TY - JOUR
T1 - The ALSFRS-R Summit
T2 - a global call to action on the use of the ALSFRS-R in ALS clinical trials
AU - Genge, Angela
AU - Cedarbaum, Jesse M.
AU - Shefner, Jeremy
AU - Chio, Adriano
AU - Al-Chalabi, Ammar
AU - Van Damme, Philip
AU - McDermott, Chris
AU - Glass, Jonathan
AU - Berry, James
AU - van Eijk, Ruben P.A.
AU - Fournier, Christina
AU - Grosskreutz, Julian
AU - Andrews, Jinsy
AU - Bertone, Vanessa
AU - Bunte, Tommy M.
AU - Couillard, Mathias
AU - Cummings, Cathy
AU - Kittle, Gale
AU - Polzer, John
AU - Salmon, Kristiana
AU - Straub, Corey
AU - van den Berg, Leonard H.
N1 - Publisher Copyright:
© 2024 The Author(s). Published by Informa UK Limited, trading as Taylor & Francis Group.
PY - 2024/5
Y1 - 2024/5
N2 - The Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS) was developed more than 25 years ago as an instrument to monitor functional change over time in patients with ALS. It has since been revised and extended to meet the needs of high data quality in ALS trials (ALSFRS-R), however a full re-validation of the scale was not completed. Despite this, the scale has remained a primary outcome measure in clinical trials. We convened a group of clinical trialists to discuss and explore opportunities to improve the scale and propose alternative measures. In this meeting report, we present a call to action on the use of the ALSFRS-Revised scale in clinical trials, focusing on the need for (1) harmonization of the ALSFRS-R administration globally, (2) alignment on a set of recommendations for clinical trial design and statistical analysis plans (SAPs), and (3) use of additional outcome measures.
AB - The Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS) was developed more than 25 years ago as an instrument to monitor functional change over time in patients with ALS. It has since been revised and extended to meet the needs of high data quality in ALS trials (ALSFRS-R), however a full re-validation of the scale was not completed. Despite this, the scale has remained a primary outcome measure in clinical trials. We convened a group of clinical trialists to discuss and explore opportunities to improve the scale and propose alternative measures. In this meeting report, we present a call to action on the use of the ALSFRS-Revised scale in clinical trials, focusing on the need for (1) harmonization of the ALSFRS-R administration globally, (2) alignment on a set of recommendations for clinical trial design and statistical analysis plans (SAPs), and (3) use of additional outcome measures.
KW - Clinical trial design
KW - clinical trial outcome measures
KW - functional rating scale
KW - harmonization
KW - statistical analysis
UR - https://www.scopus.com/pages/publications/85186570016
U2 - 10.1080/21678421.2024.2320880
DO - 10.1080/21678421.2024.2320880
M3 - Article
C2 - 38396337
AN - SCOPUS:85186570016
SN - 2167-8421
VL - 25
SP - 382
EP - 387
JO - Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration
JF - Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration
IS - 3-4
ER -