Ponatinib in pediatric patients with Philadelphia chromosome-positive leukemia: a retrospective survey of the Japan Children's Cancer Group.
Ponatinib in pediatric patients with Philadelphia chromosome-positive leukemia: a retrospective survey of the Japan Children's Cancer Group.
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帕纳替尼治疗费城染色体阳性白血病儿科患者：日本儿童癌症小组的回顾性调查。
DOI：
10.1007/s12185-022-03329-5
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发表时间：
2022
期刊：
影响因子：
--
通讯作者：
Shimada H.
中科院分区：
文献类型：
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作者：
Kodama Y;Sato A;Kato K;Sakaguchi H;Kato M;Kawasaki H;Hiramatsu H;Kato I;Taga T;Shimada H.
Ponatinib对费城染色体阳性（Ph+）白血病成人有效，对第二代酪氨酸激酶抑制剂耐药或不耐受，但其在儿童中的使用数据有限。回顾性分析了接受泊那替尼治疗的9例Ph+急性淋巴细胞白血病（Ph+ ALL）和4例慢性粒细胞白血病（CML）患儿的临床病程。泊那替尼治疗开始时的中位年龄为12岁（范围8-16岁）。9例患者为男性，4例为女性。6例患者单独接受泊那替尼治疗，3例患者接受泊那替尼联合泼尼松龙治疗，1例患者接受泊那替尼联合利妥昔单抗鞘内治疗，1例患者接受泊那替尼联合常规化疗。2例患者接受了泊那替尼单药治疗和与化疗联合治疗。泊那替尼的中位剂量和持续时间分别为16.9 mg/m2（7-34.3）和1.1个月（0.2-22.7）。6例Ph+ ALL患者和2例CML患者对泊那替尼有应答。8例接受泊那替尼单药治疗的患者中有1例出现4级脂肪酶水平升高。3级非血液学毒性包括丙氨酸转氨酶水平升高（25%）、天冬氨酸转氨酶水平升高（25%）、γ-谷氨酰转移酶水平升高（12.5%）、高血压（12.5%）和多形性红斑（12.5%）。泊那替尼在儿童Ph+白血病患者中可能安全有效。
Ponatinib is effective in adults with Philadelphia chromosome-positive (Ph+) leukemia, resistant or intolerant to second-generation tyrosine kinase inhibitors, but there are limited data on its use in children. The clinical courses of nine pediatric patients with Ph+ acute lymphoblastic leukemia (Ph+ ALL) and four with chronic myeloid leukemia (CML) who received ponatinib therapy were retrospectively reviewed. The median age at the start of ponatinib therapy was 12 years (range 8–16 years). Nine patients were male and four were female. Six patients received ponatinib alone, three received ponatinib with prednisolone, one received ponatinib with rituximab intrathecal therapy, and one received ponatinib with conventional chemotherapy. Two patients received ponatinib both alone and in combination with chemotherapy. The median dose and duration of ponatinib were 16.9 mg/m2(7–34.3) and 1.1 months (0.2–22.7), respectively. Six patients with Ph+ ALL and two with CML responded to ponatinib. One of the eight patients who received ponatinib alone had grade 4 increased lipase levels. Grade 3 non-hematologic toxicities included elevated alanine aminotransferase levels (25%), elevated aspartate aminotransferase levels (25%), elevated gamma-glutamyl transferase levels (12.5%), hypertension (12.5%), and polymorphic erythema (12.5%). Ponatinib may be safe and effective in pediatric patients with Ph+ leukemia.