SUMMARY

• A prospective, descriptive study evaluating the safety and efficacy of OPSUMIT in patients with precapillary pulmonary hypertension (PH) due to underlying sickle cell disease (SCD; N=5) showed improvement in pulmonary hemodynamics and exercise capacity. Adverse events (AEs) were reported in all patients, with 4/5 reported as mild to moderate. No patients required hospitalization for pulmonary arterial hypertension (PAH).¹

CLINICAL DATA

Villamizar et al (2024)¹ conducted a prospective, descriptive study to determine the safety and efficacy of a 4-month treatment with OPSUMIT in patients with precapillary PH due to underlying SCD (N=5). All patients were in a stable phase of their disease at the time of evaluation, with an average age of 42.6 years, and 60% of patients were male. Prospective follow-up evaluation was performed with scheduled appointments at weeks 4, 8, 12, and 16 using laboratory analysis, 6-minute walk test (6MWT), Borg Dyspnea Scale, 36-item short form survey instrument questionnaire (SF-36), and functional class assessment. Baseline characteristics of the patients are presented in Table: Characteristics of Patients at Baseline.

Efficacy

Mean pulmonary artery pressure (mPAP), pulmonary vascular resistance (PVR), and right atrial pressure (RAP) decreased by 15.6%, 22.5%, and 25.5%, respectively. In 4 out of 5 patients, the 6-minute walk distance increased over 16 weeks. Median N-terminal prohormone of brain natriuretic peptide (NT-proBNP) levels improved by 71.3%. Serum lactate dehydrogenase (LDH) levels decreased by 7.1%. There was no significant increase in aspartate aminotransferase and alanine aminotransferase from baseline. Effect of OPSUMIT on Borg Dyspnea Scale was variable, which is summarized in Table: Effect of OPSUMIT on Hemodynamics and Exercise Capacity.

Safety

Five patients had AEs with OPSUMIT, of which 4 patients had mild to moderate AEs (headache, skin rash, dyspnea, nasopharyngitis, epistaxis, lower extremity edema, decreased hemoglobin) that did not lead to drug discontinuation. Therapy was discontinued in 1 patient due to worsening anemia (serious event), however it was uncertain whether it was attributable to study drug. During the 16 week study period, there were no hospitalizations for PAH.

Literature Search

A literature search of MEDLINE^®, EMBASE^®, BIOSIS Previews^®, DERWENT^® (and/or other resources, including internal/external databases) was conducted on 27 May 2024.