Vanikar A V, Modi P R, Patel R D, Kanodia K V, Shah V R, Trivedi V B, Trivedi H L
Department of Pathology, Laboratory Medicine, Transfusion Services and Immunohematology, Smt Gulabben Rasiklal Doshi and Smt Kamlaben Mafatlal Mehta Institute of Kidney Diseases and Research Center, Ahmedabad, Gujarat, India.
Transplant Proc. 2007 Apr;39(3):703-8. doi: 10.1016/j.transproceed.2007.01.070.
Autoimmune disease represents a (AD) breakdown of natural tolerance against autoreactive antigens leading to a high mortality and morbidity. The reaction is usually polyclonal; T- and B-cell components of the hematopoietic system are responsible for disease progression. Allogeneic/autologous hematopoietic stem cell transplantation (HSCT) are the current modalities for treating drug-resistant AD.
We present a single-center retrospective evaluation of allogeneic HSCT with nonmyeloablative, low-intensity conditioning in nine patients (five males, four females) with pemphigus vulgaris (PV) and 27 patients with systemic lupus erythematosus (SLE; 3 males, 24 females). The mean follow-up period was 4.24 years for PV and 4.9 years for SLE. Cytokine-mobilized HSC from unmatched related donors, with mean dose of 21.3 x 10(8) nucleated cells/kg body weight (BW; mean CD34(+) count, 6 x 10(6)/kg BW) was administered in to the thymus as well as the portal and peripheral circulations of recipients. Cyclosporine (4 +/- 1 mg/kg BW per day) and prednisolone (10 mg/kg BW per day) were administered for 6 months to protect mixed chimerism. A subset of patients with cross-gender donors were analyzed for peripheral blood chimerism at 1 month post-HSCT and every 3 months thereafter.
Sustained clinical remission with peripheral lymphohematopoietic chimerism of 0.7 +/- 0.3% was observed in PV, whereas SLE relapsed after mean of 7.35 months of disease-free interval associated with fall in chimerism from 5 +/- 3% to < or =0.08 +/- 0.03%.
HSCT was effective to achieve early clinical remission of PV; and in SLE relapsed after a 7.35-month disease-free interval accompanied by a fall in mixed lymphohematopoietic chimerism.
自身免疫性疾病（AD）代表了对自身反应性抗原的天然耐受性的破坏，导致高死亡率和发病率。这种反应通常是多克隆的；造血系统的T细胞和B细胞成分负责疾病进展。同种异体/自体造血干细胞移植（HSCT）是目前治疗耐药性AD的方法。
我们对9例寻常型天疱疮（PV）患者（5例男性，4例女性）和27例系统性红斑狼疮（SLE；3例男性，24例女性）患者进行了单中心回顾性评估，这些患者接受了非清髓性、低强度预处理的同种异体HSCT。PV患者的平均随访期为4.24年，SLE患者为4.9年。将来自不匹配相关供体的细胞因子动员的造血干细胞以平均剂量21.3×10⁸有核细胞/千克体重（BW；平均CD34⁺计数，6×10⁶/kg BW）注入受者的胸腺以及门静脉和外周循环。给予环孢素（4±1毫克/千克体重/天）和泼尼松龙（10毫克/千克体重/天）6个月以保护混合嵌合体。对一部分有跨性别供体的患者在HSCT后1个月及此后每3个月分析外周血嵌合体情况。
PV患者出现持续临床缓解，外周淋巴造血嵌合体为0.7±0.3%，而SLE患者在平均7.35个月的无病间隔期后复发，同时嵌合体从5±3%降至≤0.08±0.03%。
HSCT有效地实现了PV的早期临床缓解；而在SLE患者中，在7.35个月的无病间隔期后复发，同时混合淋巴造血嵌合体下降。
