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一名快速进展性局限性硬皮病患者成功接受自体造血干细胞移植。

Successful autologous hematopoietic stem cell transplantation for a patient with rapidly progressive localized scleroderma.

作者信息

Nair Velu, Sharma Ajay, Sharma Sanjeevan, Das Satyaranjan, Bhakuni Darshan S, Narayanan Krishnan, Nair Vivek, Shankar Subramanian

机构信息

Armed Forces Medical College, Pune, India.

出版信息

Int J Rheum Dis. 2015 Mar;18(3):366-71. doi: 10.1111/1756-185X.12555.

Abstract

Autologous hematopoietic stem cell transplant (HSCT) for rapidly progressive disease has not been reported in localized scleroderma. Our patient, a 16-year-old girl had an aggressive variant of localized scleroderma, mixed subtype (linear-generalized) with Parry Romberg syndrome, with no internal organ involvement, that was unresponsive to immunosuppressive therapy and was causing rapid disfigurement. She was administered autologous HSCT in June 2011 and has maintained drug-free remission with excellent functional status at almost 3.5 years of follow-up.

摘要

自体造血干细胞移植(HSCT)用于局限性硬皮病快速进展型疾病的情况尚未见报道。我们的患者是一名16岁女孩,患有局限性硬皮病的侵袭性变异型,为混合亚型(线状-泛发型)并伴有帕里-龙贝格综合征,无内脏器官受累,对免疫抑制治疗无反应且导致快速毁容。她于2011年6月接受了自体HSCT,在近3.5年的随访中维持了无药缓解状态,功能状态良好。

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