Comparison of Haploidentical-allogeneic Hematopoietic Stem Cell Transplantation and Intensive Immunosuppressive Therapy for Patients with Severe Aplastic Anemia with an Absolute Neutrophil Count of Zero: a Retrospective Study

Overview

Journal Ann Hematol

Specialty Hematology

Date 2023 May 16

PMID 37193759

Authors

Li-Qiang Wu

Li-Fang Huang

Hui Yang

Bao-Dong Ye

Jian-Ping Sheng

Qing-Hong Yu

Yan Yang

Jin-Song Jia

Dong-Hua Zhang

Sheng-Yun Lin

Guang-Sheng He

Jian-Yong Li

Affiliations

Soon will be listed here.

Abstract

A retrospective analysis was conducted based on the clinical data from 60 patients older than 16 years from January 2016 to January 2021. All the patients were newly diagnosed with severe aplastic anemia (SAA) with an absolute neutrophil count (ANC) of zero. We compared the hematological response and survival of haploidentical-allogeneic hematopoietic stem cell transplantation (HID-HSCT) (n = 25) and intensive immunosuppressive therapy (IST) (n = 35) treatments. At six months, the overall response rate and complete response were significantly higher in the HID-HSCT group than those in the IST group (84.0% vs. 40.0%, P = 0.001; 80.0% vs. 17.1%, P = 0.001). With a median follow-up of 18.5 months (4.3~30.8 months), patients in the HID-HSCT group had longer overall survival and event-free survival (80.0% vs. 47.9%, P = 0.0419; 79.2% vs. 33.5%, P = 0.0048). These data suggested that HID-HSCT might be an effective alternative treatment option for adult patients with SAA with an ANC of zero, which requires further validation in an additional prospective study.

Citing Articles

Significance of absolute neutrophil count before allogeneic hematopoietic stem cell transplantation in adult patients with aplastic anemia.

Nakamura Y, Zaimoku Y, Yamaguchi H, Yamazaki H, Kanaya M, Uchida N Ann Hematol. 2024; 103(8):3121-3133.

PMID: 38750374 DOI: 10.1007/s00277-024-05800-1.

References

Killick S, Bown N, Cavenagh J, Dokal I, Foukaneli T, Hill A . Guidelines for the diagnosis and management of adult aplastic anaemia. Br J Haematol. 2015; 172(2):187-207. DOI: 10.1111/bjh.13853. View

Young N . Aplastic Anemia. N Engl J Med. 2018; 379(17):1643-1656. PMC: 6467577. DOI: 10.1056/NEJMra1413485. View

Bacigalupo A . How I treat acquired aplastic anemia. Blood. 2017; 129(11):1428-1436. DOI: 10.1182/blood-2016-08-693481. View

Kojima S, Hibi S, Kosaka Y, Yamamoto M, Tsuchida M, Mugishima H . Immunosuppressive therapy using antithymocyte globulin, cyclosporine, and danazol with or without human granulocyte colony-stimulating factor in children with acquired aplastic anemia. Blood. 2000; 96(6):2049-54. View

Rosenfeld S, Follmann D, Nunez O, Young N . Antithymocyte globulin and cyclosporine for severe aplastic anemia: association between hematologic response and long-term outcome. JAMA. 2003; 289(9):1130-5. DOI: 10.1001/jama.289.9.1130. View

Yoshizato T, Dumitriu B, Hosokawa K, Makishima H, Yoshida K, Townsley D . Somatic Mutations and Clonal Hematopoiesis in Aplastic Anemia. N Engl J Med. 2015; 373(1):35-47. PMC: 7478337. DOI: 10.1056/NEJMoa1414799. View

Kulasekararaj A, Jiang J, Smith A, Mohamedali A, Mian S, Gandhi S . Somatic mutations identify a subgroup of aplastic anemia patients who progress to myelodysplastic syndrome. Blood. 2014; 124(17):2698-704. PMC: 4383793. DOI: 10.1182/blood-2014-05-574889. View

Bejanyan N, Kim S, Hebert K, Kekre N, Abdel-Azim H, Ahmed I . Choice of conditioning regimens for bone marrow transplantation in severe aplastic anemia. Blood Adv. 2019; 3(20):3123-3131. PMC: 6849938. DOI: 10.1182/bloodadvances.2019000722. View

Yang D, Yang J, Hu X, Chen J, Gao L, Cheng H . Aplastic Anemia Preconditioned with Fludarabine, Cyclophosphamide, and Anti-Thymocyte Globulin. Ann Transplant. 2019; 24:461-471. PMC: 6705177. DOI: 10.12659/AOT.915696. View

10.

Kekre N, Zhang Y, Zhang M, Carreras J, Ahmed P, Anderlini P . Effect of antithymocyte globulin source on outcomes of bone marrow transplantation for severe aplastic anemia. Haematologica. 2017; 102(7):1291-1298. PMC: 5566045. DOI: 10.3324/haematol.2017.164459. View

11.

Zhang Y, Wu L, Mo W, Zhou M, Li Y, Chen X . Comparable Outcomes of First-Line Hematopoietic Stem Cell Transplantation from Unrelated and Matched Sibling Donors in Adult Patients with Aplastic Anemia: A Retrospective Single-Center Study. Biol Blood Marrow Transplant. 2019; 25(8):1567-1575. DOI: 10.1016/j.bbmt.2019.03.020. View

12.

Liu L, Zhang Y, Jiao W, Zhou H, Wang Q, Jin S . Comparison of efficacy and health-related quality of life of first-line haploidentical hematopoietic stem cell transplantation with unrelated cord blood infusion and first-line immunosuppressive therapy for acquired severe aplastic anemia. Leukemia. 2020; 34(12):3359-3369. DOI: 10.1038/s41375-020-0933-7. View

13.

Esteves I, Bonfim C, Pasquini R, Funke V, Pereira N, Rocha V . Haploidentical BMT and post-transplant Cy for severe aplastic anemia: a multicenter retrospective study. Bone Marrow Transplant. 2015; 50(5):685-9. DOI: 10.1038/bmt.2015.20. View

14.

Xu L, Wang S, Wu D, Wang J, Gao S, Jiang M . Haplo-identical transplantation for acquired severe aplastic anaemia in a multicentre prospective study. Br J Haematol. 2016; 175(2):265-274. DOI: 10.1111/bjh.14225. View

15.

Xu L, Jin S, Wang S, Xia L, Bai H, Gao S . Upfront haploidentical transplant for acquired severe aplastic anemia: registry-based comparison with matched related transplant. J Hematol Oncol. 2017; 10(1):25. PMC: 5251320. DOI: 10.1186/s13045-017-0398-y. View

16.

Bacigalupo A, Hows J, Gluckman E, Nissen C, Marsh J, Van Lint M . Bone marrow transplantation (BMT) versus immunosuppression for the treatment of severe aplastic anaemia (SAA): a report of the EBMT SAA working party. Br J Haematol. 1988; 70(2):177-82. DOI: 10.1111/j.1365-2141.1988.tb02460.x. View

17.

Xu L, Wang S, Ma Y, Gao S, Cheng Y, Zhang Y . Who is the best haploidentical donor for acquired severe aplastic anemia? Experience from a multicenter study. J Hematol Oncol. 2019; 12(1):87. PMC: 6719398. DOI: 10.1186/s13045-019-0775-9. View

18.

Yagasaki H, Shichino H, Ohara A, Kobayashi R, Yabe H, Ohga S . Immunosuppressive therapy with horse anti-thymocyte globulin and cyclosporine as treatment for fulminant aplastic anemia in children. Ann Hematol. 2013; 93(5):747-52. DOI: 10.1007/s00277-013-1984-x. View

19.

Liu J, Lu X, Cheng L, Yang Y, Lin S, Yin H . Clinical outcomes of immunosuppressive therapy for severe aplastic anemia patients with absolute neutrophil count of zero. Hematology. 2019; 24(1):492-497. DOI: 10.1080/16078454.2019.1631424. View

20.

Peffault de Latour R, Tabrizi R, Marcais A, Leblanc T, Lamy T, Mohty M . Nationwide survey on the use of horse antithymocyte globulins (ATGAM) in patients with acquired aplastic anemia: A report on behalf of the French Reference Center for Aplastic Anemia. Am J Hematol. 2018; 93(5):635-642. DOI: 10.1002/ajh.25050. View