Is Allogeneic Stem Cell Transplantation a Good Option for Paroxysmal Nocturnal Haemoglobinuria?

Abstract

Paroxysmal nocturnal haemoglobinuria (PNH) is a rare, nonmalignant, haematopoietic clonal disorder that manifests with haemolytic anaemia, thrombosis, and peripheral blood cytopenias. The diagnosis is based on laboratory findings of intravascular haemolysis and flow cytometry. Clinical findings in PNH include haemolytic anaemia, thrombosis in atypical sites, or nonspecific symptoms attributable to the consequences of haemolysis. Thrombosis is the leading cause of death in PNH. Terminal complement pathway inhibition with eculizumab controls most of the symptoms of haemolysis and the life-threatening complications of PNH. However, there is still no consensus about haematopoietic stem cell transplantation (HSCT) in the management of PNH; it is the only potentially curative therapy for PNH. There are limited data and few case series about both the long-term outcomes of HSCT for PNH and the impacts of conditioning regimens on PNH clones. The authors have reviewed the findings of these studies which report on HSCT for the treatment of PNH.