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

Gianluigi Ardissino; Stefania Salardi; Silvia Berra; Giacomo Colussi; Massimo Cugno; Marco Zecca; Fabio Giglio; Jacopo Peccatori; Elisa Diral; Francesca Tel; Alberto Clivio; Silvana Tedeschi

doi:10.1016/j.bbmt.2017.05.013

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

Gianluigi Ardissino, Stefania Salardi, Silvia Berra, Giacomo Colussi, Massimo Cugno, Marco Zecca, Fabio Giglio, Jacopo Peccatori, Elisa Diral, Francesca Tel, Alberto Clivio, Silvana Tedeschi

Research output: Contribution to journal › Article › peer-review

Abstract

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.

Original language	English
Pages (from-to)	1580-1582
Number of pages	3
Journal	Biology of Blood and Marrow Transplantation
Volume	23
Issue number	9
DOIs	https://doi.org/10.1016/j.bbmt.2017.05.013
Publication status	Published - Sept 1 2017

Keywords

Complement
Complement gene mutations
Hematopoietic stem cell transplantation (HSCT)
Hemolytic uremic syndrome
Thrombotic microangiopathy (TMA)

ASJC Scopus subject areas

Hematology
Transplantation

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Ardissino, G., Salardi, S., Berra, S., Colussi, G., Cugno, M., Zecca, M., Giglio, F., Peccatori, J., Diral, E., Tel, F., Clivio, A., & Tedeschi, S. (2017). Acquired Complement Regulatory Gene Mutations and Hematopoietic Stem Cell Transplant–Related Thrombotic Microangiopathy. Biology of Blood and Marrow Transplantation, 23(9), 1580-1582. https://doi.org/10.1016/j.bbmt.2017.05.013

Ardissino, G, Salardi, S, Berra, S, Colussi, G, Cugno, M , Zecca, M, Giglio, F, Peccatori, J, Diral, E, Tel, F, Clivio, A & Tedeschi, S 2017, 'Acquired Complement Regulatory Gene Mutations and Hematopoietic Stem Cell Transplant–Related Thrombotic Microangiopathy', Biology of Blood and Marrow Transplantation, vol. 23, no. 9, pp. 1580-1582. https://doi.org/10.1016/j.bbmt.2017.05.013

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abstract = "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.",

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AU - Ardissino, Gianluigi

AU - Salardi, Stefania

AU - Berra, Silvia

AU - Colussi, Giacomo

AU - Cugno, Massimo

AU - Zecca, Marco

AU - Giglio, Fabio

AU - Peccatori, Jacopo

AU - Diral, Elisa

AU - Tel, Francesca

AU - Clivio, Alberto

AU - Tedeschi, Silvana

PY - 2017/9/1

Y1 - 2017/9/1

N2 - 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.

AB - 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.

KW - Complement

KW - Complement gene mutations

KW - Hematopoietic stem cell transplantation (HSCT)

KW - Hemolytic uremic syndrome

KW - Thrombotic microangiopathy (TMA)

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

Abstract

Keywords

ASJC Scopus subject areas

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