Arthritis & Tissue Degeneration Program, Hospital for Special Surgery at Weill Medical College of Cornell University, New York, NY, USA.
Haemophilia. 2014 May;20 Suppl 4:11-4. doi: 10.1111/hae.12416.
One of the main complications of haemophilia A is haemophilic arthropathy (HA), a debilitating disease with a significant negative impact on motility and quality of life. Despite major advances in the treatment of haemophilia A, many patients still suffer from HA. We wish to develop new treatments for HA, but must first better understand its causes. Our laboratory studies molecular scissors that release the pro-inflammatory cytokine tumour necrosis factor alpha (TNFα) from cells. TNFα is considered the 'fire alarm' of the body - it helps to fight infections, but can also cause diseases such as inflammatory arthritis. We know that the molecular scissors, called TNFα convertase (TACE), and its newly discovered regulator termed iRhom2 can be rapidly activated by small amounts of cytokines, growth factors, and pro-inflammatory mediators present in the blood. We hypothesize that the rapid activation of TACE could help explain one of the unsolved mysteries regarding the development of HA, which is how even small amounts of blood can provoke a persistent inflammatory response. We propose that once blood enters the joint, iRhom2 and TACE are activated to release TNFα and that this could promote the development of HA in a similar manner to that in which it promotes rheumatoid arthritis (RA). We are currently using immune cells stimulated with blood degradation products, and mouse models of HA, to test this hypothesis. If successful, our study could provide the rationale for testing anti-TNF antibodies, which are already used to treat RA, for the treatment of HA. In addition, they might uncover iRhom2 and TACE as attractive new candidate targets for the treatment of HA.
血友病 A 的主要并发症之一是血友病性关节病 (HA),这是一种使人衰弱的疾病,对运动能力和生活质量有重大负面影响。尽管在血友病 A 的治疗方面取得了重大进展,但许多患者仍患有 HA。我们希望为 HA 开发新的治疗方法,但首先必须更好地了解其病因。我们的实验室研究从细胞中释放促炎细胞因子肿瘤坏死因子 alpha (TNFα) 的分子剪刀。TNFα 被认为是身体的“火警”——它有助于对抗感染,但也会导致炎症性关节炎等疾病。我们知道,分子剪刀称为 TNFα 转化酶 (TACE),及其新发现的调节剂称为 iRhom2,可以被血液中存在的少量细胞因子、生长因子和促炎介质迅速激活。我们假设 TACE 的快速激活可以帮助解释 HA 发展中一个未解决的谜团,即即使少量血液也可以引发持续的炎症反应。我们提出,一旦血液进入关节,iRhom2 和 TACE 就会被激活以释放 TNFα,并且这可能以类似于促进类风湿性关节炎 (RA) 的方式促进 HA 的发展。我们目前正在使用用血液降解产物刺激的免疫细胞和 HA 小鼠模型来测试这一假设。如果成功,我们的研究可以为测试抗 TNF 抗体提供依据,抗 TNF 抗体已用于治疗 RA,也可用于治疗 HA。此外,它们可能会发现 iRhom2 和 TACE 是治疗 HA 的有吸引力的新候选靶点。