SLIDE 10 4/21/2018 10
PATENT trials
Riociguat vs placebo in adults with PAH
treatment naïve or pre-treated with ERAs or non-IV prostanoids demonstrated significant improvement of exercise capacity, PVR, serum NT-proBNP, WHO functional class, time to clinical worsening, and Borg dyspnea score
PATENT-1: phase II - safe and well tolerated; phase III at 12 weeks steady dose demonstrated significantly decreased PVR and increased CI PATENT-2: well tolerated; sustained improvements in exercise capacity and functional capacity for up to 1 year
Gail N et al. J Heart Lung Transplant 2017 Rubin L et al. Eur Respir J 2015
Peds and Riociguat
no clinical data on the use of Riociguat and its titration, adverse effects, and efficiency in children with PAH not recommended by Cochrane review based on lack
case report by Spreeman et al of successful introduction of Riociguat in a now 4 yo boy with severe IPAH failing traditional therapy
Spreeman et al. Pulmonary Circ 2017
Peds and Riociguat
Spreeman et al. Pulmonary Circ 2017
10 mo 4 y Birth 2y 10 mo 3.5 y
Suprasystemic RVsP RV failure RHC severe PAH AVT positive CCB/Bosentan Systemic PAH AVT negative Bosentan/Sildenafil Rpt RHC - very vasoreactive 1) PVRi 23.48 WU/m2 and PVR/SVR 1.59 2) PVRi 5.89WU/m2 and PVR/SVR 0.93 Bosentan/Riociguat Decreased PVR/SVR to 1.21 and TPG, RVH, PAAT, and LVei improvement Functional Class II/III to I
Peds and Riociguat
The rationale to “switch this child with suprasystemic PAH and high PVR/SVR from Sildenafil to Riociguat was based on: (1) the impressive hemodynamic response to iNO & O2 (vs. O2 alone) during cardiac catheterization (for PAP reduction) (2) the preceding insufficient response to the combination of ERA and Sildenafil despite normal plasma L-arginine concentration”
Spreeman et al. Pulmonary Circ 2017
