OUTPATIENT SUBCUTANEOUS ALEMTUZUMAB is FEASIBLE and SAFE for APLASTIC ANEMIA and ASSOCIATED with HIGH RESPONSE RATES.
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All Authors
Fonseca, A.R.
Oliveira, C.C.
Yamakwa, P.
Molla, V.
Rabelo, I.
Arnold, L.
Kelly, R.
Munir, T.
Griffin, M.
Velloso, E.
LTHT Author
Arnold, Louise
Kelly, Richard
Munir, Talha
Griffin, Morag
Kelly, Richard
Munir, Talha
Griffin, Morag
LTHT Department
Oncology
Haematology
PNH Service
Haematology
PNH Service
Non Medic
Advanced Clinical Practitioner
0
0
Publication Date
2024
Item Type
Conference Abstract
Language
Subject
AGED , ANAEMIA, APLASTIC , CONTROLLED CLINICAL TRIALS AS TOPIC , DRUG THERAPY , WOMEN , FOLLOW-UP-STUDIES , IMMUNOSUPPRESSIVE AGENTS , OUTPATIENTS , SURVIVAL RATE , PROSPECTIVE STUDIES , RETROSPECTIVE STUDIES , DRUG-RELATED SIDE EFFECTS AND ADVERSE REACTIONS , ANTIBODIES, MONOCLONAL , ANTIBODIES
Subject Headings
Abstract
Background: Immunosuppressive therapy (IST) using horse antithymocyte globulin (hATG) combined with cyclosporine (CsA) is currently the standard of care for patients diagnosed with severe aplastic anemia (SAA) who do not have a suitable matched donor with addition of eltrombopag to IST improving responses. However, in many Asian, South American, and certain European countries, the use of hATG has been discontinued, and only rabbit ATG (rATG) is available as an alternative. Prospective studies have shown that rATG, although a more potent immunosuppressant, is associated with a lower rate of hematological response and poorer survival compared to hATG. Another alternative is alemtuzumab (ALZ), a humanized monoclonal antibody that targets CD52. The use of ALZ in monotherapy yielded an overall response rate (ORR) comparable to that of r-ATG when applied as salvage therapy after hATG failure. Besides, the association of subcutaneous (SC) ALZ and CsA led to a high ORR in SAA patients after in first line. Aim(s): Here we describe a multicenter international retrospective analysis of ALZ to treat patients with AA. Method(s): We retrospectively analyzed all patients who received ALZ for the treatment of aplastic anemia in 4 centers in 2 countries (Brazil and United Kingdom) from March 2009 until October 2022. In Brazil, ALZ total dose was 103 mg in an escalating dose of 3-10-30-30-30 mg, except for elderly patients who received a total dose of 73. In the United Kingdom, total dose varied from 100 mg to 150 mg. Result(s): We analyzed 60 treatments with ALZ in 57 AA patients, 74% of which had SAA or very severe aplastic anemia. Median age was 52 years (range: 18-81), and 12 (21%) were older than 65 years. Most treatments (N=34, 57%) were in first line, in 16 cases (26%) in second line, and in 10 cases (17%) after at least two lines of therapy. Median follow up was 48 months (range 6-141 months). ORR was 54% (complete response: 7%, partial response: 47%) at 6 months, and 57% (complete response: 18%, partial response: 39%) at 12 months. ORR at 12 months was higher in patients younger than 65 years (65%) that in elderly patients (25%, P=0.01). Cumulative incidence (CI) of response was 53% at 6 months, and 59% at 12 months, also significantly higher in patients younger than 65 years (68%) that in elderly patients (25%, P=0.025), and in those who received the 103 mg SC ALZ dose (61%) than in those who received different doses (41%, P=0, 045). Overall survival (OS) was 84% at 1 year, 76% at 2 years, and 71% at 4 years. OS at 4 years was significantly higher in patients who responded to ALZ (hazard ratio: 9.88, 95% confidence interval 2.12-46.0, P=0.004). Adverse events were of low grade and infectious events were infrequent Summary/Conclusion: Subcutaneous ALZ appears to be a feasible, effective, and safe alternative to hATG in patients with AA requiring immunosuppressive treatment, especially in centers and countries with limited access to hATG.
Journal
HemaSphere