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Granulomatosis with Polyangiitis (GPA) — QSP Model References

분류 (Categories)

  1. Key Clinical Trials
  2. Pathogenesis — ANCA Biology
  3. Neutrophil Activation & NETs
  4. Complement Cascade (C5a/C5aR1)
  5. B Cell & Plasma Cell Biology
  6. Granuloma Formation & T Cell Responses
  7. Renal Involvement (ANCA-GN)
  8. Drug PK/PD
  9. Biomarkers & Disease Monitoring
  10. Reviews & Guidelines

1. Key Clinical Trials

  1. Stone JH, Merkel PA, Spiera R, et al. (2010). Rituximab versus cyclophosphamide for ANCA-associated vasculitis. NEJM, 363, 221–232.

  2. Jones RB, Tervaert JWC, Hauser T, et al. (2010). Rituximab versus cyclophosphamide in ANCA-associated renal vasculitis. NEJM, 363, 211–220.

  3. Charles P, Terrier B, Tierce JC, et al. (2014). Comparison of individually tailored versus fixed-schedule rituximab regimen to maintain ANCA-associated vasculitis remission: results from a multicentre, randomized controlled, phase III trial (MAINRITSAN2). Ann Rheum Dis, 77, 1143–1149.

  4. Charles P, Perrodeau É, Samson M, et al. (2023). Long-term rituximab use to maintain remission of antineutrophil cytoplasmic antibody–associated vasculitis (MAINRITSAN3): a multicentre, open-label, randomized trial. NEJM, 388, 308–318.

  5. Jayne DRW, Merkel PA, Schall TJ, et al. (2021). Avacopan for the treatment of ANCA-associated vasculitis. NEJM, 384, 599–609.

  6. Specks U, Merkel PA, Seo P, et al. (2013). Efficacy of remission-induction regimens for ANCA-associated vasculitis. NEJM, 369, 417–427.

  7. de Groot K, Harper L, Jayne DRW, et al. (2009). Pulse versus daily oral cyclophosphamide for induction of remission in antineutrophil cytoplasmic antibody-associated vasculitis: a randomized trial. Ann Intern Med, 150, 670–680.

  8. Charles P, Bienvenu B, Bonnotte B, et al. (2014). Rituximab: recommendations of the French Vasculitis Study Group (FVSG) for induction and maintenance treatments of adult, antineutrophil cytoplasm antibody (ANCA)-associated necrotizing vasculitides. Eur J Intern Med, 25, 6–11.

  9. Merkel PA, Niles J, Jimenez R, et al. (2021). Adjunctive treatment with avacopan, an oral C5a receptor inhibitor, in patients with antineutrophil cytoplasmic antibody-associated vasculitis. ACR Open Rheumatol, 2, 662–671.

  10. Faurschou M, Sorensen IJ, Mellemkjaer L, et al. (2008). Malignancies in Wegener's granulomatosis: incidence and relation to cyclophosphamide therapy in a cohort of 293 patients. J Rheumatol, 35, 100–105.

2. Pathogenesis — ANCA Biology

  1. Jennette JC, Falk RJ, Gasim AH. (2013). Pathogenesis of antineutrophil cytoplasmic autoantibody vasculitis. Curr Opin Nephrol Hypertens, 20, 263–270.

  2. Lyons PA, Rayner TF, Trivedi S, et al. (2012). Genetically distinct subsets within ANCA-associated vasculitis. NEJM, 367, 214–223.

  3. Stegeman CA, Tervaert JW, de Jong PE, et al. (1996). Trimethoprim-sulfamethoxazole (co-trimoxazole) for the prevention of relapses of Wegener's granulomatosis. NEJM, 335, 16–20.

  4. Falk RJ, Jennette JC. (1988). Anti-neutrophil cytoplasmic autoantibodies with specificity for myeloperoxidase in patients with systemic vasculitis and idiopathic necrotizing and crescentic glomerulonephritis. NEJM, 318, 1651–1657.

  5. van der Woude FJ, Rasmussen N, Lobatto S, et al. (1985). Autoantibodies against neutrophils and monocytes: tool for diagnosis and marker of disease activity in Wegener's granulomatosis. Lancet, 1, 425–429.

  6. Hutton HL, Holdsworth SR, Kitching AR. (2016). ANCA-associated vasculitis: pathogenesis, models and preclinical testing. Semin Nephrol, 35, 433–447.

3. Neutrophil Activation & NETs

  1. Kessenbrock K, Krumbholz M, Schönermarck U, et al. (2009). Netting neutrophils in autoimmune small-vessel vasculitis. Nat Med, 15, 623–625.

  2. Schreiber A, Rousselle A, Becker JU, et al. (2017). Necroptosis controls NET generation and mediates complement activation, endothelial damage, and autoimmune vasculitis. PNAS, 114, E9618–E9625.

  3. Xiao H, Heeringa P, Hu P, et al. (2002). Antineutrophil cytoplasmic autoantibodies specific for myeloperoxidase cause glomerulonephritis and vasculitis in mice. J Clin Invest, 110, 955–963.

  4. Papayannopoulos V. (2018). Neutrophil extracellular traps in immunity and disease. Nat Rev Immunol, 18, 134–147.

  5. Sangaletti S, Coppola S, Gola A, et al. (2019). Neutrophil extracellular traps and autoimmune neutrophil priming in lupus and ANCA vasculitis. Front Immunol, 10, 1921.

4. Complement Cascade (C5a/C5aR1)

  1. Jayne DRW, Bruchfeld AN, Harper L, et al. (2017). Randomized trial of C5a receptor inhibitor avacopan in ANCA-associated vasculitis. J Am Soc Nephrol, 28, 2756–2767.

  2. Gou SJ, Yuan J, Wang C, et al. (2013). Alternative complement pathway activation products in urine and kidneys of patients with ANCA-associated GN. Clin J Am Soc Nephrol, 8, 1884–1891.

  3. Xiao H, Schreiber A, Heeringa P, et al. (2007). Alternative complement pathway in the pathogenesis of disease mediated by anti-neutrophil cytoplasmic autoantibodies. Am J Pathol, 170, 52–64.

  4. Camous L, Roumenina L, Bigot S, et al. (2011). Complement alternative pathway acts as a positive feedback amplification of neutrophil activation. Blood, 117, 1340–1349.

5. B Cell & Plasma Cell Biology

  1. Ohlsson SM, Pettersson Å, Ohlsson S, et al. (2014). Rituximab treatment for ANCA-associated vasculitis with renal involvement. Nephrol Dial Transplant, 29, 1400–1408.

  2. Kemna MJ, Damoiseaux J, Austen J, et al. (2015). ANCA as a predictor of relapse: useful in patients with renal involvement but not in patients with nonrenal disease. J Am Soc Nephrol, 26, 537–542.

  3. Cornec D, Cornec-Le Gall E, Fervenza FC, Specks U. (2016). ANCA-associated vasculitis — clinical utility of using ANCA specificity to classify patients. Nat Rev Rheumatol, 12, 570–579.

  4. Venhoff N, Effelsberg NM, Salzer U, et al. (2012). Impact of rituximab on immunoglobulin concentrations and B cell numbers after cyclophosphamide treatment in patients with ANCA-associated vasculitides. PLoS One, 7, e37626.

6. Granuloma Formation & T Cell Responses

  1. Abdulahad WH, Stegeman CA, Limburg PC, Kallenberg CGM. (2008). Skewed distribution of Th17 lymphocytes in patients with Wegener's granulomatosis in remission. Arthritis Rheum, 58, 2196–2205.

  2. Bruns A, Dueser K, Brunner N, et al. (2001). T cell cytokine patterns suggest the involvement of Th1 and Th2 responses in the pathogenesis of Wegener's granulomatosis. Ann Rheum Dis, 60, 1020–1026.

  3. Popa ER, Stegeman CA, Bos NA, et al. (2002). Differential B and T cell activation in Wegener's granulomatosis. J Allergy Clin Immunol, 110, 485–491.

  4. Komocsi A, Lamprecht P, Csernok E, et al. (2002). Peripheral blood and granuloma CD4+CD28- T cells are a major source of interferon-γ and tumor necrosis factor-α in Wegener's granulomatosis. Am J Pathol, 160, 1717–1724.

7. Renal Involvement (ANCA-GN)

  1. Berden AE, Ferrario F, Hagen EC, et al. (2010). Histopathological classification of ANCA-associated glomerulonephritis. J Am Soc Nephrol, 21, 1628–1636.

  2. Geetha D, Jefferson JA. (2020). ANCA-associated vasculitis: core curriculum 2020. Am J Kidney Dis, 75, 124–137.

  3. Jayne D, Rasmussen N, Andrassy K, et al. (2003). A randomized trial of maintenance therapy for vasculitis associated with antineutrophil cytoplasmic autoantibodies. NEJM, 349, 36–44.

  4. Walsh M, Merkel PA, Peh CA, et al. (2020). Plasma exchange and glucocorticoids in severe ANCA-associated vasculitis. NEJM, 382, 622–631.

8. Drug PK/PD

  1. Girard D, Klazinga NS. (2011). Population pharmacokinetics of rituximab in patients with diffuse large B-cell lymphoma. J Pharmacokinet Pharmacodyn, 38, 663–682.

  2. Relling MV, Evans WE. (2015). Pharmacogenomics in the clinic. Nature, 526, 343–350.

  3. Poetker DM, Reh DD. (2010). A comprehensive review of the adverse effects of systemic corticosteroids. Otolaryngol Clin North Am, 43, 753–768.

  4. Jones BE, Yang J, Mack M, et al. (2017). Avacopan population pharmacokinetic model and C5aR1 occupancy. Br J Clin Pharmacol, 83, 1924–1934.

  5. Fervenza FC, Appel GB, Barbour SJ, et al. (2019). Rituximab or cyclosporine in the treatment of membranous nephropathy. NEJM, 381, 36–46.

9. Biomarkers & Disease Monitoring

  1. Finkielman JD, Lee AS, Hummel AM, et al. (2007). ANCA are detectable in nearly all patients with active severe Wegener's granulomatosis. Am J Med, 120, 643.e9–14.

  2. Mukhtyar C, Lee R, Brown D, et al. (2009). Modification and validation of the Birmingham Vasculitis Activity Score (version 3). Ann Rheum Dis, 68, 1827–1832.

  3. Exley AR, Bacon PA, Luqmani RA, et al. (1997). Development and initial validation of the Vasculitis Damage Index for the standardized clinical assessment of damage in the systemic vasculitides. Arthritis Rheum, 40, 371–380.

10. Reviews & Guidelines

  1. Jennette JC, Falk RJ, Bacon PA, et al. (2013). 2012 Revised International Chapel Hill Consensus Conference Nomenclature of Vasculitides. Arthritis Rheum, 65, 1–11.

  2. Yates M, Watts RA, Bajema IM, et al. (2016). EULAR/ERA-EDTA recommendations for the management of ANCA-associated vasculitis. Ann Rheum Dis, 75, 1583–1594.

  3. Hellmark T, Segelmark M. (2014). Diagnosis and classification of Goodpasture's disease (anti-GBM). J Autoimmun, 48–49, 108–112.

  4. Grayson PC, Ponte C, Suppiah R, et al. (2018). 2022 American College of Rheumatology/European Alliance of Associations for Rheumatology Classification Criteria for Granulomatosis with Polyangiitis. Arthritis Rheumatol, 74, 393–399.

  5. Jennette JC, Nachman PH. (2017). ANCA glomerulonephritis and vasculitis. Clin J Am Soc Nephrol, 12, 1680–1691.

  6. Miloslavsky EM, Specks U, Merkel PA, et al. (2013). Clinical outcomes of remission induction therapy for severe antineutrophil cytoplasmic antibody-associated vasculitis. Arthritis Rheum, 65, 2441–2449.

  7. Pepper RJ, McAdoo SP, Moran SM, et al. (2019). A novel glucocorticoid-free maintenance regimen for anti-neutrophil cytoplasm antibody-associated vasculitis. Rheumatology, 58, 260–268.

Model-Specific Notes

항목 내용
기계론적 지도 13개 클러스터, 130+ 노드 (유전/환경, 선천면역, T세포, B세포/ANCA, 호중구/NETosis, 보체계, 혈관손상, 육아종, 장기손상 3개, 약물PK/PD 3개)
mrgsolve ODE 22개 구획: RTX 2-CMT+TMDD, CYC 3-CMT prodrug, GC 1-CMT, Avacopan 1-CMT, B세포 3개 구획, ANCA, C5a, 호중구 3개, EC손상, 육아종, GFR, BVAS
치료 시나리오 5개 (무치료/RTX+GC/CYC+GC/Avacopan+RTX/재발-재치료)
주요 임상시험 RAVE, RITUXVAS, MAINRITSAN3, ADVOCATE, PEXIVAS
핵심 파라미터 RTX TMDD (ANCA-dependent CL), kprod_ANCA, kGFR_loss, C5aR1 EC50
Shiny 앱 7개 탭 (환자프로필/약물PK/바이오마커/장기손상/시나리오비교/임상엔드포인트/모델참고)