Department of Neurology, Nordland Hospital Trust, Bodø, Norway2Department of Clinical Medicine, UiT The Arctic University of Tromsø, Tromsø, Norway.
Cytheris SA, Issy-les-Moulineaux, France.
JAMA Neurol. 2014 Aug;71(8):1030-5. doi: 10.1001/jamaneurol.2014.825.
No reliable treatment options are known for progressive multifocal leukoencephalopathy with underlying immunodeficiency. We describe successful compassionate use of recombinant human interleukin 7 in a patient with idiopathic CD4+ T-cell lymphocytopenia.
After the diagnoses of progressive multifocal leukoencephalopathy and idiopathic CD4+ T-cell lymphocytopenia were established, a 61-year-old man was treated with recombinant human interleukin 7 on November 1, 2012. Except for an episode of epilepsia partialis continua on January 16, 2013, a gradual clinical improvement was observed until March. Abnormalities shown on magnetic resonance imaging regressed; JC virus DNA in plasma, likely originating from the brain based on sequencing data, cleared; and increases in peripheral CD4+ T cells and JC virus intrathecal antibodies were observed. One year after treatment, the CD4+ T-cell count returned to baseline and the clinical improvement waned, possibly due to the patient's complex epilepsy. On the latest evaluation on January 14, 2014, the patient's condition was unchanged, with no signs of ongoing central nervous system infection.
The present case argues strongly for proof of the treatment concept. However, deeper insight into the JC virus and its pathogenesis and the immune response during central nervous system infection as well as further clinical studies are needed before recombinant human interleukin 7 can be recommended for the treatment of other cases of immunodeficiency and progressive multifocal leukoencephalopathy.
对于潜在免疫缺陷的进行性多灶性白质脑病，目前尚无可靠的治疗选择。我们描述了一种成功的同情使用重组人白细胞介素 7 的方法，用于治疗特发性 CD4+ T 细胞淋巴细胞减少症患者。
在确诊进行性多灶性白质脑病和特发性 CD4+ T 细胞淋巴细胞减少症后，一名 61 岁男性于 2012 年 11 月 1 日接受重组人白细胞介素 7 治疗。除了 2013 年 1 月 16 日出现部分癫痫持续状态外，一直观察到逐渐的临床改善，直至 3 月。磁共振成像显示的异常消退；血浆中的 JC 病毒 DNA，根据测序数据可能源自大脑，清除；外周血 CD4+ T 细胞和 JC 病毒鞘内抗体增加。治疗 1 年后，CD4+ T 细胞计数恢复到基线水平，临床改善减弱，可能是由于患者的复杂癫痫。在 2014 年 1 月 14 日的最新评估中，患者的病情没有变化，没有持续的中枢神经系统感染迹象。
目前的病例强烈支持治疗概念的证据。然而，在推荐重组人白细胞介素 7 用于治疗其他免疫缺陷和进行性多灶性白质脑病病例之前，还需要更深入地了解 JC 病毒及其发病机制以及中枢神经系统感染期间的免疫反应，并进行进一步的临床研究。
