June 2024 PVRI Community Call with Julien Grynblat, David Montani and Stravros Zanos

Pulmonary arterial hypertension (PAH) requires lifelong medication, with patients taking an average of 12 tablets/day. Several chronic diseases can be treated with a fixed-dose combination (FDC) tablet, decreasing patient pill burden and increasing adherence.

Substantial evidence from animal models supports the concept of inhibiting peripheral serotonin synthesis for the treatment of pulmonary arterial hypertension (PAH), as demonstrated by pharmacological blockade or genetic deletion of tryptophan hydroxylase 1 (TPH1) [1-5]. Most recently, we have shown that daily inhalation of TPT-004, a novel class TPH1 inhibitor, can alleviate PAH in the Sugen-hypoxia (SuHx) rat model [1]. 

The aim of the open label extension (OLE) of CTREPH study was to characterize multimodal treatment in patients with severe inoperable CTEPH, to describe long-term subcutaneous (SC) treprostinil safety and tolerability, and to evaluate change in functional class and exercise capacity over 24 months since completion of the blinded phase of CTREPH.

Kholdani and colleagues describe patient characteristics, completeness of diagnostic testing, treatment patterns, risk characteristics, and outcomes of a large cohort of patients with pulmonary arterial hypertension seen at an academic pulmonary hypertension center over a 20-year period.