Outpatient subcutaneous alemtuzumab is feasible and safe for aplastic anemia and is associated with high response rates | Aplastic Anemia and MDS International Foundation (AAMDSIF) Return to top.

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

Journal Title: 
Blood Advances
Primary Author: 
da Fonseca AR
Author(s): 
da Fonseca AR, Justino CC, Campos de Molla V, Yamakawa PE, Rabelo IB, Arnold L, Kelly R, Griffin M, Munir T, Hill A, Velloso E, Dalessandro T, Risitano AM, Scheinberg P, Kulasekararaj A, Arrais-Rodrigues C
Original Publication Date: 
Tuesday, February 25, 2025
Bone Marrow Disease(s): 

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.