December 27, 2018
Fewer patients in the high-dose treprostinil group had clinical worsening.
Patients with chronic thromboembolic pulmonary hypertension (CTEPH) treated with high-dose subcutaneous treprostinil, a prostacyclin analogue, had increased 6-minute walk distances compared with low-dose subcutaneous treprostinil, according to the results of a study published in Lancet Respiratory Medicine.
Six-minute walk distances were recorded from patients with CTEPH enrolled in a clinical trial (ClinicalTrials.gov Identifier: NCT01416636) from 6 European expert centers in Austria, the Czech Republic, Germany, and Poland. Participants were randomly assigned into either high-dose (target dose 30 ng/kg at week 12; n=53) or low-dose (target dose 3 ng/kg at week 12; n=52) subcutaneous treprostinil treatment groups. The primary end point was the change in baseline 6-minute walk distance from baseline between the 2 groups at 24 weeks.
Of the 138 patients screened, 105 patients met the inclusion/exclusion criteria and were enrolled into the study. At week 12, patients who received high-dose subcutaneous treprostinil had reached a mean dose of 29.15 ng/kg and patients who received low-dose subcutaneous treprostinil had reached a mean dose of 3.04 ng/kg.
At 12 weeks, the 6-minute walk distance improvement from baseline was greater, but nonsignificant, in the high-dose group compared with the low-dose group. Seven patients in the high-dose group and 12 patients in the low-dose group had clinical worsening (P =.21). At week 24, marginal mean 6-minute walk distance improved by 44.98 m in the high-dose group and by 4.29 m in the low-dose group (treatment effect, 40.69 m; 95% CI, 15.86-65.53; P =.0016).
One serious adverse event resulting from mild diarrhea or nausea was classified as possibly related to subcutaneous treprostinil.
“[O]ur data show that long-term subcutaneous treprostinil is safe and effective, leading to concentration-dependent improvements of 6-min walk distance, haemodynamics, [World Health Organization] functional class, and N-terminal pro-brain natriuretic peptide amounts in patients with severe non-operable CTEPH,” the researchers concluded.
Disclosures: Funding for this study was provided by SciPharm Sárl.
Sadushi-Kolici R, Jansa P, Kopec G et al. Subcutaneous treprostinil for the treatment of severe non-operable chronic thromboembolic pulmonary hypertension (CTREPH): a double-blind, phase 3, randomized controlled trial [published November 23, 2018]. Lancet Respir Med. doi:10.1016/S2213-2600(18)30367-9