Mierkienė Gintarė, Vaitkevičienė Goda Elizabeta, Ažukaitis Karolis, Jankauskienė Augustina, Rascon Jelena
Clinic for Children's Diseases, Institute of Clinical Medicine, Faculty of Medicine, Vilnius University, Vilnius, Lithuania.
Pediatr Transplant. 2025 Aug;29(5):e70108. doi: 10.1111/petr.70108.
Hematopoietic stem cell transplantation (HSCT) in solid organ transplant recipients has been reported in adults. However, data on children are scarce. We report a case of an allogeneic HSCT in a 14-year-old girl to treat idiopathic very severe aplastic anemia (SAA).
The girl developed end-stage renal disease at the age of 4 years following Shiga toxin hemolytic-uremic syndrome. The cadaveric kidney was grafted at the age of 7 years. Three years later, the patient was successfully treated for active humoral graft rejection and continued with tacrolimus and antihypertensive treatment. At 10 years, an absence epilepsy manifested; therefore, lamotrigine and ethosuximide were added. After 7 years of having a kidney transplant, the patient developed very severe pancytopenia and was diagnosed with SAA. Parvovirus B19 and EBV infections were documented. At the age of 14 years, she received allogeneic hematopoietic stem cells from a matched unrelated CMV-seronegative donor. Neutrophils engrafted on Day +19 and full donor chimerism was achieved. An acute graft-versus-host disease grade II regressed after the escalation of immune suppression, which aggravated arterial hypertension and triggered CMV reactivation treated with glomerular filtration rate-adjusted ganciclovir. Antiviral therapy deteriorated renal graft function. A high-risk transplantation-associated thrombotic microangiopathy was diagnosed on Day +42 and treated with eculizumab. Despite adoptive therapy with CMV-specific cytotoxic T-lymphocytes (Day +62) the pericardial effusion developed and required surgical drainage. Nevertheless, CMV viremia and polyserositis gradually progressed to multiorgan failure. The patient died on Day +95 after HSCT.
Despite reported favorable outcomes in children who received allogeneic HSCT after kidney transplantation, there is a lack of evidence on how to overcome numerous challenges in these ultrarare cases.
成人实体器官移植受者进行造血干细胞移植（HSCT）已有报道。然而，关于儿童的数据却很稀少。我们报告一例14岁女孩接受异基因HSCT治疗特发性极重型再生障碍性贫血（SAA）的病例。
该女孩4岁时因志贺毒素溶血尿毒综合征发展为终末期肾病。7岁时接受了尸体肾移植。三年后，患者成功治疗了活动性体液移植排斥反应，并继续使用他克莫司和抗高血压治疗。10岁时，出现失神癫痫；因此，加用了拉莫三嗪和乙琥胺。肾移植7年后，患者出现极重度全血细胞减少，被诊断为SAA。检测到细小病毒B19和EBV感染。14岁时，她接受了来自匹配的CMV血清阴性无关供者的异基因造血干细胞。中性粒细胞在+19天植入，并实现了完全供者嵌合。免疫抑制升级后，II级急性移植物抗宿主病消退，但加重了动脉高血压并引发了CMV重新激活，用根据肾小球滤过率调整剂量的更昔洛韦治疗。抗病毒治疗使肾移植功能恶化。+42天诊断出高危移植相关血栓性微血管病，并用依库珠单抗治疗。尽管采用了CMV特异性细胞毒性T淋巴细胞过继治疗（+62天），仍出现心包积液并需要手术引流。尽管如此，CMV病毒血症和多浆膜炎逐渐发展为多器官功能衰竭。患者在HSCT后第95天死亡。
尽管有报道称肾移植后接受异基因HSCT的儿童预后良好，但在这些极为罕见的病例中，如何克服众多挑战仍缺乏证据。
