Chronic Granulomatous Disease-Associated IBD Resolves and Does Not Adversely Impact Survival Following Allogeneic HCT
Chronic Granulomatous Disease-Associated IBD Resolves and Does Not Adversely Impact Survival Following Allogeneic HCT
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DOI：
10.1007/s10875-019-00659-8
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发表时间：
2019-10-01
影响因子：
9.1
通讯作者：
Kang, Elizabeth M.
中科院分区：
文献类型：
--
作者：
Marsh, Rebecca A.;Leiding, Jennifer W.;Kang, Elizabeth M.
简介 大约 1/3 的慢性肉芽肿病 (CGD) 患者患有炎症性肠病 (IBD)。异基因造血细胞移植（HCT）对 CGD IBD 的影响以及 IBD 对移植结果的影响尚缺乏全面的研究。方法 回顾性收集 2005 年 1 月 1 日至 2016 年 6 月 30 日期间在 26 个初级免疫缺陷治疗联盟 (PIDTC) 中心接受同种异体 HCT 的 145 例 CGD 患者的数据。结果 49 例伴有 IBD 的 CGD 患者和 96 例无 IBD 的患者接受了同种异体 HCT。 89% 的 IBD 患者和 93% 的非 IBD 患者进行了移植 (p = 0.476)。 8.5% 的 IBD 患者和 3.5% 的非 IBD 患者发生上消化道急性 GVHD (p = 0.246)。 10.6% 的 IBD 患者和 11.8% 的无 IBD 患者发生下消化道急性 GVHD (p = 0.845)。 IBD 患者中 II-IV 级急性 GVHD 的累积发生率为 30% (CI 17-43%)，无 IBD 患者为 20% (CI 12-29%) (p = 0.09)。患有和不患有 IBD 的患者的五年总生存率相当：分别为 80% [CI 66-89%] 和 83% [CI 72-90%]（p = 0.689）。所有 33 名有 IBD 病史的存活可评估患者在同种异体 HCT 后 2 年内 IBD 均得到缓解。结论 在该队列中，同种异体 HCT 对 CGD 相关 IBD 有疗效。 IBD 不应禁忌 HCT，因为它不会导致死亡风险增加。这项研究注册号为 NCT02082353。
Introduction Inflammatory bowel disease (IBD) affects approximately 1/3 of patients with chronic granulomatous disease (CGD). Comprehensive investigation of the effect of allogeneic hematopoietic cell transplantation (HCT) on CGD IBD and the impact of IBD on transplant outcomes is lacking. Methods We collected data retrospectively from 145 patients with CGD who had received allogeneic HCT at 26 Primary Immune Deficiency Treatment Consortium (PIDTC) centers between January 1, 2005 and June 30, 2016. Results Forty-nine CGD patients with IBD and 96 patients without IBD underwent allogeneic HCT. Eighty-nine percent of patients with IBD and 93% of patients without IBD engrafted (p = 0.476). Upper gastrointestinal acute GVHD occurred in 8.5% of patients with IBD and 3.5% of patients without IBD (p = 0.246). Lower gastrointestinal acute GVHD occurred in 10.6% of patients with IBD and 11.8% of patients without IBD (p = 0.845). The cumulative incidence of acute GVHD grades II-IV was 30% (CI 17-43%) in patients with IBD and 20% (CI 12-29%) in patients without IBD (p = 0.09). Five-year overall survival was equivalent for patients with and without IBD: 80% [CI 66-89%] and 83% [CI 72-90%], respectively (p = 0.689). All 33 surviving evaluable patients with a history of IBD experienced resolution of IBD by 2 years following allogeneic HCT. Conclusions In this cohort, allogeneic HCT was curative for CGD-associated IBD. IBD should not contraindicate HCT, as it does not lead to an increased risk of mortality. This study is registered at NCT02082353.