Add-on sotatercept beneficial in paediatric pulmonary hypertension

Sotatercept, when added to background therapy, helps improve outcomes for children with pulmonary hypertension, as shown in a retrospective study.

The study included 66 children and young adults ≤21 years of age (median age at sotatercept initiation 15.3 years, 60 percent female) with paediatric pulmonary hypertension treated by programmes in the Pediatric PH Network and institutional registries. These patients received sotatercept therapy for a median of 323 days.

Before starting sotatercept, World Health Organization (WHO) functional class (FC) was class I in 3 percent of patients, class II in 48 percent, class III in 42 percent, and class IV in 6 percent. The median 6-min walk distance (6MWD) was 466 m, the median b-type natriuretic peptide (BNP) was 34.2 pg/ml, and the median N-terminal Pro-BNP (NT-proBNP) was 301 pg/ml.

Over a median sotatercept treatment duration of 150 days, WHO FC improved in 51 percent of patients, with no worsening observed in any of the remaining patients (p<0.0001). Patients also showed significant improvements in 6MWD (median, 31 m; p=0.0003), NT-proBNP (median, –378 pg/ml; p< 0.0001), mean pulmonary artery pressure (median, –17 mm Hg; p<0.0001), and pulmonary vascular resistance (median, –4.4 WUi; p=0.0011). Parenteral prostacyclin was weaned or discontinued in 13 of 38 patients.

Adverse events occurred in 50 percent of patients, with bleeding being the most common (23 percent). None of the patients required escalation of pulmonary hypertension therapy, had to be hospitalized for clinical worsening, underwent lung transplant, or died.