Pulmonary vascular remodeling and arterial hypertension (PAH) correlate to increased platelet-derived growth factor (PDGF) activity and elevated KIT expression. Imatinib has emerged as a potential therapeutic agent for PAH. The purpose of this systematic review and meta-analysis was to assess the effectiveness of imatinib in treatment of PAH. A literature search was conducted with the PubMed, Embase, Web of Science, and Cochrane Library to obtain randomized controlled trials (RCTs) where the efficacy of imatinib and placebo in PAH patients was compared. Three RCTs that involved 262 patients were finally included in this study. Results showed that imatinib significantly improved six-minute walk distance (MD = 42.76, 95% CI [9.20 - 76.32], P = 0.01), reduced pulmonary vascular resistance (MD = -396.68, 95% CI [-474.50 - -318.85], P < 0.00001), and lowered mean pulmonary arterial pressure (MD = -7.29, 95% CI [-13.97 - -0.61], P = 0.03) in PAH patients. No significant difference was found between the imatinib and placebo groups in terms of mortality (OR = 1.25, 95% CI [0.49 - 3.18]) or adverse events (OR = 1.82, 95% CI [0.76 - 4.36], P = 0.18). Despite the significant improvement of key hemodynamic parameters, there was no advantage in reducing clinical adverse events or mortality. The prolonged efficacy and safety of imatinib in PAH patients warrants further studies.
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