García Morin Marina, Cela Elena, Garrido Carmen, Bardón Cancho Eduardo, Aguado Del Hoyo Alejandra, Pascual Cristina, Pérez-Corral Ana, Beléndez Cristina
Hematología-Oncología Pediátricas, Hospital General Universitario Gregorio Marañón, Madrid, España.
Hematología-Oncología Pediátricas, Hospital General Universitario Gregorio Marañón, Madrid, España.
An Pediatr (Barc). 2017 Mar;86(3):142-150. doi: 10.1016/j.anpedi.2016.03.014. Epub 2016 Apr 29.
Sickle cell disease (SCD), despite the improvement in the medical management, is still associated with severe morbidity and decreased survival. Allogenic hematopoietic stem cell transplantation (Allo-HSCT) currently provides the only curative therapy. A report is presented on our experience in children with SCD, who underwent Allo-HSCT in a single centre.
A single centre descriptive study was conducted on patients with SCD who underwent a bone marrow transplant from an HLA-identical sibling donor between January 2010 and December 2014. Epidemiological, clinical and analytical parameters were collected with a follow-up to December 2015. Data are presented as frequencies, percentages, and medians (range).
Allo-HCST was performed in 11 patients (8 males) with a median age of 7 years (2-13), all of them with comorbidity prior to the HCST. A stable graft was achieved in 10 out of 11 patients, 9 of them with complete donor chimerism, and one patient with stable mixed chimerism after 1 year of allo-HSCT. One patient has secondary graft failure with re-appearance of symptoms associated with SCD on day 180. Complications of Allo-HSCT are: arterial hypertension 7/11, acute renal failure 3/11, CMV reactivation 9/11, neurological complications 4/11 (subarachnoid haemorrhage, seizure), and acute graft versus host disease (aGVHD) of the skin 6/11, one of whom developed grade iv intestinal aGVHD, causing his death (day 51). None of the patients developed chronic GVHD. The overall survival and event-free survival was 90.9% and 81.9%, respectively, with a median follow-up of 3.1 (1-5.7) years.
Allo-HSCT, the only curative therapy, remains associated with morbidity. There was a transplant related mortality in our study, consistent with multicentre studies (1/11), and with aGVHD being the main cause. Other problems still include graft failure (1/11), and neurological complications (4/11), although the permanent sequelae are mild.
尽管镰状细胞病（SCD）的医疗管理有所改善，但仍与严重的发病率和生存率降低相关。异基因造血干细胞移植（Allo-HSCT）目前是唯一的治愈性疗法。本文报告了我们在单一中心对接受Allo-HSCT的SCD患儿的经验。
对2010年1月至2014年12月期间接受来自HLA匹配同胞供体骨髓移植的SCD患者进行了一项单一中心描述性研究。收集了流行病学、临床和分析参数，并随访至2015年12月。数据以频率、百分比和中位数（范围）表示。
11例患者（8例男性）接受了Allo-HCST，中位年龄为7岁（2-13岁），所有患者在进行HSCT之前都患有合并症。11例患者中有10例实现了稳定的移植物植入，其中9例为完全供体嵌合体，1例在Allo-HSCT 1年后为稳定的混合嵌合体。1例患者在第180天出现继发性移植物失败，并再次出现与SCD相关的症状。Allo-HSCT的并发症包括：动脉高血压7/11、急性肾衰竭3/11、巨细胞病毒再激活9/11、神经系统并发症4/11（蛛网膜下腔出血、癫痫发作）以及皮肤急性移植物抗宿主病（aGVHD）6/11，其中1例发展为IV级肠道aGVHD并导致死亡（第51天）。所有患者均未发生慢性GVHD。总体生存率和无事件生存率分别为90.9%和81.9%，中位随访时间为3.1（1-5.7）年。
Allo-HSCT作为唯一的治愈性疗法，仍然与发病率相关。在我们的研究中有与移植相关的死亡，与多中心研究一致（1/11），且aGVHD是主要原因。其他问题还包括移植物失败（1/11）和神经系统并发症（4/11），尽管永久性后遗症较轻。
