背景:血小板生成素受体激动剂(TPO-RA)用于治疗原发性免疫性血小板减少症(ITP)。一些患者在维持止血血小板计数的同时停止治疗。
目标:就ITP中何时考虑逐渐减少TPO-RA达成专家共识,如何减少患者的治疗,停药后如何监测患者,以及如何重新开始治疗。
方法:我们使用了RAND/UCLA修改的Delphi面板方法。评级由每个专家在会议之前和之后独立完成。第二轮评级用于制定小组的指导。该小组是双盲的:赞助商和非主席专家不知道彼此的身份。
结果:根据患者血小板计数制定了关于儿童和成人TPO-RA何时适合锥化的指南,出血史,加强治疗,创伤风险,和使用抗凝剂/血小板抑制剂。例如,在血小板计数正常/高于正常的患者中逐渐减少TPO-RA是合适的,没有大出血史,并且在过去6个月中不需要加强治疗;对于血小板计数低的患者,不宜逐渐减少TPO-RA。ITP持续时间,在TPO-RA上的几个月,或血小板对TPO-RA的反应时机对小组关于锥度适当性的指导没有影响。关于如何减少患者的治疗的指导,停药后如何监测患者,以及如何重新开始治疗也提供了。
结论:本指南可以支持临床决策和临床试验的发展,前瞻性测试逐渐减少TPO-RA的安全性。
BACKGROUND: Thrombopoietin receptor agonists (TPO-RAs) are used to treat primary immune thrombocytopenia (ITP). Some patients have discontinued treatment while maintaining a hemostatic platelet count.
OBJECTIVE: To develop expert
consensus on when it is appropriate to consider tapering TPO-RAs in ITP, how to taper patients off therapy, how to monitor patients after discontinuation, and how to restart therapy.
METHODS: We used a RAND/UCLA modified Delphi panel method. Ratings were completed independently by each expert before and after a meeting. Second-round ratings were used to develop the panel\'s guidance. The panel was double-blinded: The sponsor and nonchair experts did not know each other\'s identities.
RESULTS: Guidance on when it is appropriate to taper TPO-RAs in children and adults was developed based on patient platelet count, history of bleeding, intensification of treatment, trauma risk, and use of anticoagulants/platelet inhibitors. For example, it is appropriate to taper TPO-RAs in patients who have normal/above-normal platelet counts, have no history of major bleeding, and have not required an intensification of treatment in the past 6 months; it is inappropriate to taper TPO-RAs in patients with low platelet counts. Duration of ITP, months on TPO-RA, or timing of platelet response to TPO-RA did not have an impact on the panel\'s guidance on appropriateness to taper. Guidance on how to taper patients off therapy, how to monitor patients after discontinuation, and how to restart therapy is also provided.
CONCLUSIONS: This guidance could support clinical decision making and the development of clinical trials that prospectively test the safety of tapering TPO-RAs.