PURPOSE: Despite the potential benefit of SQ treprostinil for patients with PAH, its use is not widespread in part because of side effects (e.g. site pain). Furthermore, there is limited literature describing long-term outcomes with this drug.
METHODS: We identified 33 patients who have received treatment with SQ Treprostinil since 2002. We report data comparing six-minute walk test (6MWT) at baseline vs. three months and 12 months of therapy, and brain natriuretic peptide (BNP) during the same period. We also compared changes in hemodynamics and functional class.
RESULTS: Mean age was 55 years and most patients were women (82%). Most common etiologies of PH were idiopathic PAH (39%), collagen vascular disease (24%) and chronic liver disease (18%). Twenty-eight patients were naïve to prostacyclins and 5 were transitioned from IV prostacyclin. Most of our patients were WHO functional class III and IV. Only two patients (6%) discontinued treatment because of site pain.6MWT went from 243m at baseline to 301m at 3months (n=25, p=0.002) and 336m at 12months (n=17, p=0.0002). BNP also improved from 240 pg/ml at baseline to 155 at 3months (n=23,p=0.001) and 92 (n=17, p=0.02) at 12 months. The mean peak Treprostinil dose at 12 months was 44 ng/kg/min (26-72 range). Cardiac Index went from 2.96 at baseline to 3.56 (p=0.01) after mean treatment duration of 9 months. WHO FC improved from mean of 3 to 2.1 (p=0.001).
CONCLUSION: Our data support the short and long-term clinical and hemodynamic benefit of SQ treprostinil in patients with PAH. Our success rates are in part related to faster dose escalation and higher treatment doses than those initially recommended.
CLINICAL IMPLICATIONS: Our study is somewhat unique given the limited literature describing the long-term effects of this drug. SQ treprostinil is an excellent alternative for severe PAH. It has a safe profile, good tolerance and clinical and hemodynamic benefits.
DISCLOSURE: Francisco Soto, None.