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Table 1 Characteristics of Selected Studies

From: Rate of neuropathic progression in hereditary transthyretin amyloidosis with polyneuropathy and other peripheral neuropathies: a systematic review and meta-analysis

First Author /Principal Investigator Year Study Name/Identifier Study Type Disease Type Age: Mean (SD) Male Gender:
N (%)
Benson [6] 2018 NEURO-TTR: NCT01737398 RCT ATTRv-PN 60 (14) 41 (68%)
Berk [7] 2013 NA RCT ATTRv-PN 59 (12) 44 (67%)
Coelho [8] 2012 FX005: NCT00409175 RCT ATTRv-PN 38 (13) 26 (43%)
Hernandez-Ojeda [28] 2012 NA RCT DPN 57 (9) 6 (24%)
Hor [29] 2018 NCT01973400 RCT DPN 57 (9) 63 (42%)
Karama 2020 NA Non-interventional study CMT 51 (13) 6 (50%)
DPN 65 (11) 4 (36%)
ATTRv-PN 67 (7) 9 (90%)
IPN 66 (13) 7 (58%)
Luigetti [30] 2018 NA Non-interventional study ATTRv-PN 69 (9) 15 (83%)
Mundayat [31] 2018 THAOS Non-interventional study ATTRv-PN 41 (9) NA
Sahenk [19] 2005 NA RCT CMT 33 (5) 3 (75%)
Shy [32] 2008 NA Non-interventional study CMT 40 (19) 30 (42%)
Tesfaye [33] 2007 Rochester Diabetic Neuropathy Study: NCT00044408 and NCT00044395 RCT DPN 48 (9) 115 (44%)
Windebank [34] 2004 NA RCT Small fiber peripheral neuropathy 62 (11) NA
Ziegler [35] 1999 ALADIN 3 RCT DPN 57 (6) 83 (50%)
Ziegler [37] 2009 AV-007-IM: NCT00483730 RCT DPN 56 (6) 77 (27%)
Ziegler [36] 2011 NATHAN 1: NCT00977483 RCT DPN 54 (8) 150 (67%)
  1. Abbreviations: CMT Charcot-Marie-Tooth disease, DPN diabetic peripheral neuropathy, ATTRv-PN hereditary transthyretin amyloidosis with polyneuropathy, NA not applicable, RCT randomized controlled trial, SD standard deviation
  2. aUnpublished dataset