TY - JOUR
T1 - Novel optical spectral transmission (OST)-guided versus conventionally disease activity-guided treatment
T2 - Study protocol of a randomized clinical trial on guidance of a treat-to-target strategy for early rheumatoid arthritis
AU - Besselink, N. J.
AU - Westgeest, A. A.A.
AU - Klaasen, R.
AU - Gamala, M.
AU - Van Woerkom, J. M.
AU - Tekstra, J.
AU - Verhoeven, M. M.A.
AU - Van Spil, W. E.
AU - Lafeber, F. P.J.G.
AU - Marijnissen, A. C.A.
AU - Van Laar, J. M.
AU - Jacobs, J. W.G.
N1 - Funding Information:
The study is funded by unrestricted grants from “Topconsortia Knowledge and Innovation Life Sciences & Health” (ZonMW LSH-2Treat 436001001), the Dutch Arthritis Foundation, and by Hemics BV. There are no restrictions on analyses and publications of study results in peer-reviewed journals (in accordance with the CONSORT criteria). The Department of Rheumatology of the University Medical Center Utrecht is sponsor and is the coordinating study centre.
Publisher Copyright:
© 2019 The Author(s).
PY - 2019/4/17
Y1 - 2019/4/17
N2 - Background: Assessment of disease activity is a critical component of tight-control, treat-to-target treatment strategies of rheumatoid arthritis (RA). Recently, the HandScan has been validated as a novel method for objectively assessing RA disease activity in only 1.5 min, using optical spectral transmission (OST) in hands and wrists. We describe the protocol of a randomized controlled clinical trial (RCT) to investigate whether HandScan-guided treatment aimed at 'HandScan remission' (HandScan arm) is at least as effective as and more cost-effective than clinically guided treatment aimed at ACR/EULAR 2011 Boolean remission (DAS arm). Methods/design: The study is a multi-center, double-blind, non-inferiority RCT of 18 months duration. Patients ≥ 18 years with newly diagnosed, disease-modifying antirheumatic drug (DMARD)-naïve RA according to the ACR 2010 classification criteria, will be randomized to the DAS arm or the HandScan arm. The efficacy of the arms will be compared by evaluating Health Assessment Questionnaire (HAQ) scores (primary outcome) after 18 months of DMARD therapy, aimed at remission. The equivalence margin in HAQ scores between study arms is 0.2. Secondary outcomes are differences in cost-effectiveness and radiographic joint damage between treatment arms. The non-inferiority sample size calculation to obtain a power of 80% at a one-sided p value of 0.05, with 10% dropouts, resulted in 61 patients per arm. In both arms, DMARD strategy will be intensified monthly according to predefined steps until remission is achieved; in both arms DMARDs and treatment steps are identical. If sustained remission, defined as remission that persists consistently over three consecutive months, is achieved, DMARD therapy will be tapered. Discussion: The study protocol and the specifically designed decision-making software application allow for implementation of this RCT. To test a novel method of assessing disease activity and comparing (cost-)effectiveness with the contemporary method in treat-to-target DMARD strategies in early RA patients. Trial registration: Dutch Trial Register, NTR6388. Registered on 6 April 2017 (NL50026.041.14). Protocol version 3.0, 19-01-2017.
AB - Background: Assessment of disease activity is a critical component of tight-control, treat-to-target treatment strategies of rheumatoid arthritis (RA). Recently, the HandScan has been validated as a novel method for objectively assessing RA disease activity in only 1.5 min, using optical spectral transmission (OST) in hands and wrists. We describe the protocol of a randomized controlled clinical trial (RCT) to investigate whether HandScan-guided treatment aimed at 'HandScan remission' (HandScan arm) is at least as effective as and more cost-effective than clinically guided treatment aimed at ACR/EULAR 2011 Boolean remission (DAS arm). Methods/design: The study is a multi-center, double-blind, non-inferiority RCT of 18 months duration. Patients ≥ 18 years with newly diagnosed, disease-modifying antirheumatic drug (DMARD)-naïve RA according to the ACR 2010 classification criteria, will be randomized to the DAS arm or the HandScan arm. The efficacy of the arms will be compared by evaluating Health Assessment Questionnaire (HAQ) scores (primary outcome) after 18 months of DMARD therapy, aimed at remission. The equivalence margin in HAQ scores between study arms is 0.2. Secondary outcomes are differences in cost-effectiveness and radiographic joint damage between treatment arms. The non-inferiority sample size calculation to obtain a power of 80% at a one-sided p value of 0.05, with 10% dropouts, resulted in 61 patients per arm. In both arms, DMARD strategy will be intensified monthly according to predefined steps until remission is achieved; in both arms DMARDs and treatment steps are identical. If sustained remission, defined as remission that persists consistently over three consecutive months, is achieved, DMARD therapy will be tapered. Discussion: The study protocol and the specifically designed decision-making software application allow for implementation of this RCT. To test a novel method of assessing disease activity and comparing (cost-)effectiveness with the contemporary method in treat-to-target DMARD strategies in early RA patients. Trial registration: Dutch Trial Register, NTR6388. Registered on 6 April 2017 (NL50026.041.14). Protocol version 3.0, 19-01-2017.
KW - Optical spectral transmission (OST) treat-to-target
KW - Randomized controlled trial (RCT)
KW - remission
KW - Rheumatoid arthritis
KW - Tight-control treatment
UR - http://www.scopus.com/inward/record.url?scp=85064526482&partnerID=8YFLogxK
U2 - 10.1186/s13063-019-3285-8
DO - 10.1186/s13063-019-3285-8
M3 - Article
C2 - 30999969
AN - SCOPUS:85064526482
SN - 1745-6215
VL - 20
JO - Trials
JF - Trials
IS - 1
M1 - 226
ER -