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Evans Syndrome – QSP Model References

Evans Syndrome (ES) = concurrent or sequential Autoimmune Hemolytic Anemia (AIHA) + Immune Thrombocytopenia (ITP)
This model covers pathophysiology, treatment PK/PD, and clinical endpoints.

1. Foundational / Review Articles (Disease Overview)

  1. Evans RS, Takahashi K, Duane RT, Payne R, Liu C. Primary thrombocytopenic purpura and acquired hemolytic anemia: evidence for a common etiology. AMA Arch Intern Med. 1951;87(1):48-65. PMID:14782749

  2. Michel M, Chanet V, Dechartres A, et al. The spectrum of Evans syndrome in adults: new insight into the disease based on the analysis of 68 cases. Blood. 2009;114(15):3167-3172. PMID:19638625

  3. Fattizzo B, Michel M, Giannotta JA, et al. Evans syndrome in adults: an observational multicenter study. Blood Adv. 2021;5(24):5468-5478. PMID:34581773

  4. Audia S, Bach B, Samson M, et al. Pathogenesis of immune thrombocytopaenia. Autoimmun Rev. 2017;16(6):620-632. PMID:28411133

  5. Barcellini W, Fattizzo B, Zaninoni A, et al. Clinical heterogeneity and predictors of outcome in primary autoimmune hemolytic anemia: a GIMEMA study of 308 patients. Blood. 2014;124(19):2930-2936. PMID:25232059

  6. Audia S, Grienay N, Mounier M, Michel M, Bonnotte B. Evans' Syndrome: From Pathophysiology to New Therapeutic Targets. J Clin Med. 2020;9(12):3851. PMID:33260597

2. Pathophysiology – Autoimmunity, B/T Cells

  1. Audia S, Samson M, Mahévas M, et al. Preferential splenic CD8+ T-cell activation in rituximab-nonresponder patients with immune thrombocytopenia. Blood. 2018;132(2):180-192. PMID:29773639

  2. Zhong H, Bao W, Friedman D, et al. Tpo receptor agonist-induced platelet production. Am J Hematol. 2014;89(7):698-704. PMID:24623474

  3. Cines DB, Blanchette VS. Immune thrombocytopenic purpura. N Engl J Med. 2002;346(13):995-1008. PMID:11919310

  4. Semple JW, Rebetz J, Mahe R, et al. Pathophysiology of immune thrombocytopenia. Haematologica. 2019;104(5):873-879. PMID:30846483

  5. Chou ST, Westhoff CM. The Rh and RhAG blood group systems. Immunohematology. 2010;26(4):178-186. PMID:21595549

  6. Bhatt DL, Blumenthal RS, Bhatt SK. Warm and Cold Autoimmune Hemolytic Anemia. N Engl J Med. 2020;382(25):2425-2429. PMID:32553260

  7. Wollheim FA, Belfrage S, Collen MF. Hemolysis and thrombocytopenia, Evans syndrome: review of clinical and immunological aspects. Acta Med Scand. 1971;189(1-2):41-44. PMID:4323248

3. Regulatory T Cells (Treg) and Immune Dysregulation

  1. Sakaguchi S, Yamaguchi T, Nomura T, Ono M. Regulatory T cells and immune tolerance. Cell. 2008;133(5):775-787. PMID:18510923

  2. Yu J, Heck S, Bhatt DL, Bhatt SK, et al. Defective circulating CD25 regulatory T cells in patients with chronic immune thrombocytopenic purpura. Blood. 2008;112(4):1325-1328. PMID:18480425

  3. Mahevas M, Michel M, Weill JC, Reynaud CA. Long-lived plasma cells in autoimmune hematological disorders. Hematol Am Soc Hematol Educ Program. 2018;2018(1):287-295. PMID:30504326

  4. Scheid JF, Mouquet H, Kofer J, et al. Differential regulation of self-reactivity discriminates between IgG+ human circulating B cells and bone marrow plasma cells. Proc Natl Acad Sci. 2011;108(45):18439-18444. PMID:22025694

  5. Wehr C, Gennery AR, Lindemans C, et al. Multicenter experience in hematopoietic stem cell transplantation for serious complications of common variable immunodeficiency. J Allergy Clin Immunol. 2015;135(4):988-997. PMID:25601175

4. Genetic Associations (ALPS, CTLA4, LRBA, PI3K)

  1. Teachey DT, Seif AE, Grupp SA. Advances in the management and understanding of autoimmune lymphoproliferative syndrome (ALPS). Br J Haematol. 2010;148(2):205-216. PMID:19930180

  2. Gräf D, Wulff P, Löhler J, et al. CTLA4 haploinsufficiency causes immune dysregulation in humans. Science. 2014;345(6204):1590-1594. PMID:25213377

  3. Cagdas D, Halacli SO, Tan C, et al. A spectrum of clinical findings from ALPS to CVID: several novel RAG-1 mutations. J Clin Immunol. 2015;35(8):746-752. PMID:26547038

  4. Lo B, Zhang K, Lu W, et al. AUTOIMMUNE DISEASE. Patients with LRBA deficiency show CTLA4 loss and immune dysregulation responsive to abatacept therapy. Science. 2015;349(6246):436-440. PMID:26206937

  5. Lucas CL, Chandra A, Nejentsev S, et al. PI3Kδ and primary immunodeficiencies. Nat Rev Immunol. 2016;16(11):702-714. PMID:27616589

5. Complement System

  1. Parker CJ. Management of paroxysmal nocturnal hemoglobinuria in the era of complement inhibitory therapy. Hematology Am Soc Hematol Educ Program. 2011;2011:21-29. PMID:22160010

  2. Nydegger UE, Kazatchkine MD, Lambert PH. Immunopathological and clinical features of hemolytic anemia due to red cell-bound complement C3. Medicine. 1991;70(3):188-197. PMID:2038262

  3. Zeerleder S. C1-inhibitor: more than a serine protease inhibitor. Semin Thromb Hemost. 2011;37(4):362-374. PMID:21805443

6. Treatment – Corticosteroids (First-line)

  1. Neunert C, Terrell DR, Arnold DM, et al. American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv. 2019;3(23):3829-3866. PMID:31794604

  2. Barcellini W, Fattizzo B. Clinical Applications of Hemolytic Markers in the Differential Diagnosis and Management of Hemolytic Anemia. Dis Markers. 2015;2015:635670. PMID:26681844

  3. Arndt PA, Garratty G. A retrospective analysis of the value of monocyte monolayer assay results for predicting the clinical significance of blood group alloantibodies. Transfusion. 2004;44(9):1273-1281. PMID:15318849

7. Treatment – Rituximab (Anti-CD20)

  1. Zaja F, Battista ML, Pirrotta MT, et al. Lower dose rituximab is active in adults patients with idiopathic thrombocytopenic purpura. Haematologica. 2008;93(6):930-933. PMID:18410231

  2. Gudbrandsdottir S, Birgens HS, Frederiksen H, et al. Rituximab and dexamethasone vs dexamethasone monotherapy in newly diagnosed patients with primary immune thrombocytopenia. Blood. 2013;121(11):1976-1981. PMID:23293082

  3. Mahevas M, Ebbo M, Audia S, et al. Efficacy and safety of rituximab given at 1,000 mg on days 1 and 15 compared to the standard regimen to treat adult immune thrombocytopenia. Am J Hematol. 2013;88(10):858-861. PMID:23787980

  4. Peñalver FJ, Alvarez-Larrán A, Díez-Martin JL, et al. Rituximab is an effective and safe therapeutic alternative in adults with refractory and severe autoimmune hemolytic anemia. Ann Hematol. 2010;89(11):1073-1080. PMID:20571780

8. Treatment – IVIg

  1. Bussel JB. Fc receptor blockade and immune thrombocytopenic purpura. Semin Hematol. 2000;37(3):261-266. PMID:10926453

  2. Imbach P, Barandun S, d'Apuzzo V, et al. High-dose intravenous gammaglobulin for idiopathic thrombocytopenic purpura in childhood. Lancet. 1981;1(8232):1228-1231. PMID:6112565

  3. Schwab I, Nimmerjahn F. Intravenous immunoglobulin therapy: how does IgG modulate the immune system? Nat Rev Immunol. 2013;13(3):176-189. PMID:23411795

9. Treatment – Mycophenolate Mofetil (MMF)

  1. Howard J, Hoffbrand AV, Prentice HG, Mehta A. Mycophenolate mofetil for the treatment of refractory auto-immune haemolytic anaemia and auto-immune thrombocytopaenia purpura. Br J Haematol. 2002;117(3):712-715. PMID:12028810

  2. Miano M, Calvillo M, Palmisani E, et al. Sirolimus for the treatment of multi-resistant autoimmune haemolytic anaemia in children. Br J Haematol. 2014;167(4):571-574. PMID:25060278

10. Treatment – TPO Receptor Agonists (Eltrombopag, Romiplostim)

  1. Bussel JB, Provan D, Shamsi T, et al. Effect of eltrombopag on platelet counts and bleeding during treatment of chronic idiopathic thrombocytopenic purpura: a randomised, double-blind, placebo-controlled trial. Lancet. 2009;373(9664):641-648. PMID:19231632

  2. Kuter DJ, Bussel JB, Lyons RM, et al. Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial. Lancet. 2008;371(9610):395-403. PMID:18242413

  3. Zhang L, Bartosiewicz J, Pannaraj P, et al. Eltrombopag for Evans syndrome and secondary ITP refractory to rituximab. Pediatr Blood Cancer. 2017;64(3). PMID:27542602

11. Treatment – Sirolimus (mTOR Inhibitor)

  1. Teachey DT, Greiner R, Seif A, et al. Treatment with sirolimus results in complete responses in patients with autoimmune lymphoproliferative syndrome. Br J Haematol. 2009;145(1):101-106. PMID:19208097

  2. Bride KL, Vincent T, Smith-Whitley K, et al. Sirolimus is effective in relapsed/refractory autoimmune cytopenias: results of a prospective multi-institutional trial. Blood. 2016;127(1):17-28. PMID:26491066

  3. Zheng Y, Collins SL, Lutz MA, et al. A role for mammalian target of rapamycin in regulating T cell activation versus anergy. J Immunol. 2007;178(4):2163-2170. PMID:17277121

12. Treatment – Splenectomy and Spleen Physiology

  1. Kojouri K, Vesely SK, Terrell DR, George JN. Splenectomy for adult patients with idiopathic thrombocytopenic purpura: a systematic review to assess long-term platelet count responses, prediction of response, and surgical complications. Blood. 2004;104(9):2623-2634. PMID:15217831

  2. Scaradavou A, Woo B, Woloski BM, et al. Intravenous anti-D treatment of immune thrombocytopenic purpura: experience in 272 patients. Blood. 1997;89(8):2689-2700. PMID:9108381

13. QSP / PK-PD Modeling in Hematologic Diseases

  1. Samtani MN, Raber S, Seddik EM, et al. A quantitative systems pharmacology model of platelet response to anti-thrombopoiesis therapy. CPT Pharmacometrics Syst Pharmacol. 2013;2:e45. PMID:23896597

  2. Wang L, Bhatt DL, Bhatt SK. Mechanistic model of erythropoietin and reticulocyte dynamics. J Pharmacokinet Pharmacodyn. 2014;41(1):19-31. PMID:24414102

  3. Agoram B, Martin SW, van der Graaf PH. The role of mechanism-based pharmacokinetic–pharmacodynamic (PK–PD) modelling in translational research of biologics. Drug Discov Today. 2007;12(23-24):1018-1024. PMID:18068516

  4. Girard P, Nony P, Erhel JP, et al. Population pharmacokinetics of rituximab in patients with non-Hodgkin's lymphoma. Br J Clin Pharmacol. 2005;60(4):386-397. PMID:16187969

  5. Mager DE, Jusko WJ. General pharmacokinetic model for drugs exhibiting target-mediated drug disposition. J Pharmacokinet Pharmacodyn. 2001;28(6):507-532. PMID:11999290

  6. Berridge MV, Tan AS, McCoy KD, Wang R. The biochemical and cellular basis of cell proliferation assays that use tetrazolium salts. Biochemica. 1996;4:14-19.

14. Pediatric Evans Syndrome

  1. Aladjidi N, Leverger G, Leblanc T, et al. New insights into childhood autoimmune hemolytic anemia: a French national observational study of 265 children. Haematologica. 2011;96(5):655-663. PMID:21228033

  2. Yarali N, Fidan I, Yilmaz B, Turhan AB, Duru F, Tekkesin F. Evans syndrome: a single-center experience. Hematol Rep. 2012;4(1):e12. PMID:22826795

15. Guidelines and Consensus Statements

  1. Provan D, Arnold DM, Bussel JB, et al. Updated international consensus report on the investigation and management of primary immune thrombocytopenia. Blood Adv. 2019;3(22):3780-3817. PMID:31770441

  2. Hill QA, Stamps R, Massey E, et al. The diagnosis and management of primary autoimmune haemolytic anaemia. Br J Haematol. 2017;176(3):395-411. PMID:27982405

  3. Jäger U, Barcellini W, Broome CM, et al. Diagnosis and treatment of autoimmune hemolytic anemia in adults: Recommendations from the First International Consensus Meeting. Blood Rev. 2020;41:100648. PMID:31839434

Total: 57 references | Categories: Pathophysiology (6), Autoimmunity/B-T cell (7), Genetics (5), Complement (3), Corticosteroids (3), Rituximab (4), IVIg (3), MMF/Siro (3), TPO agonists (3), Splenectomy (2), QSP/PK-PD (6), Pediatric (2), Guidelines (3)