P132 | Is Anti-thymocyte Globulin Still a Viable Treatment Option for Older Patients With Aplastic Anemia? a Single-center Experience
Year: 2025
Type: article
Abstract: Patients over 60 years old affected by acquired aplastic anemia (AAA) face challenges related to comorbidities and reduced tolerance to intensive treatments. Standard therapy includes immunosuppressive treatment (IST) with anti-thymocyte globulin plus cyclosporine (ATG+CSA), with or without eltrombopag (ELT), or hematopoietic stem cell transplantation (HSCT) in selected cases. We retrospectively analyzed 20 patients >60 years diagnosed with AAA (Camitta criteria) and treated at our center between January 2014 and December 2024. This study evaluates the feasibility and safety of ATG+CSA in elderly AAA pts. Horse-ATG was administered at 40 mg/kg/day x4 days and CSA at 5 mg/kg. ELT (up to 150 mg/day) was introduced after 3 months in non-responders. Responses were assessed by EBMT criteria: complete response (CR, hemoglobin >10 g/dL, ANC >1.5×10⁹/L, platelets >100×10⁹/L); partial response (PR, transfusion independence); no response (NR, persistent transfusion dependence). Median age was 71y (range 60–82). Two patients (10%) had non-severe, transfusion-dependent AA; 9 (45%) had severe AA; and 9 (45%) had very severe AA. Eight patients (40%) had a small PNH clone. In patients >70 (30% of the cohort), ATG dose was reduced by 50%. At three months, the overall response rate (ORR, CR + PR) was 30% (6/20). Five patients (median age 76y; range 69-77) died due to complications (aspergillus pneumonia 2, cardiac arrest, cerebral hemorrhage, septic shock). Of 9 non-responders, 7 received ELT. After an additional four months, the ORR increased to 60% (12/20), with all initial responders maintaining their response and 6/7 ELT-treated patients responded (3 CRs, 9 PRs in total). Major complications within the first three months among survivors included fever of unknown origin (5/14, 35%), cardiac events (3/14, 21%)—two cases of atrial fibrillation and one of heart failure post-ATG—and one instance of grade 3 hepatic toxicity post-ELT (1/14, 7%). At 39 months median follow-up (range 0-154), the median survival has not been reached (Fig. 1). Despite hematologic responses, ATG treatment was associated with notable early mortality (25%) and morbidity in the elderly. The addition of eltrombopag improved response rates with manageable toxicity. Further studies are warranted to evaluate whether a combination of CSA and eltrombopag, excluding ATG, could represent a safer and effective first-line strategy in elderly AAA patients.
Authors: B. De Marco, A. Peli, E. Borlenghi, C. Cattaneo, E. Cerqui, M. Oberti, S. Bagnasco, L. Masina, L. Donatoni, G. Magliano, and A. Tucci
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: green
OpenAlex ID: W7093121256
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