Inotersen treatment for patients with Hereditary transthyretin amyloidosis

New England Journal of Medicine

Volumn 379, Issue 1, 2018, Pages 22-31

  Benson, Merrill D ^b   Waddington Cruz, Márcia ^c   Berk, John L ^d   Polydefkis, Michael ^e   Dyck, Peter J ^f   Wang, Annabel K ^g   Planté Bordeneuve, Violaine ^h   Barroso, Fabio A ⁱ   Merlini, Giampaolo ^j   Obici, Laura ^j   Scheinberg, Morton ^l   Brannagan, Thomas H ^m   Litchy, William J ^f   Whelan, Carol ⁿ   Drachman, Brian M ^o   Adams, David ^p   Heitner, Stephen B ^q   Conceição, Isabel ^r   Schmidt, Hartmut H ^t   Vita, Giuseppe ^k   Campistol, Josep M ^u   Gamez, Josep ^u   Gorevic, Peter D ^m   Gane, Edward ^v   Shah, Amil M ^d   Solomon, Scott D ^d   Monia, Brett P ^w   Hughes, Steven G ^w   Jesse Kwoh, T ^w   McEvoy, Bradley W ^w   Jung, Shiangtung W ^w   Baker, Brenda F ^w   Ackermann, Elizabeth J ^w   Gertz, Morie A ^f   Coelho, Teresa ^a,s   more..

^a HOSPITAL DE SANTO ANTÓNIO   (Portugal)

^b INDIANA UNIVERSITY   (United States)

^c FEDERAL UNIVERSITY OF RIO DE JANEIRO   (Brazil)

^d BOSTON UNIVERSITY SCHOOL OF MEDICINE   (United States)

^e JOHNS HOPKINS UNIVERSITY   (United States)

^f MAYO CLINIC   (United States)

^g UNIVERSITY OF CALIFORNIA   (United States)

^h HENRI MONDOR HOSPITAL   (France)

ⁱ FLENI   (Argentina)

^j UNIVERSITY OF PAVIA   (Italy)

^k UNIVERSITY OF MESSINA   (Italy)

^l AACD   (Brazil)

^m NewYork Presbyterian Hospital   (United States)

ⁿ UNIVERSITY COLLEGE LONDON   (United Kingdom)

^o PENN PRESBYTERIAN MEDICAL CENTER   (United States)

^p Section of Radiology   (France)

^q OREGON HEALTH AND SCIENCE UNIVERSITY   (United States)

^r HOSPITAL DE SANTA MARIA   (Portugal)

^s Centro Hospitalar Universitario do Porto   (Portugal)

^t UNIVERSITY HOSPITAL MÜNSTER   (Germany)

^u UNIVERSITY OF BARCELONA   (Spain)

^v AUCKLAND CITY HOSPITAL   (New Zealand)

^w ISIS PHARMACEUTICALS   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

INOTERSEN; METHIONINE; PLACEBO; PREALBUMIN; VALINE; ANTISENSE OLIGONUCLEOTIDE;

ADULT; AGED; AMYLOIDOSIS; ARTICLE; BRAIN HEMORRHAGE; CARDIOMYOPATHY; CEREBROVASCULAR ACCIDENT; CLINICAL ASSESSMENT; COMPARATIVE STUDY; CONTROLLED STUDY; DEATH; DEEP VEIN THROMBOSIS; DISEASE ASSOCIATION; DISEASE CLASSIFICATION; DISEASE EXACERBATION; DOUBLE BLIND PROCEDURE; DRUG EFFICACY; DRUG RESPONSE; DRUG SAFETY; FEMALE; GENE MUTATION; GLOMERULONEPHRITIS; HEREDITARY TRANSTHYRETIN AMYLOIDOSIS; HUMAN; INCIDENCE; INTERSTITIAL NEPHRITIS; LUNG EMBOLISM; MAJOR CLINICAL STUDY; MALE; MODIFIED NEUROPATHY IMPAIRMENT SCORE 7; NEUROLOGIC DISEASE ASSESSMENT; NORFOLK QUALITY OF LIFE DIABETIC NEUROPATHY QUESTIONNAIRE; PATIENT MONITORING; POLYNEUROPATHY; PRIORITY JOURNAL; PROTEIN BLOOD LEVEL; QUALITY OF LIFE ASSESSMENT; RANDOMIZED CONTROLLED TRIAL; SPINAL CORD DISEASE; THROMBOCYTOPENIA; TREATMENT DURATION; ANTAGONISTS AND INHIBITORS; BLOOD; CHEMICALLY INDUCED; CLINICAL TRIAL; COMPLICATION; FAMILIAL AMYLOID POLYNEUROPATHY; GENETICS; LEAST SQUARE ANALYSIS; MIDDLE AGED; MULTICENTER STUDY; PHASE 3 CLINICAL TRIAL; QUALITY OF LIFE; RNAI THERAPEUTICS; SEVERITY OF ILLNESS INDEX; SUBCUTANEOUS DRUG ADMINISTRATION; VERY ELDERLY;

ADULT; AGED; AGED, 80 AND OVER; AMYLOID NEUROPATHIES, FAMILIAL; DISEASE PROGRESSION; DOUBLE-BLIND METHOD; FEMALE; GLOMERULONEPHRITIS; HUMANS; INJECTIONS, SUBCUTANEOUS; LEAST-SQUARES ANALYSIS; MALE; MIDDLE AGED; OLIGONUCLEOTIDES, ANTISENSE; POLYNEUROPATHIES; PREALBUMIN; QUALITY OF LIFE; RNAI THERAPEUTICS; SEVERITY OF ILLNESS INDEX; THROMBOCYTOPENIA;

EID: 85049654759     PISSN: 00284793     EISSN: 15334406     Source Type: Journal    
DOI: 10.1056/NEJMoa1716793     Document Type: Article

References (29)

1. Sekijima Y. Transthyretin (ATTR) amyloidosis: clinical spectrum, molecular pathogenesis and disease-modifying treatments. J Neurol Neurosurg Psychiatry 2015;86:1036-43.
2. Lobato L, Beirão I, Silva M, et al. Familial ATTR amyloidosis: microalbumin-uria as a predictor of symptomatic disease and clinical nephropathy. Nephrol Dial Transplant 2003;18:532-8.
3. Rapezzi C, Quarta CC, Riva L, et al. Transthyretin-related amyloidoses and the heart: a clinical overview. Nat Rev Cardiol 2010;7:398-408.
4. Sekijima Y, Dendle MA, Kelly JW. Orally administered diflunisal stabilizes transthyretin against dissociation required for amyloidogenesis. Amyloid 2006;13: 236-49.
5. 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.
6. Berk JL, Suhr OB, Obici L, et al. Re-purposing diflunisal for familial amyloid polyneuropathy: a randomized clinical trial. JAMA 2013;310:2658-67.
7. Ionis and international neurologists' proficiency in a familial amyloidotic polyneuropathy trial. Muscle Nerve 2017;56:901-11.
8. Vinik EJ, Hayes RP, Oglesby A, et al. The development and validation of the Norfolk QOL-DN, a new measure of patients' perception of the effects of diabetes and diabetic neuropathy. Diabetes Technol Ther 2005;7:497-508.
9. Planté-Bordeneuve V, Said G. Familial amyloid polyneuropathy. Lancet Neurol 2011;10:1086-97.
10. Lobato L, Rocha A. Transthyretin amyloidosis and the kidney. Clin J Am Soc Nephrol 2012;7:1337-46.
11. Gertz MA. Hereditary ATTR amyloidosis: burden of illness and diagnostic challenges. Am J Manag Care 2017;23: Suppl:S107-S112.
12. Coelho T, Ericzon B, Falk R, et al. A physician's guide to transthyretin amyloidosis. Clarkston, MI: Amyloidosis Foundation, 2008:1-16.
13. Merlini G, Bellotti V. Molecular mechanisms of amyloidosis. N Engl J Med 2003; 349:583-96.
14. Liepnieks JJ, Zhang LQ, Benson MD. Progression of transthyretin amyloid neuropathy after liver transplantation. Neurology 2010;75:324-7.
15. Ando Y, Coelho T, Berk JL, et al. Guideline of transthyretin-related hereditary amyloidosis for clinicians. Orphanet J Rare Dis 2013;8:31.
16. Ericzon BG, Wilczek HE, Larsson M, et al. Liver transplantation for hereditary transthyretin amyloidosis: after 20 years still the best therapeutic alternative? Transplantation 2015;99:1847-54.
17. Barroso FA, Judge DP, Ebede B, et al. Long-term safety and efficacy of tafamidis for the treatment of hereditary transthyretin amyloid polyneuropathy: results up to 6 years. Amyloid 2017;24:194-204.
18. Alexander KM, Singh A, Falk RH. Novel pharmacotherapies for cardiac amyloidosis. Pharmacol Ther 2017;180: 129-38.
19. Ackermann EJ, Guo S, Benson MD, et al. Suppressing transthyretin production in mice, monkeys and humans using 2nd-Generation antisense oligonucleotides. Amyloid 2016;23:148-57.
20. Diabetic polyneuropathy in controlled clinical trials: consensus report of the Peripheral Nerve Society. Ann Neurol 1995; 38:478-82.
21. Dyck PJ, Kratz KM, Lehman KA, et al. The Rochester Diabetic Neuropathy Study: design, criteria for types of neuropathy, selection bias, and reproducibility of neuropathic tests. Neurology 1991; 41:799-807.
22. Dyck PJ, Overland CJ, Low PA, et al. “Unequivocally abnormal” vs “usual” signs and symptoms for proficient diagnosis of diabetic polyneuropathy: Cl vs N Phys Trial. Arch Neurol 2012;69:1609-14.
23. 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.
24. Vinik EJ, Vinik AI, Paulson JF, et al. Norfolk QOL-DN: validation of a patient reported outcome measure in transthyretin familial amyloid polyneuropathy. J Peripher Nerv Syst 2014;19:104-14.
25. Adams D, Gonzalez-Duarte A, O'Riordan WD, et al. Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis. N Engl J Med 2018;379:11-21.
26. Crooke ST, Baker BF, Kwoh TJ, et al. Integrated safety assessment of 2′-O-methoxyethyl chimeric antisense oligonucleotides in nonhuman primates and healthy human volunteers. Mol Ther 2016; 24:1771-82.
27. Crooke ST, Baker BF, Witztum JL, et al. The effects of 2′-O-methoxyethyl containing antisense oligonucleotides on platelets in human clinical trials. Nucleic Acid Ther 2017;27:121-9.
28. Crooke ST, Baker BF, Pham NC, et al. The effects of 2′-O-methoxyethyl oligonucleotides on renal function in humans. Nucleic Acid Ther 2018;28:10-22.
29. Maurer MS, Elliott P, Merlini G, et al. Design and rationale of the phase 3 ATTR-ACT clinical trial (Tafamidis in Transthyretin Cardiomyopathy Clinical Trial). Circ Heart Fail 2017;10(6):e003815.