Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis

New England Journal of Medicine

Volumn 379, Issue 1, 2018, Pages 11-21

  Adams, David ^a   Gonzalez Duarte, Alejandra ^d   O'Riordan, William D ^e   Yang, Chih Chao ^f   Ueda, Mitsuharu ^h   Kristen, Arnt V ^e   Tournev, Ivailo ^l   Schmidt, Hartmut H ^k   Coelho, Teresa ^m   Berk, John L ⁿ   Lin, Kon Ping ^g   Vita, Giuseppe ^p   Attarian, Shahram ^b   Planté Bordeneuve, Violaine ^c   Mezei, Michelle M ^q   Campistol, Josep M ^r   Buades, Juan ^s   Brannagan, Thomas H ^t   Kim, Byoung J ^u   Oh, Jeeyoung ^v   Parman, Yesim ^w   Sekijima, Yoshiki ⁱ   Hawkins, Philip N ^x   Solomon, Scott D ^o   Polydefkis, Michael ^y   Dyck, Peter J ^z   Gandhi, Pritesh J ^aa   Goyal, Sunita ^aa   Chen, Jihong ^aa   Strahs, Andrew L ^aa   Nochur, Saraswathy V ^aa   Sweetser, Marianne T ^aa   Garg, Pushkal P ^z,aa   Vaishnaw, Akshay K ^j,aa   Gollob, Jared A ^aa   Suhr, Ole B ^ab   more..

^a BICÊTRE HOSPITAL   (France)

^b TIMONE HOSPITAL   (France)

^c HENRI MONDOR HOSPITAL   (France)

^d INSTITUTO NACIONAL DE CIENCIAS MÉDICAS Y NUTRICIÓN SALVADOR ZUBIRÁN   (Mexico)

^e EStudySite ^*

^f NATIONAL TAIWAN UNIVERSITY HOSPITAL   (Taiwan)

^g TAIPEI VETERANS GENERAL HOSPITAL   (Taiwan)

^h KUMAMOTO UNIVERSITY HOSPITAL   (Japan)

ⁱ SHINSHU UNIVERSITY SCHOOL OF MEDICINE   (Japan)

^j UNIVERSITY OF HEIDELBERG   (Germany)

^k UNIVERSITY HOSPITAL MÜNSTER   (Germany)

^l Neurology Clinic   (Bulgaria)

^m HOSPITAL DE SANTO ANTÓNIO   (Portugal)

ⁿ BOSTON MEDICAL CENTER   (United States)

^o HARVARD MEDICAL SCHOOL   (United States)

^p UNIVERSITY OF MESSINA   (Italy)

^q VANCOUVER GENERAL HOSPITAL   (Canada)

^r HOSPITAL CLÍNIC   (Spain)

^s HOSPITAL SON LLÀTZER   (Spain)

^t College of Physicians and Surgeons ^*  (United States)

^u Samsung Medical Center   (South Korea)

^v Konkuk University Hospital   (South Korea)

^w ISTANBUL UNIVERSITY   (Turkey)

^x UNIVERSITY COLLEGE LONDON   (United Kingdom)

^y JOHNS HOPKINS BAYVIEW MEDICAL CENTER   (United States)

^z MAYO CLINIC   (United States)

^aa ALNYLAM PHARMACEUTICALS   (United States)

^ab UMEÅ UNIVERSITY   (Sweden)

more..

Author keywords

[No Author keywords available]

Indexed keywords

ALBUMIN; PATISIRAN; PLACEBO; PREALBUMIN; SMALL INTERFERING RNA;

ADULT; ADVERSE EVENT; AGED; ANEMIA; ARTICLE; ASTHENIA; ATTR AMYLOIDOSIS; BODY MASS; CLINICAL FEATURE; CONSTIPATION; CONTROLLED STUDY; COUGHING; DIARRHEA; DIZZINESS; DOUBLE BLIND PROCEDURE; DRUG EFFICACY; DRUG SAFETY; FAINTNESS; FALLING; FATIGUE; FEMALE; GAIT; HEADACHE; HEREDITARY ATTR AMYLOIDOSIS; HUMAN; INJECTION SITE REACTION; INSOMNIA; MAJOR CLINICAL STUDY; MALE; MODIFIED NEUROPATHY IMPAIRMENT SCORE; MUSCLE WEAKNESS; NAUSEA; NEUROLOGIC EXAMINATION; NORFOLK QUALITY OF LIFE DIABETIC NEUROPATHY QUESTIONNAIRE; NUTRITIONAL STATUS; PAIN; PERIPHERAL EDEMA; PHARMACODYNAMICS; PHASE 3 CLINICAL TRIAL; PRIORITY JOURNAL; QUALITY OF LIFE; QUESTIONNAIRE; RANDOMIZED CONTROLLED TRIAL; RHINOPHARYNGITIS; RNA INTERFERENCE; SIDE EFFECT; URINARY TRACT INFECTION; VOMITING; BLOOD; CHEMICALLY INDUCED; CLINICAL TRIAL; COMPLICATION; DISEASE EXACERBATION; EDEMA; FAMILIAL AMYLOID POLYNEUROPATHY; GENETICS; INTRAVENOUS DRUG ADMINISTRATION; LEAST SQUARE ANALYSIS; MIDDLE AGED; MULTICENTER STUDY; NEUROLOGIC GAIT DISORDER; POLYNEUROPATHY; RNAI THERAPEUTICS; SEVERITY OF ILLNESS INDEX; VERY ELDERLY; WALK TEST;

ADMINISTRATION, INTRAVENOUS; ADULT; AGED; AGED, 80 AND OVER; AMYLOID NEUROPATHIES, FAMILIAL; DISEASE PROGRESSION; DOUBLE-BLIND METHOD; EDEMA; FEMALE; GAIT DISORDERS, NEUROLOGIC; HUMANS; INFUSIONS, INTRAVENOUS; LEAST-SQUARES ANALYSIS; MALE; MIDDLE AGED; POLYNEUROPATHIES; PREALBUMIN; QUALITY OF LIFE; RNA, SMALL INTERFERING; RNAI THERAPEUTICS; SEVERITY OF ILLNESS INDEX; WALK TEST;

EID: 85049655756     PISSN: 00284793     EISSN: 15334406     Source Type: Journal    
DOI: 10.1056/NEJMoa1716153     Document Type: Article

References (26)

1. Hawkins PN, Ando Y, Dispenzeri A, Gonzalez-Duarte A, Adams D, Suhr OB. Evolving landscape in the management of transthyretin amyloidosis. Ann Med 2015; 47:625-38.
2. Ruberg FL, Berk JL. Transthyretin (TTR) cardiac amyloidosis. Circulation 2012;126:1286-300.
3. Conceição I, González-Duarte A, Obici L, et al. “Red-flag” symptom clusters in transthyretin familial amyloid polyneuropathy. J Peripher Nerv Syst 2016;21:5-9.
4. Ando Y, Coelho T, Berk JL, et al. Guideline of transthyretin-related hereditary amyloidosis for clinicians. Orphanet J Rare Dis 2013;8:31.
5. Dungu JN, Anderson LJ, Whelan CJ, Hawkins PN. Cardiac transthyretin amyloidosis. Heart 2012;98:1546-54.
6. Adams D, Coelho T, Obici L, et al. Rapid progression of familial amyloidotic polyneuropathy: a multinational natural history study. Neurology 2015;85:675-82.
7. Koike H, Tanaka F, Hashimoto R, et al. Natural history of transthyretin Val-30Met familial amyloid polyneuropathy: analysis of late-onset cases from non-endemic areas. J Neurol Neurosurg Psychiatry 2012;83:152-8.
8. Mariani LL, Lozeron P, Théaudin M, et al. Genotype-phenotype correlation and course of transthyretin familial amyloid polyneuropathies in France. Ann Neurol 2015;78:901-16.
9. Ruberg FL, Maurer MS, Judge DP, et al. Prospective evaluation of the morbidity and mortality of wild-type and V122I mutant transthyretin amyloid cardiomyopathy: the Transthyretin Amyloidosis Cardiac Study (TRACS). Am Heart J 2012; 164(2):222-228.e1.
10. Castaño A, Drachman BM, Judge D, Maurer MS. Natural history and therapy of TTR-cardiac amyloidosis: emerging disease-modifying therapies from organ transplantation to stabilizer and silencer drugs. Heart Fail Rev 2015;20:163-78.
11. Liepnieks JJ, Zhang LQ, Benson MD. Progression of transthyretin amyloid neuropathy after liver transplantation. Neurology 2010;75:324-7.
12. Adams D, Buades J, Suhr O, Obici L, Coelho T. Preliminary assessment of neuropathy progression in patients with hereditary ATTR amyloidosis after orthotopic liver transplantation (OLT). Orphanet J Rare Dis 2015;10:Suppl 1. abstract.
13. Coelho T, Maia LF, da Silva AM, et al. Long-term effects of tafamidis for the treatment of transthyretin familial amyloid polyneuropathy. J Neurol 2013;260: 2802-14.
14. Cortese A, Vita G, Luigetti M, et al. Monitoring effectiveness and safety of Tafamidis in transthyretin amyloidosis in Italy: a longitudinal multicenter study in a non-endemic area. J Neurol 2016;263:916-24.
15. Planté-Bordeneuve V, Gorram F, Salhi H, et al. Long-term treatment of transthyretin familial amyloid polyneuropathy with Tafamidis: a clinical and neurophys-iological study. J Neurol 2017;264:268-76.
16. Lozeron P, Théaudin M, Mincheva Z, Ducot B, Lacroix C, Adams D. Effect on disability and safety of Tafamidis in late onset of Met30 transthyretin familial amyloid polyneuropathy. Eur J Neurol 2013;20:1539-45.
17. Coelho T, Adams D, Silva A, et al. Safety and efficacy of RNAi therapy for transthyretin amyloidosis. N Engl J Med 2013;369:819-29.
18. Suhr OB, Coelho T, Buades J, et al. Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study. Orphanet J Rare Dis 2015;10:109.
19. Partisano AM, Berk JL, Adams D, et al. Long-term, open-label clinical experience with patisiran, an investigational RNAi therapeutic for patients with hereditary transthyretin-mediated (hATTR) amyloidosis with polyneuropathy. Orphanet J Rare Dis 2017;12:Suppl 1:165. abstract.
20. Adams D, Suhr OB, Dyck PJ, et al. Trial design and rationale for APOLLO, a Phase 3, placebo-controlled study of patisiran in patients with hereditary ATTR amyloidosis with polyneuropathy. BMC Neurol 2017;17:181.
21. Suanprasert N, Berk JL, Benson MD, et al. Retrospective study of a TTR FAP cohort to modify NIS+7 for therapeutic trials. J Neurol Sci 2014;344:121-8.
22. Ionis and international neurologists' proficiency in a familial amyloidotic polyneuropathy trial. Muscle Nerve 2017;56:901-11.
23. Benson MD, Waddington-Cruz M, Berk JL, et al. Inotersen treatment for patients with hereditary transthyretin amyloidosis. N Engl J Med 2018;379:22-31.
24. Coelho T, Maia LF, Martins da Silva A, et al. Tafamidis for transthyretin familial amyloid polyneuropathy: a randomized, controlled trial. Neurology 2012;79:785-92.
25. Berk JL, Suhr OB, Obici L, et al. Re-purposing diflunisal for familial amyloid polyneuropathy: a randomized clinical trial. JAMA 2013;310:2658-67.
26. Sepp-Lorenzino L, Ruddy M. Challenges and opportunities for local and systemic delivery of siRNA and antisense oligonucleotides. Clin Pharmacol Ther 2008;84:628-32.