Common-sense combination therapy in refractory immune thrombocytopaenia.
Common-sense combination therapy in refractory immune thrombocytopaenia.
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难治性免疫性血小板减少症的常识性联合治疗。
DOI：
10.1111/bjh.18919
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发表时间：
2023
影响因子：
6.5
通讯作者：
Bussel,JamesB
中科院分区：
文献类型：
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作者：
Al-Samkari,Hanny;Bussel,JamesB
1979年，瑞士两名患有维斯科特-奥尔德里奇综合征的男孩接受了静脉注射免疫球蛋白（IVIG），以帮助治疗病毒性疾病。令所有人吃惊的是，两个男孩的血小板计数都显著增加了。Drs。Imbach、Barandun等人认识到了这一点，Imbach博士随后给他的一位慢性免疫性血小板减少症（ITP）患者注射了IVIG。该患者的血小板计数也有所改善，最终于1981年6月首次发表了IVIG在13例急性和慢性ITP患儿中的成功应用(1)。IVIG已成为ITP和其他自身免疫性疾病的一种众所周知且被广泛接受的治疗方法，但与此相关的是，这是最后一种偶然发现的ITP治疗方法。从那时起，基本上所有治疗病人的尝试都是由行动机制驱动的。这并不是说每种治疗方法的确切作用机制都是完全已知的；远非如此，但至少对主要针对的疾病病理生理学方面有合理的理解。因此，当考虑ITP中的同时多药治疗（所谓的“联合治疗”）时，如果有一种“疯狂的方法”而不是简单的方便组合，成功的前景可能会得到优化。这就是我们在Crickx及其同事在这一期《BJH》上的研究中所看到的(2)：一种治疗通过免疫抑制来减少血小板破坏，另一种治疗通过促血小板生成来增加血小板产生，这种常见性的组合可以达到治疗“难治性”ITP的高成功率。
In 1979, two boys with Wiskott-Aldrich syndrome in Switzerland were given intravenous immunoglobulin (IVIG) to help treat a viral illness. To everyone’s surprise, the platelet counts of both boys increased dramatically. Drs. Imbach, Barandun and others recognized this and Dr. Imbach subsequently gave IVIG to one of his patients with chronic immune thrombocytopenia (ITP). This patient’s platelet count also improved, and eventually the first publication on the successful use of IVIG in 13 children with acute and chronic ITP appeared in June 1981 (1). IVIG has become a well-known and widely accepted treatment in ITP and now other autoimmune diseases, but the relevance of the story is that this was the last ITP treatment “discovered” serendipitously. Since then, essentially all attempts to treat patients have been largely mechanism-of-action-driven. This is not to say that the exact mechanism of effect is completely known for each therapeutic; far from it, but there is at least reasonable understanding as to the aspect of the disease pathophysiology that is being primarily targeted. Thus, when considering simultaneous multidrug therapy (so-called “combination therapy”) in ITP, prospects for success may be optimized when there is a “method to the madness” rather than simply combinations of convenience. This is what we see in the study by Crickx and colleagues in this issue of BJH (2): high rates of success in treating “refractory” ITP may be achieved with a common-sense combination of one treatment to reduce platelet destruction via immunosuppression with another to increase platelet production via thrombopoietic stimulation.