Abul Mehtap Haktanir, Tuano Karen, Healy C Mary, Vece Timothy J, Quintanilla Norma M, Davis Carla M, Seeborg Filiz O, Hanson Imelda Celine
Section of Immunology, Allergy and Rheumatology, Department of Pediatrics, Baylor College of Medicine, Texas Children's Hospital, Houston, Texas, USA.
Allergy Asthma Proc. 2015 Sep-Oct;36(5):407-11. doi: 10.2500/aap.2015.36.3876.
Hematopoietic stem cell transplantation (HSCT) outcomes in X-linked severe combined immune deficiency are most effective when performed with patients <3 months of age and without coexisting morbidity, and with donor cells from a matched sibling. Even under such favorable circumstances, outcomes can be suboptimal, and full cellular engraftment may not be complete, which results in poor B or natural killer cell function. Protein losing enteropathies can accompany persistent immune deficiency disorders with resultant low serum globulins (immunoglobulin A [IgA], IgG, IgM) and lymphopenia. Patients with immune disorders acquire infections that can be predicted by their immune dysfunction. Fungal infections are typically noted in neutropenic (congenital or acquired) and T-cell deficient individuals. Coexisting fungal infections are rare, even in hosts who are immunocompromised, and they require careful evaluation. Antifungal treatment may result in drug-drug interactions with significant complications.
对于X连锁重症联合免疫缺陷患者,造血干细胞移植(HSCT)在以下情况下效果最佳:患者年龄小于3个月且无并存疾病,供体细胞来自匹配的同胞。即使在这种有利情况下,结果也可能不理想,完全的细胞植入可能不完全,这会导致B细胞或自然杀伤细胞功能不佳。蛋白质丢失性肠病可伴随持续性免疫缺陷疾病,导致血清球蛋白(免疫球蛋白A [IgA]、IgG、IgM)降低和淋巴细胞减少。免疫紊乱患者会发生可由其免疫功能障碍预测的感染。真菌感染通常见于中性粒细胞减少(先天性或后天性)和T细胞缺陷个体。并存真菌感染很少见,即使在免疫受损宿主中也是如此,需要仔细评估。抗真菌治疗可能导致药物相互作用并引发严重并发症。