Vox Sanguinis (2018) ORIGINAL PAPER © 2018 International Society of Blood Transfusion DOI: 10.1111/vox.12726 aPCC vs. rFVIIa for the treatment of bleeding in patients with acquired haemophilia – a cost-effectiveness model Chong H. Kim, 1, * Sierra C. Simmons, 2, * Chau M. Bui, 2 Ning Jiang 3 & Huy P. Pham 4 1 Department of Clinical Pharmacy, University of Colorado Anschutz Medical Campus, Aurora, CO, USA 2 Independent Researcher, Reno, NV, USA 3 Center for Family Life at Sunset Park, SCO Family of Services, Brooklyn, NY, USA 4 Department of Pathology, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA Received: 31 May 2018, revised 23 October 2018, accepted 29 October 2018 Background Acquired haemophilia A (AHA) is an autoimmune bleeding disorder with significant morbidity and mortality. Bleeding AHA patients with high titre inhibitors can be treated with either activated prothrombin complex concentrate (aPCC) or recombinant activated factor VII (rFVIIa). Given that both replacement therapies have inherent benefits and limitations, a cost-effectiveness analysis (CEA) was performed in this population to compare rFVIIa with aPCC. Methods In high-titered AHA patients with bleeding treated with either aPCC or rFVIIa, during a 5-day study period, a Markov model was developed such that these patients were transitioned into four different health states: (1) continuous bleeding, (2) thrombosis, (3) stop bleeding and (4) death, with states (2), (3) and (4) modelled as absorbing states. Model parameters, including probabilities, health utility index and costs, were gathered from the medical literature, except for the costs of aPCC and rFVIIa, which were obtained from our institutional data. Results During the 5-day period, the total treatment cost of rFVIIa was substan- tially more than the cost of aPCC ($13 635 vs. $1741). The average quality- adjusted life days (QALDs) gained for rFVIIa were slightly lower compared to aPCC (408 vs. 409). Overall, aPCC prevailed over rFVIIa. Sensitivity analysis confirmed the robustness of the model across tested ranges of all input variables. Conclusion In high-titered AHA patients with bleeding, aPCC is a cost-effective treatment option when compared to rFVIIa. Thus, aPCC may be considered in these patients, if available, and provided there is no clinical contraindication. Key words: acquired haemophilia, autoimmune, bleeding, coagulation factor con- centrates, cost-effectiveness, factor VIII deficiency. Introduction Acquired haemophilia A (AHA) is an acquired bleeding disorder with significant morbidity and mortality [1–3]. It is a rare autoimmune disorder affecting 13–15 cases per million annually, usually in the elderly population (median age between 64 and 78 years) [2, 4, 5]. The con- dition results from the spontaneous formation of autoan- tibodies against factor VIII [6]. Patients with AHA usually present with large haematomas, extensive ecchymoses or severe mucosal bleeding [4]. Many of the haemorrhages can be limb- or life-threatening [4, 7, 8], and the mortal- ity can be greater than 20% in high-risk patients [9]. For example, according to one study, 22% of patients died from haemorrhage or complications attributed directly to the disease [10]. Therapies include immunosuppression, bleeding control, eradication of the inhibitors and treat- ment of the underlying condition (if applicable). In Correspondence: Huy P. Pham, Department of Pathology, Keck School of Medicine of USC, 1450 San Pablo Street, Building HC4 – Room 2426, Los Angeles, CA 90033, USA E-mail: phamh@usc.edu *Contributed equally to this work 1