scholarly journals AB0413 HIGH-RESOLUTION COMPUTED TOMOGRAPHY FOR THE SCREENING, RE-SCREENING AND FOLLOW-UP OF SYSTEMIC SCLEROSIS RELATED INTERSTITIAL LUNG DISEASE: RESULTS OF A EUSTAR-SCTC SURVEY

2021 ◽  
Vol 80 (Suppl 1) ◽  
pp. 1234.2-1235
Author(s):  
C. Bruni ◽  
L. Chung ◽  
A. M. Hoffmann-Vold ◽  
S. Assassi ◽  
A. Gabrielli ◽  
...  
Keyword(s):  
Systemic Sclerosis ◽  
Medical Specialty ◽  
Working Environment ◽  
Research Support ◽  
High Resolution Computed Tomography ◽  
Awareness Programs ◽  
Scientific Group ◽  
Relative Decline ◽  
New Onset

Background:High-resolution computed tomography (HRCT) is the gold standard diagnostic test for Interstitial lung disease (ILD), a significant cause of morbidity and mortality in systemic sclerosis (SSc). Different algorithms have been proposed for the screening of SSc-ILD, including the use of pulmonary function tests (Forced Vital Capacity - FVC, Lung Diffusion of Carbone Monoxyde - DLCO). A prior survey reported that 50-66% of general rheumatologists and SSc experts ordered HRCT for ILD screening in newly diagnosed SSc patients (1).Objectives:Given the recent availability of on-label treatment for SSc-ILD (2), the publication of consensus recommendations for the identification of SSc-ILD (3) and recent awareness programs for the use of HRCT to detect SSc-ILD, we aimed to re-evaluate the use of HRCT for screening, re-screening and follow-up of SSc-ILD.Methods:An invitation was sent to the European Scleroderma Trials and Research (EUSTAR) and Scleroderma Clinical Trials Consortium (SCTC) members, also advertised through social media. Answers were recorded between Nov 25th and Dec 31st 2020. Questions were asked on the use of chest HRCT at baseline, the re-screening of patients with a negative baseline HRCT and the follow-up of HRCT positive SSc-ILD patients. When HRCT was not routinely requested, additional details were collected about the parameters guiding its use. The results of the survey were tested for association with geographical origin, medical specialty, working environment, SSc referral institute and scientific group membership of the responders, using Chi-squared test.Results:205/630 (32.5%) physicians replied to the survey. Participants were widely distributed in terms of geographical origin (130 Europe, 23 Asia, 23 North America, 31 other continents), medical specialty (156 rheumatology, 21 internal medicine, 14 clinical immunology, 14 other), working environment (176 University Hospital, 12 community hospital, 17 other), SSc dedicated clinic (179 referral and 26 non-referral) and scientific group membership (98 EUSTAR, 42 SCTC, 42 EUSTAR and SCTC, 23 not declared).At SSc diagnosis, 95.7% of responders would perform HRCT: 66.7% as routine screening for ILD (67,4% of SSc referral and 62% for non-referral physicians) and 29% for diagnostic purposes (among the latter, if crackles on auscultation – 92.5%, FVC<80% predicted - 86.6%, FVC±DLCO relative decline reaching the current definition of ILD progression, 86.6% or dyspnea at rest/exercise - 85.1/83.3%).During follow-up, 78.8% of responders would repeat an HRCT in baseline negative cases: 20.3% as a yearly routine screening and 64.5% for diagnostic aims (decision on the latter group was more frequently driven by FVC±DLCO relative decline indicative of ILD progression– 90.6%, new onset or worsening of dyspnoea at rest/exercise – 80.5/86.6%, new onset or worsening of lung crackles on auscultation – 82.6%).Finally, 94.5% of responders would repeat a chest HRCT after SSc-ILD diagnosis: 36.8% as a yearly routine and 56.7% according to clinical evaluation (driven by new FVC±DLCO relative decline based ILD progression – 90.8%, new onset or worsening of dyspnoea at rest/exercise – 83.2/81.7%; 5.2% to evaluate treatment effects). We found no difference in the distribution of answers among groups.Conclusion:The use of baseline HRCT for the screening of SSc-ILD has slightly increased in non-referral and remained stable in referral centers compared to previous surveys, indicating that the implementation of guidelines might be successful and awareness programs should be continued. In addition, we provide new data on use of HRCT in re-screening and follow-up. The development of validated algorithms to further support the appropriate application of HRCT at follow-up is highly needed.References:[1]Bernstein EJ et al. Arthritis Rheumatol. 2018 Jun;70(6):971-972.[2]Distler O et al. N Engl J Med. 2019 Jun 27;380(26):2518-2528.[3]Hoffmann-Vold AM et al. The Lancet Rheumatology, Volume 2, Issue 2, e71 - e83.Disclosure of Interests:Cosimo Bruni Speakers bureau: Actelion, Consultant of: Eli Lilly, Grant/research support from: Foundation for Research in Rheumatology (FOREUM), Gruppo Italiano Lotta alla Sclerodermia (GILS), Fondazione Italiana per la Ricerca sull’Artrite (FIRA), New Horizon Fellowship, European Sclerodermia Trial and Reserach (EUSTAR) Group., Lorinda Chung Consultant of: Boehringer Ingelheim, Eicos, Mitsubishi Tanabe, Reata., Anna-Maria Hoffmann-Vold Consultant of: Actelion, ARXX therapeutics, Bayer, Boehringer-Ingelheim, Medscape, MSD, Lilly, Roche, Shervin Assassi Speakers bureau: Integrity Continuing Education, Consultant of: Boehringer Ingelheim, Novartis, and Corbus, Armando Gabrielli: None declared, Dinesh Khanna Consultant of: Acceleron, Actelion, Abbvie, Amgen, Bayer, Boehringer Ingelheim, CSL Behring, Corbus, Gilead, Galapagos, Genentech/Roche, GSK, Horizon, Merck, Mitsubishi Tanabe Pharma, Sanofi-Aventis, and United Therapeutics Leadership, Grant/research support from: NIH, Immune Tolerance Network, Bayer, BMS, Horizon, Pfizer, Employee of: Equity position – Chief Medical Officer, Eicos Sciences, Inc., Elana Bernstein Consultant of: Boehringer Ingelheim, Oliver Distler Consultant of: Abbvie, Acceleron Pharma, Amgen, AnaMar, Arxx Therapeutics, Baecon Discovery, Blade Therapeutics, Bayer, Boehringer Ingelheim, ChemomAb, Corbus Pharmaceuticals, CSL Behring, Galapagos NV, Glenmark Pharmaceuticals, GSK, Horizon (Curzion) Pharmaceuticals, Inventiva, iQvia, Italfarmaco, iQone, Kymera Therapeutics, Lilly, Medac, Medscape, Mitsubishi Tanabe Pharma, MSD, Novartis, Pfizer, Roche, Sanofi, Serodapharm, Topadur, Target Bioscience and UCB., Grant/research support from: Abbvie, Acceleron Pharma, Amgen, AnaMar, Arxx Therapeutics, Baecon Discovery, Blade Therapeutics, Bayer, Boehringer Ingelheim, ChemomAb, Corbus Pharmaceuticals, CSL Behring, Galapagos NV, Glenmark Pharmaceuticals, GSK, Horizon (Curzion) Pharmaceuticals, Inventiva, iQvia, Italfarmaco, iQone, Kymera Therapeutics, Lilly, Medac, Medscape, Mitsubishi Tanabe Pharma, MSD, Novartis, Pfizer, Roche, Sanofi, Serodapharm, Topadur, Target Bioscience and UCB. Patent issued “mir-29 for the treatment of systemic sclerosis” (US8247389, EP2331143).

scholarly journals SAT0500 HOW THE ADULT CRISS WORKS IN PEDIATRIC jSSc PATIENTS - RESULTS FROM THE JUVENILE SCLERODERMA INCEPTION COHORT

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 1206.2-1206
Author(s):  
J. Klotsche ◽  
I. Foeldvari ◽  
O. Kasapcopur ◽  
A. Adrovic ◽  
K. Torok ◽  
...  
Keyword(s):  
Systemic Sclerosis ◽  
Area Under Curve ◽  
Health Assessment ◽  
Organ Damage ◽  
Left Ventricular ◽  
Inception Cohort ◽  
Research Support ◽  
Predicted Probability ◽  
New Onset

Background:The Composite Response Index in Systemic Sclerosis (CRISS) was developed by Dinesh Khanna as a response measure in patients with adult systemic sclerosis. CRISS aims to capture the complexity of systemic sclerosis and to provide a sensitive measure for change in disease activity. The CRISS score is based on a two-step approach. First, significant disease worsening or new-onset organ damage is defined as non-responsiveness. In patients who did not fulfill the criteria of part one, a probability of improvement is calculated for each patient based the Rodnan Skin Score (mRSS), percent predicted forced vital capacity (FVC%), patient and physician global assessments (PGA), and the Health Assessment Questionnaire Disability Index (HAQ-DI). A probability of 0.6 or higher indicates improvement.Objectives:The objective of this study was to validate the CRISS in a prospectively followed cohort of patients with juvenile systemic sclerosis (jSSc).Methods:Data from the prospective international inception cohort for jSSc was used to validate the CRISS. Patients with an available 12-months follow-up were included in the analyses. Clinically improvement was defined by the anchor question about improvement (much better or little better versus almost the same, little worse or much worse) in patients overall health due to scleroderma since the last visit provided by the treating physician.Results:Forty seven jSSc patients were included in the analysis. 74.2% had diffuse subtype. The physician rated the disease as improved in 34 patients (72.3%) since the last visit. No patient had a renal crisis or new onset of left ventricular failure during the 12-months follow-up. Three patients (3.4%) each had a new onset or worsening of lung fibrosis and new onset of pulmonary arterial hypertension. In total, 6 patients resulted in a rating of not improved based on the CRISS in part I. The mRSSS, FVC%, CHAQ and PGA significantly improved during the 12-months follow-up in patients who were rated as improved. The predicted probability based on the CRISS algorithm resulted in an area under curve of 0.77 predicting the anchor question of improvement. In summary, 33 (70.0%) patients were correctly classified by the adult CRISS score resulting in an overall area under curve of 0.7.Conclusion:The CRISS score was evaluated in a pediatric jSSc cohort for the first time. It showed a good performance. However, it seems that the formula of part II of the CRISS score needs a calibration to pediatric jSSc patients.Disclosure of Interests:Jens Klotsche: None declared, Ivan Foeldvari Consultant of: Novartis, Ozgur Kasapcopur: None declared, Amra Adrovic: None declared, Kathryn Torok: None declared, Valda Stanevicha: None declared, Jordi Anton Grant/research support from: grants from Pfizer, abbvie, Novartis, Sobi. Gebro, Roche, Novimmune, Sanofi, Lilly, Amgen, Grant/research support from: Pfizer, abbvie, Novartis, Sobi. Gebro, Roche, Novimmune, Sanofi, Lilly, Amgen, Consultant of: Novartis, Sobi, Pfizer, abbvie, Consultant of: Novartis, Sobi, Pfizer, abbvie, Speakers bureau: abbvie, Pfizer, Roche, Novartis, Sobi, Gebro, Speakers bureau: abbvie, Pfizer, Roche, Novartis, Sobi, Gebro, edoardo marrani: None declared, Maria T. Terreri: None declared, Flávio R. Sztajnbok: None declared, Cristina Battagliotti: None declared, Lillemor Berntson Consultant of: paid by Abbvie as a consultant, Speakers bureau: paid by Abbvie for giving speaches about JIA, Despina Eleftheriou: None declared, Gerd Horneff Grant/research support from: AbbVie, Chugai, Merck Sharp & Dohme, Novartis, Pfizer, Roche, Speakers bureau: AbbVie, Bayer, Chugai, Merck Sharp & Dohme, Novartis, Pfizer, Roche, Farzana Nuruzzaman: None declared, Nicola Helmus: None declared

scholarly journals FRI0495 FOLLOW UP OF INTERSTITIAL PNEUMONIA WITH AUTOIMMUNE FEATURES – THE EXPERIENCE OF ONE CENTRE

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 846.3-846
Author(s):  
N. Madeira ◽  
M. Alvarenga Santos ◽  
L. Cunha Miranda ◽  
S. Clemente ◽  
S. Furtado
Keyword(s):  
Interstitial Pneumonia ◽  
Respiratory Symptoms ◽  
Interstitial Lung Diseases ◽  
European Respiratory Society ◽  
High Resolution Computed Tomography ◽  
Disease Evolution ◽  
Clinical Criteria ◽  
Clinical Evolution ◽  
Relative Decline

Background:Interstitial Lung Diseases (ILD) may present features suggesting an underlying autoimmune process, which seem to differentiate them from idiopathic interstitial pneumonias, although without fully meeting the classification criteria (CC) for a specific connective tissue disease. Different terms had been used to describe these conditions and, to reach a consensus, the European Respiratory Society/American Thoracic Society proposed the CC for an entity named Interstitial Pneumonia with Autoimmune Features (IPAF). Clinical evolution and prognosis of this entity are still poorly understood.Objectives:To evaluate clinical evolution and prognosis of a population of patients with IPAF.Methods:Retrospective analysis of clinical files of patients followed by the Pulmonology Department since 02/2012 until 06/2019, who met the CC for IPAF, regarding clinical, functional and radiological evolution. Patients were considered to have a progressive phenotype in 24±3 months from their 1stevaluation if they fulfil 1 of the 4 criteria: relative decline in FVC ≥10% predicted; relative decline in FVC ≥5–<10% predicted and worsened respiratory symptoms; relative decline in FVC ≥5–<10% predicted and increased extent of fibrosis on High-resolution Computed Tomography (HRCT); worsened respiratory symptoms and increased extent of fibrosis on HRCT.Results:22 (7.4%) of 296 ILD patients met IPAF CC. 59.0% were female with an age at the 1stevaluation of 66.7±12.4 years. They were all non-smokers (63.6%) or ex-smokers (36.4%). Serologic and morphologic criteria were both present in 21 (95.4%) and clinical criteria in 5 patients (22.7%). Antinuclear antibodies (ANA) were identified in 19, rheumatoid factor in 4, SSA in 3 and anti-Jo-1 in 1 patient. HRCT patterns were identified in 21 patients: 15 nonspecific interstitial pneumonia (NSIP), 5 organizing pneumonia (OP) and 2 lymphocytic interstitial pneumonia (LIP). One NSIP and 1 LIP identified on HRCT were confirmed by histopathology. Three patients had inflammatory arthritis and 2 had Raynaud’s phenomenon. Immunosuppressive therapy was introduced in most cases (18 patients, including systemic corticotherapy in 17, azathioprine in 4, mycophenolate mofetil in 1), azithromycin was prescribed in 2 patients and 3 remained without therapy. Regarding the follow up at 24±3 months from the 1stevaluation (3 patients were excluded due to too recent follow-up), 4 patients (18.2%) had progressive phenotype, 7 (31.8%) had a favourable evolution and 3 (13.6%) patients had died. During a follow-up of 31.1±19.8 months, this number rose to 6 patients (27.3%), all of them died by respiratory cause and had NSIP pattern. No differences were found in age, last FVC, therapy and time of disease evolution between those who died and the others.Conclusion:Our study showed that a small proportion of IPAF patients had a progressive phenotype and the NSIP pattern seemed to be a poor prognosis factor for survival.References:[1]Ito Y, Arita M, Kumagai S, et al. Serological and morphological prognostic factors in patients with interstitial pneumonia with autoimmune features. BMC Pulm Med 2017; 17:111 10.1186/s12890-017-0453-zDisclosure of Interests:None declared

scholarly journals SAT0032 INCIDENCE OF RHEUMATOID ARTHRITIS IN PATIENTS WITH NEW ONSET OF MUSCULOSKELETAL SYMPTOMS AND ANTI-CPP POSITIVITY COMPARED TO ANTI-CPP NEGATIVE PATIENTS

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 946.1-946
Author(s):  
S. Dauth ◽  
M. Köhm ◽  
T. Oberwahrenbrock ◽  
U. Henkemeier ◽  
T. Rossmanith ◽  
...  
Keyword(s):  
Rheumatoid Arthritis ◽  
Point Of Care ◽  
Early Arthritis ◽  
Clinical Parameters ◽  
Research Support ◽  
Point Of Care Test ◽  
Test Elisa ◽  
Patient Reported ◽  
New Onset

Background:Rheumatoid Arthritis (RA) is a chronic inflammatory joint disease. Strategies for its early detection and diagnosis are of high importance as prompt treatment improves clinical and structural outcome. Autoantibodies against cyclic citrullinated proteins (anti-CCP) have been associated with RA-development. Non-specific musculoskeletal (nsMSK) symptoms are often described prior to RA development. Majority of patients with nsMSK symptoms present to their general practice (GP) first. Studies of early arthritis cohorts have shown that many early arthritis patients cannot be accurately diagnosed at their first visit and are often referred as undifferentiated arthritis patients.Objectives:To evaluate the incidence of anti-CCP positivity in patients with new onset of nsMSK symptoms and the incidence of RA in these patients over a 3-year follow-up period compared to anti-CPP negative patients.Methods:In this prospective study (PANORA), 978 patients with new onset of nsMSK symptoms were included in 77 GP sites in Germany. Patients with a positive anti-CCP rapid-test (CCPoint®) were referred to Rheumatology Department (RD) for rheumatological assessment, RA-evaluation and an anti-CCP validation test (ELISA). ELISA anti-CCP positive patients without RA were monitored every 6 months for a total follow-up of 36 months or until RA-diagnosis. Patients with a negative anti-CPP result (CCPoint® or ELISA) are followed up with a questionnaire after 1 and 3 y.Results:From 978 included patients, 105 (10.7%) were CCPoint® positive. 96 were tested with ELISA and 27 (28.1%) were confirmed anti-CCP positive. 9 (33.3%) were diagnosed with RA at the first RD visit (study visit 2); 4 further patients were diagnosed with RA during the follow-up (FU) period so far. Overall, 48.1% of ELISA-positive (ELISA+) patients were diagnosed with RA up to now; 11 ELISA+ patients are still in the FU period of the study. Of the 868 CCPoint® negative patients, currently, 282 have filled out a 1-year FU questionnaire; 3.5% of those reported a RA diagnosis (Table 1). As expected, clinical parameters at V2 (e.g. CRP, swollen and tender joint count) were worse in the ELISA+/RA+ group compared to the ELISA-/RA- group, but no obvious differences were detected between ELISA+ patients who were diagnosed with RA during the FU period (after V2) and ELISA-/RA- patientsTable 1.Number and percentage of patients with a RA diagnosisAnti-CCP statusVisit 2Follow-up*TotalPoint-of-Care Test --3.5% (10 of 282)#3.5% (10 of 282)#Point-of-Care Test + / ELISA -2.9% (2 of 69)0% (0 of 34)#2.9% (2 of 69)Point-of-Care Test + / ELISA +33.3% (9 of 27)14.8% (4 of 27)48.1% (13 of 27)$* 1 year-questionnaire for Point-of-Care Test and ELISA negative patients or every 6 months follow-up for ELISA positive patients;#Patient-reported;$11 patients are still in the follow-up phase of the studyConclusion:Currently, 48.1% of anti-CCP+ (ELISA) patients have received a RA diagnosis, whereas 3.5% of the anti-CCP- (CCPoint®) received a RA diagnosis (patient reported), which underlines, that anti-CCP can be used as a marker to identify high-risk patients in GP setting. While clinical parameters are correlated with the diagnosis of RA, they are not suited for predicting future RA development alone. Anti-CCP, possibly in combination with additional parameters imaging, might increase the likelihood to early diagnose or predict RA development.Figure 1.Study overview: Patient distribution depending on anti-CCP results and RA diagnosis.Disclosure of Interests:Stephanie Dauth Grant/research support from: BMS, Michaela Köhm Grant/research support from: Pfizer, Janssen, BMS, LEO, Consultant of: BMS, Pfizer, Speakers bureau: Pfizer, BMS, Janssen, Novartis, Timm Oberwahrenbrock Grant/research support from: BMS, Ulf Henkemeier: None declared, Tanja Rossmanith Grant/research support from: Janssen, BMS, LEO, Pfizer, Karola Mergenthal Grant/research support from: BMS, Juliana J. Petersen Grant/research support from: BMS, Harald Burkhardt Grant/research support from: Pfizer, Roche, Abbvie, Consultant of: Sanofi, Pfizer, Roche, Abbvie, Boehringer Ingelheim, UCB, Eli Lilly, Chugai, Bristol Myer Scripps, Janssen, and Novartis, Speakers bureau: Sanofi, Pfizer, Roche, Abbvie, Boehringer Ingelheim, UCB, Eli Lilly, Chugai, Bristol Myer Scripps, Janssen, and Novartis, Frank Behrens Grant/research support from: Pfizer, Janssen, Chugai, Celgene, Lilly and Roche, Consultant of: Pfizer, AbbVie, Sanofi, Lilly, Novartis, Genzyme, Boehringer, Janssen, MSD, Celgene, Roche and Chugai

scholarly journals OP0252 CIRCULATING COLLAGEN TURNOVER MARKERS ARE SPECIFICALLY CHANGED IN VERY EARLY SYSTEMIC SCLEROSIS

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 159.1-159
Author(s):  
R. Dobrota ◽  
S. Jordan ◽  
P. Juhl ◽  
B. Maurer ◽  
M. O. Becker ◽  
...  
Keyword(s):  
Systemic Sclerosis ◽  
Roc Analysis ◽  
Cox Regression ◽  
Classification Criteria ◽  
Full Time ◽  
Research Support ◽  
Type Iii ◽  
Turnover Markers ◽  
Full Time Employee

Background:Timely diagnosis of patients with very early systemic sclerosis (veSSc) is essential for their personalized and optimal management. We hypothesise that changes in serum-based extracellular matrix (ECM) turnover biomarkers are already detectable in patients with veSSc, even before occurrence of specific clinical signs.Objectives:To investigate circulating ECM turnover markers as potential biomarkers for veSSc.Methods:Patients with veSSc, n=42, defined as presence of Raynaud’s syndrome and at least one of puffy fingers, positive antinuclear antibodies or pathological nailfold capillaroscopy, who did not meet any classification criteria for SSc, were compared to healthy controls (HC, n=29). Longitudinal assessment, data and sera collection were conducted by EUSTAR standards. ECM-degradation (BGM, C3M, C4M, C6M) and ECM-formation biomarkers (PRO-C3, PRO-C4, PRO-C5) were measured in serum using ELISA assays. The statistical analyses included Mann-Whitney U, Spearman correlation and ROC analysis. Using Kaplan-Meier plots and univariable Cox regression, we explored if biomarkers can predict progression towards definite SSc (fulfillment of ACR/EULAR criteria or minimum two points increase in the criteria score) during the longitudinal follow-up.Results:Compared to HC, veSSc patients showed a deregulated turnover of type III and IV collagen, with higher degradation (higher C3M, C4M, both p<0.0001 and PRO-C3, p=0.004, Figure 1a, resulting in lower turnover ratios PRO-C3/C3M and PRO-C4/C4M, both p<0.0001). The biglycan degradation biomarker BGM was also higher in veSSc (p=0.006), whereas the degradation biomarker for type VI collagen, C6M, was lower than in HC (p=0.002). In the ROC analysis, biomarkers of type III and IV collagen distinguished between veSSc and HC: C3M, AUC=0.95, p<0.0001; C4M, AUC=0.97, p<0.0001; turnover ratios PRO-C3/C3M, AUC=0.80, p<0.0001; PRO-C4/C4M, AUC=0.97; p<0.0001 (Figure 1b).Median follow up was 4.5 years (range 0.5-7.9 years), mean age was 50±2.2 years, 88% female gender, 24% with puffy fingers, 92% were ANA positive, 64% had an abnormal capillaroscopy, none had organ involvement or skin fibrosis. 14/42 veSSc patients fulfilled the ACR/EULAR classification criteria at follow-up (time to fulfilment of criteria ranged between 0.5 and 6.8 years from inclusion) and in addition, 18/42 veSSc patients gained at least two classification criteria-points. This resulted in 14, respectively 18 progressors for the longitudinal analysis. However, in univariable Cox regression, the baseline levels of the markers did not predict progression over time.Conclusion:ECM turnover is already altered in veSSc patients compared to HC. Biomarkes of type III and IV collagen distinguished between veSSc patients and HC, which may indicate them as potential biomarkers for the detection of veSSc in addition to the established immunological and capillaroscopic criteria.Disclosure of Interests:Rucsandra Dobrota: None declared, Suzana Jordan: None declared, Pernille Juhl Employee of: Nordic Bioscience, Britta Maurer Grant/research support from: AbbVie, Protagen, Novartis, congress support from Pfizer, Roche, Actelion, and MSD, Speakers bureau: Novartis, Mike O. Becker: None declared, Carina Mihai: None declared, Anne-Christine Bay-Jensen Shareholder of: Nordic Bioscience A/S, Employee of: Full time employee at Nordic Bioscience A/S., Morten Karsdal Shareholder of: Nordic Bioscience A/S., Employee of: Full time employee at Nordic Bioscience A/S., Anne Sofie Siebuhr Employee of: Nordic Bioscience, Oliver Distler Grant/research support from: Grants/Research support from Actelion, Bayer, Boehringer Ingelheim, Competitive Drug Development International Ltd. and Mitsubishi Tanabe; he also holds the issued Patent on mir-29 for the treatment of systemic sclerosis (US8247389, EP2331143)., Consultant of: Consultancy fees from Actelion, Acceleron Pharma, AnaMar, Bayer, Baecon Discovery, Blade Therapeutics, Boehringer, CSL Behring, Catenion, ChemomAb, Curzion Pharmaceuticals, Ergonex, Galapagos NV, GSK, Glenmark Pharmaceuticals, Inventiva, Italfarmaco, iQvia, medac, Medscape, Mitsubishi Tanabe Pharma, MSD, Roche, Sanofi and UCB, Speakers bureau: Speaker fees from Actelion, Bayer, Boehringer Ingelheim, Medscape, Pfizer and Roche

scholarly journals POS0866 TWO-DIMENSIONAL HRCT-BASED RADIOMIC FEATURES IN SSC-ILD DISTINGUISH DRUG RESPONDERS FROM NON-RESPONDERS

2021 ◽  
Vol 80 (Suppl 1) ◽  
pp. 688-689
Author(s):  
C. Meier ◽  
M. Maciukiewicz ◽  
M. Brunner ◽  
J. Schniering ◽  
H. Gabrys ◽  
...  
Keyword(s):  
Computed Tomography ◽  
Systemic Sclerosis ◽  
Interstitial Lung Disease ◽  
Linear Regression ◽  
Lung Disease ◽  
Treatment Response ◽  
Two Dimensional ◽  
Research Support ◽  
Pre Treatment

Background:Management of patients with systemic sclerosis-associated interstitial lung disease (SSc-ILD) is complicated by high inter-patient variability. To date, no validated predictors of treatment response are available for routine use. High resolution computed tomography (HRCT)-based radiomics, i.e. the high-dimensional, quantitative analysis of imaging metadata, have previously been shown to be successful in discriminating (SSc-)ILD phenotypes in preclinical and clinical studies1. Since HRCT is an integral part of the routine work-up in SSc, HRCT-based radiomic features may hold potential as non-invasive biomarkers.Objectives:To predict treatment response using two-dimensional (2D) HRCT-based radiomics in SSc-ILD patients from a prospectively followed cohort.Methods:Inclusion criteria were diagnosis of SSc-ILD in HRCT, availability of a suitable chest HRCT scan within 12 months prior to initiation of a new treatment, and availability of clinical baseline and follow-up information. Treatment response was defined as the absence of all of the following over a follow-up period of 12-24 months: relative decrease in forced vital capacity (FVC) ≥5%, increase of ILD in HRCT as assessed by a radiologist, change in treatment regimen due to insufficient response, ILD-related death or lung transplantation. Of each pre-treatment HRCT, 6 slices (15±5 mm apart, starting from the basal lung margin) were manually segmented and 1513 2D radiomic features were extracted using the in-house software Z-Rad (Python 2.7). Features were Z-score transformed and pre-filtered for inter- and intra-reader robustness (intraclass correlation coefficient >0.85) and inter-feature correlation (Spearman’s rho <0.9). A categorical linear regression model was created using 3-fold cross-validated elastic nets for feature selection. Features were then summarized and divided by their number. For generation of a score cut-off, Youden’s score was used. For two-group analyses of continuous variables, Wilcoxon’s test was performed, whereas categorical data was assessed using Fisher’s exact test.Results:A total of 64 pre-treatment HRCTs from 54 patients were analyzed. In 9 patients, >1 asynchronous treatments were assessed, while 45 patients had only 1 eligible treatment approach. The response rate within the assessed follow-up period was 45.3% (n=29). For score generation, 13 radiomic features were selected and an optimal cut-off value of -0.1589 was determined. Univariate linear regression showed significant association between our categorical radiomics-based score and treatment response (p=0.007, area under the curve = 0.65 (0.51-0.79), sensitivity=0.90, specificity=0.43), whereby a high score was predictive for treatment response.No differences between patients with high (n=46) or low (n=18) scores were detected for baseline age (mean±SD=55.5±12.0 and 55.5±13.6 years, p=0.84), duration of SSc (mean±SD=6.2±8.4 and 4.7±4.4 years, p=0.79), time since ILD diagnosis (2.7±2.9 and 2.4±3.1 years, p=0.59), FVC (77.6±20.6 and 80.1±17.9, p=0.41) or DLco (54.4±21.0 and 57.6±18.9, p=0.40). Distribution of anti-Scl-70 positivity (45.7% vs. 55.6%, p=0.58) and diffuse cutaneous disease (47.7% vs. 61.1%, p=0.41) was not significantly different between patients with high and low scores, respectively, although a trend towards higher percentages in the high score group was observed.Conclusion:Our results indicate that, following validation in external cohorts, radiomics may be a promising tool for future pre-treatment patient stratification. Moreover, our radiomics-based score seems not to be associated with commonly studied clinical predictors such as anti-Scl-70 positivity or lung function, underlining a possible additive value to ‘traditional’ clinical parameters.References:[1]Schniering, J., et al. Resolving phenotypic and prognostic differences in interstitial lung disease related to systemic sclerosis by computed tomography-based radiomics. medRxiv [Preprint] doi:10.1101/2020.06.09.20124800 (2020).Disclosure of Interests:Chantal Meier: None declared, Malgorzata Maciukiewicz: None declared, Matthias Brunner: None declared, Janine Schniering: None declared, Hubert Gabrys: None declared, Anja Kühnis: None declared, Oliver Distler Speakers bureau: Speaker fee on Scleroderma and related complications: Bayer, Boehringer Ingelheim, Medscape, Novartis, Roche. Speaker fee on rheumatology topic other than Scleroderma: MSD, iQone, Novartis, Pfizer, Roche, Consultant of: Consultancy fee for Scleroderma and its complications: Abbvie, Acceleron Pharma, Amgen, AnaMar, Arxx Therapeutics, Bayer, Baecon Discovery, Boehringer, CSL Behring, ChemomAb, Corbus Pharmaceuticals, Horizon Pharmaceuticals, Galapagos NV, GSK, Glenmark Pharmaceuticals, Inventiva, Italfarmaco, iQvia, Kymera, Medac, Medscape, Mitsubishi Tanabe Pharma, MSD, Roche, Roivant Sciences, Sanofi, UCB. Consultancy fee for rheumatology topic other than Scleroderma: Abbvie, Amgen, Lilly, Pfizer, Grant/research support from: Research Grants to investigate the pathophysiology and potential treatment of Scleroderma and its complications: Kymera Therapeutics, Mitsubishi Tanabe, Thomas Frauenfelder: None declared, Stephanie Tanadini-Lang: None declared, Britta Maurer Speakers bureau: Speaker fees from Boehringer-Ingelheim, Grant/research support from: Grant/research support from AbbVie, Protagen, Novartis Biomedical Research, congress support from Pfizer, Roche, Actelion, mepha, and MSD

scholarly journals OP0251 THE EULAR SYSTEMIC SCLEROSIS IMPACT OF DISEASE (SCLEROID) SCORE – A NEW PATIENT-REPORTED OUTCOME MEASURE FOR PATIENTS WITH SYSTEMIC SCLEROSIS

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 158-159
Author(s):  
M. O. Becker ◽  
R. Dobrota ◽  
K. Fligelstone ◽  
A. Roennow ◽  
Y. Allanore ◽  
...  
Keyword(s):  
Clinical Practice ◽  
Systemic Sclerosis ◽  
Validation Study ◽  
Hand Function ◽  
Patient Reported Outcome ◽  
Sensitivity To Change ◽  
Research Support ◽  
Patient Reported ◽  
Disease Impact

Background:Patient reported outcome measures (PROM) are important for clinical practice and research. Given the unmet need for a comprehensive PROM for systemic sclerosis (SSc), the ScleroID questionnaire was developed by a joint team of patients with SSc and medical experts. This is intended as a brief, specific, patient-derived, disease impact score for research and clinical use in SSc.Objectives:Here, we present the validation and final version of the ScleroID.Methods:This EULAR-endorsed project involves 9 European expert SSc centers. Patients fulfilling the ACR/EULAR 2013 criteria were prospectively included since 05/16 in a large observational cohort study. Patients completed the ScleroID and comparators SHAQ, EQ5D, SF36. They also weighted the 10 dimensions of the ScleroID by distributing 100 points according to the perceived impact on their health. The final score calculation is based on the ranking of the weights. The validation study included a reliability arm and a longitudinal arm, looking at sensitivity to change at follow-up.Results:Of the 472 patients included at baseline, 109 patients also had a reliability visit and 113 patients a follow-up visit. 84.5% of patients were female, 29.8% had diffuse SSc, mean age was 54.6 years, and mean disease duration 9.5 years. The highest weights were assigned by the patients to Raynaud`s phenomenon, fatigue, hand function and pain, confirming our previous results. The total ScleroID score showed good Spearman correlation coefficients with the comparators (SHAQ, 0.73; EQ5D -0.48; Patient’s global assessment, VAS 0.77; HAQ-DI 0.62; SF36 physical score -0.62; each p<0.001). The internal consistency was good: Crohnbach’s alpha 0.866, similar to SS-HAQ (0.88) and higher than EQ5D (0.77). The ScleroID had a very good reliability: intra-class correlation coefficient 0.839 (ranging 0.608 to 0.788 for the individual items), superior to all comparators. Twenty of 113 patients reported a change in their disease status at follow up. Sensitivity to change: the standardized response mean was 0.34 for the total ScleroID score and highest for lower GI (0.633) and life choices domains (0.521), superior to all other PROM. Figure 1 shows the final ScleroID.Figure 1.Conclusion:The EULAR ScleroID is a novel PROM designed for use in clinical practice and clinical trials to reflect the disease impact of SSc, showing good performance in the validation study. Importantly, Raynaud syndrome, impaired hand function, pain and fatigue were the main patient reported drivers of disease impact.Disclosure of Interests:Mike O. Becker: None declared, Rucsandra Dobrota: None declared, Kim Fligelstone: None declared, Annelise Roennow: None declared, Yannick Allanore Grant/research support from: BMS, Inventiva, Roche, Sanofi, Consultant of: Actelion, Bayer AG, BMS, BI, Patricia Carreira Grant/research support from: Actelion, Roche, MSD, Consultant of: GlaxoSmithKline, VivaCell Biotechnology, Emerald Health Pharmaceuticals, Boehringer Ingelheim, Roche, Speakers bureau: Actelion, GlaxoSmithKline, Roche, László Czirják Consultant of: Actelion, BI, Roche-Genentech, Lilly, Medac, Novartis, Pfizer, Bayer AG, Christopher Denton Grant/research support from: GlaxoSmithKline, CSL Behring, and Inventiva, Consultant of: Medscape, Roche-Genentech, Actelion, GlaxoSmithKline, Sanofi Aventis, Inventiva, CSL Behring, Boehringer Ingelheim, Corbus Pharmaceuticals, Acceleron, Curzion and Bayer, Roger Hesselstrand: None declared, Gunnel Sandqvist: None declared, Otylia Kowal-Bielecka Consultant of: Bayer, Boehringer Ingelheim, Inventiva, MSD, Medac, Novartis, Roche and Sandoz, Speakers bureau: Bayer, Boehringer Ingelheim, Inventiva, MSD, Medac, Novartis, Roche and Sandoz, Cosimo Bruni Speakers bureau: Actelion, Eli Lilly, Marco Matucci Cerinic: None declared, Carina Mihai: None declared, Ana Maria Gheorghiu: None declared, Ulf Müller-Ladner Speakers bureau: Biogen, Joe Sexton: None declared, Turid Heiberg: None declared, Oliver Distler Grant/research support from: Grants/Research support from Actelion, Bayer, Boehringer Ingelheim, Competitive Drug Development International Ltd. and Mitsubishi Tanabe; he also holds the issued Patent on mir-29 for the treatment of systemic sclerosis (US8247389, EP2331143)., Consultant of: Consultancy fees from Actelion, Acceleron Pharma, AnaMar, Bayer, Baecon Discovery, Blade Therapeutics, Boehringer, CSL Behring, Catenion, ChemomAb, Curzion Pharmaceuticals, Ergonex, Galapagos NV, GSK, Glenmark Pharmaceuticals, Inventiva, Italfarmaco, iQvia, medac, Medscape, Mitsubishi Tanabe Pharma, MSD, Roche, Sanofi and UCB, Speakers bureau: Speaker fees from Actelion, Bayer, Boehringer Ingelheim, Medscape, Pfizer and Roche

THU0330 EFFECTS OF NINTEDANIB IN PATIENTS WITH SYSTEMIC SCLEROSIS-ASSOCIATED ILD (SSC-ILD) AND DIFFERING EXTENTS OF SKIN FIBROSIS: FURTHER ANALYSES OF THE SENSCIS TRIAL

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 395-396
Author(s):  
Y. Allanore ◽  
V. Steen ◽  
M. Kuwana ◽  
C. Denton ◽  
M. Matucci-Cerinic ◽  
...  
Keyword(s):  
Placebo Group ◽  
Systemic Sclerosis ◽  
Topoisomerase I ◽  
Statistical Testing ◽  
Skin Fibrosis ◽  
Annual Rate ◽  
Research Support ◽  
High Resolution Computed Tomography ◽  
Significant Difference ◽  
Rate Of Decline

Background:In the SENSCIS trial, nintedanib reduced the progression of SSc-ILD compared with placebo, as shown by a significantly lower rate of decline in forced vital capacity (FVC) over 52 weeks. There was no significant difference between treatment groups in change in modified Rodnan skin score (mRSS) at week 52. An mRSS of 18–25 has been proposed as an upper cut-off to enrich a cohort for skin-progressive patients. Progression of skin fibrosis has been associated with later progression of ILD.Objectives:To assess the effects of nintedanib on the rate of FVC decline and change in mRSS in the SENSCIS trial in subgroups by mRSS <18 and ≥18 at baseline.Methods:Patients with SSc-ILD with onset of first non-Raynaud symptom <7 years before screening and ≥10% fibrosis of the lungs on a high-resolution computed tomography scan were randomised to receive nintedanib or placebo. We analysed the rate of decline in FVC (ml/year) over 52 weeks and the change from baseline in mRSS at week 52 in subgroups by mRSS (<18; ≥18) at baseline.Results:In the nintedanib and placebo groups, respectively, 219/288 (76.0%) and 226/288 (78.5%) patients had mRSS <18 at baseline. Compared with those with mRSS <18, patients with mRSS ≥18 had a lower mean FVC % predicted (68.3% vs 73.7%) and greater proportions were taking mycophenolate at baseline (58.1% vs 45.6%), were anti-topoisomerase I antibody positive (67.4% vs 58.7%) and had diffuse cutaneous SSc (100% vs 37.8%). The mean (SE) annual rate of decline in FVC in the placebo group was numerically greater in patients who had mRSS ≥18 than mRSS <18 at baseline (-131.7 [29.2] mL/year vs -81.4 [15.4] mL/year). The effect of nintedanib vs placebo on reducing the annual rate of decline in FVC was numerically more pronounced in patients with mRSS ≥18 (difference: 88.7 mL/year [95% CI 7.7, 169.8]) than mRSS <18 (difference: 26.4 mL/year (95% CI -16.8, 69.6) at baseline, but statistical testing did not indicate heterogeneity in the treatment effect of nintedanib between subgroups (p=0.18 for treatment-by-time-by-subgroup interaction) (Figure). In the nintedanib and placebo groups, respectively, changes in mRSS at week 52 were -2.2 (0.3) and -2.1 (0.3) (difference -0.1 [95% CI -1.0, 0.7]) in patients with mRSS <18 at baseline and -2.1 (0.7) and -1.6 (0.7) (difference -0.6 [95% CI -2.1, 1.0]) in patients with mRSS ≥18 at baseline (p=0.62 for treatment-by-visit-by-subgroup interaction).Conclusion:In the placebo group of the SENSCIS trial, the rate of decline in FVC over 52 weeks was numerically greater in patients with mRSS ≥18 than <18 at baseline, while reductions in mRSS were similar. A lower rate of FVC decline was observed in patients treated with nintedanib than placebo both in patients with mRSS ≥18 and <18 at baseline.Acknowledgments:The SENSCIS trial was funded by Boehringer IngelheimDisclosure of Interests:Yannick Allanore Grant/research support from: Yannick Allanore has received grants from Inventiva, Roche and Sanofi, Consultant of: Yannick Allanore has received fees from Actelion, Bayer, Boehringer Ingelheim, Bristol-Myers Squibb, Curzion, Inventiva, Roche, Sanofi, Viginia Steen Grant/research support from: The associated affiliation has received grants/research from Boehringer Ingelheim, Corbus Pharmaceuticals, CSL Behring, Eicos, Galapagos, Immune Tolerance Network, Reata, Consultant of: Virginia Steen has acted as a consultant for Boehringer Ingelheim, Corbus, CSL Behring, Eicos, Forbius, Masataka Kuwana Grant/research support from: Acetelion, Consultant of: Acetelion, Bayer, Chugai, Corbus Pharmaceuticals, CSL Behring and Reata Pharmaceuticals. He was a member of the SENSCIS trial Steering Committee (Boehringer Ingelheim), Christopher Denton Grant/research support from: GlaxoSmithKline, CSL Behring, and Inventiva, Consultant of: Medscape, Roche-Genentech, Actelion, GlaxoSmithKline, Sanofi Aventis, Inventiva, CSL Behring, Boehringer Ingelheim, Corbus Pharmaceuticals, Acceleron, Curzion and Bayer, Marco Matucci-Cerinic Grant/research support from: Actelion, MSD, Bristol-Myers Squibb, Speakers bureau: Acetelion, Lilly, Boehringer Ingelheim, Elizabeth Volkmann Grant/research support from: Forbius, Corbus Pharmaceuticals, Consultant of: Boehringer Ingelheim, Forbius, Speakers bureau: Boehringer Ingelheim, Dinesh Khanna Shareholder of: Eicos Sciences, Inc./Civi Biopharma, Inc., Grant/research support from: Dr Khanna was supported by NIH/NIAMS K24AR063120, Consultant of: Acceleron, Actelion, Bayer, Boehringer Ingelheim, Bristol-Myers Squibb, Corbus Pharmaceuticals, Horizon Therapeutic, Galapagos, Roche/Genentech, GlaxoSmithKline, Mitsubishi Tanabe, Sanofi-Aventis/Genzyme, UCB, Daniel Wachtlin Employee of: Employee of Boehringer Ingelheim, Martina Gahlemann Employee of: Employee of Boehringer Ingelheim, Manuel Quaresma Employee of: Employee of Boehringer Ingelheim, Margarida Alves Employee of: Employee of Boehringer Ingelheim, Oliver Distler Grant/research support from: Grants/Research support from Actelion, Bayer, Boehringer Ingelheim, Competitive Drug Development International Ltd. and Mitsubishi Tanabe; he also holds the issued Patent on mir-29 for the treatment of systemic sclerosis (US8247389, EP2331143)., Consultant of: Consultancy fees from Actelion, Acceleron Pharma, AnaMar, Bayer, Baecon Discovery, Blade Therapeutics, Boehringer, CSL Behring, Catenion, ChemomAb, Curzion Pharmaceuticals, Ergonex, Galapagos NV, GSK, Glenmark Pharmaceuticals, Inventiva, Italfarmaco, iQvia, medac, Medscape, Mitsubishi Tanabe Pharma, MSD, Roche, Sanofi and UCB, Speakers bureau: Speaker fees from Actelion, Bayer, Boehringer Ingelheim, Medscape, Pfizer and Roche

scholarly journals THU0334 LASER SPECKLE CONTRAST ANALYSIS FOR MEASUREMENT OF PERIPHERAL BLOOD PERFUSION IN SYSTEMIC SCLEROSIS PATIENTS: CAN IT PREDICT FUTURE ISCHEMIC DIGITAL TROPHIC LESIONS?

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 398.1-398
Author(s):  
C. Debusschere ◽  
A. Vanhaecke ◽  
M. Cutolo ◽  
E. Deschepper ◽  
V. Smith
Keyword(s):  
Logistic Regression ◽  
Systemic Sclerosis ◽  
Peripheral Blood ◽  
Blood Perfusion ◽  
Laser Speckle ◽  
Research Support ◽  
Speckle Contrast ◽  
Contrast Analysis ◽  
Laser Speckle Contrast Analysis

Background:Vasculopathy is a hallmark of systemic sclerosis (SSc). Laser speckle contrast analysis (LASCA) is a research tool to assess peripheral blood perfusion (PBP) (1). At this moment, its reliability has been attested in SSc patients, but its predictive value for future ischemic digital trophic lesions (DTL) is unknown (1).Objectives:To investigate in an unselected, prospective SSc cohort if baseline LASCA PBP measurements can discriminate between patients who will develop ischemic DTL (iDTL) and those who will not.Methods:Patients (fulfilling 2013 ACR/EULAR criteria and/or 2001 LeRoy and Medsger criteria) were recruited during the period of December 2017 to September 2018. LASCA was performed at baseline, in standardized conditions (1). Regions of interest (ROIs) (diameter 1 cm) were outlined at the 2nd-5thfingertip both volar and dorsal. The ‘average PBP’ of these ROIs was calculated (expressed in arbitrary perfusion units [PU]). A monthly telephone survey was conducted for 1 year to investigate DTL occurrence. DTL were considered ‘ischemic’ if not related to calcinosis. Logistic regression and ROC analysis were used to assess if average PBP is predictive of future iDTL.Results:Of the 106 patients with complete follow-up (92 women [86,8%]; 18 limited SSc [17,0%], 82 limited cutaneous SSc [77,4%], 6 diffuse cutaneous SSc [5,7%]), 29 patients (27,4%) had a DTL history. Forty-nine patients (46,2%) were on vasodilator therapy. Only 7 patients developed at least 1 iDTL during follow-up (6,6%) (Figure 1a). Performing univariate logistic regression (ULR), average PBP was not predictive for future iDTL (Table 1). Of note, analyzing only the patients not taking vasodilators, average PBP in the ‘iDTL group’ (n = 3) was median 46,8 PU (min. 45,6 - max. 68,8) vs. median 141,4 PU (min. 24,4 - max. 269,5) in the ‘no iDTL group’ (n = 54) (Figure 1b). In this subgroup, all 3 patients who developed iDTL (100%) had an average PBP ≤ 70 PU whereas only 9 of the 54 patients without iDTL development (16,7%) had such PBP values.Table 1.Results of ULRSummary statisticsULRVariableiDTL cases(n = 7)Non-iDTL cases(n = 99)ParameterOR(95% CI)pROC AUC(95% CI)Average PBP (PU) mean (+/- SD)123,0 (74,6)142,9 (61,9)Average PBP (linear)0,995(0,982-1,007)0,4180,597(0,352-0,843)Conclusion:In this pilot study with an unselected day-to-day SSc population, where patients were allowed to continue vasodilators, there was an unexpected low iDTL incidence, undermining the power of our study. Even though, the observations in the subgroup of patients not taking vasodilators deserve future investigation to assess whether low PBP values, as measured by LASCA, are associated with a higher iDTL incidence.References:[1]Cutolo M, Vanhaecke A, et al. Autoimmun Rev. 2018;17(8):775-80.Figure 1.Distribution of ‘average PBP’ as measured by LASCA for ‘no iDTL group’ and ‘iDTL group’Disclosure of Interests:Claire Debusschere: None declared, Amber Vanhaecke: None declared, Maurizio Cutolo Grant/research support from: Bristol-Myers Squibb, Actelion, Celgene, Consultant of: Bristol-Myers Squibb, Speakers bureau: Sigma-Alpha, Ellen Deschepper: None declared, Vanessa Smith Grant/research support from: The affiliated company received grants from Research Foundation - Flanders (FWO), Belgian Fund for Scientific Research in Rheumatic diseases (FWRO), Boehringer Ingelheim Pharma GmbH & Co and Janssen-Cilag NV, Consultant of: Boehringer-Ingelheim Pharma GmbH & Co, Speakers bureau: Actelion Pharmaceuticals Ltd, Boehringer-Ingelheim Pharma GmbH & Co and UCB Biopharma Sprl

Mycophenolate in scleroderma-associated interstitial lung disease: Real-world data from rheumatology and pulmonology clinics in South Asia

2021 ◽  
pp. 239719832110244
Author(s):  
Ramya Janardana ◽  
Aparna Irodi ◽  
Pramod P Chebbi ◽  
Ruchika Goel ◽  
Leena R Vimala ◽  
...  
Keyword(s):  
Computed Tomography ◽  
Mycophenolate Mofetil ◽  
Systemic Sclerosis ◽  
High Resolution ◽  
Interstitial Lung Disease ◽  
Lung Disease ◽  
Forced Vital Capacity ◽  
Vital Capacity ◽  
High Resolution Computed Tomography

Introduction: There is a paucity of real-world data on mycophenolate mofetil/mycophenolate sodium in systemic sclerosis-related interstitial lung disease. Aim: To study the efficacy of mycophenolate mofetil/ mycophenolate sodium in systemic sclerosis-related interstitial lung disease. Methods: In this single-centre study, clinical, laboratory and imaging details of consecutive patients with systemic sclerosis-related interstitial lung disease receiving mycophenolate mofetil/mycophenolate sodium from rheumatology and pulmonology clinics between January 2008 and March 2017 were retrospectively retrieved. The change in percentage of predicted normal forced vital capacity at last follow-up visit as compared with baseline was studied. In addition, high-resolution computed tomography scans at baseline and 2-year follow-up visit were scored as either stable/improved or worsened by experienced thoracic radiologists blinded to the clinical details of patients. Results: Altogether, 88 patients (85.2% females) with mean age (SD) of 33.8 years (± 11.3) and median (interquartile range) duration of disease since non-Raynaud’s symptoms of 36 months (13.5–60) were studied. Diffuse systemic sclerosis comprised 85.2% of them. The mean baseline forced vital capacity was 61.2 ± 17.9% and median scores for ground glass opacities and fibrosis in high-resolution computed tomography were 0.5 (0–1.3) and 1 (0–1.3), respectively. At a median follow-up duration of 30 months (interquartile range = 16.5–49), the percentage of forced vital capacity improved by 1.8% (–3.82 to 9.07) as compared with baseline visit ( p = 0.02). In the 2-year follow-up, the ground glass opacity and fibrosis scores in high-resolution computed tomography improved in 17.3% and 7.7% of patients and stabilized in 63.5% and 78.8% patients, respectively. Conclusion: Mycophenolate mofetil/mycophenolate sodium was efficacious in improving /stabilizing forced vital capacity irrespective of the baseline high-resolution computed tomography lung scores in our patients with systemic sclerosis-related interstitial lung disease during the ⩾ 2-year follow-up period.

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