Patel Niraj C, Chinen Javier, Rosenblatt Howard M, Hanson Imelda C, Brown Betty S, Paul Mary E, Abramson Stuart L, Ritz Jerome, Shearer William T
Department of Pediatrics, Section of Allergy and Immunology, Baylor College of Medicine, Texas Children's Hospital, Houston, TX 77039, USA.
J Allergy Clin Immunol. 2008 Dec;122(6):1185-93. doi: 10.1016/j.jaci.2008.10.030.
Between 1981 and 1995, 20 children with severe combined immunodeficiency (SCID; median age at transplant, 6.5 [range, 0.5-145] mo, 12 with serious infection) were treated with haploidentical T cell-depleted (anti-CD6 antibody) bone marrow (median number of 5.7 [0.8-18.8] x 10(8) nucleated cells/kg) from mismatched related donors (MMRDs), and 5 children with SCID (median age at transplant, 1.8 [0.5-5.0] mo, 1 with serious infection) were given unmanipulated bone marrow from matched related donors (MRDs). No conditioning or graft-versus-host disease (GvHD) prophylaxis was used.
To assess the outcomes of patients with SCID who received bone marrow from MMRDs or MRDs.
We reviewed the medical records of these 25 consecutive patients with SCID (4 with Omenn syndrome).
Of the 20 patients who received bone marrow from MMRDs, 12 engrafted, 10 survived at a median age of 15.2 [10.0-19.1] years, 4 had chronic GvHD (lung, intestine, skin), 5 required intravenous immunoglobulin, and 8 attended school or college. Two of 5 patients who died had chronic GvHD, and 2 developed lymphoproliferative disease. Of the 5 patients who received bone marrow from MRDs, 5 engrafted, 5 survived at a median age of 23.3 [18.5-26] years, 1 had chronic GvHD (lung, skin), 2 required intravenous immunoglobulin, and 4 attended school or college.
Treatment of critically ill patients with SCID with anti-CD6 antibody T cell-depleted MMRD marrow resulted in an overall 50% long-term survival of patients (83% survival of those engrafted). The principal barriers to long-term survival were delay in diagnosis, life-threatening infection, failure to engraft, and chronic GvHD. Educational goals were achieved in most of the survivors.
1981年至1995年间，20例患有严重联合免疫缺陷（SCID；移植时中位年龄为6.5[范围0.5 - 145]个月，12例有严重感染）的儿童接受了来自不匹配相关供者（MMRD）的单倍体相合T细胞去除（抗CD6抗体）骨髓移植（中位数量为5.7[0.8 - 18.8]×10⁸有核细胞/kg），5例患有SCID（移植时中位年龄为1.8[0.5 - 5.0]个月，1例有严重感染）的儿童接受了来自匹配相关供者（MRD）的未处理骨髓移植。未采用预处理或移植物抗宿主病（GvHD）预防措施。
评估接受MMRD或MRD骨髓移植的SCID患者的治疗结果。
我们回顾了这25例连续的SCID患者（4例患有奥门综合征）的病历。
在接受MMRD骨髓移植的20例患者中，12例植入成功，10例存活，中位年龄为15.2[10.0 - 19.1]岁，4例有慢性GvHD（肺部、肠道、皮肤），5例需要静脉注射免疫球蛋白，8例上学或上大学。死亡的5例患者中有2例有慢性GvHD，2例发生了淋巴增殖性疾病。在接受MRD骨髓移植的5例患者中，5例植入成功，5例存活，中位年龄为23.3[18.5 - 26]岁，1例有慢性GvHD（肺部、皮肤），2例需要静脉注射免疫球蛋白，4例上学或上大学。
用抗CD6抗体去除T细胞的MMRD骨髓治疗重症SCID患者，患者的长期总体生存率为50%（植入成功患者的生存率为83%）。长期生存的主要障碍是诊断延迟、危及生命的感染、植入失败和慢性GvHD。大多数幸存者实现了教育目标。
