Late effects after allogeneic hematopoietic cell transplantation with busulphan conditioning in 33 childhood leukemia survivors (1987-2013)

F. Freycon, L. Casagranda, B. Trombert-Paviot, C. Berger
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Abstract

Late effects (LE) after busulphan (BU) conditioning Hematopoietic Cell Transplantation (HCT) in childhood leukemia survivors are rarely studied. LE were retrospectively collected for 33 childhood leukemia survivors (median age 21y at follow-up) treated with BU myeloablative conditioning allogeneic HCT. Median delay after HCT was 14.0y and median age at evaluation was 21.0y (17.0-33.5). Patients had a chronic Graft versus Host Disease (cGvHD) (n=3). The mean number of severe late effects was 1.1 per patient: decreased height at adulthood (n=4); Growth Hormone deficit (n=1); risk of impaired fertility (n=14/18 females; n=7/15 males; 2 women had a child); iron overload (n=3); mellitus diabetes (n=1); pulmonary LE (n=2); psychiatric disorders (n=3); sclerodermic cGvHD (n=1); severe academic difficulties (n=3). On the contrary, we found no cardiac, renal or CNS LE, and no secondary cancer. Among the other non-severe LE, there was: alopecia (n=3); treated hypogonadism (n=13 all females); low weight (n=10); minor overweight (n=13); hepatic focal nodular hyperplasia (n=9); dental hypoplasia (n=6); osteopenia (n=4); hypertriglyceridemia (n=4); moderate cataract (n=1). Conclusion: The mean number of severe late effects was 1.1 (±1.0) per patient, significantly less than in our other cohort of 71 children grafted after TBI conditioning published in 2019 (2.3 ±1.5; p<0.0001). *Correspondence to: Léonie Casagranda, Department of Pediatric Hematology and Oncology Unit, University Hospital of Saint-Etienne, 42055 Saint-Etienne Cedex 02, France, Tel: +33 477 127 937, E-mail: leonie.casagranda@chu-stetienne.fr
异基因造血细胞移植配合布硫芬治疗33例儿童白血病幸存者的后期效应(1987-2013)
布硫芬(BU)调节造血细胞移植(HCT)对儿童白血病幸存者的晚期效应(LE)研究很少。回顾性收集33例儿童白血病幸存者(随访时中位年龄21岁)接受BU清髓调节异基因HCT治疗的LE。HCT后的中位延迟为14.0岁,评估时的中位年龄为21.0岁(17.0-33.5岁)。患者有慢性移植物抗宿主病(cGvHD) (n=3)。严重晚期效应的平均数量为1.1例:成年后身高下降(n=4);生长激素缺乏(n=1);生育能力受损风险(n=14/18名女性;n = 7/15男性;2个女人有一个孩子);铁过载(n=3);糖尿病(n=1);肺LE (n=2);精神疾病(n=3);硬皮病cGvHD (n=1);严重的学习困难(n=3)。相反,我们没有发现心脏、肾脏或中枢神经系统LE,也没有继发性癌症。其他非重度LE患者有:脱发(n=3);治疗性腺功能减退(n=13,均为女性);低重量(n=10);轻度超重(n=13);肝局灶性结节增生(n=9);牙齿发育不全(n=6);骨量减少(n = 4);高甘油三酯血症(n = 4);中度白内障(n=1)。结论:严重晚期效应的平均数量为1.1(±1.0)例/例患者,显著低于2019年发表的71例TBI后移植儿童队列(2.3±1.5;p < 0.0001)。*通讯:法国圣埃蒂安大学医院儿童血液学和肿瘤科l 937;电话:+33 477 127 937;电子邮件:leonie.casagranda@chu-stetienne.fr
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