PURPOSE Haploidentical related donor (HRD) transplantation was performed in 7 recipients with chronic granulomatous disease (CGD) who had no matched-related or unrelated donor. METHODS Peripheral blood cell (PBC) products were used with a conditioning regimen consisting of low-dose cyclophosphamide, fludarabine, total body irradiation, and busulfan. Graft-versus-host disease (GVHD) prophylaxis consisted of high-dose post-transplant cyclophosphamide and sirolimus. Recipients were ages 14-26 years, and 3 had severe infections active at transplant. RESULTS All 7 recipients achieved full engraftment with complete donor chimerism early in the post-transplant period. Acute GVHD occurred in all cases and was grade 3 or steroid refractory in 3. Two patients with steroid-refractory GVHD died. Three patients with severe infectious complications active at transplant, 1 Nocardia pneumonia and 2 extensive invasive fungal infections), survived and were cured of their infection at last follow-up. Bacterial disease occurred post-transplant in all recipients, and viral infections/reactivation were common, including 4 cases of BK virus-associated hemorrhagic cystitis. CONCLUSIONS Seven patients with CGD achieved rapid and full-donor engraftment from HRDs utilizing PBCs and a conditioning regimen with PTCy and sirolimus GVHD prophylaxis. However, the incidence of grade 3 and steroid-refractory GVHD was high and led to 2 deaths. Patients with active infections at transplant had successful transplant courses and were cured of their disease. Although there was an initial success with this regimen, the cumulative experience does not support its use in CGD due to an unacceptable rate of severe GVHD.

中文翻译：

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在慢性肉芽肿病中使用移植后环磷酰胺无法预防单倍体相合造血细胞移植中的严重移植物抗宿主病。

目的 单倍体相关供体 (HRD) 移植在 7 名慢性肉芽肿病 (CGD) 受者中进行，这些受者没有匹配的相关或无关供体。方法 外周血细胞 (PBC) 产品与由低剂量环磷酰胺、氟达拉滨、全身照射和白消安组成的预处理方案一起使用。移植物抗宿主病 (GVHD) 预防包括大剂量移植后环磷酰胺和西罗莫司。接受者的年龄为 14-26 岁，其中 3 人在移植时有严重感染。结果 所有 7 位受者在移植后早期均实现了完全移植并具有完全的供体嵌合体。所有病例均发生急性 GVHD，其中 3 例为 3 级或 3 例类固醇难治性。2 例类固醇难治性 GVHD 患者死亡。3 名移植时出现严重感染并发症的患者，1 名诺卡菌肺炎和 2 名广泛侵袭性真菌感染），在最后一次随访时存活并治愈感染。所有受者在移植后均发生细菌性疾病，病毒感染/再激活很常见，包括 4 例 BK 病毒相关的出血性膀胱炎。结论 7 名 CGD 患者利用 PBC 和 PTCy 和西罗莫司 GVHD 预防的预处理方案从 HRD 中实现了快速和完全供体移植。然而，3 级和类固醇难治性 GVHD 的发生率很高，导致 2 人死亡。移植时活动性感染的患者移植成功，疾病治愈。虽然这个方案取得了初步成功，