Hematology 2022: Nonmalignant diseases

CME

Key Studies in Nonmalignant Hematology: Independent Conference Coverage of ASH 2022

Physicians: Maximum of 1.00 AMA PRA Category 1 Credit

Released: March 23, 2023

Expiration: March 22, 2024

Activity

Progress
1
Course Completed
References

  1. De Latour RP, Roeth A, Kulasekaraj A, et al. Oral monotherapy with iptacopan, a proximal complement inhibitor of factor B, has superior efficacy to intravenous terminal complement inhibition with standard of care eculizumab or ravulizumab and favorable safety in patients with paroxysmal nocturnal hemoglobinuria and residual anemia: results from the randomized, active-comparator-controlled, open-label, multicenter, phase III APPLY-PNH study. Presented at: 2022 American Society of Hematology Annual Meeting; December 10-13, 2022. Abstract LBA-2.
  2. Kenet G, Nolan B, Zulfikar B, et al. A phase 3 study (ATLAS-PPX) to evaluate efficacy and safety of fitusiran, an siRNA therapeutic, in people with haemophilia A or B who have switched from prior factor or bypassing agent prophylaxis. Presented at: 2022 International Society on Thrombosis and Haemostasis; July 9-13, 2022. Abstract LB 01.1.
  3. Berentsen S. Complement activation and inhibition in autoimmune hemolytic anemia: focus on cold agglutinin disease. Semin Hematol. 2018;55:141-149.
  4. Mullins M, Jiang X, Bylsma LC, et al. Cold agglutinin disease burden: a longitudinal analysis of anemia, medications, transfusions, and health care utilization. Blood Adv. 2017;1:839-848.
  5. Roeth A, Berentsen S, Barcellini W, et al. Sutimlimab in patients with cold agglutinin disease: results of the randomized placebo-controlled phase 3 CADENZA trial. Blood. 2022;140:980-991.
  6. Roeth A, Broome CM, Barcellini W, et al. Sutimlimab provides sustained improvements in patient-reported outcomes and quality of life in patients with cold agglutinin disease: open-label extension of the randomized, phase 3 CADENZA study. Presented at: 2022 American Society of Hematology Annual Meeting; December 10-13, 2022. Abstract 31.
  7. Shah N, Bhatt H. Paroxysmal nocturnal hemoglobinuria. In: StatPearls [Internet]. StatPearls Publishing; 2022.
  8. Risitano AM, Frieri C, Urciuoli E, et al. The complement alternative pathway in paroxysmal nocturnal hemoglobinuria: from a pathogenic mechanism to a therapeutic target. Immunol Rev. 2022;[Online ahead of print].
  9. Eculizumab [prescribing information]. Boston, MA: Alexion; 2020.
  10. Ravulizumab [prescribing information]. Boston, MA: Alexion; 2022.
  11. Pegcetacoplan [prescribing information]. Waltham, MA: Apellis; 2021.
  12. Risitano AM, Marotta S, Ricci P, et al. Anti-complement treatment for paroxysmal nocturnal hemoglobinuria: time for proximal complement inhibition? A position paper from the SAAWP of the EBMT. Front Immunol. 2019;10:1157.
  13. Risitano AM, Roeth A, Soret J, et al. Addition of iptacopan, an oral factor B inhibitor, to eculizumab in patients with paroxysmal nocturnal haemoglobinuria and active haemolysis: an open-label, single-arm, phase 2, proof-of-concept trial. Lancet Haematol. 2021;8:e344-e354.
  14. Browett PJ, Kulasekaraj A, Notaro R, et al. Vemircopan (ALXN2050) monotherapy in paroxysmal nocturnal hemoglobinuria: Interim data from a phase 2 open-label proof-of-concept study. Presented at: 2022 American Society of Hematology Annual Meeting; December 10-13, 2022. Abstract 294.
  15. Risitano AM, Kulasekaraj AG, Lee JW, et al. Danicopan: an oral complement factor D inhibitor for paroxysmal nocturnal hemoglobinuria. Haematologica. 2021;106:3188-3197.
  16. Kulasekararaj AG, Risitano AM, Maciejewski JP, et al. Phase 2 study of danicopan in patients with paroxysmal nocturnal hemoglobinuria with an inadequate response to eculizumab. Blood. 2021;138:1928-1938.
  17. Storb R, Deeg HJ, Farewell V, et al. Marrow transplantation for severe aplastic anemia: methotrexate alone compared with a combination of methotrexate and cyclosporine for prevention of acute graft-versus-host disease. Blood. 1986;68:119-125.
  18. Storb R, Deeg HJ, Whitehead J, et al. Methotrexate and cyclosporine compared with cyclosporine alone for prophylaxis of acute graft versus host disease after marrow transplantation for leukemia. New Engl J Med. 1986;314:729735.
  19. Ratanatharathorn V, Nash RA, Przepiorka D, et al. Phase III study comparing methotrexate and tacrolimus (Prograf, FK506) with methotrexate and cyclosporine for graft-versus-host disease prophylaxis after HLA-identical sibling bone marrow transplantation. Blood. 1998;92:2303-2314.
  20. Holtan SG, Hamadani M, Wu J, et al. Post-transplant cyclophosphamide, tacrolimus, and mycophenolate mofetil as the new standard for graft-versus-host disease (GVHD) prophylaxis in reduced intensity conditioning: results from phase III BMT CTN 1703. Presented at: 2022 American Society of Hematology Annual Meeting; December 10-13, 2022. Abstract LBA-4.
  21. Bolanños-Meade J, Reshef R, Fraser R, et al. Lancet Haematol. Three prophylaxis regimens (tacrolimus, mycophenolate mofetil, and cyclophosphamide; tacrolimus, methotrexate, and bortezomib; or tacrolimus, methotrexate, and maraviroc) versus tacrolimus and methotrexate for prevention of graft-versus-host disease with haemopoietic cell transplantation with reduced-intensity conditioning: a randomised phase 2 trial with a non-randomised contemporaneous control group (BMT CTN 1203). Lancet Haematol. 2019;6:e132-e143.
  22. Gatza E, Reddy P, and Choi SW. Prevention and treatment of acute graft-versus-host disease in children, adolescents, and young adults. Biol Blood Marrow Transplant. 2020;26:e101-e112.
  23. Martin PJ, Rizzo JD, Wingard JR, et al. First- and second-line systemic treatment of acute graft-versus-host disease: recommendations of the American Society of Blood and Marrow Transplantation. Biol Blood Marrow Transplant. 2012;18:1150-1163.
  24. Zeiser R, von Bubnoff N, Butler J, et al. Ruxolitinib for glucocorticoid-refractory acute graft-versus-host disease. N Engl J Med. 2020;382:1800-1810.
  25. Ruxolitinib [prescribing information]. Wilmington, DE: Incyte; 2021.
  26. Locatelli F, Kang HJ, Bruno B, et al. Ruxolitinib in pediatric patients with treatment-naïve or steroid-refractory acute graft-versus-host disease: primary findings from the phase I/II REACH4 study. Presented at: 2022 American Society of Hematology Annual Meeting; December 10-13, 2022. Abstract 572.
  27. Bhatt VR, Shostrum VK, Saad A, et al. Ruxolitinib for treatment of steroid refractory sclerotic chronic graft-versus-host disease (cGVHD): results of a multicenter phase II trial. Presented at: 2022 American Society of Hematology Annual Meeting; December 10-13, 2022. Abstract 573.
  28. Dou L, Zhao Y, Deng L, et al. Addition of ruxolitinb to corticosteroids as first-line therapy for high-risk acute graft versus host disease in allogeneic peripheral blood stem cell transplantation. Presented at: 2022 American Society of Hematology Annual Meeting; December 10-13, 2022. Abstract 574.
  29. Im A, Wolff D, Cutler C, et al. Itacitinib and corticosteroids as initial treatment for chronic graft-versus-host disease: phase 1/2 results from Gravitas-309. Presented at: 2022 American Society of Hematology Annual Meeting; December 10-13, 2022. Abstract 771.
  30. Etra AM, Capellini A, Alousi AM, et al. Effective treatment of low risk acute GVHD with itacitinib monotherapy. Presented at: 2022 American Society of Hematology Annual Meeting; December 10-13, 2022. Abstract 770.
  31. Hill Q and Newland AC. Fatigue in immune thrombocytopenia. Br J Haematol. 2015;170:141-149.
  32. Zufferey A, Kapur R, and Semple JW. Pathogenesis and therapeutic mechanism in immune thrombocytopenia (ITP). J Clin Med. 2017;6:16.
  33. Kashiwagi H, Tomiyama Y. Pathophysiology and management of primary immune thrombocytopenia. Int J Hematol. 2013;98:24-33.
  34. Swinkels M, Rijkers M, Voorberg J, et al. Emerging concepts in immune thrombocytopenia. Front Immunol. 2018;30:880.
  35. van Leeuwen EF, van der Ven JT, Engelfriet CP, et al Specificity of autoantibodies in autoimmune thrombocytopenia. Blood. 1982;59:23.
  36. McMillan R, Tani P, Millard F, et al. Platelet-associated and plasma anti-glycoprotein autoantibodies in chronic ITP. Blood. 1987;70:1040-1045.
  37. He R, Reid DM, Jones Cem, et al. Spectrum of Ig classes, specificitiesm, and titers of serum antiglycoproteins in chronic idiopathic thrombocytopenic purpura. Blood. 1994;83:1024-1032.
  38. 38. Efgartigimod [prescribing information]. Boston, MA: Argenx; 2022.
  39. 39. O’Hara S, Castro FA, Black J, et al. Disease burden and remaining unmet need in patients with haemophilia A treated with primary prophylaxis. Haemophilia. 2021;27:113-119.
  40. Malec LM, Cheng D, Witmer CM, et al. The impact of extended half-life factor concentrates on prophylaxis for severe hemophilia in the United States. Am J Hematology. 2020;95:960-965.
  41. von Drygalski A, Chowdary P, Kulkarni R, et al. Efficacy, safety, and pharmacokinetics of once-weekly efanesoctocog alfa (BIVV001) prophylaxis in previously treated patients with severe hemophilia A: results from the phase 3 XTEND-1 study. Presented at: 2022 International Society on Thrombosis and Haemostasis; July 9-13, 2022. Abstract LB 01.4.
  42. Wilson A, Kragh N, Dumont J, et al. Efficacy of efanesoctocog alfa on pain in patients with hemophilia A: results from the XTEND-1 phase 3 clinical trial in previously treated patients with hemophilia A. Presented at: 2022 American Society of Hematology Annual Meeting; December 10-13, 2022. Abstract 2474.
  43. Srivastava A, Santagostino E, Dougall A, et al. WFH guidelines for the management of hemophilia, 3rd edition. Haemophilia. 2020;26:1-158.
  44. Warrier I, Ewenstein BM, Koerper MA, et al. Factor IX inhibitors and anaphylaxis in hemophilia B. J Pediatric Hematol Oncol. 1997;19:23-27.
  45. Witmer C and Young G. Factor VIII inhibitors in hemophilia A: rationale and latest evidence. Ther Adv Hematol. 2013 ;4:59-72.
  46. Pasi KJ, Lissitchkov T, Mamonov V, et al. Targeting of antithrombin in hemophilia A or B with investigational siRNA therapeutic fitusiran-Results of the phase 1 inhibitor cohort. J Thromb Haemost. 2021;19:1436-1446.
  47. Kenet G, Nolan B, Matsushita T, et al. Fitusiran prophylaxis improves health-related quality of life in people with hemophilia A or B, with or without inhibitors: Results of ATLAS-PPX study. Presented at: 2022 American Society of Hematology Annual Meeting; December 10-13, 2022. Abstract 3559.