Rosewich Hendrik, Nessler Stefan, Brück Wolfgang, Gärtner Jutta
Division of Pediatric Neurology, Department of Pediatrics and Adolescent Medicine, University Medical Center Göttingen, Georg August University, Robert Koch Strasse 40, Göttingen, 37075, Germany.
Institute of Neuropathology, University Medical Center Göttingen, Georg August University, Germany.
Ther Adv Neurol Disord. 2019 Aug 14;12:1756286419868133. doi: 10.1177/1756286419868133. eCollection 2019.
Rituximab exerts its clinical efficacy by its specific pattern of depletion of CD20 B lymphocytes and it has been demonstrated that rituximab is an effective treatment for relapsing remitting multiple sclerosis. X-linked adrenoleukodystrophy (X-ALD), the most common monogenetic neuroinflammatory disorder, shares substantial overlap with multiple sclerosis in the neuropathological changes found in brain tissues in advanced stages of the disease. While there is no effective therapy for these patients, we hypothesized that rituximab might be effective in arresting the neuroinflammatory process. Our detailed clinical, imaging and immunological data revealed that rituximab is not effective in advanced stages of X-ALD and consequently should not be applied for compassionate use in these patients.
利妥昔单抗通过其特异性清除CD20 B淋巴细胞的方式发挥临床疗效，并且已经证明利妥昔单抗是复发缓解型多发性硬化症的有效治疗方法。X连锁肾上腺脑白质营养不良（X-ALD）是最常见的单基因神经炎症性疾病，在疾病晚期脑组织的神经病理变化方面与多发性硬化症有很大重叠。虽然这些患者目前没有有效的治疗方法，但我们推测利妥昔单抗可能对阻止神经炎症过程有效。我们详细的临床、影像学和免疫学数据显示，利妥昔单抗在X-ALD晚期无效，因此不应在这些患者中出于同情用药的目的使用。
