Aplastic Anemia: Insight from a Single Center Experience

CONTEXT: Aplastic anemia (AA) is a rare disease with changing treatment modalities over time. Patients often do not respond to first line therapy and require multiple lines of therapy. Treatment has also evolved over time, with the addition of thrombopoietin receptor agonists. Understanding treatment response and outcomes remains imperative. AIMS: To categorize treatments and outcomes of aplastic anemia patients DESIGN: Single center retrospective study of 96 patients who were seen at Moffitt Cancer Center with a diagnosis of Aplastic Anemia between January 2009 and January 2023. RESULTS: Our study included 96 patients (mean age at diagnosis, 54 years) diagnosed with AA. 56 patients (58%) were male, while 41 patients (42%) were female. Seventy-four (74%) were Caucasian, 7 (7%) were African American, 10 (10%) were Hispanic. At diagnosis, mean white blood count was 2.60, with an absolute neutrophil count of 1.02, a hemoglobin of 8.45, and platelet count of 26. At diagnosis, 67/91 (70%) patients had required packed red blood cell transfusion, while 68/91 (71%) had required platelet transfusion. In total, 77/91 (80%) required either a packed red blood cell or platelet transfusion at the time of diagnosis. At diagnosis, 27 patients had infectious complications while 27 also had bleeding complications. Bone marrow cellularity was 10% or less in 59/86 patients (69%), and under 15% in 71 (83%). Forty-two patients were tested for large granular lymphocyte (LGL) clones, and 14 (33%) had clones present. Paroxysmal nocturnal hematuria (PNH) clones were detected in 35 out of the 75 patients tested (47%). The clones progressed in 14 patients. Seven patients had transformation from AA into Myelodysplastic syndrome (MDS), while 3 had transformation into Acute Myeloid Leukemia (AML). Of the 96 patients, 6 patients were managed with observation. The most used frontline therapies were equine ATG (eATG) + cyclosporine (CSA) in 41 patients, followed by eATG + CSA + eltrombopag in 20 patients. In total, 65 patients received eATG based therapy first line (4 patients received eATG alone frontline). Eleven patients received an eltrombopag based therapy without concomitant ATG. Between singlet/doublet eATG based therapy and triplet therapy, the overall response rate to singlet/doublet was 66% (complete response rate (CR) 23%, partial response rate (PR) 43%) and 67% for triplet (CR 28%, PR 39%) (p=.91). There was no difference to date in OS. There was also no difference in response