Predisposition to mycobacterial infection is a key presenting feature of several rare inborn errors of intrinsic and innate immunity. Hematopoietic stem cell transplantation (HSCT) can be curative for such conditions, but published reports are few. We present a retrospective survey of the outcome of 11 affected patients (7 males, 4 females) who underwent HSCT between 2007 and 2019. Eight patients had disseminated mycobacterial infection prior to transplant. Median age at first transplant was 48 months (9 -192); three patients were successfully re-transplanted due to secondary graft failure. Donors were matched family (1), matched unrelated (3), and mismatched unrelated and haploidentical family (5 each). Stem cell source was peripheral blood (9), bone marrow (4), and cord blood (1). TCRαβ/CD19 + depletion was performed in 6. Conditioning regimens were treosulfan, fludarabine (4), with additional thiotepa (in 8), and fludarabine, melphalan (2); all had serotherapy with alemtuzumab (8) or anti T-lymphocyte globulin (6). Median hospital stay was 113 days (36–330). Three patients developed acute grade I-II skin and one grade IV skin graft versus host disease. Four patients had immune-reconstitution syndrome. Two reactivated cytomegalovirus (CMV), 1 Epstein-Barr virus, and 3 adenovirus post HSCT. Nine are alive, 1 died early post-transplant from CMV, and the other was a late death from pneumococcal sepsis. Patients with active mycobacterial infection at HSCT continued anti-mycobacterial therapy for almost 12 months. In conclusion, HSCT is a successful treatment for patients with mycobacterial susceptibility even with disseminated mycobacterial infection and in the absence of an HLA matched donor.

分枝杆菌感染的易感性是几种罕见的先天性内在和先天免疫缺陷的关键表现特征。造血干细胞移植 (HSCT) 可以治愈这种情况，但发表的报告很少。我们对 2007 年至 2019 年期间接受 HSCT 的 11 名受影响患者（7 名男性，4 名女性）的结果进行了回顾性调查。8 名患者在移植前有播散性分枝杆菌感染。首次移植的中位年龄为 48 个月 (9 -192)；3名患者因继发性移植失败而成功再次移植。供体是匹配的家庭（1），匹配的无关（3）和不匹配的无关和单倍体家庭（每个5）。干细胞来源是外周血 (9)、骨髓 (4) 和脐带血 (1)。TCRαβ/CD19 + 耗尽在 6 中进行。调理方案是硫丹、氟达拉滨 (4)、额外的噻替帕 (8) 和氟达拉滨、美法仑 (2)；所有人都接受了阿仑单抗 (8) 或抗 T 淋巴细胞球蛋白 (6) 的血清疗法。中位住院时间为 113 天 (36-330)。3 名患者出现急性 I-II 级皮肤病和 1 名 IV 级皮肤移植物抗宿主病。四名患者患有免疫重建综合征。HSCT 后 2 种重新激活的巨细胞病毒 (CMV)、1 种 Epstein-Barr 病毒和 3 种腺病毒。9 人还活着，1 人死于 CMV 移植后早期，另外 1 人死于肺炎球菌败血症。HSCT 中存在活动性分枝杆菌感染的患者继续抗分枝杆菌治疗近 12 个月。综上所述，