Eculizumab Bridging before Bone Marrow Transplant for Marrow Failure Disorders Is Safe and Does Not Limit Engraftment

Abstract

Paroxysmal nocturnal hemoglobinuria (PNH) often develops secondary to other bone marrow failure (BMF) disorders, especially aplastic anemia (AA). Patients with the AA/PNH syndrome may require treatment with both eculizumab to reduce intravascular hemolysis and the risk of thrombosis and allogeneic stem cell transplant for the severe BMF. There has been concern that eculizumab could adversely affect the outcomes for transplant in these patients. This is a retrospective, single-center study of severe AA (SAA)/PNH patients treated with eculizumab immediately before the start of conditioning for transplant. Metrics of engraftment and infectious outcomes are described. Eight patients with SAA/PNH and PNH-related symptoms were treated with eculizumab and then proceeded to transplant. All were successfully transplanted without adverse events related to C5 blockage before conditioning. All were also cured of their both PNH and SAA. Eculizumab is safe and efficacious in patients with PNH clones who require transplant. This is sometimes required to "bridge" patients before bone marrow transplantation and does not appear to adversely impact outcomes even when using HLA matched unrelated or haploidentical donors.

Keywords: Aplastic anemia; Eculizumab; Paroxysmal nocturnal hemoglobinuria; Transplant.