” 有了治疗新办法

2017-08-10 10:06:30   

幼年型粒单核细胞白血病(JMML)素有“儿童白血病之王”的称号,患儿往往在发病一年内就死亡,许多家长只能选择放弃治疗。目前最佳的治疗方式是造血干细胞移植,但复发率仍高达50%。如今,广东科学家找到了这一疾病的新疗法。

近两年来,南方医科大学南方医院儿科主任李春富、主治医生彭智勇及其团队成员利用去甲基化联合造血干细胞移植,目前已成功使得13例患儿存活,无一例复发。

去年,小宇被确诊患上了JMML,父亲曾先生抱着最后一线希望找到了李春富。李春富为小宇进行了去甲基化联合HCT的治疗,目前小宇的白血病已经治愈,也没有复发。

据介绍,JMML主要侵袭婴幼儿,患儿血液中粒单核两系细胞恶性增殖,发病的男女幼儿比例为13:1。虽然这种病只占儿童白血病的2%左右,但病死率高,化疗效果差,长期生存率不到10%。

李春富的团队以前也曾尝试治疗JMML病人,但在2014年前,所有治疗的JMML患儿无一例生存。

2014年4月,受到国外临床研究的启迪,李春富开始尝试用地西他滨和造血干细胞移植两者结合起来治疗JMML。效果令人鼓舞,包括小宇在内,李春富团队目前治疗了14例JMML病童,除了一例因移植失败无力再次移植而死亡外,其余13例病人均已存活,无一例复发,并无中度以上并发症,存活最长的已达39个月。

李春富解释,JMML病人通常甲基化水平很高,而使用去甲基化药物能抑制癌细胞生长,有利于造血干细胞的移植。另一方面,甲基化水平高也有利于癌细胞发生“免疫逃逸”,使用地西他滨也能降低肿瘤复发的几率。

李春富的团队在使用去甲基化药物的同时,还首创了互补性移植方法,为14名移植患儿同时输注父母造血干细胞与非亲缘脐血,两种造血干细胞“适者生存”,为患儿提供了“双保险”。最终,这些患儿一半以上非血缘脐带血成功植入,剩下的患儿父母半相合造血干细胞成功植入。

南方日报记者李秀婷

实习生梁嘉韵通讯员李晓姗

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