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CME

Key Studies in Nonmalignant Hematology: Independent Conference Coverage of ASH 2020

Physicians: Maximum of 1.00 AMA PRA Category 1 Credit™

Released: February 11, 2021

Expiration: February 10, 2022

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  1. Bradbury CA, Greenwood R, Pell J, et al, Multicenter randomized trial of first-line treatment pathways for newly diagnosed immune thrombocytopenia: standard steroid treatment versus combined steroid and mycophenolate. The FLIGHT trial. Program and abstracts of the 2020 American Society of Hematology Annual Meeting; December 5-8, 2020. Abstract LBA-2.
  2. Neunert C, Terrell DR, Arnold DM, et al. American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv. 2019;3:3829-3866.
  3. Panigrahi A, Clark A, Myers J, et al. A novel immunomodulatory treatment involving mycophenolate mofetil and corticosteroids for pediatric autoimmune cytopenias. Pediatr Blood Cancer. 2017;64:287-293.
  4. Colović M, Suvajdzic N, Colović N, et al. Mycophenolate mofetil therapy for chronic immune thrombocytopenic purpura resistant to steroids, immunosuppressants, and/or splenectomy in adults. Platelets. 2011;22:153-156.
  5. Smith PF, Krishnarajah J, Nunn PA, et al. A phase I trial of PRN1008, a novel reversible covalent inhibitor of Bruton's tyrosine kinase, in healthy volunteers. Br J Clin Pharmacol. 2017;83:2367
  6. Langrish C, Francesco M, Xing Y, et al. Rilzabrutinib (PRN1008) shows BTK-mediated mechanisms of action supporting clinical development for immune-mediated diseases. SID Annual Meeting Virtual Conference; May13-16, 2020. Abstract 569.
  7. Caron F, Leong DP, Hillis C, et al. Current understanding of bleeding with ibrutinib use: a systematic review and meta-analysis. Blood Adv. 2017;772-778.
  8. Langrish CL, Bradshaw JM, Owens TD, et al. PRN1008, a reversible covalent BTK inhibitor in clinical development for immune thrombocytopenic purpura. Program and abstracts of the 2017 American Society of Hematology Annual Meeting; December 9-12, 2017; Atlanta, Georgia. Abstract 1052.
  9. Kuter DJ, Efraim M, Mayer J, et al. Oral rilzabrutinib, a Bruton’s tyrosine kinase inhibitor, shows clinically active and durable platelet responses and was well-tolerated in patients with heavily pretreated immune thrombocytopenia. Program and abstracts of the 2020 American Society of Hematology Annual Meeting; December 5-8, 2020. Abstract 22.
  10. ClinicalTrials.gov. Ph3, randomized, double-blind, placebo-controlled, parallel-group study with open-label extension to evaluate the efficacy and safety of oral rilzabrutinib in adults and adolescents with persistent or chronic immune thrombocytopenia (ITP). Available at: https://clinicaltrials.gov/ct2/show/NCT04562766. Accessed February 4, 2021.
  11. Broome CM, Röth A, Kuter D, et al. Inhibition of the classical pathway of complement with sutimlimab in patients with chronic immune thrombocytopenia without adequate response to two or more prior therapies. Program and abstracts of the 2019 American Society of Hematology Annual Meeting; December 7-10, 2019. Abstract 898.
  12. Newland A, Lilebman HA, McDonald V, et al. Efficacy and safety of intravenous efgartigimod 10 mg/kg in adult patients with primary immune thrombocytopenia: advance, a phase 3 clinical trial in progress. Program and abstracts of the 2020 American Society of Hematology Annual Meeting; December 5-8, 2020. Abstract 1757.
  13. Pipe SW, Recht M, Key NS, et al. First data from the phase III HOPE-B gene therapy trial: efficacy and safety of etranacogene dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in adults with severe or moderate-severe hemophilia B treated irrespective of preexisting anti-capsid neutralizing antibodies. Program and abstracts of the 2020 American Society of Hematology Annual Meeting; December 5-8, 2020. Abstract LBA-6.
  14. Miesbach W, Meijer K, Coppens M, et al. Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B. Blood. 2018;131:1022-1031.
  15. Von Drygalski A, Giermasz A, Castaman G, et al. Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B. Blood Adv. 2019;3:3241-3247.
  16. Von Drygalski A, Giermasz A, Castaman G, et al. Etranacogene dezaparvovec (AAV5-Padua hFIX variant), an enhanced vector for gene transfer in adults with severe or moderate-severe hemophilia B: two-year data from a phase IIb trial. Program and abstract .of the 2020 American Society of Hematology Annual Meeting; December 5-8, 2020. Abstract 672.
  17. Frangoul H, Bobruff Y, Cappellini MD, et al. Safety and efficacy of CTX001 in patients with transfusion-dependent β-thalassemia and sickle cell disease: early results from the Climb THAL-111 and Climb SCD-121 studies of autologous CRISPR-CAS9-modified CD34+ hematopoietic stem and progenitor cells. Program and abstracts of the 2020 American Society of Hematology Annual Meeting; December 5-8, 2020. Abstract 4.
  18. Frangoul H, Altshuler D, Cappellini MD, et al. CRISPR-Cas9 gene editing for sickle cell disease and β-thalassemia. N Engl J Med. 2021;384:252-260.
  19. Thompson A, Walters M, Kwiatkowski J, et al. Gene therapy in patients with transfusion-dependent β-thalassemia. N Engl J Med. 2018;378:1479-1493.
  20. Ribeil JA, Hacein-Bey-Abina S, Payen E, et al. Gene therapy in patients with sickle cell disease. N Engl J Med. 2017;376:848-855.
  21. Steinberg MH, Chui DHK, Dover GJ, et al. Fetal hemoglobin in sickle cell anemia: a glass half full? Blood. 2014;123:481-485.
  22. ClinicalTrials.gov. A long-term follow-up study of subjects with β-thalassemia or sickle cell disease treated with autologous CRISPR-Cas9 modified hematopoietic stem cells (CTX001). Available at: https://clinicaltrials.gov/ct2/show/NCT04208529. Accessed February 4, 2021.
  23. Xu JZ, Conrey A, Frey I, et al. Phase I multiple ascending dose study of safety, tolerability, and pharmacokinetics/pharmacodynamics of mitapivat (AG-348) in subjects with sickle cell disease. Program and abstracts of the 2020 American Society of Hematology Annual Meeting; December 5-8, 2020. Abstract 681.
  24. Grace RF, Rose C, Layton DM, et al. Safety and efficacy of mitapivat in pyruvate kinase deficiency. N Engl J Med. 2019;381:933.
  25. Kuo K, Layton DM, Lai A, et al. Proof of concept for the oral pyruvate kinase activator mitapivat in adults with non–transfusion-dependent thalassemia: interim results from an ongoing, phase 2, open-label, multicenter study. Program and abstracts of the 2020 European Hematology Association Annual Meeting; June 11-21, 2020. Abstract S297.
  26. ClinicalTrials.gov. Evaluation of the safety, tolerability, pharmacokinetics, and pharmacodynamics of long-term mitapivat dosing in subjects with stable sickle cell disease: An extension of a phase I pilot study of mitapivat. Available at: https://clinicaltrials.gov/ct2/show/NCT04610866. Accessed February 4, 2021.
  27. Cappellini MD, Taher AT, Piga A, et al. Health-related quality of life outcomes for patients with transfusion-dependent β-thalassemia treated with luspatercept in the BELIEVE trial. Program and abstract of the 2020 American Society of Hematology Annual Meeting; December 5-8, 2020. Abstract 364.
  28. Ruxolitinib tablets for oral use [package insert]. Wilmington, DE. Incyte Corporation; 2019.
  29. Arora M, Cutler CS, Jagasia MH, et al. Late acute and chronic graft-versus-host disease after allogeneic hematopoietic cell transplantation. Biol Blood Marrow Transplant. 2016;22:449-455.
  30. Axt L, Naumann A, Toennies J, et al. Retrospective single center analysis of outcome, risk factors and therapy in steroid refractory graft-versus-host disease after allogeneic hematopoietic cell transplantation. Bone Marrow Transplant. 2019;54:1805-1814.
  31. Jaglowski S, Devine S. Graft-versus-host disease: why have we not made more progress? Curr Opin Hematol. 2014;21:141-147.
  32. Ibrutinib capsules for oral use [package insert]. Horsham, PA. Janssen Biotech. 2020.
  33. Waller EK, Miklos D, Cutler C, et al. Ibrutinib for chronic graft-versus-host disease after failure of prior therapy: 1-year update of a phase 1b/2 study. Biol Blood Marrow Transplant. 2019;25:2002-2007.
  34. Zeiser R, Polverelli N, Ram R, et al. Ruxolitinib (RUX) vs best available therapy (BAT) in patients with steroid refractory/steroid-dependent chronic graft-vs-host disease (cGVHD): primary findings from the phase 3, randomized REACH3 study. Program and abstract of the 2020 American Society of Hematology Annual Meeting; December 5-8, 2020. Abstract 77.
  35. ClinicalTrials.gov. A study of ruxolitinib vs best available therapy (BAT) in patients with steroid-refractory chronic graft vs. host disease (GVHD) after bone marrow transplantation (REACH3). Available at: https://clinicaltrials.gov/ct2/show/NCT03112603. Accessed February 4, 2021.
  36. Holtzman N, Im A, Ostojic A, et al. Efficacy and safety of baricitinib in refractory chronic graft-vs-host disease (cGVHD): preliminary analysis results of a phase I/II study. Program and abstract of the 2020 American Society of Hematology Annual Meeting; December 5-8, 2020. Abstract 357.
  37. Zanin-Zhorov A, Weis JM, Nyuydzefe MS, et al. Selective oral ROCK2 inhibitor down-regulates IL-21 and IL-17 secretion in human T cells via STAT3-dependent mechanism. Proc Natl Acad Sci USA. 2014;111:16814-16819.
  38. Riches DWH, Backos DS, and Redente EF. ROCK and Rho: promising therapeutic targets to ameliorate pulmonary fibrosis. Am J Pathol. 2015; 185:909-912.
  39. Cutler C, Lee SJ, Arai S, et al. Belumosudil for chronic graft-vs-host disease (cGVHD) after 2 or more prior lines of therapy: the ROCKstar study (KD025-213). Program and abstracts of the 2020 American Society of Hematology Annual Meeting; December 5-8, 2020. Abstract 353.
  40. Pidala J, Walton K, Elmariah H, et al. Biological and clinical impact of JAK2/mTOR blockade in GVHD prevention: preclinical and phase I trial results. Program and abstracts of the 2020 American Society of Hematology Annual Meeting; December 5-8, 2020. Abstract 355.
  41. Choe H, Shah NN, Chevallier P, et al. A single-arm, open-label, phase I study of itacitinib (ITA) with calcineurin inhibitor (CNI)-based interventions for prophylaxis of graft-vs-host disease (GVHD). Program and abstracts of the 2020 American Society of Hematology Annual Meeting; December 5-8, 2020. Abstract 356.

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