Division of Pediatric Hematology and Oncology, Ibaraki Children's Hospital, Futaba-dai, 3-3-1, Mito, Ibaraki 311-4145, Japan.
Int J Hematol. 2011 Nov;94(5):479-82. doi: 10.1007/s12185-011-0932-6. Epub 2011 Oct 21.
We report two patients with chronic granulomatous disease (CGD). The first patient presented with granulomatous colitis and pulmonary aspergillosis, and the second patient with liver abscess and restrictive pulmonary disorder. Both patients underwent allogeneic hematopoietic stem cell transplantation, the first from an HLA-matched sibling donor, and the second from an HLA-matched unrelated donor, after preconditioning with fludarabine, anti-thymocyte globulin, cyclophosphamide, and total-body irradiation of 3 Gy. The engraftment was prompt and the regimen-related toxicity was mild. The patients are able to return to their daily lives with full donor chimerism, although the second patient underwent a living-related-donor orthotopic liver transplantation from his mother for chronic liver graft-versus-host disease. The conditioning regimen we used was feasible and applicable to patients with CGD accompanied by inflammatory disease and severe infection.
我们报告了两例慢性肉芽肿病（CGD）患者。第一例患者表现为肉芽肿性结肠炎和肺曲霉病，第二例患者表现为肝脓肿和限制性肺疾病。两名患者均在氟达拉滨、抗胸腺细胞球蛋白、环磷酰胺和 3 Gy 全身照射预处理后，接受了同种异体造血干细胞移植，第一例来自 HLA 匹配的同胞供体，第二例来自 HLA 匹配的无关供体。移植后造血恢复迅速，治疗相关毒性较轻。两名患者均完全供者嵌合，能够恢复正常生活，尽管第二例患者因慢性肝移植物抗宿主病，从母亲那里接受了活体相关供体原位肝移植。我们使用的预处理方案是可行的，适用于伴有炎症性疾病和严重感染的 CGD 患者。
