Kawano Noriaki, Yokota-Ikeda Naoko, Yoshida Shuro, Kuriyama Takuro, Yamashita Kiyoshi, Sugio Yasuhiro, Makino Shigeyoshi, Ono Nobuyuki, Inoue Yasushi, Himeji Daisuke, Kodama Kieko, Uezono Shigehiro, Shimao Yoshiya, Ueda Akira, Matsumoto Masanori, Iino Hisayo, Fujimura Yoshihiro
Department of Internal Medicine, Miyazaki Prefectural Hospital, Japan.
Intern Med. 2013;52(17):1883-91. doi: 10.2169/internalmedicine.52.8253.
Thrombotic thrombocytopenic purpura (TTP) is a life-threatening generalized disease with pathological features that are termed thrombotic microangiopathies. Since the discovery of the von Willebrand factor-cleaving protease [a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13 (ADAMTS13)], it is widely known that approximately two-thirds of TTP patients have a severe deficiency of ADAMTS13 activity due to gene mutations or acquired autoantibodies to this enzyme. However, the remaining one-third of TTP patients have only moderately reduced or almost normal ADAMTS13 activity. To elucidate the clinical characteristics and outcomes of these two types of TTP, we have retrospectively analyzed the cases of acquired TTP patients treated in a single institution from 2000 to 2011.
Our case studies include 11 TTP patients, of which 5 were considered idiopathic and 6 had cases of TTP associated with underlying diseases such as non-Hodgkin lymphoma or connective tissue diseases.
These patients were treated with a combination therapy of plasma exchange and steroids and with several adjunctive therapeutic regimens including the on-label use of cyclophosphamide and cyclosporine and the off-label use of high-dose steroid or immunoglobulin with rituximab. Splenectomies were not performed. As a result of these treatments, 6 out of the 7 patients with ADAMTS13 activity deficient TTP achieved a complete remission without relapse, but the remaining 4 patients with non-ADAMTS13 activity deficient TTP all died without complete remission.
We present herein the detailed clinical courses of 11 patients with TTP and address our experiences with the efficacy of various therapeutic regimens. This case-oriented study should be helpful to the physicians who directly care for TTP patients, and may provide a future direction for developing a more efficient treatment modality.
血栓性血小板减少性紫癜（TTP）是一种危及生命的全身性疾病，其病理特征被称为血栓性微血管病。自从发现血管性血友病因子裂解蛋白酶[具有血小板反应蛋白1型基序的解整合素和金属蛋白酶13（ADAMTS13）]以来，众所周知，约三分之二的TTP患者由于基因突变或针对该酶的获得性自身抗体而存在严重的ADAMTS13活性缺乏。然而，其余三分之一的TTP患者ADAMTS13活性仅中度降低或几乎正常。为了阐明这两种类型TTP的临床特征和结局，我们回顾性分析了2000年至2011年在单一机构接受治疗的获得性TTP患者的病例。
我们的病例研究包括11例TTP患者，其中5例被认为是特发性的，6例TTP病例与潜在疾病如非霍奇金淋巴瘤或结缔组织病相关。
这些患者接受了血浆置换和类固醇的联合治疗以及几种辅助治疗方案，包括环磷酰胺和环孢素的标签内使用以及高剂量类固醇或免疫球蛋白与利妥昔单抗的标签外使用。未进行脾切除术。经过这些治疗，7例ADAMTS13活性缺乏的TTP患者中有6例实现了完全缓解且无复发，但其余4例非ADAMTS13活性缺乏的TTP患者均未完全缓解而死亡。
我们在此展示了11例TTP患者的详细临床病程，并阐述了我们对各种治疗方案疗效的经验。这项以病例为导向的研究应对直接照料TTP患者的医生有所帮助，并可能为开发更有效的治疗方式提供未来方向。
