Recovery From Severe Heart Failure in a Patient With Primary Hyperoxaluria Type 1 After Treatment With Lumasiran, Pyridoxine, and Kidney Transplant
Combined liver and kidney transplant has long been the only therapeutic option for patients with primary hyperoxaluria type 1 (PH1) and advanced chronic kidney disease. The development of lumasiran, a liver-directed RNA interference therapy, has led to effective reduction of hepatic oxalate producti...
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| Format: | Article |
| Language: | English |
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American College of Physicians
2024-06-01
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| Series: | Annals of Internal Medicine: Clinical Cases |
| Online Access: | https://www.acpjournals.org/doi/10.7326/aimcc.2023.1428 |
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| author | Mira Choi Andreas Kahl Gerlineke Hawkins-van der Cingel Maria de las Mercedes Noriega Karin Klingel Patrick Doeblin Felix Schoenrath Kai-Uwe Eckardt Robert Öllinger Felix Knauf Fabian Halleck |
| author_facet | Mira Choi Andreas Kahl Gerlineke Hawkins-van der Cingel Maria de las Mercedes Noriega Karin Klingel Patrick Doeblin Felix Schoenrath Kai-Uwe Eckardt Robert Öllinger Felix Knauf Fabian Halleck |
| author_sort | Mira Choi |
| collection | DOAJ |
| description | Combined liver and kidney transplant has long been the only therapeutic option for patients with primary hyperoxaluria type 1 (PH1) and advanced chronic kidney disease. The development of lumasiran, a liver-directed RNA interference therapy, has led to effective reduction of hepatic oxalate production in patients with PH1. Despite reports of single cases successfully treated with isolated kidney transplant and lumasiran therapy, data are still scarce. Here, we report the case of a patient with PH1, severe cardiomyopathy, and chronic kidney disease, whose cardiac function improved remarkably after receiving an isolated kidney transplant and therapy with lumasiran and pyridoxine. |
| format | Article |
| id | doaj-art-733efa87f4d4490a8af19f8dab899710 |
| institution | OA Journals |
| issn | 2767-7664 |
| language | English |
| publishDate | 2024-06-01 |
| publisher | American College of Physicians |
| record_format | Article |
| series | Annals of Internal Medicine: Clinical Cases |
| spelling | doaj-art-733efa87f4d4490a8af19f8dab8997102025-08-20T02:11:45ZengAmerican College of PhysiciansAnnals of Internal Medicine: Clinical Cases2767-76642024-06-013610.7326/aimcc.2023.1428Recovery From Severe Heart Failure in a Patient With Primary Hyperoxaluria Type 1 After Treatment With Lumasiran, Pyridoxine, and Kidney TransplantMira Choi0Andreas Kahl1Gerlineke Hawkins-van der Cingel2Maria de las Mercedes Noriega3Karin Klingel4Patrick Doeblin5Felix Schoenrath6Kai-Uwe Eckardt7Robert Öllinger8Felix Knauf9Fabian Halleck101Department of Nephrology and Medical Intensive Care, Charité – Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany1Department of Nephrology and Medical Intensive Care, Charité – Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany1Department of Nephrology and Medical Intensive Care, Charité – Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany2Institute of Pathology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany3Cardiopathology, Institute for Pathology and Neuropathology, University Hospital Tuebingen, Tuebingen, Germany4Department of Cardiology, Angiology and Intensive Care Medicine, Deutsches Herzzentrum der Charité, Berlin, Germany5Department of Cardiothoracic and Vascular Surgery, Deutsches Herzzentrum der Charité, Berlin, Germany1Department of Nephrology and Medical Intensive Care, Charité – Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany6Department of Surgery, Charité – Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany1Department of Nephrology and Medical Intensive Care, Charité – Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany1Department of Nephrology and Medical Intensive Care, Charité – Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, GermanyCombined liver and kidney transplant has long been the only therapeutic option for patients with primary hyperoxaluria type 1 (PH1) and advanced chronic kidney disease. The development of lumasiran, a liver-directed RNA interference therapy, has led to effective reduction of hepatic oxalate production in patients with PH1. Despite reports of single cases successfully treated with isolated kidney transplant and lumasiran therapy, data are still scarce. Here, we report the case of a patient with PH1, severe cardiomyopathy, and chronic kidney disease, whose cardiac function improved remarkably after receiving an isolated kidney transplant and therapy with lumasiran and pyridoxine.https://www.acpjournals.org/doi/10.7326/aimcc.2023.1428 |
| spellingShingle | Mira Choi Andreas Kahl Gerlineke Hawkins-van der Cingel Maria de las Mercedes Noriega Karin Klingel Patrick Doeblin Felix Schoenrath Kai-Uwe Eckardt Robert Öllinger Felix Knauf Fabian Halleck Recovery From Severe Heart Failure in a Patient With Primary Hyperoxaluria Type 1 After Treatment With Lumasiran, Pyridoxine, and Kidney Transplant Annals of Internal Medicine: Clinical Cases |
| title | Recovery From Severe Heart Failure in a Patient With Primary Hyperoxaluria Type 1 After Treatment With Lumasiran, Pyridoxine, and Kidney Transplant |
| title_full | Recovery From Severe Heart Failure in a Patient With Primary Hyperoxaluria Type 1 After Treatment With Lumasiran, Pyridoxine, and Kidney Transplant |
| title_fullStr | Recovery From Severe Heart Failure in a Patient With Primary Hyperoxaluria Type 1 After Treatment With Lumasiran, Pyridoxine, and Kidney Transplant |
| title_full_unstemmed | Recovery From Severe Heart Failure in a Patient With Primary Hyperoxaluria Type 1 After Treatment With Lumasiran, Pyridoxine, and Kidney Transplant |
| title_short | Recovery From Severe Heart Failure in a Patient With Primary Hyperoxaluria Type 1 After Treatment With Lumasiran, Pyridoxine, and Kidney Transplant |
| title_sort | recovery from severe heart failure in a patient with primary hyperoxaluria type 1 after treatment with lumasiran pyridoxine and kidney transplant |
| url | https://www.acpjournals.org/doi/10.7326/aimcc.2023.1428 |
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