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Real-world evidence in rheumatic diseases: relevance and lessons learnt

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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

  1. 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

  2. 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

    Article  PubMed  PubMed Central  Google Scholar 

  3. 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

    Article  PubMed  Google Scholar 

  4. 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

    Article  PubMed  Google Scholar 

  5. 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

    Article  Google Scholar 

  6. 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

    Article  PubMed  Google Scholar 

  7. Marelli L, Testa G (2018) Scrutinizing the EU general data protection regulation. Science 360:496–498. https://doi.org/10.1126/science.aar5419

    Article  CAS  PubMed  Google Scholar 

  8. 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

    Article  PubMed  Google Scholar 

  9. 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

    Article  PubMed  PubMed Central  Google Scholar 

  10. 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

    Article  CAS  PubMed  Google Scholar 

  11. 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

    Article  PubMed  PubMed Central  Google Scholar 

  12. 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)

    Article  Google Scholar 

  13. 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

    Article  PubMed  PubMed Central  Google Scholar 

  14. Ford I, Norrie J (2016) Pragmatic trials. N Engl J Med 375:454–463. https://doi.org/10.1056/NEJMra1510059

    Article  PubMed  Google Scholar 

  15. 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

    Article  PubMed  PubMed Central  Google Scholar 

  16. 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

    Article  PubMed  Google Scholar 

  17. 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

    Article  PubMed  Google Scholar 

  18. Ravindran V, Kataria S (2018) Digital health in rheumatology. Ann Rheum Dis. https://doi.org/10.1136/annrheumdis-2018-214146

    Article  PubMed  Google Scholar 

  19. 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

    Article  PubMed  Google Scholar 

  20. Dutton RP (2014) Quality management and registries. Anesthesiol Clin 32:577–586. https://doi.org/10.1016/j.anclin.2014.02.014

    Article  PubMed  Google Scholar 

  21. 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

    Article  PubMed  Google Scholar 

  22. Tovino SA (2017) The HIPAA privacy rule and the EU GDPR: illustrative comparisons. Seton Hall Law Rev 47:973–993

    PubMed  Google Scholar 

  23. 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

    Article  PubMed  Google Scholar 

  24. 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

    Article  PubMed  Google Scholar 

  25. 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]

    Article  PubMed  Google Scholar 

  26. 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

    Article  PubMed  PubMed Central  Google Scholar 

  27. 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

    Article  PubMed  Google Scholar 

  28. 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

    PubMed  Google Scholar 

  29. 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

    Article  PubMed  PubMed Central  Google Scholar 

  30. 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

    Article  PubMed  Google Scholar 

  31. 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

    Article  CAS  PubMed  Google Scholar 

  32. 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

    Article  PubMed  Google Scholar 

  33. 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

    Article  PubMed  Google Scholar 

  34. 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

    Article  PubMed  PubMed Central  Google Scholar 

  35. 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

    Article  PubMed  Google Scholar 

  36. 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

    Article  PubMed  Google Scholar 

  37. 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

    Article  PubMed  Google Scholar 

  38. 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

    PubMed  Google Scholar 

  39. 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

    PubMed  Google Scholar 

  40. 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

    Article  CAS  PubMed  Google Scholar 

  41. 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

    Article  CAS  Google Scholar 

  42. 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)

    Article  Google Scholar 

  43. 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

    Article  CAS  PubMed  Google Scholar 

  44. 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

    Article  PubMed  Google Scholar 

  45. 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

    Article  PubMed  Google Scholar 

  46. 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)

    Article  Google Scholar 

  47. 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

    Article  PubMed  Google Scholar 

  48. 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

    Article  PubMed  PubMed Central  Google Scholar 

  49. 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

    Article  PubMed  PubMed Central  Google Scholar 

  50. 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

    Article  PubMed  PubMed Central  Google Scholar 

  51. 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

    Article  PubMed  Google Scholar 

  52. 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

  53. 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

    Article  PubMed  Google Scholar 

  54. 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

    Article  CAS  PubMed  Google Scholar 

  55. 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

    Article  Google Scholar 

  56. 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

    Article  CAS  PubMed  Google Scholar 

  57. 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]

    Article  PubMed  Google Scholar 

  58. 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

    Article  CAS  PubMed  Google Scholar 

  59. 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

    Article  Google Scholar 

  60. 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

    Article  PubMed  Google Scholar 

  61. 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

    Article  CAS  PubMed  Google Scholar 

  62. 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

    Article  CAS  PubMed  Google Scholar 

  63. 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

    Article  PubMed  Google Scholar 

  64. 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

    Article  CAS  PubMed  Google Scholar 

  65. 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

    Article  CAS  PubMed  Google Scholar 

  66. 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

    Article  CAS  PubMed  Google Scholar 

  67. 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

    Article  CAS  Google Scholar 

  68. 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

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  69. 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

    PubMed  Google Scholar 

  70. 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

    Article  PubMed  Google Scholar 

  71. 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)

    Article  Google Scholar 

  72. 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

    Article  CAS  PubMed  Google Scholar 

  73. 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

    Article  CAS  PubMed  Google Scholar 

  74. 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

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  75. 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

    Article  CAS  PubMed  Google Scholar 

  76. 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

    PubMed  Google Scholar 

  77. 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

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  78. 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

    Article  CAS  PubMed  Google Scholar 

  79. 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

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  80. 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

    Article  PubMed  PubMed Central  Google Scholar 

  81. 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

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  82. 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

    PubMed  Google Scholar 

  83. 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

    Article  PubMed  Google Scholar 

  84. 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

    Article  CAS  PubMed  Google Scholar 

  85. 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

    Article  PubMed  PubMed Central  Google Scholar 

  86. 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]

    Article  PubMed  Google Scholar 

  87. 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

    Article  PubMed  Google Scholar 

  88. 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

    Article  PubMed  Google Scholar 

  89. 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

    Article  PubMed  Google Scholar 

  90. 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

    Article  CAS  PubMed  Google Scholar 

  91. 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

    Article  PubMed  Google Scholar 

  92. 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

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  93. 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

    Article  CAS  PubMed  Google Scholar 

  94. 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

    Article  CAS  PubMed  Google Scholar 

  95. 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)

    Article  PubMed  PubMed Central  Google Scholar 

  96. 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

    Article  PubMed  Google Scholar 

  97. 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]

    Article  PubMed  PubMed Central  Google Scholar 

  98. 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

    Article  CAS  PubMed  Google Scholar 

  99. 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

    Article  CAS  PubMed  Google Scholar 

  100. 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

    CAS  PubMed  PubMed Central  Google Scholar 

  101. 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

    Article  CAS  PubMed  Google Scholar 

  102. 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

    Article  Google Scholar 

  103. 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

    Article  PubMed  Google Scholar 

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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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Correspondence to Durga Prasanna Misra.

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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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