» Articles » PMID: 38993743

Acquired Aplastic Anemia Therapies: Immunosuppressive Therapy Versus Alternative Donor Hematopoietic Cell Transplantation

Overview
Journal J Hematol
Specialty Hematology
Date 2024 Jul 12
PMID 38993743
Authors
Affiliations
Soon will be listed here.
Abstract

Immunosuppressive therapy for acquired severe aplastic anemia improves pancytopenia but has a significant risk of relapse (40%) and clonal evolution to myeloid neoplasms (15%), especially in patients older than 40. Yet, current guidelines for newly diagnosed severe aplastic anemia patients over the age of 40 recommend immunosuppressive therapy instead of curative allogeneic stem cell transplantation. Upfront allogeneic stem cell transplants are restricted to the rare patient who is not only young but also has a matched sibling donor. This article will discuss practice-changing data on the recent advances in upfront alternative donor hematopoietic cell transplants that could rewrite current treatment algorithms.

References
1.
Peffault de Latour R, Kulasekararaj A, Iacobelli S, Terwel S, Cook R, Griffin M . Eltrombopag Added to Immunosuppression in Severe Aplastic Anemia. N Engl J Med. 2022; 386(1):11-23. DOI: 10.1056/NEJMoa2109965. View

2.
Gurnari C, Pagliuca S, Prata P, Galimard J, Catto L, Larcher L . Clinical and Molecular Determinants of Clonal Evolution in Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria. J Clin Oncol. 2022; 41(1):132-142. PMC: 10476808. DOI: 10.1200/JCO.22.00710. View

3.
Gurnari C, Pagliuca S, Maciejewski J . Clonal evolution in aplastic anemia: failed tumor surveillance or maladaptive recovery?. Leuk Lymphoma. 2023; 64(8):1389-1399. PMC: 11104022. DOI: 10.1080/10428194.2023.2215614. View

4.
Mizumaki H, Hosomichi K, Hosokawa K, Yoroidaka T, Imi T, Zaimoku Y . A frequent nonsense mutation in exon 1 across certain HLA-A and -B alleles in leukocytes of patients with acquired aplastic anemia. Haematologica. 2020; 106(6):1581-1590. PMC: 8168509. DOI: 10.3324/haematol.2020.247809. View

5.
Babushok D, Duke J, Xie H, Stanley N, Atienza J, Perdigones N . Somatic HLA Mutations Expose the Role of Class I-Mediated Autoimmunity in Aplastic Anemia and its Clonal Complications. Blood Adv. 2017; 1(22):1900-1910. PMC: 5621748. DOI: 10.1182/bloodadvances.2017010918. View