Patisiran treatment in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy after liver transplantation
Schmidt, Hartmut Hans-Jürgen (University Hospital Essen (Alemanya))
Wixner, Jonas (Umeå University (Suècia))
Planté-Bordeneuve, Violaine (Mondor Biomedical Research Institute (França))
Muñoz-Beamud, Francisco (Hospital Universitario Juan Ramón Jimenez (Huelva))
Lladó, Laura (Institut d'Investigació Biomèdica de Bellvitge)
Gillmore, Julian D. (University College London Medical School (Regne Unit))
Mazzeo, Anna (University of Messina (Itàlia))
Li, Xingyu (Alnylam Pharmaceuticals. Inc (Cambridge, Estats Units d'Amèrica))
Arum, Seth (Alnylam Pharmaceuticals. Inc (Cambridge, Estats Units d'Amèrica))
Jay, Patrick Y. (Alnylam Pharmaceuticals. Inc (Cambridge, Estats Units d'Amèrica))
Adams, David H. (Université Paris-Saclay (França))
Langestroer, Christel (Universitätsklinikum Münster)
Huesing-Kabar, Anna (Universitätsklinikum Münster)
Schilling, Matthias (Universitätsklinikum Münster)
Kabar, IyadF (Universitätsklinikum Münster)
Backlund, Rolf (Umeå University Hospital (Suècia))
Anan, Intissar (Umeå University Hospital (Suècia))
Nordh, Erik (Umeå University Hospital (Suècia))
Uneus, Erika (Umeå University Hospital (Suècia))
Pilebro, Björn Umeå University Hospital (Suècia)
Englund, Ulrika (Umeå University Hospital (Suècia))
Coelho, Teresa (Centro Hospitalar Universitário do Porto (Portugal))
Novais, Marta (Centro Hospitalar Universitário do Porto (Portugal))
Perez, Javier (Centro Hospitalar Universitário do Porto (Portugal))
Martins da Silva, Ana (Centro Hospitalar Universitário do Porto (Portugal))
Pesseguerio Miranda, Helena (Centro Hospitalar Universitário do Porto (Portugal))
Ramalho, Joana (Centro Hospitalar Universitário do Porto (Portugal))
Monte, Raquel (Centro Hospitalar Universitário do Porto (Portugal))
Alves, Cristina (Centro Hospitalar Universitário do Porto (Portugal))
Cardaso, Ines (Centro Hospitalar Universitário do Porto (Portugal))
Guimaraes, Nádia (Centro Hospitalar Universitário do Porto (Portugal))
Gentile, Luca (Azienda Ospedaliera Universitaria Gaetano Martino (Messina, Itàlia))
Russo, Massimo (Azienda Ospedaliera Universitaria Gaetano Martino (Messina, Itàlia))
Di Bella, Gianluca (Azienda Ospedaliera Universitaria Gaetano Martino (Messina, Itàlia))
Gaouar, Amina (CHU Bicêtre (París, França))
Cauquil-Michon, Cécile (CHU Bicêtre (París, França))
Kounis, Ilias (CHU Bicêtre (París, França))
Echaniz-Laguna, Andoni (CHU Bicêtre (París, França))
Stéphant, Maëva (CHU Bicêtre (París, França))
Rakotondratafika, Fetra (CHU Bicêtre (París, França))
Boubrit, Yasmine (CHU Bicêtre (París, França))
Labeyrie, Celine (CHU Bicêtre (París, França))
Focsenaunu, Cecile (Hôpitaux Universitaires Henri Mondor (Créteil, França))
Le Corvoisier, Phillippe (Hôpitaux Universitaires Henri Mondor (Créteil, França))
Ayache, Samar S. (Hôpitaux Universitaires Henri Mondor (Créteil, França))
Gendre, Thierry (Hôpitaux Universitaires Henri Mondor (Créteil, França))
Vervoitte, Laetitia (Hôpitaux Universitaires Henri Mondor (Créteil, França))
Arrouasse, Raphaele (Hôpitaux Universitaires Henri Mondor (Créteil, França))
Gragera Martinez, Alvaro (Hospital Universitario Juan Ramón Jimenez (Huelva))
Borrachero, Cristina (Hospital Universitario Juan Ramón Jimenez (Huelva))
Manovel, Ana (Hospital Universitario Juan Ramón Jimenez (Huelva))
Diaz Rodriguez, Eusebio (Hospital Universitario Juan Ramón Jimenez (Huelva))
Gutiérrez Gándara, Marta (Hospital Universitari de Bellvitge)
Fabra Jiménez, Elena (Hospital Universitari de Bellvitge)
Vélez Santamaría, Valentina (Hospital Universitari de Bellvitge)
Martínez Vilar, Yurema (Hospital Universitari de Bellvitge)
Cachero, Alba (Hospital Universitari de Bellvitge)
Rannigan, Lisa (University College London Medical School London (Regne Unit))
Fontana, Marianna (University College London Medical School London (Regne Unit))
Orrell, Richard (University College London Medical School London (Regne Unit))
Louth, Sarah (University College London Medical School London (Regne Unit))
Chacko, Liza (University College London Medical School London (Regne Unit))
Varughese, Sindhu (University College London Medical School London (Regne Unit))
Throburn, Douglas (University College London Medical School London (Regne Unit))
Cohen, Oliver (University College London Medical School London (Regne Unit))
Law, Steven (University College London Medical School London (Regne Unit))
Smit, Angelique (University College London Medical School London (Regne Unit))
Strehina, Svetla (University College London Medical School London (Regne Unit))
Universitat Autònoma de Barcelona

Data:	2022
Resum:	Hereditary transthyretin-mediated (hATTR) amyloidosis, or ATTRv amyloidosis, is a progressive disease, for which liver transplantation (LT) has been a long-standing treatment. However, disease progression continues post-LT. This Phase 3b, open-label trial evaluated efficacy and safety of patisiran in patients with ATTRv amyloidosis with polyneuropathy progression post-LT. Primary endpoint was median transthyretin (TTR) reduction from baseline. Twenty-three patients received patisiran for 12 months alongside immunosuppression regimens. Patisiran elicited a rapid, sustained TTR reduction (median reduction [Months 6 and 12 average], 91. 0%; 95% CI: 86. 1%-92. 3%); improved neuropathy, quality of life, and autonomic symptoms from baseline to Month 12 (mean change [SEM], Neuropathy Impairment Score, -3. 7 [2. 7]; Norfolk Quality of Life-Diabetic Neuropathy questionnaire, -6. 5 [4. 9]; least-squares mean [SEM], Composite Autonomic Symptom Score-31, -5. 0 [2. 6]); and stabilized disability (Rasch-built Overall Disability Scale) and nutritional status (modified body mass index). Adverse events were mild or moderate; five patients experienced ≥1 serious adverse event. Most patients had normal liver function tests. One patient experienced transplant rejection consistent with inadequate immunosuppression, remained on patisiran, and completed the study. In conclusion, patisiran reduced serum TTR, was well tolerated, and improved or stabilized key disease impairment measures in patients with ATTRv amyloidosis with polyneuropathy progression post-LT (www. clinicaltrials. gov NCT03862807).
Drets:	Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, la comunicació pública de l'obra i la creació d'obres derivades, fins i tot amb finalitats comercials, sempre i quan es reconegui l'autoria de l'obra original.
Llengua:	Anglès
Document:	Article ; recerca ; Versió publicada
Matèria:	Clinical research/practice ; Clinical trial ; Liver allograft function/dysfunction ; Liver transplantation/hepatology ; Molecular biology: small interfering RNA ; Neurology ; Patient survival ; Pharmacology
Publicat a:	American Journal of Transplantation, Vol. 22 Núm. 6 (june 2022) , p. 1646-1657, ISSN 1600-6143

DOI: 10.1111/ajt.17009
PMID: 35213769

12 p, 880.4 KB

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