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Date Published: 19/12/2024
Abstract Views: 65
Views PDF: 17
DOI: 10.52322/jocmbmh.143.0201
Issue
Vol. 2 No. 143 (2024)
Section
Review papers
How to Cite
Hoang Thi, H., Pham Quang, V., Vu Minh, P., & Duong Thi Thuy, L. (2024). ACHIEVEMENTS AND CHALLENGES IN THE DIAGNOSIS AND TREATMENT OF APLASTIC ANEMIA. Journal of Clinical Medicine Bach Mai Hospital, 2(143), 3-10. https://doi.org/10.52322/jocmbmh.143.0201

ACHIEVEMENTS AND CHALLENGES IN THE DIAGNOSIS AND TREATMENT OF APLASTIC ANEMIA

Hue Hoang Thi1, , Vinh Pham Quang2, Phuong Vu Minh1, Linh Duong Thi Thuy
1 Trường Đại học Y Hà Nội
2 Bach Mai Hospital

Author contact

  • Contact information: Hue Hoang Thi
  • Email: hoanghuehhtm@gmail.com
  • Address: Trường Đại học Y Hà Nội

Main Article Content

Abstract

Idiopathic aplastic anemia is a condition characterized by decreased bone marrow production due to an immunological mechanism involving activated autoreactive T lymphocytes attacking hematopoietic stem cells. The definitive diagnosis of idiopathic aplastic anemia (IAA) lacks a gold standard, thus presenting significant challenges in clinical diagnosis. The two primary treatment modalities comprise allogeneic stem cell transplantation and immunosuppressive therapy. The selection of treatment regimen depends on factors such as age, comorbidities, and the availability of stem cell sources. Treatment outcomes for IAA have improved through immunosuppressive therapy utilizing a three-drug regimen consisting of hATG (horse anti-thymocyte globulin), CSA (Cyclosporin A), and eltrombopag (revolade). However, a proportion of patients still experience nonresponse, relapse, or clonal evolution. Allogeneic hematopoietic stem cell transplantation from HLA (human leukocyte antigen)-matched related donors remains the most effective treatment modality. Nevertheless, only approximately 25% of patients have the possibility of finding a suitable stem cell donor. Alternative transplantation methods, such as haploidentical transplantation and umbilical cord blood transplantation, coupled with advancements in conditioning regimens and graft-versushost disease prophylaxis, are yielding promising results for patients with aplastic anemia.

Keywords

Aplastic anemia, Diagnosis and treatment, Medical advancements, Clinical challenges, Hematology

Article Details

References

1. Hartung HD, Olson TS, Bessler M. Acquired aplastic anemia in children. Pediatr Clin North Am. 2013;60(6):1311-1336.
2. Scheinberg P, Cooper JN, Sloand EM, Wu CO, Calado RT, Young NS. Association of telomere length of peripheral blood leukocytes with hematopoietic relapse, malignant transformation, and survival in severe aplastic anemia. JAMA. 2010;304:1358-1364.
3. Fattizzo B, Ireland R, Dunlop A, et al. Clinical and prognostic significance of small paroxysmal nocturnal hemoglobinuria clones in myelodysplastic syndrome and aplastic anemia. Leukemia. 2021;35:3223-3231.
4. Gurnari C, Pagliuca S, Prata PH, et al. Clinical and molecular determinants of clonal evolution in aplastic anemia and paroxysmal nocturnal hemoglobinuria. J Clin Oncol. 2023;41:132-142.
5. Gupta V, Brooker C, Tooze JA, et al. Clinical relevance of cytogenetic abnormalities at diagnosis of acquired aplastic anaemia in adults. Br J Haematol. 2006;134:95-99.
6. Hoffbrand AV, Higgs DR, Keeling DM, Mehta AB. Postgraduate Haematology. 7th ed. USA: John Wiley & Sons Ltd; 2016.
7. Piekarska A, Pyziak K, Szmigielska-Kapłon A, Matysiak M. The state of the art in the treatment of severe aplastic anemia: immunotherapy and hematopoietic cell transplantation in children and adults. Front Immunol. 2024;15:1378432.
8. Scheinberg P, Nunez O, Weinstein B. Horse versus rabbit antithymocyte globulin in acquired aplastic anemia. N Engl J Med. 2011;365:430-438.
9. Yoshimi A, Niemeyer CM, Fuhrer MM, Strahm B. Comparison of the efficacy of rabbit and horse antithymocyte globulin for the treatment of severe aplastic anemia in children. Blood. 2013;121:860.
10. Yang N, Chen J, Zhang H, et al. Horse versus rabbit antithymocyte globulin in immunosuppressive therapy of treatment-naive aplastic anemia: a systematic review and meta-analysis. Ann Hematol. 2017;96:2031-2043.
11. Desmond R, Townsley DM, Dumitriu B, Olnes MJ. Eltrombopag restores trilineage hematopoiesis in refractory severe aplastic anemia that can be sustained on discontinuation of drug. Blood. 2014;123:1818-1825.
12. Peffault de Latour R, Kulasekararaj A, Iacobelli S, et al. Eltrombopag added to immunosuppression in severe aplastic anemia. N Engl J Med. 2022;386:11-23.
13. Jang JH, Townsley Y, Miyazaki K, Nagafuji K. Efficacy and safety of romiplostim in refractory aplastic anaemia. Br J Haematol. 2021;192:190-199.
14. Chaudhry QUN, Iftikhar R, Satti TM, et al. Outcome of fludarabine-based conditioning in high-risk aplastic anemia patients undergoing matched related donor transplantation: A single-center study from Pakistan. Biol Blood Marrow Transplant. 2019;25:2375-2383.
15. DeZern AE, Zahurak M, Symons HJ, et al. Alternative donor BMT with posttransplant cyclophosphamide as initial therapy for acquired severe aplastic anemia. Blood. 2023;141:3031-3038.
16. Gong S, Chen C, Chen K, et al. Alternative transplantation with post-transplantation cyclophosphamide in aplastic anemia: A retrospective report from the BMF-WG of Hunan province, China. Transplant Cell Ther. 2023;29:48.
17. Bacigalupo A. How I treat acquired aplastic anemia. Blood. 2017;129:1428-1436.
18. Onishi Y, Matsuoka T, Yamazaki H, et al. Comparison of haploidentical stem cell transplantation with post-transplantation cyclophosphamide versus umbilical cord blood transplantation in adult patients with aplastic anemia. Transplant Cell Ther. 2023;9(9):660-669.
19. DeZern AE, Zahurak M, Symons HJ, et al. Alternative donor BMT with posttransplant cyclophosphamide as initial therapy for acquired severe aplastic anemia. Blood. 2023;141:3031-3038.