ASH 2023: Nonmalignant Hematology

CE / CME

Key Studies in Nonmalignant Hematology Disorders: Independent Conference Coverage of ASH 2023

Physician Assistants/Physician Associates: 1.00 AAPA Category 1 CME credit

Nurses: 1.00 Nursing contact hour

Physicians: maximum of 1.00 AMA PRA Category 1 Credit

Pharmacists: 1.00 contact hour (0.1 CEUs)

Released: February 22, 2024

Expiration: February 21, 2025

Catherine M. Broome
Catherine M. Broome, MD

Activity

Progress
1 2
Course Completed

References

  1. McGann PT, Tshilolo L, Santos B, et al. Hydroxyurea therapy for children with sickle cell anemia in sub-Saharan Africa: Rationale and design of the REACH trial. Pediatr Blood Cancer. 2016;63:98-104.
  2. Rees DC, Williams TN, Gladwin MT. Sickle-cell disease. Lancet. 2010;376:2018-2031.
  3. Hydroxyurea [prescribing information]. Bryn Mawr, Pennsylvania: Theravia; 2023.
  4. Aygun B, Lane AC, Smart LR, et al. Hydroxyurea dose optimization is safe and improves outcomes for children with sickle cell anemia living in sub-Saharan Africa: the reach experience. Presented at: 2023 American Society of Hematology Annual Meeting; December 9-12, 2023. Abstract 6.
  5. Kanter J, Walters MC, Krishnamurti L, et al. Biologic and clinical efficacy of lentiglobin for sickle cell disease. N Engl J Med. 2022;386:617-628.
  6. Kanter J, Thompson AA, Kwiatkowski JL, et al. Efficacy, safety, and health-related quality of life (HRQoL) in patients with sickle cell disease (SCD) who have received lovotibeglogene autotemcel (Lovo-cel) gene therapy: up to 60 months of follow-up. Presented at: 2023 American Society of Hematology Annual Meeting; December 9-12, 2023. Abstract Abstr 1051.
  7. Kassim AA, Walters MC, Eapen M, et al. Reduced intensity haploidentical bone marrow transplantation in adults with severe sickle cell disease: BMT CTN 1507. Presented at: 2023 American Society of Hematology Annual Meeting; December 9-12, 2023. Abstract Abstr LBA-4.
  8. Gluckman E, Cappelli B, Bernaudin F, et al. Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation. Blood. 2017;129:1548-1556.
  9. Chaturvedi S, Ghafuri DL, Jordan N, et al. Clustering of end-organ disease and earlier mortality in adults with sickle cell disease: A retrospective-prospective cohort study. Am J Hematol. 2018;93:1153-1160.
  10. DeBaun MR, Ghafuri DL, Rodeghier M, et al. Decreased median survival of adults with sickle cell disease after adjusting for left truncation bias: a pooled analysis. Blood. 2019;133:615-617.
  11. Shah N, Bhatt H. Paroxysmal nocturnal hemoglobinuria. ncbi.nlm.nih.gov/books/NBK562292/. Accessed February 12, 2024.
  12. Risitano AM, Frieri C, Urciuoli E, Marano L. The complement alternative pathway in paroxysmal nocturnal hemoglobinuria: From a pathogenic mechanism to a therapeutic target. Immunol Rev. 2023;313:262-278.
  13. 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.
  14. Fishman J, Kuranz S, Yeh MM, et al. Changes in hematologic lab measures observed in patients with paroxysmal nocturnal hemoglobinuria treated with C5 inhibitors, ravulizumab and eculizumab: Real-world evidence from a US based EMR network. Hematol Rep. 2023;15:266-282.
  15. Schubart A, Anderson K, Mainolfi N, et al. Small-molecule factor B inhibitor for the treatment of complement-mediated diseases. Proc Natl Acad Sci U S A. 2019;116:7926-7931.
  16. Iptacopan [prescribing information]. East Hanover, New Jersey: Novartis; 2023.
  17. Peffault de Latour R, Roeth A, Kulasekararaj 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 facorable 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.
  18. Risitano AM, Kulasekararaj AG, Röth A, et al. Factor B inhibitor with oral iptacopan monotherapy demonstrates sustained long-term efficacy and safety in anti-C5-treated patients (pts) with paroxismal nocturnal hemoglobinuria (PNH) and persistent anemia: final 48-week results from the multicenter, phase III APPLY-PNH trial. Presented at: 2023 American Society of Hematology Annual Meeting; December 9-12, 2023. Abstract 571.
  19. Kulasekararaj AG, Griffin M, Piatek CI, et al. Danicopan as add-on therapy to ravulizumab or eculizumab versus placebo in patients with paroxismlal nocturnal hemoglobiuria and clinically signifiacant extravascular hemolysis: phase 3 long-term data. Presented at: 2023 American Society of Hematology Annual Meeting; December 9-12, 2023. Abstract 576.
  20. 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.
  21. Swiecicki PL, Hegerova LT, Gertz MA. Cold agglutinin disease. Blood. 2013;122:1114-1121.
  22. Roth A, Barcellini W, D'Sa S, et al. Sutimlimab in cold agglutinin disease. N Engl J Med. 2021;384:1323-1334.
  23. Roth 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.
  24. Sutimlimab [prescribing information]. Waltman, Massachussetts: Sanofi; 2023.
  25. Roth A, Barcellini W, D'Sa S, et al. Sustained inhibition of complement C1s with sutimlimab over 2 years in patients with cold agglutinin disease. Am J Hematol. 2023;98:1246-1253.
  26. Broome CM, Barcellini W, Ueda Y, et al. Combined safety data for sutimlimab in cold agglutinin disease: a post-hoc analysis of the phase 3 cardinal and cadenza studies. Presented at: 2023 American Society of Hematology Annual Meeting; December 9-12, 2023. Abstr 3833.