| Autor: |
Ivy DD; 1 Children's Hospital of Colorado, Aurora, CO, USA., Feinstein JA; 2 Lucile Packard Children's Hospital Stanford and Stanford University School of Medicine, Palo Alto, CA, USA., Yung D; 3 Seattle Children's Hospital, Seattle, WA, USA., Mullen MP; 4 Boston Children's Hospital, Boston, MA, USA., Kirkpatrick EC; 5 Children's Hospital of Wisconsin, Milwaukee, WI, USA., Hirsch R; 6 Cincinnati Children's Hospital, Cincinnati, OH, USA., Austin ED; 7 Monroe Carell Jr. Children's Hospital at Vanderbilt, Nashville, TN, USA., Fineman J; 8 University of California San Francisco, San Francisco, CA, USA., Truong U; 1 Children's Hospital of Colorado, Aurora, CO, USA., Solum D; 9 United Therapeutics, Research Triangle Park, NC, USA., Deng CQ; 9 United Therapeutics, Research Triangle Park, NC, USA., Hopper RK; 2 Lucile Packard Children's Hospital Stanford and Stanford University School of Medicine, Palo Alto, CA, USA.; 10 Children's Hospital of Philadelphia, Philadelphia, PA, USA. |
| Abstrakt: |
Treprostinil, a prostacyclin analogue, is approved for the treatment of pulmonary arterial hypertension (PAH) in adults. Transition from parenteral to oral treprostinil has been successfully accomplished in adults with PAH but not in children. In this multicenter study, pediatric patients treated with parenteral (Cohort 1) or inhaled (Cohort 2) treprostinil were transitioned to oral treprostinil. Prostacyclin-naïve individuals on background oral PAH therapy received oral treprostinil as add-on therapy (Cohort 3). Successful transition was oral treprostinil dose maintenance through week 24. Patients were monitored for adverse events (AEs), 6-min walk distance (6MWD), PAH symptoms, World Health Organization (WHO) Functional Class (FC), cardiac magnetic resonance imaging (cMRI), cardiopulmonary exercise testing (CPET), and quality of life through 24 weeks. A total of 32 patients were enrolled in the study; 23 (72%) were girls (mean age = 12.2 years). All patients were on background oral PAH therapy. Overall, patients (96.9%) maintained transition to oral treprostinil; one patient (Cohort 1) transitioned to oral treprostinil, then back to parenteral after experiencing syncope and WHO FC change from II to III. Cohorts 1, 2, and 3 received a final mean oral treprostinil dose of 5.6, 3.3, and 4.5 mg t.i.d., respectively. All cohorts had variable changes in 6MWD, cMRI, and CPET. Overall, 12 serious AEs were reported. All patients had drug-related AEs including headache (81%), diarrhea (69%), nausea (66%), vomiting (66%), and flushing (56%). Pediatric patients maintained transition to oral treprostinil with preservation of exercise capacity and WHO FC. Prostanoid-related AEs were most common and similar to those reported in adults. |
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