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  • Schmidt, Hartmut H.Department of Gastroenterology, Hepatology and Transplant Medicine, University Hospital Essen, University of Duisburg-Essen (formerly of University Hospital Munster, Munster, Germany), Essen, Germany (författare)

Patisiran treatment in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy after liver transplantation

  • Artikel/kapitelEngelska2022

Förlag, utgivningsår, omfång ...

  • John Wiley & Sons,2022
  • electronicrdacarrier

Nummerbeteckningar

  • LIBRIS-ID:oai:DiVA.org:umu-193692
  • https://urn.kb.se/resolve?urn=urn:nbn:se:umu:diva-193692URI
  • https://doi.org/10.1111/ajt.17009DOI

Kompletterande språkuppgifter

  • Språk:engelska
  • Sammanfattning på:engelska

Ingår i deldatabas

Klassifikation

  • Ämneskategori:ref swepub-contenttype
  • Ämneskategori:art swepub-publicationtype

Anmärkningar

  • 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).

Ämnesord och genrebeteckningar

Biuppslag (personer, institutioner, konferenser, titlar ...)

  • Wixner, JonasUmeå universitet,Avdelningen för medicin(Swepub:umu)jowi0065 (författare)
  • Planté-Bordeneuve, ViolaineDepartment of Neurology, East Paris University, Hospital Henri Mondor – Public Assistance Hospital of Paris, Créteil, France; Mondor Biomedical Research Institute – IMRB, INSERM, U955 Team 10 “Biology of the Neuro-Muscular System”, Créteil, France (författare)
  • Muñoz-Beamud, FranciscoHereditary Amyloidosis Unit, Department of Internal Medicine, Juan Ramón Jiménez Hospital, Huelva, Spain (författare)
  • Lladó, LauraLiver Transplantation Unit, Department of Surgery, and the Multidisciplinary Familial Amyloidosis Unit, Hospital Universitari de Bellvitge, Barcelona, Spain; Biomedical Research Institute, IDIBELL, University of Barcelona, Barcelona, Spain (författare)
  • Gillmore, Julian D.National Amyloidosis Centre, Division of Medicine, University College London Medical School, London, United Kingdom (författare)
  • Mazzeo, AnnaUnit of Neurology and Neuromuscular Diseases, Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy (författare)
  • Li, XingyuAlnylam Pharmaceuticals, Inc, MA, Cambridge, United States (författare)
  • Arum, SethAlnylam Pharmaceuticals, Inc, MA, Cambridge, United States (författare)
  • Jay, Patrick Y.Alnylam Pharmaceuticals, Inc, MA, Cambridge, United States (författare)
  • Adams, DavidNeurology Department, Université Paris-Saclay, U1195, INSERM, Le Kremlin Bicêtre, France; Neurology Department, AP-HP, CHU Bicêtre, Le Kremlin Bicêtre, France (författare)
  • Backlund, RolfUmeå University Hospital,The Patisiran Post-LT Study Group(Swepub:umu)rofbad84 (bidragsgivare)
  • Anan, IntissarUmeå University Hospital,The Patisiran Post-LT Study Group(Swepub:umu)inrann97 (bidragsgivare)
  • Nordh, ErikUmeå University Hospital,The Patisiran Post-LT Study Group(Swepub:umu)erno0019 (bidragsgivare)
  • Unéus, EricaUmeå University Hospital,The Patisiran Post-LT Study Group(Swepub:umu)erun0001 (bidragsgivare)
  • Pilebro, BjörnUmeå University Hospital,The Patisiran Post-LT Study Group(Swepub:umu)bjpi0001 (bidragsgivare)
  • Department of Gastroenterology, Hepatology and Transplant Medicine, University Hospital Essen, University of Duisburg-Essen (formerly of University Hospital Munster, Munster, Germany), Essen, GermanyAvdelningen för medicin (creator_code:org_t)

Sammanhörande titlar

  • Ingår i:American Journal of Transplantation: John Wiley & Sons22:6, s. 1646-16571600-61351600-6143

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