Teachey David T, Greiner Robert, Seif Alix, Attiyeh Edward, Bleesing Jack, Choi John, Manno Catherine, Rappaport Eric, Schwabe Dirk, Sheen Cecilia, Sullivan Kathleen E, Zhuang Hongming, Wechsler Daniel S, Grupp Stephan A
Pediatric Hematology and Oncology, Children's Hospital of Philadelphia, University of Pennsylvania School of Medicine, Philadelphia PA 19104, USA.
Br J Haematol. 2009 Apr;145(1):101-6. doi: 10.1111/j.1365-2141.2009.07595.x. Epub 2009 Feb 4.
We hypothesized that sirolimus, an mTOR inhibitor, may be effective in patients with autoimmune lymphoproliferative syndrome (ALPS) and treated patients who were intolerant to or failed other therapies. Four patients were treated for autoimmune cytopenias; all had a rapid complete or near complete response. Two patients were treated for autoimmune arthritis and colitis, demonstrating marked improvement. Three patients had complete resolution of lymphadenopathy and splenomegaly and all patients had a reduction in double negative T cells, a population hallmark of the disease. Based on these significant responses, we recommend that sirolimus be considered as second-line therapy for patients with steroid-refractory disease.
我们推测，雷帕霉素（一种mTOR抑制剂）可能对自身免疫性淋巴细胞增生综合征（ALPS）患者以及对其他疗法不耐受或治疗失败的患者有效。4例患者接受了自身免疫性血细胞减少症的治疗；所有患者均迅速获得完全或近乎完全缓解。2例患者接受了自身免疫性关节炎和结肠炎的治疗，病情有显著改善。3例患者的淋巴结病和脾肿大完全消退，所有患者的双阴性T细胞数量均减少，而双阴性T细胞是该疾病的一个特征性细胞群。基于这些显著疗效，我们建议将雷帕霉素视为激素难治性疾病患者的二线治疗药物。
