Economic Evaluation for the US of Ibrutinib Versus Allogeneic Hematopoietic Stem-Cell Transplantation for Patients with Relapsed or Refractory Chronic Lymphocytic Leukemia with 17p Deletion

Background:Prior to targeted therapies, allogeneic hematopoietic stem cell transplantation (HSCT) was the primary therapy for relapsed/refractory (R/R) chronic lymphocytic leukemia (CLL) with 17p deletion (del(17p)). Treatment options now include oral ibrutinib, which produces significant progression free (PFS) and overall survival (OS) with acceptable safety and the convenience of being non-invasive. Complete remission is unlikely and ibrutinib is not considered curative. HSCT has curative potential, but is associated with high upfront cost, potential transplant-related morbidity and mortality (incl. graft-versus-host disease) and potential relapse. Both treatments are expensive with relative differences in cost of treatment and supportive care management. Considering these different profiles, we performed cost-effectiveness and cost-utility analyses of ibrutinib versus HSCT in R/R CLL del(17p) from a US payer perspective.