Long-Term Outcomes in Patients with Connective Tissue Disease-Associated Pulmonary Arterial Hypertension in the Modern Treatment Era: Meta-Analyses of Randomized, Controlled Trials and Observational Registries. Arthritis & rheumatology (Hoboken, N.J.) Khanna, D. n., Zhao, C. n., Saggar, R. n., Mathai, S. C., Chung, L. S., Coghlan, J. G., Shah, M. n., Hartney, J. n., McLaughlin, V. n. 2021


Data on the magnitude of benefit of modern pulmonary arterial hypertension (PAH) therapies in connective tissue disease-associated PAH (CTD-PAH) are limited. We performed meta-analyses of randomized, controlled trials (RCTs) and registries to quantify this benefit (PROSPERO# CRD42020167119).PubMed and EMBASE were searched for articles reporting data from RCTs or registries published 1/1/2000-11/25/2019. Eligibility criteria included multicenter studies with =30 CTD-PAH patients. RCTs had to evaluate approved PAH therapy and report long-term clinical morbidity/mortality or 6-minute walk distance. Registries had to report survival. Random effects models were used to pool data.Eleven RCTs (N=4329 n=1267 CTD-PAH) and 19 registries (N=9739; n=4008 CTD-PAH) were included. Investigational therapy produced a 36% reduction in risk of clinical morbidity/mortality events versus control (HR=0.64; 95%CI: 0.54-0.75; P<0.001) in all patients and a 36% reduction (HR=0.64; 95% CI: 0.51-0.81; P<0.001) in CTD-PAH patients. Survival was lower in CTD-PAH versus all patients (62%, 95% CI: 57%-67% versus 72%, 95% CI: 69%-75% at 3 years). Survival in CTD-PAH patients treated primarily after 2010 was higher than in those treated before (73%, 95% CI: 62%-81% versus 65%, 95% CI: 59%-71% at 3 years).Modern therapy provides a similar reduction in morbidity/mortality risk in CTD-PAH and the overall PAH population. Risk of death is higher in CTD-PAH than in PAH overall, but survival has improved in the last 10 years, which may be related to increased screening and/or new treatment approaches. Early detection of PAH in patients with CTD and upfront intensive treatment are warranted.

View details for DOI 10.1002/art.41669

View details for PubMedID 33538058