Serdjebi Cindy, Ciccolini Joseph, Fina Frederic, Delarue Arnauld, Verschuur Arnauld, Lacarelle Bruno, Ouafik L'Houcine, André Nicolas
aTransfert Oncology Unit, Nord Hospital bSurgery Unit, La Timone Hospital cPediatric Oncology Unit, La Timone Hospital dUMR S 911 - CRO2, Aix-Marseille Université, Marseille, France.
Pharmacogenet Genomics. 2014 Oct;24(10):527-9. doi: 10.1097/FPC.0000000000000076.
A 5-year-old girl who had undergone liver transplantation was scheduled for treatment with high-dose cytarabine for a Burkitt lymphoma. Because of impaired transplantation, a study of cytidine deaminase (CDA), the liver enzyme responsible for cytarabine detoxification, was conducted before initiating treatment to evaluate the risk for toxicity in this patient. The CDA genotype and phenotype were both studied and showed none of the polymorphisms usually associated with impaired CDA, but surprisingly functional deficiency was observed. Despite a subsequent 30% reduction in cytarabine dosing, life-threatening toxicities appeared quickly and treatment was discontinued. Further genetic investigations performed on liver biopsy showed that the donor was actually homozygous for CDA*2, a genotype associated with severe CDA deficiency. On the basis of the liver genotype, treatment was resumed with further dose reduction, which led to a better tolerance. This case report highlights the limits of searching germline polymorphisms in patients with liver transplant when the story plays in the liver.
一名接受过肝移植的5岁女孩因伯基特淋巴瘤计划接受大剂量阿糖胞苷治疗。由于移植功能受损,在开始治疗前对负责阿糖胞苷解毒的肝脏酶胞苷脱氨酶(CDA)进行了研究,以评估该患者的毒性风险。对CDA的基因型和表型均进行了研究,结果显示不存在通常与CDA受损相关的多态性,但令人惊讶的是观察到了功能缺陷。尽管随后将阿糖胞苷剂量减少了30%,但危及生命的毒性反应很快出现,治疗被迫中断。对肝活检进行的进一步基因研究表明,供体实际上是CDA*2纯合子,该基因型与严重的CDA缺乏有关。根据肝脏基因型,在进一步降低剂量的情况下恢复治疗,从而使耐受性更好。本病例报告强调了在肝脏中出现问题时,对肝移植患者进行种系多态性检测的局限性。