Mingot Castellano M E
Servicio de Hematología y Hemoterapia, Hospital Regional Universitario Carlos Haya, Malaga.
Methods Find Exp Clin Pharmacol. 2010 Dec;32 Suppl A:39-43.
Primary Immune thrombocytopenia or idiopathic thrombocytopenic purpura (ITP) is an acquired immune disorder presenting with abnormal hemorrhagic symptoms resulting from a decrease in the number of platelets. The disorder used to be attributed to increased destruction of platelets mediated by antibodies. In the past few years, the description of its etiopathology has changed. A deficiency in the marrow production of thrombocytes has been demonstrated; because it is associated with increased peripheral platelet destruction, the deficiency cannot be compensated. These findings have justified the realization of studies assessing the utility of second generation thrombopoietin analogues for the treatment of these patients. These drugs include romiplostim or AMG 537 (Nplate), a peptidic analogue that stimulates the thrombopoietin receptor and induces an increase In the production and differentiation of megakaryocytes. Data obtained from the clinical trials that led to the authorization and subsequent follow-up describe romiplostin as an effective and safe drug for adult patients with chronic ITR The overall response rate is 94%; despite variations in the levels of platelets throughout treatment, 50% of patients maintain the response 95% of the time, and 78% of patients discontinue or significantly reduce the use of rescue treatment. The most common adverse event is headache. Reticulin fibrosis has been described, which is reversible after treatment discontinuation.
原发性免疫性血小板减少症或特发性血小板减少性紫癜(ITP)是一种获得性免疫疾病,表现为因血小板数量减少而出现异常出血症状。该疾病过去被认为是由抗体介导的血小板破坏增加所致。在过去几年中,其病因病理学的描述发生了变化。已证实骨髓中血小板生成不足;由于它与外周血小板破坏增加相关,这种不足无法得到补偿。这些发现为开展评估第二代血小板生成素类似物治疗这些患者效用的研究提供了依据。这些药物包括罗米司亭或AMG 537(Nplate),一种刺激血小板生成素受体并诱导巨核细胞生成和分化增加的肽类类似物。从导致该药物获批及后续随访的临床试验中获得的数据表明,罗米司亭对成年慢性ITR患者是一种有效且安全的药物。总体缓解率为94%;尽管在整个治疗过程中血小板水平存在波动,但50%的患者在95%的时间里维持缓解状态,78%的患者停止或显著减少了急救治疗的使用。最常见的不良事件是头痛。已出现网状纤维组织增生,停药后可逆转。