Riociguat for the treatment of transthyretin cardiac amyloidosis: data from a named patient use program in Austria
Patients with transthyretin cardiac amyloidosis (TTR CA) suffer from impaired exercise capacity, have a poor quality of life (QoL), and approved treatments are lacking. Stimulators of the soluble guanylate cyclase are promising new pharmaceuticals in the treatment armamentarium of heart failure pati...
| Main Authors: | , , , , , , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Wiley
2019-11-01
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| Series: | Pulmonary Circulation |
| Online Access: | https://doi.org/10.1177/2045894019849394 |
| _version_ | 1818247743186927616 |
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| author | Franz Duca Stefan Aschauer Caroline Zotter-Tufaro Christina Binder Andreas A. Kammerlander Benedikt Börries Hermine Agis Renate Kain Christian Hengstenberg Julia Mascherbauer Diana Bonderman |
| author_facet | Franz Duca Stefan Aschauer Caroline Zotter-Tufaro Christina Binder Andreas A. Kammerlander Benedikt Börries Hermine Agis Renate Kain Christian Hengstenberg Julia Mascherbauer Diana Bonderman |
| author_sort | Franz Duca |
| collection | DOAJ |
| description | Patients with transthyretin cardiac amyloidosis (TTR CA) suffer from impaired exercise capacity, have a poor quality of life (QoL), and approved treatments are lacking. Stimulators of the soluble guanylate cyclase are promising new pharmaceuticals in the treatment armamentarium of heart failure patients. The aim of the present study was to report on the safety and efficacy of riociguat administration in patients with TTR CA. TTR CA patients received riociguat for 4–6 months within the frames of a national named patient use (NPU) program. Parameters of interest included changes in submaximal exercise capacity, invasive hemodynamic parameters, and QoL. Between March 2012 and June 2017, 86 CA patients were screened for the NPU program, of whom 13 TTR CA patients were eligible for participation. In our study cohort, riociguat had an acceptable tolerability profile. At follow-up, we could detect slight improvements in median 6-min walk distance (396 m [interquartile range (IQR) = 340–518] vs. 400 m [IQR = 350–570], P = 0.045), New York Heart Association class ≥ III (n = 7 [53.9%] vs. n = 0 [0.0%], P = 0.031), cardiac output (4.3 L/min [IQR = 3.9–5.1] vs. 4.5 L/min [IQR = 4.2–5.1], P = 0.022), diastolic pressure gradient (1.0 mmHg [IQR = −1.5–3.0) vs. −1.0 mmHg [IQR = −3.0–1.0], P = 0.049), and QoL (50.0% [IQR = 40.0–58.0] vs. 60.0% [IQR = 50.0–75.0], P = 0.021). Pulmonary arterial pressures were not altered. The present case series of TTR CA patients indicates that riociguat administration was safe and associated with minor clinical as well as hemodynamic improvements. |
| first_indexed | 2024-12-12T15:09:33Z |
| format | Article |
| id | doaj.art-89ed84ed4f8f4cbd9478e5d1589fb287 |
| institution | Directory Open Access Journal |
| issn | 2045-8940 |
| language | English |
| last_indexed | 2024-12-12T15:09:33Z |
| publishDate | 2019-11-01 |
| publisher | Wiley |
| record_format | Article |
| series | Pulmonary Circulation |
| spelling | doaj.art-89ed84ed4f8f4cbd9478e5d1589fb2872022-12-22T00:20:39ZengWileyPulmonary Circulation2045-89402019-11-01910.1177/2045894019849394Riociguat for the treatment of transthyretin cardiac amyloidosis: data from a named patient use program in AustriaFranz Duca0Stefan Aschauer1Caroline Zotter-Tufaro2Christina Binder3Andreas A. Kammerlander4Benedikt Börries5Hermine Agis6Renate Kain7Christian Hengstenberg8Julia Mascherbauer9Diana Bonderman10Division of Cardiology, Department of Internal Medicine II, Medical University of Vienna, Vienna, AustriaDivision of Cardiology, Department of Internal Medicine II, Medical University of Vienna, Vienna, AustriaDivision of Cardiology, Department of Internal Medicine II, Medical University of Vienna, Vienna, AustriaDivision of Cardiology, Department of Internal Medicine II, Medical University of Vienna, Vienna, AustriaDivision of Cardiology, Department of Internal Medicine II, Medical University of Vienna, Vienna, AustriaDivision of Cardiology, Department of Internal Medicine II, Medical University of Vienna, Vienna, AustriaDivision of Oncology, Department of Internal Medicine I, Medical University of Vienna, Vienna, AustriaClinical Institute of Pathology, Medical University of Vienna, Vienna, AustriaDivision of Cardiology, Department of Internal Medicine II, Medical University of Vienna, Vienna, AustriaDivision of Cardiology, Department of Internal Medicine II, Medical University of Vienna, Vienna, AustriaDivision of Cardiology, Department of Internal Medicine II, Medical University of Vienna, Vienna, AustriaPatients with transthyretin cardiac amyloidosis (TTR CA) suffer from impaired exercise capacity, have a poor quality of life (QoL), and approved treatments are lacking. Stimulators of the soluble guanylate cyclase are promising new pharmaceuticals in the treatment armamentarium of heart failure patients. The aim of the present study was to report on the safety and efficacy of riociguat administration in patients with TTR CA. TTR CA patients received riociguat for 4–6 months within the frames of a national named patient use (NPU) program. Parameters of interest included changes in submaximal exercise capacity, invasive hemodynamic parameters, and QoL. Between March 2012 and June 2017, 86 CA patients were screened for the NPU program, of whom 13 TTR CA patients were eligible for participation. In our study cohort, riociguat had an acceptable tolerability profile. At follow-up, we could detect slight improvements in median 6-min walk distance (396 m [interquartile range (IQR) = 340–518] vs. 400 m [IQR = 350–570], P = 0.045), New York Heart Association class ≥ III (n = 7 [53.9%] vs. n = 0 [0.0%], P = 0.031), cardiac output (4.3 L/min [IQR = 3.9–5.1] vs. 4.5 L/min [IQR = 4.2–5.1], P = 0.022), diastolic pressure gradient (1.0 mmHg [IQR = −1.5–3.0) vs. −1.0 mmHg [IQR = −3.0–1.0], P = 0.049), and QoL (50.0% [IQR = 40.0–58.0] vs. 60.0% [IQR = 50.0–75.0], P = 0.021). Pulmonary arterial pressures were not altered. The present case series of TTR CA patients indicates that riociguat administration was safe and associated with minor clinical as well as hemodynamic improvements.https://doi.org/10.1177/2045894019849394 |
| spellingShingle | Franz Duca Stefan Aschauer Caroline Zotter-Tufaro Christina Binder Andreas A. Kammerlander Benedikt Börries Hermine Agis Renate Kain Christian Hengstenberg Julia Mascherbauer Diana Bonderman Riociguat for the treatment of transthyretin cardiac amyloidosis: data from a named patient use program in Austria Pulmonary Circulation |
| title | Riociguat for the treatment of transthyretin cardiac amyloidosis: data from a named patient use program in Austria |
| title_full | Riociguat for the treatment of transthyretin cardiac amyloidosis: data from a named patient use program in Austria |
| title_fullStr | Riociguat for the treatment of transthyretin cardiac amyloidosis: data from a named patient use program in Austria |
| title_full_unstemmed | Riociguat for the treatment of transthyretin cardiac amyloidosis: data from a named patient use program in Austria |
| title_short | Riociguat for the treatment of transthyretin cardiac amyloidosis: data from a named patient use program in Austria |
| title_sort | riociguat for the treatment of transthyretin cardiac amyloidosis data from a named patient use program in austria |
| url | https://doi.org/10.1177/2045894019849394 |
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