PSY90 - Health Resource Burden Among Patients with Severe Aplastic Anemia who Have Had Insufficient Response to Immunosuppressive Therapy: A Retrospective Chart Review Study

Abstract

To examine healthcare resource utilization (HRU) among patients with severe aplastic anemia (SAA) with insufficient response to immunosuppressive therapy (IST) in real-world practice. A retrospective chart review was conducted at Dana-Farber Cancer Institute, US (DFCI) and Hôpital Saint-Louis, France (HSL). Eligible patients were ≥18 years old, diagnosed with acquired SAA between 1/1/2006-7/31/2016, had insufficient response to ≥1 course of IST, and had ≥12 months follow-up post diagnosis. SAA-related HRU was summarized with incidence rates. Among the 40 patients (NDFCI=20; NHSL=20), mean age at diagnosis was 44.1 years and 52.5% of patients were women. During follow-up, 25.0% of patients died; median follow-up time after SAA diagnosis was 48.3 months. 95.0% of patients received antithymocyte globulin (ATG) in combination with cyclosporine or tacrolimus as primary therapy prior to hematopoietic stem cell transplant (HSCT). The most common secondary SAA therapies prior to HSCT were eltrombopag (27.5%) and androgens (15.0%). 75% of patients received ≥1 HSCT with a median time of 15.7 months after first IST. Prior to HSCT, the mean frequency of transfusions per patient per month (PPPM) was 2.7 red blood cell (RBC) and 3.3 platelet transfusions. The mean SAA-related HRU rates per patient per year (PPPY) were 0.9 hospitalizations, 0.4 emergency room visits, and 12.8 office visits. Among patients treated with eltrombopag (N=11), the mean number of RBC transfusions PPPM was reduced from 2.2 to 1.3 and platelet transfusions from 2.9 to 2.1 after eltrombopag treatment. Similarly, SAA-related HRU rates appeared lower after eltrombopag initiation (∆hospitalizations: -0.5; ∆emergency room visits: -1.0; ∆office visits: -10.2 PPPY). This study is among the first to quantify the transfusion and healthcare resource burden of SAA. In a subgroup of patients receiving eltrombopag, there was a trend toward a reduction in transfusion frequency and HRU following eltrombopag initiation.