Zhu Jia, Su Gaixiu, Lai Jianming, Dong Boya, Kang Min, Li Shengnan, Zhou Zhixuan, Wu Fengqi
Division of Rheumatology and Immunology, The Affiliated Children's Hospital, Capital Institute of Pediatrics, Beijing, 100020, China.
Pediatr Rheumatol Online J. 2018 Nov 20;16(1):72. doi: 10.1186/s12969-018-0284-3.
To follow up the refractory juvenile dermatomyositis (JDM) with autologous hematopoietic stem cell transplantation (AHSCT) in a long time and to investigate whether AHSCT is effective and safe to treat refractory JDM.
We collected the AHSCT and follow-up data of three patients with refractory JDM who received autologous peripheral blood CD34+ cell transplantation in our hospital between June 2004 and July 2015. Those data include: hight, weight, routine blood and urine tests, ESR, CK, ALT, AST, LDH, renal functional tests, lymphocyte subpopulations, HRCT and muscle MRI. The last follow-up was done in June 2017.
All three patients had complete remission and could stop prednisone after 3-12 months. None of them relapsed at 144, 113 and 23 months follow-up. Twelve months after their AHSCT, all of their monitoring indexes have returned to normal and they have stopped all medications. Until the date of this article, none of them relapsed or need medicine.
Our study suggests that AHSCT is safe and effective in treating refractory JDM, and it can provides long term drug-free survival. However, more cases are needed for further confirmation.
长期随访难治性幼年皮肌炎（JDM）患者自体造血干细胞移植（AHSCT）的情况，探讨AHSCT治疗难治性JDM是否有效及安全。
收集2004年6月至2015年7月在我院接受自体外周血CD34+细胞移植的3例难治性JDM患者的AHSCT及随访资料。这些资料包括：身高、体重、血常规、尿常规、血沉、肌酸激酶（CK）、谷丙转氨酶（ALT）、谷草转氨酶（AST）、乳酸脱氢酶（LDH）、肾功能检查、淋巴细胞亚群、高分辨率CT（HRCT）及肌肉磁共振成像（MRI）。最后一次随访于2017年6月进行。
3例患者均完全缓解，3 - 12个月后可停用泼尼松。在144、113和23个月的随访中均无复发。AHSCT后12个月，所有监测指标均恢复正常，且均已停用所有药物。截至本文发表之日，均未复发或需要用药。
我们的研究表明，AHSCT治疗难治性JDM安全有效，可实现长期无药生存。然而，需要更多病例进一步证实。
