Indirect treatment comparison of the efficacy of patisiran and inotersen for hereditary transthyretin-mediated amyloidosis with polyneuropathy

被引:12
|
作者
Gorevic, Peter [1 ]
Franklin, Jaclyn [2 ]
Chen, Jihong [2 ]
Sajeev, Gautam [3 ]
Wang, Jessie C. H. [3 ]
Lin, Hollis [2 ]
机构
[1] Mt Sinai Med Ctr, Dept Med Rheumatol, New York, NY 10029 USA
[2] Alnylam Pharmaceut, Cambridge, MA USA
[3] Anal Grp Inc, Boston, MA USA
关键词
Hereditary transthyretin-mediated amyloidosis; indirect comparison; patisiran; inotersen; neuropathy; quality of life; ADJUSTED INDIRECT COMPARISONS; CARE DECISION-MAKING; TASK-FORCE; METAANALYSIS; FAP;
D O I
10.1080/14656566.2020.1811850
中图分类号
R9 [药学];
学科分类号
1007 ;
摘要
Introduction Patisiran and inotersen are two therapies approved for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis with polyneuropathy, a rapidly progressive disease with a substantial clinical burden. This analysis indirectly compares the efficacy of patisiran and inotersen on neuropathy and quality of life (QOL). Methods Published results from the NEURO-TTR study of inotersen and individual patient data from the APOLLO study of patisiran were used. Indirect comparisons were conducted for 15-month changes in neuropathy and QOL endpoints: modified Neuropathy Impairment Score +7 (mNIS+7(Ionis,)), Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QOL-DN) questionnaire, body mass index (BMI), and Polyneuropathy Disability (PND) score. Analyses were conducted under different assumptions about the impact of missing data and to adjust for baseline differences between studies. Results Patisiran showed significantly greater treatment effects than inotersen for mNIS+7(Ionis)(mean difference: -12.3 [95% confidence interval: -21.4, -3.3]), Norfolk QOL-DN (-11.3 [-19.8, -2.9]), and BMI (1.0 [0.4, 1.7]). The proportion of patients with improvement or no change from baseline on PND score was higher for patisiran-treated patients (odds ratio: 8.9 [4.6, 17.5]). Results were consistent and robust across analyses and methods. Conclusions Patisiran demonstrated greater treatment effects on neuropathy and QOL than inotersen in patients with hATTR amyloidosis with polyneuropathy.
引用
收藏
页码:121 / 129
页数:9
相关论文
共 50 条
  • [1] An indirect treatment comparison of the efficacy of patisiran and tafamidis for the treatment of hereditary transthyretin-mediated amyloidosis with polyneuropathy
    Plante-Bordeneuve, Violaine
    Lin, Hollis
    Gollob, Jared
    Agarwal, Sonalee
    Betts, Marissa
    Fahrbach, Kyle
    Chitnis, Madhura
    Polydefkis, Michael
    [J]. EXPERT OPINION ON PHARMACOTHERAPY, 2019, 20 (04) : 473 - 481
  • [2] Response to the letter to the editor on "an indirect treatment comparison of the efficacy of patisiran and tafamidis for the treatment of hereditary transthyretin-mediated amyloidosis with polyneuropathy"
    Lin, Hollis
    Agarwal, Sonalee
    Betts, Marissa
    Fahrbach, Kyle
    Chitnis, Madhura
    Polydefkis, Michael
    [J]. EXPERT OPINION ON PHARMACOTHERAPY, 2019, 20 (12) : 1529 - 1530
  • [3] COST OF INOTERSEN AND PATISIRAN FOR THE TREATMENT OF ADULTS WITH POLYNEUROPATHY OF HEREDITARY TRANSTHYRETIN-MEDIATED AMYLOIDOSIS IN CANADA, GERMANY, AND ITALY
    Brown, D.
    Vera-Llonch, M.
    Shaff, M.
    Rolli, A.
    Vega, M.
    Blum, M.
    Nestler-Parr, S.
    Jiresch, M.
    Weycker, D.
    [J]. VALUE IN HEALTH, 2020, 23 : S606 - S607
  • [4] Letter to the editor concerning the article: "An indirect treatment comparison of the efficacy of patisiran and tafamidis for the treatment of hereditary transthyretin-mediated amyloidosis with polyneuropathy"
    Salvo, E. M.
    Samjoo, I. A.
    Tran, D.
    Cameron, C.
    [J]. EXPERT OPINION ON PHARMACOTHERAPY, 2019, 20 (12) : 1527 - 1528
  • [5] Inotersen for the treatment of adults with polyneuropathy caused by hereditary transthyretin-mediated amyloidosis
    Gertz, Morie A.
    Scheinberg, Morton
    Waddington-Cruz, Marcia
    Heitner, Stephen B.
    Karam, Chafic
    Drachman, Brian
    Khella, Sami
    Whelan, Carol
    Obici, Laura
    [J]. EXPERT REVIEW OF CLINICAL PHARMACOLOGY, 2019, 12 (08) : 701 - 711
  • [6] Patisiran for the treatment of hereditary transthyretin-mediated amyloidosis
    Yang, Junyi
    [J]. EXPERT REVIEW OF CLINICAL PHARMACOLOGY, 2019, 12 (02) : 95 - 99
  • [7] Patisiran in hereditary transthyretin-mediated amyloidosis
    Luigetti, Marco
    Servidei, Serenella
    [J]. LANCET NEUROLOGY, 2021, 20 (01): : 21 - 23
  • [8] Indirect treatment comparison (ITC) of the efficacy of vutrisiran and tafamidis for hereditary transthyretin-mediated amyloidosis with polyneuropathy
    Merkel, Madeline
    Danese, David
    Chen, Chongshu
    Wang, Jessie
    Wu, Aozhou
    Yang, Hongbo
    Lin, Hollis
    [J]. EXPERT OPINION ON PHARMACOTHERAPY, 2023, 24 (10) : 1205 - 1214
  • [9] Treatment with patisiran of a patient with hereditary transthyretin-mediated amyloidosis with stage 3 polyneuropathy
    Mueschen, Lars H.
    Koerner, Gudrun
    Gingele, Stefan
    Haenselmann, Anja
    Bavendiek, Udo
    Skripuletz, Thomas
    [J]. MUSCLE & NERVE, 2021, 64 (03) : E11 - E13
  • [10] SIMULATED TREATMENT COMPARISON AND MATCHINGADJUSTED INDIRECT COMPARISON OF THE EFFICACY OF EPLONTERSEN AND VUTRISIRAN FOR THE TREATMENT OF HEREDITARY TRANSTHYRETIN-MEDIATED AMYLOIDOSIS WITH POLYNEUROPATHY
    Karam, C.
    Gillmore, J. D.
    Chen, G.
    Jenkins, N. C.
    Hale, M. J.
    Chen, J.
    Viney, N. J.
    Schneider, E.
    [J]. VALUE IN HEALTH, 2023, 26 (12) : S46 - S47