Skip to main content
Top
Published in: Annals of Hematology 8/2023

17-05-2023 | Aplastic Anemia | Review Article

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

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

Published in: Annals of Hematology | Issue 8/2023

Login to get access

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.
Literature
4.
go back to reference Kojima S, Hibi S, Kosaka Y, Yamamoto M, Tsuchida M, Mugishima H et al (2000) Immunosuppressive therapy using antithymocyte globulin, cyclosporine, and danazol with or without human granulocyte colony-stimulating factor in children with acquired aplastic anemia. Blood 96(6):2049–2054 e-pub ahead of print 2000/09/09CrossRefPubMed Kojima S, Hibi S, Kosaka Y, Yamamoto M, Tsuchida M, Mugishima H et al (2000) Immunosuppressive therapy using antithymocyte globulin, cyclosporine, and danazol with or without human granulocyte colony-stimulating factor in children with acquired aplastic anemia. Blood 96(6):2049–2054 e-pub ahead of print 2000/09/09CrossRefPubMed
11.
go back to reference Zhang Y, Wu L, Mo W, Zhou M, Li Y, Chen X et al (2019) 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 25(8):1567–1575CrossRefPubMed Zhang Y, Wu L, Mo W, Zhou M, Li Y, Chen X et al (2019) 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 25(8):1567–1575CrossRefPubMed
12.
go back to reference Liu L, Zhang Y, Jiao W, Zhou H, Wang Q, Jin S et al (2020) 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 34(12):3359–3369CrossRefPubMed Liu L, Zhang Y, Jiao W, Zhou H, Wang Q, Jin S et al (2020) 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 34(12):3359–3369CrossRefPubMed
13.
go back to reference Esteves I, Bonfim C, Pasquini R, Funke V, Pereira NF, Rocha V et al (2015) Haploidentical BMT and post-transplant Cy for severe aplastic anemia: a multicenter retrospective study. Bone Marrow Transplant 50(5):685–689CrossRefPubMed Esteves I, Bonfim C, Pasquini R, Funke V, Pereira NF, Rocha V et al (2015) Haploidentical BMT and post-transplant Cy for severe aplastic anemia: a multicenter retrospective study. Bone Marrow Transplant 50(5):685–689CrossRefPubMed
20.
go back to reference Peffault de Latour R, Tabrizi R, Marcais A, Leblanc T, Lamy T, Mohty M et al (2018) 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 93(5):635–642. e-pub ahead of print 2018/01/30. https://doi.org/10.1002/ajh.25050CrossRefPubMed Peffault de Latour R, Tabrizi R, Marcais A, Leblanc T, Lamy T, Mohty M et al (2018) 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 93(5):635–642. e-pub ahead of print 2018/01/30. https://​doi.​org/​10.​1002/​ajh.​25050CrossRefPubMed
21.
go back to reference ElGohary G, El Fakih R, de Latour R, Risitano A, Marsh J, Schrezenmeier H et al (2020) Haploidentical hematopoietic stem cell transplantation in aplastic anemia: a systematic review and meta-analysis of clinical outcome on behalf of the severe aplastic anemia working party of the European group for blood and marrow transplantation (SAAWP of EBMT). Bone Marrow Transplant 55(10):1906–1917. e-pub ahead of print 2020/04/30. https://doi.org/10.1038/s41409-020-0897-2CrossRefPubMed ElGohary G, El Fakih R, de Latour R, Risitano A, Marsh J, Schrezenmeier H et al (2020) Haploidentical hematopoietic stem cell transplantation in aplastic anemia: a systematic review and meta-analysis of clinical outcome on behalf of the severe aplastic anemia working party of the European group for blood and marrow transplantation (SAAWP of EBMT). Bone Marrow Transplant 55(10):1906–1917. e-pub ahead of print 2020/04/30. https://​doi.​org/​10.​1038/​s41409-020-0897-2CrossRefPubMed
Metadata
Title
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
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
Publication date
17-05-2023
Publisher
Springer Berlin Heidelberg
Published in
Annals of Hematology / Issue 8/2023
Print ISSN: 0939-5555
Electronic ISSN: 1432-0584
DOI
https://doi.org/10.1007/s00277-023-05256-9

Other articles of this Issue 8/2023

Annals of Hematology 8/2023 Go to the issue
Live Webinar | 27-06-2024 | 18:00 (CEST)

Keynote webinar | Spotlight on medication adherence

Live: Thursday 27th June 2024, 18:00-19:30 (CEST)

WHO estimates that half of all patients worldwide are non-adherent to their prescribed medication. The consequences of poor adherence can be catastrophic, on both the individual and population level.

Join our expert panel to discover why you need to understand the drivers of non-adherence in your patients, and how you can optimize medication adherence in your clinics to drastically improve patient outcomes.

Prof. Kevin Dolgin
Prof. Florian Limbourg
Prof. Anoop Chauhan
Developed by: Springer Medicine
Obesity Clinical Trial Summary

At a glance: The STEP trials

A round-up of the STEP phase 3 clinical trials evaluating semaglutide for weight loss in people with overweight or obesity.

Developed by: Springer Medicine