Repurposing Diflunisal for Familial Amyloid Polyneuropathy A Randomized Clinical Trial
John L. Berk,Ole B. Suhr,Laura Obici,Yoshiki Sekijima,Steven R. Zeldenrust,Taro Yamashita,Michael A. Heneghan,Peter D. Gorevic,William J. Litchy,Janice F. Wiesman,Erik Nordh,Manuel Corato,Alessandro Lozza,Andrea Cortese,Jessica Robinson-Papp,Theodore Colton,Denis Rybin,Alice Bisbee,Yukio Ando,Shu-ichi Ikeda,David C. Seldin,Giampaolo Merlini,Martha Skinner,Jeffery W. Kelly,Peter J. Dyck +24 more
Reads0
Chats0
TLDR
Among patients with familial amyloid polyneuropathy, the use of diflunisal compared with placebo for 2 years reduced the rate of progression of neurological impairment and preserved quality of life.Abstract:
RESULTS By multiple imputation, the NIS+7 score increased by 25.0 (95% CI, 18.4-31.6) points in the placebo group and by 8.7 (95% CI, 3.3-14.1) points in the diflunisal group, a difference of 16.3 points (95% CI, 8.1-24.5 points; P < .001). Mean SF-36 physical scores decreased by 4.9 (95% CI, −7.6 to −2.2) points in the placebo group and increased by 1.5 (95% CI, −0.8 to 3.7) points in the diflunisal group (P < .001). Mean SF-36 mental scores declined by 1.1 (95% CI, −4.3 to 2.0) points in the placebo group while increasing by 3.7 (95% CI, 1.0-6.4) points in the diflunisal group (P = .02). By responder analysis, 29.7% of the diflunisal group and 9.4% of the placebo group exhibited neurological stability at 2 years (<2-point increase in NIS+7 score; P = .007). CONCLUSIONS AND RELEVANCE Among patients with familial amyloid polyneuropathy, the use of diflunisal compared with placebo for 2 years reduced the rate of progression of neurological impairment and preserved quality of life. Although longer-term follow-up studies are needed, these findings suggest benefit of this treatment for familial amyloid polyneuropathy.read more
Citations
More filters
Journal ArticleDOI
Multiple Imputation for Nonresponse in Surveys
TL;DR: It is concluded that multiple Imputation for Nonresponse in Surveys should be considered as a legitimate method for answering the question of why people do not respond to survey questions.
Journal ArticleDOI
Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis
David Adams,Alejandra González-Duarte,William O'Riordan,Chih-Chao Yang,Mitsuharu Ueda,Arnt V. Kristen,Ivailo Tournev,Hartmut Schmidt,Teresa Coelho,John L. Berk,Kon Ping Lin,Giuseppe Vita,Shahram Attarian,Violaine Planté-Bordeneuve,Michelle M. Mezei,Josep M. Campistol,Juan Buades,Thomas H. Brannagan,Byoung Joon Kim,Jeeyoung Oh,Yesim Parman,Yoshiki Sekijima,Philip N. Hawkins,Scott D. Solomon,Michael Polydefkis,Peter J. Dyck,Pritesh Gandhi,Sunita Goyal,Jihong Chen,Andrew Strahs,Saraswathy V. Nochur,Marianne T. Sweetser,Pushkal Garg,Pushkal Garg,Akshay Vaishnaw,Akshay Vaishnaw,Jared Gollob,Ole B. Suhr +37 more
TL;DR: Patisiran improved multiple clinical manifestations of hereditary transthyretin amyloidosis with polyneuropathy and showed an effect on gait speed and modified BMI.
Journal ArticleDOI
2022 AHA/ACC/HFSA Guideline for the Management of Heart Failure: A Report of the American College of Cardiology/American Heart Association Joint Committee on Clinical Practice Guidelines.
Paul A. Heidenreich,Biykem Bozkurt,David Aguilar,Larry A. Allen,Joni J. Byun,Monica Colvin,Anita Deswal,Mark H. Drazner,Shannon M. Dunlay,Linda R. Evers,James C. Fang,Savitri Fedson,Gregg C. Fonarow,Salim S. Hayek,Adrian F. Hernandez,Prateeti Khazanie,Michelle M. Kittleson,Christopher S. Lee,Mark S. Link,Carmelo A. Milano,Lorraine C. Nnacheta,Alexander T. Sandhu,Lynne W. Stevenson,Orly Vardeny,Amanda R. Vest,Clyde W. Yancy +25 more
TL;DR: The "2022 AHA/ACC/HFSA Guideline for the Management of Heart Failure" as discussed by the authors provides patient-centric recommendations for clinicians to prevent, diagnose, and manage patients with heart failure.
Journal ArticleDOI
Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis
Merrill D. Benson,Márcia Waddington-Cruz,John L. Berk,Michael Polydefkis,Peter J. Dyck,Annabel K. Wang,Violaine Planté-Bordeneuve,Fabio Barroso,Giampaolo Merlini,Laura Obici,Morton A. Scheinberg,Thomas H. Brannagan,William J. Litchy,Carol J. Whelan,Brian M. Drachman,David C. Adams,Stephen B. Heitner,Isabel Conceição,Hartmut H. Schmidt,Giuseppe Vita,Josep M. Campistol,Josep Gamez,Peter D. Gorevic,Edward Gane,Amil M. Shah,Scott D. Solomon,Brett P. Monia,Steven G. Hughes,Jesse Kwoh,Bradley W. McEvoy,Shiangtung W. Jung,Brenda F. Baker,Elizabeth J. Ackermann,Morie A. Gertz,Teresa Coelho +34 more
TL;DR: Inotersen improved the course of neurologic disease and quality of life in patients with hereditary transthyretin amyloidosis and improvements were independent of disease stage, mutation type, or the presence of cardiomyopathy.
Journal ArticleDOI
Transthyretin Amyloid Cardiomyopathy: JACC State-of-the-Art Review
TL;DR: Previously treatable only by organ transplantation, pharmaceutical therapy that slows or halts ATTR-CM progression and favorably affects clinical outcomes is now available.
References
More filters
Book
Multiple imputation for nonresponse in surveys
TL;DR: In this article, a survey of drinking behavior among men of retirement age was conducted and the results showed that the majority of the participants reported that they did not receive any benefits from the Social Security Administration.
Book
Analysis of longitudinal data
TL;DR: In this paper, a generalized linear model for longitudinal data and transition models for categorical data are presented. But the model is not suitable for categric data and time dependent covariates are not considered.
Journal ArticleDOI
Multiple Imputation for Nonresponse in Surveys
TL;DR: It is concluded that multiple Imputation for Nonresponse in Surveys should be considered as a legitimate method for answering the question of why people do not respond to survey questions.
Journal ArticleDOI
Analysis of Longitudinal Data.
TL;DR: Van Der Heijden et al. as discussed by the authors used correspondence analysis for the analysis of transitions between more than two time points, where the transition matrix is the product of the margins of the table divided by the total sample size.
Journal ArticleDOI
The Prevention and Treatment of Missing Data in Clinical Trials
Roderick J. A. Little,Ralph B. D'Agostino,Michael L. Cohen,Kay Dickersin,Scott S. Emerson,John T. Farrar,Constantine Frangakis,Joseph W. Hogan,Geert Molenberghs,Susan A. Murphy,James D. Neaton,Andrea Rotnitzky,Daniel O. Scharfstein,Weichung Joe Shih,Jay P. Siegel,Hal S. Stern +15 more
TL;DR: Methods for preventing missing data and, failing that, dealing with data that are missing in clinical trials are reviewed.
Related Papers (5)
Tafamidis for transthyretin familial amyloid polyneuropathy A randomized, controlled trial
Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis
David Adams,Alejandra González-Duarte,William O'Riordan,Chih-Chao Yang,Mitsuharu Ueda,Arnt V. Kristen,Ivailo Tournev,Hartmut Schmidt,Teresa Coelho,John L. Berk,Kon Ping Lin,Giuseppe Vita,Shahram Attarian,Violaine Planté-Bordeneuve,Michelle M. Mezei,Josep M. Campistol,Juan Buades,Thomas H. Brannagan,Byoung Joon Kim,Jeeyoung Oh,Yesim Parman,Yoshiki Sekijima,Philip N. Hawkins,Scott D. Solomon,Michael Polydefkis,Peter J. Dyck,Pritesh Gandhi,Sunita Goyal,Jihong Chen,Andrew Strahs,Saraswathy V. Nochur,Marianne T. Sweetser,Pushkal Garg,Pushkal Garg,Akshay Vaishnaw,Akshay Vaishnaw,Jared Gollob,Ole B. Suhr +37 more
Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis
Merrill D. Benson,Márcia Waddington-Cruz,John L. Berk,Michael Polydefkis,Peter J. Dyck,Annabel K. Wang,Violaine Planté-Bordeneuve,Fabio Barroso,Giampaolo Merlini,Laura Obici,Morton A. Scheinberg,Thomas H. Brannagan,William J. Litchy,Carol J. Whelan,Brian M. Drachman,David C. Adams,Stephen B. Heitner,Isabel Conceição,Hartmut H. Schmidt,Giuseppe Vita,Josep M. Campistol,Josep Gamez,Peter D. Gorevic,Edward Gane,Amil M. Shah,Scott D. Solomon,Brett P. Monia,Steven G. Hughes,Jesse Kwoh,Bradley W. McEvoy,Shiangtung W. Jung,Brenda F. Baker,Elizabeth J. Ackermann,Morie A. Gertz,Teresa Coelho +34 more
Safety and efficacy of RNAi therapy for transthyretin amyloidosis
Teresa Coelho,David H. Adams,Ana Martins da Silva,Pierre Lozeron,Philip N. Hawkins,Timothy Mant,Javier Perez,Joseph Chiesa,Steve Warrington,Elizabeth Tranter,Malathy Munisamy,Rick Falzone,Jamie Harrop,Jeffrey Cehelsky,Brian Bettencourt,Mary Geissler,James Butler,Alfica Sehgal,Rachel Meyers,Qingmin Chen,Todd Borland,Renta Hutabarat,Valerie A. Clausen,Rene Alvarez,Kevin Fitzgerald,Christina Gamba-Vitalo,Saraswathy V. Nochur,Akshay Vaishnaw,Dinah W.Y. Sah,Jared Gollob,Ole B. Suhr +30 more
Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy
Mathew S. Maurer,Jeffrey H. Schwartz,Balarama Gundapaneni,Perry M. Elliott,Giampaolo Merlini,Márcia Waddington-Cruz,Arnt V. Kristen,Martha Grogan,Ronald M. Witteles,Thibaud Damy,Brian M. Drachman,Sanjiv J. Shah,Mazen Hanna,Daniel P. Judge,Alexandra I. Barsdorf,Peter Huber,Terrell A. Patterson,Steven Riley,Jennifer Schumacher,Michelle Stewart,Marla B. Sultan,Claudio Rapezzi +21 more