Acquired Complement Regulatory Gene Mutations and Hematopoietic Stem Cell Transplant–Related Thrombotic Microangiopathy

G Ardissino, S Salardi, S Berra, G Colussi, M Cugno, M Zecca, F Giglio, J Peccatori, E Diral, F Tel, A Clivio, S Tedeschi

Research output: Contribution to journalArticle


Hematopoietic stem cell transplant–related thrombotic microangiopathy (HSCT-TMA) is a severe complication whose pathophysiology is unknown. We describe 6 patients in which the disease was associated with complement regulatory gene abnormalities received from their respective donors. It is suggested that mutated and transplanted monocyte-derived cells are responsible for production of abnormal proteins, complement dysregulation, and, ultimately, for the disease. This observation might have important drawbacks as far as HSCT-TMA pathophysiology and treatment are concerned. © 2017 The American Society for Blood and Marrow Transplantation
Original languageEnglish
Pages (from-to)1580-1582
Number of pages3
JournalBiology of Blood and Marrow Transplantation
Issue number9
Publication statusPublished - 2017


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