Rodolfi Stefano, Ong Voon H, Denton Christopher P
Centre for Rheumatology and Connective Tissue Diseases, University College London Medical School, London, UK.
Department of Rheumatology and Clinical Immunology, IRCCS Humanitas Research Hospital, Milan, Italy.
Int J Cardiol Congenit Heart Dis. 2024 Apr 14;16:100513. doi: 10.1016/j.ijcchd.2024.100513. eCollection 2024 Jun.
Connective tissue disease associated pulmonary arterial hypertension (CTD-PAH) has benefited from the major treatment advances that have occurred within pulmonary hypertension over the past three decades. Inclusion of CTD-PAH cases in pivotal clinical trials led to regulatory approval and drug availability. This has improved outcomes but there are additional challenges for management. First, the multifaceted co-morbidity related to the associated CTD needs treatment alongside PAH and may impact on diagnosis and evaluation of treatment response. Secondary, cardiac involvement, interstitial lung disease and predisposition to thromboembolism in CTD may lead to compound phenotypes where PH has multiple mechanisms as well as precapillary pulmonary vasculopathy of PAH. In general, especially for systemic sclerosis, CTD-PAH has worse long-term survival than idiopathic or familial PAH. However, CTD also present an opportunity for screening and early detection and treatment for associated PAH, and this may in the future be a major advantage over idiopathic disease where presentation inevitable only occurs at symptomatic stages and diagnosis may be delayed. This article reviews and summarises some of the recent developments in investigation and management of CTD-PAH.
结缔组织病相关肺动脉高压（CTD-PAH）受益于过去三十年肺动脉高压领域取得的重大治疗进展。关键临床试验纳入CTD-PAH病例促使监管部门批准并使药物得以应用。这改善了治疗结果，但管理方面仍存在其他挑战。首先，与相关CTD相关的多方面合并症需要在治疗PAH的同时进行治疗，并且可能影响诊断和治疗反应评估。其次，CTD中的心脏受累、间质性肺疾病和血栓栓塞易感性可能导致复杂的表型，其中PH有多种机制以及PAH的毛细血管前肺血管病变。总体而言，尤其是对于系统性硬化症，CTD-PAH的长期生存率比特发性或家族性PAH更差。然而，CTD也为相关PAH的筛查及早期检测和治疗提供了机会，这在未来可能是相对于特发性疾病的一个主要优势，因为特发性疾病不可避免地仅在症状阶段出现，诊断可能会延迟。本文回顾并总结了CTD-PAH研究和管理方面的一些最新进展。
