Nakagawa R, Kawano Y, Yoshimura E, Suzuya H, Watanabe T, Kanamaru S, Onishi T, Nakayama H, Nakagawa R, Matsuoka S, Yamashita K, Kuroda Y
Department of Pediatrics, University of Tokushima School of Medicine, Japan.
Bone Marrow Transplant. 2001 Feb;27(3):333-6. doi: 10.1038/sj.bmt.1702781.
A 15-year-old boy with refractory juvenile rheumatoid arthritis (JRA) underwent intense immunosuppressive therapy followed by purified blood CD34+ cell autografting. He had been taking prednisolone (PDN) daily or every other day combined with methotrexate once a week to control the disease for 7 years. He suffered from psychological complications and a very short stature due to the adverse effects of these drugs. CD34+ cells were purified in bulk from G-CSF-mobilized PBSC using an Isolex 300. After the administration of cyclophosphamide (200 mg/kg) and anti-lymphocyte globulin (45 mg/kg), 3.6 x 10(6)/kg purified CD34+ cells were infused. His post-transplant course was uneventful except for herpes-zoster infection. He is now more than 1 year post transplant and has not taken any immunosuppressive medication. His rate of growth has increased (>10 cm/year) due to the effects of the cessation of PDN and the administration of recombinant human growth hormone (rGH), in contrast to the gain of 2 cm in the preceding 3 years with rGH treatment. Although the durability of this remission is unknown, intense immunosuppressive therapy followed by purified blood CD34+ cell autografting might be acceptable for adolescent patients with refractory JRA to achieve a drug-free period for physical and psychological maturation.
一名患有难治性幼年类风湿性关节炎（JRA）的15岁男孩接受了强化免疫抑制治疗，随后进行了纯化血液CD34+细胞自体移植。他已经每日或隔日服用泼尼松龙（PDN）并每周联合使用一次甲氨蝶呤来控制病情达7年之久。由于这些药物的副作用，他出现了心理并发症和身材极为矮小的情况。使用Isolex 300从粒细胞集落刺激因子（G-CSF）动员的外周血干细胞（PBSC）中大量纯化CD34+细胞。在给予环磷酰胺（200 mg/kg）和抗淋巴细胞球蛋白（45 mg/kg）后，输注了3.6×10(6)/kg纯化的CD34+细胞。除带状疱疹感染外，他的移植后病程平稳。现在他移植后已超过1年，未服用任何免疫抑制药物。由于停用了PDN并使用了重组人生长激素（rGH），他的生长速度加快（>10厘米/年），而在前3年接受rGH治疗时仅增长了2厘米。尽管这种缓解的持久性尚不清楚，但对于难治性JRA的青少年患者，强化免疫抑制治疗后进行纯化血液CD34+细胞自体移植可能是可以接受的，以实现身心成熟的无药期。
