Original clinical scienceLong-term treatment, tolerability, and survival with sub-cutaneous treprostinil for severe pulmonary hypertension
Section snippets
Patients and methods
The database used in this study has been under the auspices of the Ethics Committee of the Medical University of Vienna (#972/2009). Patients provided written informed consent. Patient disposition is shown in Figure 1.
Patient characteristics
Between June 1, 1999, and November 30, 2010, SC treprostinil was started in 111 patients with severe pre-capillary PH (Table 1). There was an 11.7% discontinuation rate due to side effects throughout the median observation time of 56 months (IQR, 31–91 months). At baseline by the WHO FC, 62 patients (56%) were classified as III and 49 (44%) as IV. PAH was present in 69 patients (62%), categorized by a diagnosis of IPAH in 33 (29.7%), PAH associated with congenital heart disease in 21 (18.9%),
Discussion
The ability to conduct large, prospective randomized controlled trials over years with morbidity and death as primary end points for PH is limited by patient numbers, economic constraints, and ethical constraints to using a placebo. Therefore, all information and evidence leading to novel drug approval have been based on 12- to 24-week studies and their open-label phases. This is a particular shortfall for SC treprostinil, demonstrating a between-treatment group difference in median 6MWD of
Disclosure statement
R.S.-K. has served as a paid consultant for AOPOrphan Pharmaceuticals AG and has received compensation for scientific symposia from Actelion, GlaxoSmithKline, and AOPOrphan Pharmaceuticals AG. N.S.-S. has served as a paid consultant for Actelion, Nycomed, Pfizer, GlaxoSmithKline, United Therapeutics Corporation, and AOPOrphan Pharmaceuticals AG and has received educational grants and compensations for scientific symposia from Actelion, Nycomed, Pfizer, GSK, United Therapeutics Corporation, and
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2020, International Journal of CardiologyCitation Excerpt :The average reduction in PVR after 3 to 6 months reported in the active arm of RCTs with oral monotherapies is around 20–30% from baseline [39–49]. These results were confirmed when oral drugs were tested in a real-world setting [50–55], whereas a greater reduction was reported for high dose treprostinil s.c. and epoprostenol i.v. compared with the oral drugs [34,56–60]. Sequential combination of oral drugs has been tested in the context of limited RCTs [40,42,43,46,61–63,71,72] and real-life studies [57,64,65] confirming the supplementary effect of add-on therapy on reducing PVR, thus suggesting an overall strategy effect on hemodynamic parameters rather than a class effect able to distinguish between various targeted oral drugs.
Subcutaneous treprostinil for the treatment of severe non-operable chronic thromboembolic pulmonary hypertension (CTREPH): a double-blind, phase 3, randomised controlled trial
2019, The Lancet Respiratory MedicineCitation Excerpt :Limitations of the study include the small sample size. The CTREPH trial stipulated a maximum dose, however, higher doses and more individualised treprostinil dosing schemes might have been more effective.21,37 Change in 6-min walk distance might not translate into long-term outcomes in pulmonary arterial hypertension38,39 and CTEPH.
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2018, Journal of Heart and Lung TransplantationCitation Excerpt :Standardization of the implantation procedure and peri-operative care and the further development of fully implantable pump systems merit additional evaluation. Our study included a distinct group of patients with advanced PH. Their overall survival is almost comparable to previously reported outcomes in patients receiving intravenous iloprost,22 intravenous epoprostenol,24–26 and sub-cutaneous treprostinil27 and in a registry of various PH sub-groups.28 Transplant-free survival was substantially lower and is comparable to the overall survival of patients with PAH categorized as high risk.29
Treatment strategies in pulmonary arterial hypertension
2017, Revista Colombiana de Cardiologia