Outpatient subcutaneous alemtuzumab is feasible and safe for aplastic anemia and is associated with high response rates

Immunosuppressive therapy using horse antithymocyte globulin (ATG; h-ATG) combined with cyclosporine (CsA) and eltrombopag is the standard care for aplastic anemia (AA) in patients without a suitable matched donor. However, in many countries, h-ATG use has been discontinued, leaving rabbit ATG (r-ATG), which has a lower response rates and poorer survival, as the only alternative. In previous studies, alemtuzumab (ALZ), a humanized monoclonal antibody targeting CD52, combined with CsA, resulted in an adequate overall response rate (ORR) in patients with AA. This study describes a multicenter, international retrospective analysis of ALZ for treating AA.We analyzed a series of patients who received subcutaneous ALZ for AA in 4 centers in Brazil and the United Kingdom from March 2009 to January 2024. We analyzed 64 ALZ treatments in 61 adult patients with AA, 76% with severe AA (SAA) or very SAA. The ORR was 59.4% at 12 months (complete, 21.9%; partial, 37.5%). Cumulative incidence (CI) of response was 54.7% at 6 months and 59.4% at 12 months. Younger patients (age <65 years) had higher CI of response (67% vs 31%; P = .03), as did patients treated with a total dose of 103 mg (70% vs 38%; P = .02). Overall survival was 86% at 1 year, 78% at 2 years, and 70% at 4 years, significantly higher in responders (90% vs 44%; P < .0001). Adverse events were of low grade, and infectious events were infrequent. Subcutaneous ALZ is a feasible, effective, and safe alternative to r-ATG for patients with AA requiring immunosuppressive treatment when h-ATG access is limited.

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Publication Details

Journal
Blood Advances
Published
2024-12-18
DOI
https://doi.org/10.1182/bloodadvances.2024014159
Primary Topic
Hematopoietic Stem Cell Transplantation
Type
article
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article

Outpatient subcutaneous alemtuzumab is feasible and safe for aplastic anemia and is associated with high response rates

Anita Hill, Richard Kelly, Talha Munir, Patrícia Eiko Yamakawa et al.
1 links
Blood Advances
Hematopoietic Stem Cell Transplantation
article

Outpatient subcutaneous alemtuzumab is feasible and safe for aplastic anemia and is associated with high response rates

Anita Hill, Richard Kelly, Talha Munir, Patrícia Eiko Yamakawa, Iara Baldim Rabelo, Phillip Scheinberg, Morag Griffin, Celso Arrais‐Rodrigues, Austin Gladston Kulasekararaj, Antonio M. Risitano, Ana Rita Da Fonseca, Thales Dalessandro, Vinícius Campos de Molla, Caio Cesar Justino, Elvira Deolinda Rodrigues Pereira Velloso, Louise Arnold
article en

Abstract

Immunosuppressive therapy using horse antithymocyte globulin (ATG; h-ATG) combined with cyclosporine (CsA) and eltrombopag is the standard care for aplastic anemia (AA) in patients without a suitable matched donor. However, in many countries, h-ATG use has been discontinued, leaving rabbit ATG (r-ATG), which has a lower response rates and poorer survival, as the only alternative. In previous studies, alemtuzumab (ALZ), a humanized monoclonal antibody targeting CD52, combined with CsA, resulted in an adequate overall response rate (ORR) in patients with AA. This study describes a multicenter, international retrospective analysis of ALZ for treating AA.We analyzed a series of patients who received subcutaneous ALZ for AA in 4 centers in Brazil and the United Kingdom from March 2009 to January 2024. We analyzed 64 ALZ treatments in 61 adult patients with AA, 76% with severe AA (SAA) or very SAA. The ORR was 59.4% at 12 months (complete, 21.9%; partial, 37.5%). Cumulative incidence (CI) of response was 54.7% at 6 months and 59.4% at 12 months. Younger patients (age <65 years) had higher CI of response (67% vs 31%; P = .03), as did patients treated with a total dose of 103 mg (70% vs 38%; P = .02). Overall survival was 86% at 1 year, 78% at 2 years, and 70% at 4 years, significantly higher in responders (90% vs 44%; P < .0001). Adverse events were of low grade, and infectious events were infrequent. Subcutaneous ALZ is a feasible, effective, and safe alternative to r-ATG for patients with AA requiring immunosuppressive treatment when h-ATG access is limited.

Blood AdvancesVol. 9(4)
Sunovion (United Kingdom) (GB), St James's University Hospital (GB), Universidade Nove de Julho (BR), European Society for Blood and Marrow Transplantation (NL), King's College Hospital NHS Foundation Trust (GB), Naples Anesthesia & Physician Associates (US), Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo (BR), Hospital Sírio-Libanês (BR), University of Naples Federico II (IT), Vion Pharmaceuticals (United States) (US), Universidade Federal de São Paulo (BR)
Coordenação de Aperfeiçoamento de Pessoal de Nível Superior, Conselho Nacional de Desenvolvimento Científico e Tecnológico
No poverty
Openalex Percentile: Top 57%
Hematopoietic Stem Cell Transplantation
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