Balloon pulmonary angioplasty combined with riociguat for the treatment of inoperable chronic thromboembolic pulmonary hypertension (PRACTICE study)

Recent overseas prospective studies have confirmed the efficacy of BPA in inoperable CTEPH, but studies from China remain limited. Single center, randomized controlled trial enrolling patients with inoperable CTEPH, assigned 2:1 to BPA plus riociguat (B) or riociguat monotherapy (R), with 12-month follow-up from treatment initiation. The primary endpoint is the change in pulmonary vascular resistance (PVR) from baseline to month 12. Key secondary outcomes assess 12‑month changes from baseline in mean pulmonary arterial pressure (mPAP), six‑minute walking distance, WHO functional classification, NT‑proBNP levels, as well as additional measures of cardiovascular hemodynamics. A total of 86 individuals diagnosed with inoperable CTEPH were enrolled and randomly allocated to two cohorts: the B cohort ( n = 54; age 63.5 ± 11.8 years; 38.9% male) and the R cohort ( n = 32; mean age 65.4 ± 11.0 years; 26.7% male). Patients from B group underwent an average of 3.8 (SD 1.0) BPA procedures. In both groups, 41 patients (76%) in the B group and 23 patients (72%) in the R group were able to reach a riociguat dose of 2.5 mg three times daily. At 12 months, PVR had improved by − 5.00 WU (SE 0.53) in the B group compared with − 2.61 WU (1.35) in the R group from baseline (group difference–2.97 WU (95% CI − 5.09 to − 0.84; p = 0.007). Over the 12-month follow-up, the B group experienced a mean reduction in mPAP of 16.11 mmHg (1.41), whereas the R group showed a decrease of 8.22 mmHg (3.32). The adjusted between-group difference in mPAP change was − 7.82 mmHg (95% CI − 15.3 to − 0.37; p = 0.04). Seven cases of clinical worsening of pulmonary hypertension occurred in the R group, whereas only one patient experienced clinical worsening in the B group. The predominant adverse events—comprising haemoptysis, blood-stained sputum, or pulmonary bleeding—were reported in 16 patients (30%) in the B group and in one patient (3%) in the R group. Across 204 BPA sessions performed in 54 individuals, procedure-related complications occurred in 16 sessions (8%), affecting a total of 14 patients (26%). Compared with riociguat alone, BPA combined with riociguat significantly improves hemodynamics in patients with inoperable CTEPH, while procedure-related complications were within manageable limits. Chinese Clinical Trial Registry ChiCTR2000032403. Registered on 27 April 2020.