Off-label targeted drugs improve survival in CTEPH

Off-label pulmonary arterial hypertension (PAH)-targeted drugs may increase long-term survival in patients with chronic thromboembolic pulmonary hypertension (CTEPH), as shown in a study.

CTEPH patients from a prospective multicentre national registry were enrolled in this study. PAH-targeted drugs, except for licensed riociguat and treprostinil, were off-label.

The investigators compared 5-year survival in two groups in the original and propensity score‒matched (PSM) samples: patients receiving off-label PAH-targeted drugs (treatment group) and those not receiving off-label targeted drugs (control group). The latter was assessed for the impact of initiated off-label targeted drugs at baseline (initial) or during follow-up (subsequent).

A total of 347 patients were enrolled, of whom 212 received off-label PAH-targeted drugs initially (n=173) or subsequently (n=39) and 135 did not. The treatment group showed significantly better survival than the control group at 1 (97.1 percent vs 89.4 percent), 2 (92.3 percent vs 82.1 percent), 3 (83.2 percent vs 75.1 percent), and 5 years (71.1 percent vs 55.3 percent; log-rank test, p=0.005).

Notably, initial treatment was associated with improved 5-year survival after excluding patients with subsequent treatment to reduce the immortal-time bias (hazard ratio, 0.611, 95 percent confidence interval, 0.397‒0.940; p=0.025).

In PSM, patients who received initial treatment had significantly better 5-year survival than those who did not receive treatment (68.9 percent vs 49.3 percent; log-rank, p=0.008).

“Off-label PAH-targeted drugs are commonly prescribed for nonoperated CTEPH,” the investigators said.