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Observational Study
. 2020 Oct 28;22(1):257.
doi: 10.1186/s13075-020-02329-2.

Health Assessment Questionnaire-Disability Index (HAQ-DI) use in modelling disease progression in diffuse cutaneous systemic sclerosis: an analysis from the EUSTAR database

Yannick Allanore  1 , Sylvie Bozzi  2 , Augustin Terlinden  2 , Doerte Huscher  3 , Caroline Amand  2 , Christina Soubrane  2 , Elise Siegert  4 , László Czirják  5 , Patricia E Carreira  6 , Eric Hachulla  7 , Elisabetta Zanatta  8 , Mengtao Li  9 , Paolo Airò  10 , Fabian A Mendoza  11 , Edoardo Rosato  12 , Oliver Distler  13 ; EUSTAR Collaborators
Collaborators, Affiliations
Observational Study

Health Assessment Questionnaire-Disability Index (HAQ-DI) use in modelling disease progression in diffuse cutaneous systemic sclerosis: an analysis from the EUSTAR database

Yannick Allanore et al. Arthritis Res Ther. .

Abstract

Background: Patients with diffuse cutaneous systemic sclerosis (dcSSc) have a poor prognosis. The importance of monitoring subjective measures of functioning and disability, such as the Health Assessment Questionnaire-Disability Index (HAQ-DI), is important as dcSSc is rated by patients as worse than diabetes or hemodialysis for quality of life impairment. This European Scleroderma Trials and Research (EUSTAR) database analysis was undertaken to examine the importance of impaired functionality in dcSSc prognosis. The primary objectives were to identify predictors of death and HAQ-DI score progression over 1 year. HAQ-DI score, major advanced organ involvement, and death rate were also used to develop a comprehensive model to predict lifetime dcSSc progression.

Methods: This was an observational, longitudinal study in patients with dcSSc registered in EUSTAR. Death and HAQ-DI scores were, respectively, analyzed by Cox regression and linear regression analyses in relation to baseline covariates. A microsimulation Markov model was developed to estimate/predict natural progression of dcSSc over a patient's lifetime.

Results: The analysis included dcSSc patients with (N = 690) and without (N = 4132) HAQ-DI score assessments from the EUSTAR database. Baseline HAQ-DI score, corticosteroid treatment, and major advanced organ involvement were predictive of death on multivariable analysis; a 1-point increase in baseline HAQ-DI score multiplied the risk of death by 2.7 (p < 0.001) and multiple advanced major organ involvement multiplied the risk of death by 2.8 (p < 0.05). Multivariable analysis showed that baseline modified Rodnan Skin Score (mRSS) and baseline HAQ-DI score were associated with HAQ-DI score progression at 1 year (p < 0.05), but there was no association between baseline organ involvement and HAQ-DI score progression at 1 year. HAQ-DI score, major advanced organ involvement, and death were successfully used to model long-term disease progression in dcSSc.

Conclusions: HAQ-DI score and major advanced organ involvement were comparable predictors of mortality risk in dcSSc. Baseline mRSS and baseline HAQ-DI score were predictive of HAQ-DI score progression at 1 year, indicating a correlation between these endpoints in monitoring disease progression. It is hoped that this EUSTAR analysis may change physician perception about the importance of the HAQ-DI score in dcSSc.

Keywords: Diffuse cutaneous systemic sclerosis; EUSTAR registry; HAQ-DI score; Health Assessment Questionnaire-Disability Index.

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Conflict of interest statement

Yannick Allanore received fees or grants from Actelion, Bayer, Boehringer Ingelheim, Bristol Myers Squibb, Curzion, Inventiva, Roche, and Sanofi.

Oliver Distler had consultancy relationship and/or has received research funding from AbbVie, Actelion, Acceleron Pharma, Amgen, AnaMar, Baecon Discovery, Blade Therapeutics, Bayer, Boehringer Ingelheim, Catenion, Competitive Drug Development International Ltd., CSL Behring, ChemomAb, Curzion Pharmaceuticals, Ergonex, Galapagos NV, Glenmark Pharmaceuticals, GSK, Inventiva, Italfarmaco, iQone, iQvia, Lilly, Medac, Medscape, Mitsubishi Tanabe Pharma, MSD, Novartis, Pfizer, Roche, Sanofi, Target Bio Science, and UCB in the area of potential treatments of scleroderma and its complications. In addition, Oliver Distler has a patent mir-29 for the treatment of systemic sclerosis issued (US8247389, EP2331143).

Elise Siegert has received speaker fees/compensation for advisory board meetings from Actelion and Boehringer Ingelheim and a grant from Actelion.

László Czirják receives honoraria for consulting services (educational services, scientific articles, participation in advisory boards, clinical trials, other) from AbbVie, Actelion, Boehringer Ingelheim, Lilly, MSD, Novartis, Pfizer, Richter, Roche, and UCB.

Patricia E Carreira received fees or grants from Actelion, Boehringer Ingelheim, Emmerald Health Pharmaceuticals, Sanofi, AbbVie, Bristol Myers Squibb, and Mitsubishi Tanabe Pharma.

Eric Hachulla has no conflicts of interest to declare.

Elisabetta Zanatta had consultancy relationship and/or received fees from Actelion and GSK.

Mengtao Li declares no conflict of interest.

Paolo Airò has received consultancy and/or travel expenses from Bristol Myers Squibb, CSL Behring, Janssen, Novartis, Pfizer, Roche, and SOBI.

Fabian A Mendoza had a consultancy relationship with AbbVie, Genentech, and Gilead.

Edoardo Rosato has received research funding from Actelion.

Sylvie Bozzi, Augustin Terlinden, Caroline Amand, and Christina Soubrane are employees of Sanofi R&D, France.

Doerte Huscher has received traveling expenses from Actelion Pharmaceuticals, Boehringer Ingelheim, and Shire International GmbH.

Figures

Fig. 1
Fig. 1
EUSTAR cohort with dcSSc (February 11, 2019). ACR American College of Rheumatology, ACR/EULAR American College of Rheumatology/European League Against Rheumatism, dcSSc diffuse cutaneous systemic sclerosis, EUSTAR European Scleroderma Trials and Research, HAQ-DI Health Assessment Questionnaire-Disability Index, RP Raynaud’s phenomenon, SSc systemic sclerosis
Fig. 2
Fig. 2
Calculation of SMR using death rates from the EUSTAR dcSSc cohort (observed deaths) and deaths in the general population (expected deaths from the Italian life tables) in patients with ≥ 1 HAQ-DI score measurement. CI confidence interval, dcSSc diffuse cutaneous systemic sclerosis, EUSTAR European Scleroderma Trials and Research, GI gastrointestinal, HAQ-DI Health Assessment Questionnaire-Disability Index, PH pulmonary arterial hypertension, SMR standardized mortality ratio
Fig. 3
Fig. 3
Illustration of HAQ-DI transition states (0–0.5, 0.5–1.0, 1.0–1.5, 1.5–2.0, and 2.0–3.0), risk of advanced lung involvement, and death for a patient with dcSSc. HAQ-DI state in t − 1 influences the lung status in t. Similarly, the lung status in t − 1 influences the HAQ-DI state in t. The lung status in t − 1 (which is influenced by the HAQ-DI state in t − 2) influences the mortality in t. t = time in years, and f = “is a function of”. Note: Each HAQ-DI state should be included in a “triangle” as they all influence the lung and no lung states. However, due to lack of space on the diagram, two triangles were omitted. dcSSc diffuse cutaneous systemic sclerosis, HAQ-DI Health Assessment Questionnaire-Disability Index
Fig. 4
Fig. 4
Distribution of dcSSc patients over time between the various HAQ-DI, lung and mortality states as per an illustrative microsimulation that was run over 40 years for a cohort of dcSSc patients. Note: Model was based on 1000 simulated patients with specific baseline characteristics (33% male, age 50 years, HAQ-DI score < 0.5, no lung involvement, and known time since disease onset). dcSSc diffuse cutaneous systemic sclerosis, HAQ-DI Health Assessment Questionnaire-Disability Index
See this image and copyright information in PMC

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