Zver Samo, Zupan Irena Preloznik, Cernelc Peter
Department of Haematology, University Medical Center Ljubljana, Ljubljana, Slovenija.
Int J Hematol. 2006 Apr;83(3):238-42. doi: 10.1532/IJH97.05149.
The treatment of autoimmune thrombocytopenic purpura (AITP) remains unsatisfactory in patients refractory to first-line management such as corticosteroid therapy and/or splenectomy. Patients with refractory AITP usually require unacceptably high doses of corticosteroids to maintain a safe platelet count. Immunosuppressive treatment with cyclosporin A (CsA) is a relatively new treatment modality, and no large studies of this drug have been conducted. We used CsA in 6 patients with refractory AITP who had platelet counts of less than 20 x 10(9)/L without any therapy or who had evidence of subcutaneous and mucosal bleeding. All 6 patients had undergone splenectomy. When CsA therapy was begun, 5 of the patients were receiving methylprednisolone (MP) at a daily dose of 32 mg or greater. During the following months, the MP dosage was tapered, or the drug was withdrawn. Three patients achieved a complete remission (CR), whereupon CsA treatment was gradually discontinued. Two of these 3 patients later relapsed, but both responded to an additional course of CsA and achieved a second CR. The remaining 3 patients achieved a partial remission (PR). One patient, a woman with an AITP history of more than 30 years, obtained a stable PR with a platelet count substantially greater than 20 x 10(9)/L, which was successfully maintained by low doses of CsA and MP. The most frequent side effect of CsA therapy in our patients was a painful edema of the lower extremities. Our experience shows that CsA is a safe and effective treatment option for patients with refractory (chronic) AITP. It may be given at a low dose as maintenance therapy, and remissions may be sustained even after the drug has been discontinued.
对于一线治疗（如皮质类固醇疗法和/或脾切除术）无效的自身免疫性血小板减少性紫癜（AITP）患者，其治疗效果仍不尽人意。难治性AITP患者通常需要使用高得难以接受的皮质类固醇剂量来维持安全的血小板计数。环孢素A（CsA）免疫抑制治疗是一种相对较新的治疗方式，目前尚未有关于该药物的大型研究。我们对6例难治性AITP患者使用了CsA，这些患者在未接受任何治疗时血小板计数低于20×10⁹/L，或有皮下及黏膜出血的证据。所有6例患者均已接受脾切除术。开始CsA治疗时，5例患者正在接受每日剂量32毫克或更高的甲泼尼龙（MP）治疗。在接下来的几个月里，逐渐减少MP剂量或停用该药物。3例患者实现完全缓解（CR），随后逐渐停用CsA治疗。这3例患者中有2例后来复发，但二者对再次使用CsA治疗均有反应并实现第二次CR。其余3例患者实现部分缓解（PR）。1例有超过30年AITP病史的女性患者获得了稳定的PR，血小板计数大幅高于20×10⁹/L，通过低剂量的CsA和MP成功维持。在我们的患者中，CsA治疗最常见的副作用是下肢疼痛性水肿。我们的经验表明，CsA是难治性（慢性）AITP患者的一种安全有效的治疗选择。它可以低剂量作为维持治疗给药，甚至在停药后缓解仍可持续。
