Abstract
An emerging trend in the medical literature, including the Rheumatology literature, is that of accumulating large, multicentric, multi-national data based on registries of patients seen in real life situations. Such real-world evidence (RWE) may help provide valuable insights into the long-term outcomes of disease in unselected patients seen in daily practice, including patients belonging to vulnerable populations such as extremes of age, during pregnancy and lactation. Evidences gathered from real life practice settings can help understand drug prescription patterns, including adherence to treatment guidelines, cost-effectiveness of therapy, and real-life long-term outcomes, and adverse effects of treatment with particular medications. Registry-based data also helps analyze comorbidities in patients with rheumatic diseases, and their impact on quality of life, morbidity and mortality. Traditionally, a randomized controlled trial (RCT), or systematic reviews of multiple, homogenous RCTs, have been considered the cornerstone of evidence-based medicine, and RWE does, at times, provide differing viewpoints from the results of particular drugs in clinical trial settings. Therefore, in the present day, it is prudent to consider the complementary nature of information derived from RWE to that obtained from rigorous, clinical trial settings. Future guidelines for disease management may consider it relevant to include information from RWE in addition to that available from clinical trials, to help devise management guidelines that are harmonious with routine practice settings.
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Abbreviations
- AAV:
-
ANCA-associated vasculitis
- ANCA:
-
Anti-neutrophil cytoplasmic antibody
- Anti-MPO:
-
Anti-myeloperoxidase
- Anti-PR3:
-
Anti-proteinase 3
- AS:
-
Ankylosing spondylitis
- bDMARD:
-
Biological disease-modifying antirheumatic drug
- cDMARD:
-
Conventional disease-modifying antirheumatic drug
- BILAG:
-
British Isles Lupus Assessment Group
- BMI:
-
Body mass index
- DAS28-CRP:
-
Disease activity score using 28 joints with C-reactive protein
- DAS28-ESR:
-
Disease activity score using 28 joints with erythrocyte sedimentation rate
- DMARD:
-
Disease-modifying anti-rheumatic drug
- EBM:
-
Evidence-based medicine
- EMR:
-
Electronic medical records
- ESCEO:
-
European Society for Clinical and Economic Aspects of Osteoporosis and Osteoarthritis
- GCA:
-
Giant cell arteritis
- GDPR:
-
General data protection regulation
- IIM:
-
Idiopathic inflammatory myositis
- ILD:
-
Interstitial lung disease
- LDA:
-
Low disease activity
- LFA-REAL:
-
Lupus Foundation of America: rapid evaluation of activity in lupus
- LTBI:
-
Latent tuberculosis infection
- MDA:
-
Minimal disease activity
- MMF:
-
Mycophenolate mofetil
- nrAxSpA:
-
non-radiographic axial spondyloarthritis
- NSAID:
-
Non-steroidal anti-inflammatory drug
- PAH:
-
Pulmonary arterial hypertension
- PET-CT:
-
positron emission tomography computerized tomography
- PsA:
-
Psoriatic arthritis
- RA:
-
Rheumatoid arthritis
- RCT:
-
Randomized controlled trial
- RWE:
-
Real-world evidence
- SDAI:
-
Simplified disease activity index
- SLE:
-
Systemic lupus erythematosus
- SLEDAI:
-
Systemic lupus erythematosus disease activity index
- SpA:
-
Spondyloarthritis
- TNF:
-
Tumor necrosis factor alpha
- UIP:
-
Usual interstitial pneumonia
- UK:
-
United Kingdom
- USA:
-
United States of America
- VERA:
-
Very early rheumatoid arthritis
References
Levels of evidence—Oxford Centre for evidence based medicine [Internet] http://www.cebm.net/oxford-centre-evidence-based-medicine-levels-evidence-march-2009/. Accessed 20 December 2018
Misra DP, Agarwal V (2018) Systematic reviews: challenges for their justification, related comprehensive searches, and implications. J Korean Med Sci 33:e92. https://doi.org/10.3346/jkms.2018.33.e92
van der Heijde D, Aletaha D, Carmona L et al (2015) 2014 Update of the EULAR standardised operating procedures for EULAR-endorsed recommendations. Ann Rheum Dis 74:8–13. https://doi.org/10.1136/annrheumdis-2014-206350
Misra DP, Sharma A, Agarwal V (2018) Guidelines for management of rheumatic diseases in developing countries from basics to real-world situation: relevance, need, and processes for development. Rheumatol Int 38:549–556. https://doi.org/10.1007/s00296-018-3996-2
Evans RS (2016) Electronic health records: then, now, and in the future. Yearb Med Inform 25 (Suppl 1):S48–S61. https://doi.org/10.15265/IYS-2016-s006
Burmester GR (2018) Rheumatology 4.0: big data, wearables and diagnosis by computer. Ann Rheum Dis 77:963–965. https://doi.org/10.1136/annrheumdis-2017-212888
Marelli L, Testa G (2018) Scrutinizing the EU general data protection regulation. Science 360:496–498. https://doi.org/10.1126/science.aar5419
Gasparyan AY, Ayvazyan L, Blackmore H, Kitas GD (2011) Writing a narrative biomedical review: considerations for authors, peer reviewers, and editors. Rheumatol Int 31:1409–1417. https://doi.org/10.1007/s00296-011-1999-3
Kim HS, Lee S, Kim JH (2018) Real-world evidence versus randomized controlled trial: clinical research based on electronic medical records. J Korean Med Sci 33:e213. https://doi.org/10.3346/jkms.2018.33.e213
Monti S, Grosso V, Todoerti M, Caporali R (2018) Randomized controlled trials and real-world data: differences and similarities to untangle literature data. Rheumatology 57:vii54–vii58. https://doi.org/10.1093/rheumatology/key109
Wong SE, North SA, Sweeney CJ, Stockler MR, Sridhar SS (2017) Screen failure rates in contemporary randomized clinical phase ii/iii therapeutic trials in genitourinary malignancies. Clin Genitourin Cancer. https://doi.org/10.1016/j.clgc.2017.08.019
Aggarwal R, Ringold S, Khanna D et al (2015) Distinctions between diagnostic and classification criteria? Arthritis Care Res 67:891–897. https://doi.org/10.1002/acr.22583 (Hoboken)
Walters SJ, Henriques-Cadby dos IBA, Bortolami O et al (2017) Recruitment and retention of participants in randomised controlled trials: a review of trials funded and published by the United Kingdom Health Technology Assessment Programme. BMJ Open 7:e015276. https://doi.org/10.1136/bmjopen-2016-015276
Ford I, Norrie J (2016) Pragmatic trials. N Engl J Med 375:454–463. https://doi.org/10.1056/NEJMra1510059
Kennedy-Martin T, Curtis S, Faries D, Robinson S, Johnston J (2015) A literature review on the representativeness of randomized controlled trial samples and implications for the external validity of trial results. Trials 16:495. https://doi.org/10.1186/s13063-015-1023-4
Carter JT (2015) Electronic medical records and quality improvement. Neurosurg Clin N Am 26:245–251. https://doi.org/10.1016/j.nec.2014.11.018
Kataria S, Ravindran V (2018) Digital health: a new dimension in rheumatology patient care. Rheumatol Int 38:1949–1957. https://doi.org/10.1007/s00296-018-4037-x
Ravindran V, Kataria S (2018) Digital health in rheumatology. Ann Rheum Dis. https://doi.org/10.1136/annrheumdis-2018-214146
Hauswaldt J, Kempter V, Himmel W, Hummers E (2018) Obstacles in secondary analysis of routine data from primary care. Gesundheitswesen 80:987–993. https://doi.org/10.1055/a-0668-5817
Dutton RP (2014) Quality management and registries. Anesthesiol Clin 32:577–586. https://doi.org/10.1016/j.anclin.2014.02.014
Hyman J (2015) The limitations of using insurance data for research. J Am Dent Assoc 146:283–285. https://doi.org/10.1016/j.adaj.2015.02.010
Tovino SA (2017) The HIPAA privacy rule and the EU GDPR: illustrative comparisons. Seton Hall Law Rev 47:973–993
Chico V (2018) The impact of the general data protection regulation on health research. Br Med Bull 128:109–118. https://doi.org/10.1093/bmb/ldy038
Favalli EG, Becciolini A, Biggioggero M, Marchesoni A, Meroni PL (2015) Is there a need for new thresholds to define remission and low disease activity by disease activity score 28 calculated with C reactive protein? Real life data from a local registry. Ann Rheum Dis 74:e5. https://doi.org/10.1136/annrheumdis-2014-206651
Kaltsonoudis E, Pelechas E, Voulgari PV, Drosos AA (2018) Unmet needs in the treatment of rheumatoid arthritis. An observational study and a real-life experience from a single university center. Semin Arthritis Rheum. https://doi.org/10.1016/j.semarthrit.2018.06.003. [Epub ahead of print]
Norvang V, Sexton J, Kristianslund EK et al (2018) Predicting achievement of the treatment targets at 6 months from 3-month response levels in rheumatoid arthritis: data from real-life follow-up in the NOR-DMARD study. RMD Open 4:e000773. https://doi.org/10.1136/rmdopen-2018-000773
Gremese E, Salaffi F, Bosello SL et al (2013) Very early rheumatoid arthritis as a predictor of remission: a multicentre real life prospective study. Ann Rheum Dis 72:858–862. https://doi.org/10.1136/annrheumdis-2012-201456
Aydin SZ, Pay S, Inanc N et al (2017) Which joints and why do rheumatologists scan in rheumatoid arthritis by ultrasonography? A real life experience. Clin Exp Rheumatol 35:508–511
Haavardsholm EA, Aga AB, Olsen IC et al (2016) Ultrasound in management of rheumatoid arthritis: ARCTIC randomised controlled strategy trial. BMJ 354:i4205. https://doi.org/10.1136/bmj.i4205
Zufferey P, Scherer A, Nissen MJ et al (2018) Can ultrasound be used to predict loss of remission in patients with RA in a real-life setting? A multicenter cohort study. J Rheumatol 45:887–894. https://doi.org/10.3899/jrheum.161193
Naranjo A, Caceres L, Hernandez-Beriain JA et al (2015) Factors associated with the intensification of treatment in rheumatoid arthritis in clinical practice. Rheumatol Int 35:1851–1855. https://doi.org/10.1007/s00296-015-3332-z
An Y, Liu T, He D et al (2017) The usage of biological DMARDs and clinical remission of rheumatoid arthritis in China: a real-world large scale study. Clin Rheumatol 36:35–43. https://doi.org/10.1007/s10067-016-3424-5
Favalli EG, Becciolini A, Meroni PL (2017) Change over time in the pattern of clinical response to first-line biologic drugs in patients with rheumatoid arthritis: observational data in a real-life setting. J Rheumatol 44:262–263. https://doi.org/10.3899/jrheum.161045
Santos-Moreno P, Sanchez G, Gomez D, Bello-Gualtero J, Castro C (2016) Direct comparative effectiveness among 3 anti-tumor necrosis factor biologics in a real-life cohort of patients with rheumatoid arthritis. J Clin Rheumatol 22:57–62. https://doi.org/10.1097/rhu.0000000000000358
Monti S, Klersy C, Gorla R et al (2017) Factors influencing the choice of first- and second-line biologic therapy for the treatment of rheumatoid arthritis: real-life data from the Italian LORHEN Registry. Clin Rheumatol 36:753–761. https://doi.org/10.1007/s10067-016-3528-y
Abasolo L, Leon L, Rodriguez-Rodriguez L et al (2015) Safety of disease-modifying antirheumatic drugs and biologic agents for rheumatoid arthritis patients in real-life conditions. Semin Arthritis Rheum 44:506–513. https://doi.org/10.1016/j.semarthrit.2014.11.003
Iannone F, Sinigaglia L, Favalli EG et al (2016) Drug survival of adalimumab in patients with rheumatoid arthritis over 10 years in the real-world settings: high rate remission together with normal function ability. Clin Rheumatol 35:2649–2656. https://doi.org/10.1007/s10067-016-3349-z
Vassilopoulos D, Delicha EM, Settas L et al (2016) Safety profile of repeated rituximab cycles in unselected rheumatoid arthritis patients: a long-term, prospective real-life study. Clin Exp Rheumatol 34:893–900
Sharma B (2017) Clinical use of ZRC3197 (Adalimumab Biosimilar) in patients with inflammatory arthritis: a real-life experience. J Assoc Physicians India 65:22–25
Codreanu C, Sirova K, Jarosova K, Batalov A (2018) Assessment of effectiveness and safety of biosimilar infliximab (CT-P13) in a real-life setting for treatment of patients with active rheumatoid arthritis or ankylosing spondylitis. Curr Med Res Opin 34:1763–1769. https://doi.org/10.1080/03007995.2018.1441144
Scherlinger M, Germain V, Labadie C et al (2018) Switching from originator infliximab to biosimilar CT-P13 in real-life: the weight of patient acceptance. Jt Bone Spine 85:561–567. https://doi.org/10.1016/j.jbspin.2017.10.003
Mease PJ, Heijde DV, Karki C et al (2018) Characterization of patients with ankylosing spondylitis and nonradiographic axial spondyloarthritis in the US-based corrona registry. Arthritis Care Res 70:1661–1670. https://doi.org/10.1002/acr.23534. (Hoboken)
Varkas G, Vastesaeger N, Cypers H et al (2018) Association of inflammatory bowel disease and acute anterior uveitis, but not psoriasis, with disease duration in patients with axial spondyloarthritis: results from two belgian nationwide axial spondyloarthritis cohorts. Arthritis Rheumatol 70:1588–1596. https://doi.org/10.1002/art.40551
Madsen OR (2018) Stability of fatigue, pain, patient global assessment and the Bath Ankylosing Spondylitis Functional Index (BASFI) in spondyloarthropathy patients with stable disease according to the Bath Ankylosing Spondylitis Disease Activity Index (BASDAI). Rheumatol Int 38:425–432. https://doi.org/10.1007/s00296-017-3920-1
Kim SK, Choe JY, Lee SS, Shin K (2017) Body mass index is related with the presence of syndesmophyte in axial spondyloarthritis: data from the Korean College of Rheumatology BIOlogics (KOBIO) registry. Mod Rheumatol 27:855–861. https://doi.org/10.1080/14397595.2016.1265637
Jones GT, Ratz T, Dean LE, Macfarlane GJ, Atzeni F (2017) Disease severity in never smokers, ex-smokers, and current smokers with axial spondyloarthritis: results from the scotland registry for ankylosing spondylitis. Arthritis Care Res 69:1407–1413. https://doi.org/10.1002/acr.23157. (Hoboken)
Macfarlane GJ, Barnish MS, Pathan E et al (2017) Co-occurrence and characteristics of patients with axial spondyloarthritis who meet criteria for fibromyalgia: results from a UK national register. Arthritis Rheumatol 69:2144–2150. https://doi.org/10.1002/art.40185
Shah K, Paris M, Mellars L, Changolkar A, Mease PJ (2017) Real-world burden of comorbidities in US patients with psoriatic arthritis. RMD Open 3:e000588. https://doi.org/10.1136/rmdopen-2017-000588
Coates LC, Moverley AR, McParland L et al (2015) Effect of tight control of inflammation in early psoriatic arthritis (TICOPA): a UK multicentre, open-label, randomised controlled trial. Lancet 386:2489–2498. https://doi.org/10.1016/s0140-6736(15)00347-5
Rahman P, Zummer M, Bessette L et al (2017) Real-world validation of the minimal disease activity index in psoriatic arthritis: an analysis from a prospective, observational, biological treatment registry. BMJ Open 7:e016619. https://doi.org/10.1136/bmjopen-2017-016619
Barra L, Pope JE, Payne M (2009) Real-world anti-tumor necrosis factor treatment in rheumatoid arthritis, psoriatic arthritis, and ankylosing spondylitis: cost-effectiveness based on number needed to treat to improve health assessment questionnaire. J Rheumatol 36:1421–1428. https://doi.org/10.3899/jrheum.081122
Glintborg B, Lindstrom U, Aaltonen K et al (2018) Biological treatment in ankylosing spondylitis in the Nordic countries during 2010–2016: a collaboration between five biological registries. Scand J Rheumatol:1–10. https://doi.org/10.1080/03009742.2018.1444199
Chiowchanwisawakit P, Katchamart W, Osiri M et al (2019) Effectiveness and drug survival of anti-tumor necrosis factor alpha therapies in patients with spondyloarthritis: analysis from the thai rheumatic disease prior authorization registry. J Clin Rheumatol 25:9–15. https://doi.org/10.1097/rhu.0000000000000741
Fong W, Holroyd C, Davidson B et al (2016) The effectiveness of a real life dose reduction strategy for tumour necrosis factor inhibitors in ankylosing spondylitis and psoriatic arthritis. Rheumatology 55:1837–1842. https://doi.org/10.1093/rheumatology/kew269
Purmonen T, Tormalehto S, Wahlman H, Puolakka K (2018) Budget impact analysis of secukinumab versus adalimumab in the treatment of ankylosing spondylitis. J Med Econ 22:1–14. https://doi.org/10.1080/13696998.2018.1551227
Benucci M, Gobbi FL, Bandinelli F et al (2017) Safety, efficacy and immunogenicity of switching from innovator to biosimilar infliximab in patients with spondyloarthritis: a 6-month real-life observational study. Immunol Res 65:419–422. https://doi.org/10.1007/s12026-016-8843-5
Glintborg B, Loft AG, Omerovic E et al (2018) To switch or not to switch: results of a nationwide guideline of mandatory switching from originator to biosimilar etanercept. One-year treatment outcomes in 2061 patients with inflammatory arthritis from the DANBIO registry. Ann Rheum Dis. https://doi.org/10.1136/annrheumdis-2018-213474. [Epub ahead of print]
Chiu YM, Tang CH, Hung ST, Yang YW, Fang CH, Lin HY (2017) A real-world risk analysis of biological treatment (adalimumab and etanercept) in a country with a high prevalence of tuberculosis and chronic liver disease: a nationwide population-based study. Scand J Rheumatol 46:236–240. https://doi.org/10.1080/03009742.2016.1202318
Malaviya A, Thakaran R, Rawat R et al (2018) Real life experience of a screening strategy for latent tuberculosis before treatment with biologicals in indian patients with rheumatic diseases. Indian J Rheumatol 13:233–239. https://doi.org/10.4103/injr.injr_66_18
Conaghan PG, Peloso PM, Everett SV et al (2015) Inadequate pain relief and large functional loss among patients with knee osteoarthritis: evidence from a prospective multinational longitudinal study of osteoarthritis real-world therapies. Rheumatology 54:270–277. https://doi.org/10.1093/rheumatology/keu332
Angiolillo DJ, Weisman SM (2017) Clinical pharmacology and cardiovascular safety of naproxen. Am J Cardiovasc Drugs 17:97–107. https://doi.org/10.1007/s40256-016-0200-5
Bruyere O, Altman RD, Reginster JY (2016) Efficacy and safety of glucosamine sulfate in the management of osteoarthritis: Evidence from real-life setting trials and surveys. Semin Arthritis Rheum 45:S12–S17. https://doi.org/10.1016/j.semarthrit.2015.11.011
Bruyere O, Cooper C, Pelletier JP et al (2016) A consensus statement on the European Society for Clinical and Economic Aspects of Osteoporosis and Osteoarthritis (ESCEO) algorithm for the management of knee osteoarthritis-From evidence-based medicine to the real-life setting. Semin Arthritis Rheum 45:S3–S11. https://doi.org/10.1016/j.semarthrit.2015.11.010
Maheu E, Rannou F, Reginster JY (2016) Efficacy and safety of hyaluronic acid in the management of osteoarthritis: evidence from real-life setting trials and surveys. Semin Arthritis Rheum 45:S28–S33. https://doi.org/10.1016/j.semarthrit.2015.11.008
Pelletier JP, Martel-Pelletier J, Rannou F, Cooper C (2016) Efficacy and safety of oral NSAIDs and analgesics in the management of osteoarthritis: evidence from real-life setting trials and surveys. Semin Arthritis Rheum 45:S22–S27. https://doi.org/10.1016/j.semarthrit.2015.11.009
Rannou F, Pelletier JP, Martel-Pelletier J (2016) Efficacy and safety of topical NSAIDs in the management of osteoarthritis: Evidence from real-life setting trials and surveys. Semin Arthritis Rheum 45:S18–S21. https://doi.org/10.1016/j.semarthrit.2015.11.007
Zhang W, Moskowitz RW, Nuki G et al (2008) OARSI recommendations for the management of hip and knee osteoarthritis, Part II: OARSI evidence-based, expert consensus guidelines. Osteoarthr Cartil 16:137–162. https://doi.org/10.1016/j.joca.2007.12.013
Dreinhofer KE, Dieppe P, Sturmer T et al (2006) Indications for total hip replacement: comparison of assessments of orthopaedic surgeons and referring physicians. Ann Rheum Dis 65:1346–1350. https://doi.org/10.1136/ard.2005.047811
Van Manen MD, Nace J, Mont MA (2012) Management of primary knee osteoarthritis and indications for total knee arthroplasty for general practitioners. J Am Osteopath Assoc 112:709–715
Maillefert JF, Roy C, Cadet C, Nizard R, Berdah L, Ravaud P (2008) Factors influencing surgeons’ decisions in the indication for total joint replacement in hip osteoarthritis in real life. Arthritis Rheum 59:255–262. https://doi.org/10.1002/art.23331
Askanase AD, Nguyen SC, Costenbader K et al (2018) Comparison of the lupus foundation of America-rapid evaluation of activity in lupus to more complex disease activity instruments as evaluated by clinical investigators or real-world clinicians. Arthritis Care Res 70:1058–1063. https://doi.org/10.1002/acr.23445. (Hoboken)
Schneider M, Mosca M, Pego-Reigosa JM et al (2016) Understanding remission in real-world lupus patients across five European countries. Lupus 25:505–512. https://doi.org/10.1177/0961203315619030
Rovin BH, Furie R, Latinis K et al (2012) Efficacy and safety of rituximab in patients with active proliferative lupus nephritis: the lupus nephritis assessment with rituximab study. Arthritis Rheum 64:1215–1226. https://doi.org/10.1002/art.34359
Merrill JT, Neuwelt CM, Wallace DJ et al (2010) Efficacy and safety of rituximab in moderately-to-severely active systemic lupus erythematosus: the randomized, double-blind, phase II/III systemic lupus erythematosus evaluation of rituximab trial. Arthritis Rheum 62:222–233. https://doi.org/10.1002/art.27233
Fernandez-Nebro A, de la Fuente JL, Carreno L et al (2012) Multicenter longitudinal study of B-lymphocyte depletion in refractory systemic lupus erythematosus: the LESIMAB study. Lupus 21:1063–1076. https://doi.org/10.1177/0961203312446627
Iaccarino L, Bartoloni E, Carli L et al (2015) Efficacy and safety of off-label use of rituximab in refractory lupus: data from the Italian Multicentre Registry. Clin Exp Rheumatol 33:449–456
Furie R, Petri M, Zamani O et al (2011) A phase III, randomized, placebo-controlled study of belimumab, a monoclonal antibody that inhibits B lymphocyte stimulator, in patients with systemic lupus erythematosus. Arthritis Rheum 63:3918–3930. https://doi.org/10.1002/art.30613
Navarra SV, Guzman RM, Gallacher AE et al (2011) Efficacy and safety of belimumab in patients with active systemic lupus erythematosus: a randomised, placebo-controlled, phase 3 trial. Lancet 377:721–731. https://doi.org/10.1016/s0140-6736(10)61354-2
Stohl W, Schwarting A, Okada M et al (2017) Efficacy and safety of subcutaneous belimumab in systemic lupus erythematosus: a fifty-two-week randomized, double-blind, placebo-controlled study. Arthritis Rheumatol 69:1016–1027. https://doi.org/10.1002/art.40049
Schwarting A, Schroeder JO, Alexander T et al (2016) First Real-World Insights into Belimumab Use and Outcomes in Routine Clinical Care of Systemic Lupus Erythematosus in Germany: Results from the OBSErve Germany Study. Rheumatol Ther 3:271–290. https://doi.org/10.1007/s40744-016-0047-x
Touma Z, Sayani A, Pineau CA et al (2017) Belimumab use, clinical outcomes and glucocorticoid reduction in patients with systemic lupus erythematosus receiving belimumab in clinical practice settings: results from the OBSErve Canada Study. Rheumatol Int 37:865–873. https://doi.org/10.1007/s00296-017-3682-9
Gualtierotti R, Borghi MO, Gerosa M et al (2018) Successful sequential therapy with rituximab and belimumab in patients with active systemic lupus erythematosus: a case series. Clin Exp Rheumatol 36:643–647
Rubio-Rivas M, Corbella X, Pestana-Fernandez M et al (2018) First clinical symptom as a prognostic factor in systemic sclerosis: results of a retrospective nationwide cohort study. Clin Rheumatol 37:999–1009. https://doi.org/10.1007/s10067-017-3936-7
Sanchez-Cano D, Ortego-Centeno N, Callejas JL et al (2018) Interstitial lung disease in systemic sclerosis: data from the spanish scleroderma study group. Rheumatol Int 38:363–374. https://doi.org/10.1007/s00296-017-3916-x
Kolstad KD, Li S, Steen V, Chung L (2018) Long-Term outcomes in systemic sclerosis-associated pulmonary arterial hypertension from the Pulmonary Hypertension Assessment and Recognition of Outcomes in Scleroderma Registry (PHAROS). Chest 154:862–871. https://doi.org/10.1016/j.chest.2018.05.002
Hruskova Z, Pippias M, Stel VS et al (2018) Characteristics and outcomes of patients with systemic sclerosis (scleroderma) requiring renal replacement therapy in Europe: results from the ERA-EDTA Registry. Am J Kidney Dis. https://doi.org/10.1053/j.ajkd.2018.05.016. [Epub ahead of print]
Ram Poudel D, George M, Dhital R, Karmacharya P, Sandorfi N, Derk CT (2018) Mortality, length of stay and cost of hospitalization among patients with systemic sclerosis: results from the National Inpatient Sample. Rheumatology 57:1611–1622. https://doi.org/10.1093/rheumatology/key150
Lilleker JB, Vencovsky J, Wang G et al (2018) The EuroMyositis registry: an international collaborative tool to facilitate myositis research. Ann Rheum Dis 77:30–39. https://doi.org/10.1136/annrheumdis-2017-211868
Nuno L, Joven B, Carreira P et al (2017) Multicenter registry on inflammatory myositis from the Rheumatology Society in Madrid, Spain: descriptive analysis. Reumatol Clin 13:331–337. https://doi.org/10.1016/j.reuma.2016.07.010
Limaye V, Hakendorf P, Woodman RJ, Blumbergs P, Roberts-Thomson P (2012) Mortality and its predominant causes in a large cohort of patients with biopsy-determined inflammatory myositis. Intern Med J 42:191–198. https://doi.org/10.1111/j.1445-5994.2010.02406.x
Aggarwal R, McBurney C, Schneider F et al (2017) Myositis-associated usual interstitial pneumonia has a better survival than idiopathic pulmonary fibrosis. Rheumatology 56:384–389. https://doi.org/10.1093/rheumatology/kew426
Oddis CV, Reed AM, Aggarwal R et al (2013) Rituximab in the treatment of refractory adult and juvenile dermatomyositis and adult polymyositis: a randomized, placebo-phase trial. Arthritis Rheum 65:314–324. https://doi.org/10.1002/art.37754
Couderc M, Gottenberg JE, Mariette X et al (2011) Efficacy and safety of rituximab in the treatment of refractory inflammatory myopathies in adults: results from the AIR registry. Rheumatology 50:2283–2289. https://doi.org/10.1093/rheumatology/ker305
Unger L, Kampf S, Luthke K, Aringer M (2014) Rituximab therapy in patients with refractory dermatomyositis or polymyositis: differential effects in a real-life population. Rheumatology 53:1630–1638. https://doi.org/10.1093/rheumatology/keu024
Lariviere D, Benali K, Coustet B et al (2016) Positron emission tomography and computed tomography angiography for the diagnosis of giant cell arteritis: a real-life prospective study. Medicine 95:e4146. https://doi.org/10.1097/md.0000000000004146. (Baltimore)
Broder MS, Sarsour K, Chang E et al (2016) Corticosteroid-related adverse events in patients with giant cell arteritis: a claims-based analysis. Semin Arthritis Rheum 46:246–252. https://doi.org/10.1016/j.semarthrit.2016.05.009
Gale S, Wilson JC, Chia J et al (2018) Risk associated with cumulative oral glucocorticoid use in patients with giant cell arteritis in real-world databases from the USA and UK. Rheumatol Ther. https://doi.org/10.1007/s40744-018-0112-8. [Epub ahead of print]
Vitiello G, Orsi Battaglini C, Carli G et al (2018) Tocilizumab in giant cell arteritis: a real-life retrospective study. Angiology 69:763–769. https://doi.org/10.1177/0003319717753223
Stone JH, Tuckwell K, Dimonaco S et al (2017) Trial of tocilizumab in giant-cell arteritis. N Engl J Med 377:317–328. https://doi.org/10.1056/NEJMoa1613849
Pagnoux C, Carette S, Khalidi NA et al (2015) Comparability of patients with ANCA-associated vasculitis enrolled in clinical trials or in observational cohorts. Clin Exp Rheumatol 33:S–S77
Watad A, Bragazzi NL, Sharif K et al (2018) Borderline positive antineutrophil cytoplasmic antibodies (ANCA)-PR3/MPO detection in a large cohort tertiary center: lessons learnt from a real-life experience. Clin Chem Lab Med 56:947–953. https://doi.org/10.1515/cclm-2017-1053
Barbour S, Lo C, Espino-Hernandez G et al (2017) The population-level costs of immunosuppression medications for the treatment of glomerulonephritis are increasing over time due to changing patterns of practice. Nephrol Dial Transpl. https://doi.org/10.1093/ndt/gfx185
Raimundo K, Farr AM, Kim G, Duna G (2015) Clinical and economic burden of antineutrophil cytoplasmic antibody-associated vasculitis in the United States. J Rheumatol 42:2383–2391. https://doi.org/10.3899/jrheum.150479
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The conception and design of the study, or acquisition of data, or analysis and interpretation of data—DPM, VA. Drafting the article—DPM; Revising it critically for important intellectual content—VA. Final approval of the version to be submitted—DPM, VA. Agreement to be accountable for all aspects of the work in ensuring that questions related to the accuracy or integrity of any part of the work are appropriately investigated and resolved—DPM, VA.
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Durga Prasanna Misra declares that he has no conflict of interest. Vikas Agarwal declares that he has no conflict of interest.
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Misra, D.P., Agarwal, V. Real-world evidence in rheumatic diseases: relevance and lessons learnt. Rheumatol Int 39, 403–416 (2019). https://doi.org/10.1007/s00296-019-04248-1
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DOI: https://doi.org/10.1007/s00296-019-04248-1