Efficacy and safety of balloon pulmonary angioplasty in chronic thromboembolic pulmonary hypertension associated with sickle cell disease.

BACKGROUND

Pulmonary hypertension (pH) is a severe complication of sickle cell disease (SCD) and may result from chronic thromboembolic sequelae. Balloon pulmonary angioplasty (BPA) is an established treatment for inoperable chronic thromboembolic pulmonary hypertension (CTEPH), but data on safety and efficacy in SCD-associated CTEPH are scarce.

OBJECTIVES

To assess the clinical, functional, and hemodynamic effects of BPA, and its safety profile, in patients with SCD-associated CTEPH.

METHODS

We retrospectively analyzed patients with SCD-associated CTEPH enrolled in the French pH Registry (2014-2023). Clinical status, exercise capacity, and hemodynamics were compared before and after BPA.

RESULTS

Nineteen patients with SCD-associated CTEPH were identified, 14 of whom underwent BPA. Median age was 41 years, 50% were male, and 72% had hemoglobin SC (HbSC) disease. Ten patients (71%) received pulmonary arterial hypertension-targeted therapy before BPA. After BPA, NYHA functional class improved in 10 patients. Six-minute walk distance increased from 432 m (IQR 387-469) to 473 m (IQR 431-512; p = 0.02), and pulmonary vascular resistance decreased from 5.8 WU (IQR 4.5-7.4) to 2.6 WU (IQR 1.6-3.8; p = 0.002). BPA-related complications occurred in 6 patients (43%), mainly mild hemoptysis and mild lung injury; none were life-threatening. This per-patient complication rate was similar to that in a contemporaneous non-SCD CTEPH cohort undergoing BPA.

CONCLUSIONS

This retrospective single-centre series provides evidence that BPA is a safe and effective therapeutic option for SCD-associated CTEPH, particularly in patients with HbSC disease. Larger multicenter or international registry-based studies are warranted to confirm these results and guide management of this challenging population.