Tesidolumab (LFG316) for treatment of C5-variant patients with
paroxysmal nocturnal hemoglobinuria

J 2022

Tesidolumab (LFG316) for treatment of C5-variant patients with paroxysmal nocturnal hemoglobinuria

NISHIMURA, Jun-ichi, Kiyoshi ANDO, Masayoshi MASUKO, Hideyoshi NOJI, Yoshikazu ITO et. al.

Basic information

Original name

Tesidolumab (LFG316) for treatment of C5-variant patients with paroxysmal nocturnal hemoglobinuria

Authors

NISHIMURA, Jun-ichi (guarantor), Kiyoshi ANDO, Masayoshi MASUKO, Hideyoshi NOJI, Yoshikazu ITO, Jiří MAYER (203 Czech Republic, belonging to the institution), Laimonas GRISKEVICIUS, Christoph BUCHER, Florian MULLERSHAUSEN, Peter GERGELY, Izabela ROZENBERG, Anna SCHUBART, Raghav CHAWLA, Jean-Michel RONDEAU, Michael ROGUSKA, Igor SPLAWSKI, Mark T KEATING, Leslie JOHNSON, Rambabu DANEKULA, Morten BAGGER, Yoko WATANABE, Borje HARALDSSON and Yuzuru KANAKURA

Edition

Haematologica, PAVIA, FERRATA STORTI FOUNDATION, 2022, 0390-6078

Other information

Language

English

Type of outcome

Článek v odborném periodiku

Field of Study

30205 Hematology

Country of publisher

Italy

Confidentiality degree

není předmětem státního či obchodního tajemství

References:

URL

Impact factor

Impact factor: 10.100

RIV identification code

RIV/00216224:14110/22:00128073

Organization unit

Faculty of Medicine

DOI

http://dx.doi.org/10.3324/haematol.2020.265868

UT WoS

000836816100029

Keywords in English

Tesidolumab; paroxysmal nocturnal hemoglobinuria; C5-variant patients

Abstract

V originále

Paroxysmal nocturnal hemoglobinuria (PNH) is caused by clonal expansion of hematopoietic stem cells that carry a somatic mutation in the X-linked gene PIG-A (phosphatidylinositol glycan anchor biosynthesis class A). The mutation leads to a deficiency of glycosylphosphatidylinositol-anchored membrane proteins. The loss of membrane-associated complement regulatory proteins CD55 and CD59 increases susceptibility of red blood cells and platelets to complement-mediated lysis, leading to hemolytic anemia, thrombophilia and reduced life expectancy in untreated patients.

Citovat

NISHIMURA, Jun-ichi, Kiyoshi ANDO, Masayoshi MASUKO, Hideyoshi NOJI, Yoshikazu ITO, Jiří MAYER, Laimonas GRISKEVICIUS, Christoph BUCHER, Florian MULLERSHAUSEN, Peter GERGELY, Izabela ROZENBERG, Anna SCHUBART, Raghav CHAWLA, Jean-Michel RONDEAU, Michael ROGUSKA, Igor SPLAWSKI, Mark T KEATING, Leslie JOHNSON, Rambabu DANEKULA, Morten BAGGER, Yoko WATANABE, Borje HARALDSSON and Yuzuru KANAKURA. Tesidolumab (LFG316) for treatment of C5-variant patients with paroxysmal nocturnal hemoglobinuria. Haematologica. PAVIA: FERRATA STORTI FOUNDATION, 2022, vol. 107, No 6, p. 1483-1488. ISSN 0390-6078. Available from: https://dx.doi.org/10.3324/haematol.2020.265868.

@article{2247544,
   author = {Nishimura, Junandichi and Ando, Kiyoshi and Masuko, Masayoshi and Noji, Hideyoshi and Ito, Yoshikazu and Mayer, Jiří and Griskevicius, Laimonas and Bucher, Christoph and Mullershausen, Florian and Gergely, Peter and Rozenberg, Izabela and Schubart, Anna and Chawla, Raghav and Rondeau, JeanandMichel and Roguska, Michael and Splawski, Igor and Keating, Mark T and Johnson, Leslie and Danekula, Rambabu and Bagger, Morten and Watanabe, Yoko and Haraldsson, Borje and Kanakura, Yuzuru},
   article_location = {PAVIA},
   article_number = {6},
   doi = {http://dx.doi.org/10.3324/haematol.2020.265868},
   keywords = {Tesidolumab; paroxysmal nocturnal hemoglobinuria; C5-variant patients},
   language = {eng},
   issn = {0390-6078},
   journal = {Haematologica},
   title = {Tesidolumab (LFG316) for treatment of C5-variant patients with paroxysmal nocturnal hemoglobinuria},
   url = {https://haematologica.org/article/view/haematol.2020.265868},
   volume = {107},
   year = {2022}
}

TY  - JOUR
ID  - 2247544
AU  - Nishimura, Jun-ichi - Ando, Kiyoshi - Masuko, Masayoshi - Noji, Hideyoshi - Ito, Yoshikazu - Mayer, Jiří - Griskevicius, Laimonas - Bucher, Christoph - Mullershausen, Florian - Gergely, Peter - Rozenberg, Izabela - Schubart, Anna - Chawla, Raghav - Rondeau, Jean-Michel - Roguska, Michael - Splawski, Igor - Keating, Mark T - Johnson, Leslie - Danekula, Rambabu - Bagger, Morten - Watanabe, Yoko - Haraldsson, Borje - Kanakura, Yuzuru
PY  - 2022
TI  - Tesidolumab (LFG316) for treatment of C5-variant patients with paroxysmal nocturnal hemoglobinuria
JF  - Haematologica
VL  - 107
IS  - 6
SP  - 1483-1488
EP  - 1483-1488
PB  - FERRATA STORTI FOUNDATION
SN  - 03906078
KW  - Tesidolumab
KW  - paroxysmal nocturnal hemoglobinuria
KW  - C5-variant patients
UR  - https://haematologica.org/article/view/haematol.2020.265868
N2  - Paroxysmal nocturnal hemoglobinuria (PNH) is caused by clonal expansion of hematopoietic stem cells that carry a somatic mutation in the X-linked gene PIG-A (phosphatidylinositol glycan anchor biosynthesis class A). The mutation leads to a deficiency of glycosylphosphatidylinositol-anchored membrane proteins. The loss of membrane-associated complement regulatory proteins CD55 and CD59 increases susceptibility of red blood cells and platelets to complement-mediated lysis, leading to hemolytic anemia, thrombophilia and reduced life expectancy in untreated patients.
ER  -

NISHIMURA, Jun-ichi, Kiyoshi ANDO, Masayoshi MASUKO, Hideyoshi NOJI, Yoshikazu ITO, Jiří MAYER, Laimonas GRISKEVICIUS, Christoph BUCHER, Florian MULLERSHAUSEN, Peter GERGELY, Izabela ROZENBERG, Anna SCHUBART, Raghav CHAWLA, Jean-Michel RONDEAU, Michael ROGUSKA, Igor SPLAWSKI, Mark T KEATING, Leslie JOHNSON, Rambabu DANEKULA, Morten BAGGER, Yoko WATANABE, Borje HARALDSSON and Yuzuru KANAKURA. Tesidolumab (LFG316) for treatment of C5-variant patients with paroxysmal nocturnal hemoglobinuria. \textit{Haematologica}. PAVIA: FERRATA STORTI FOUNDATION, 2022, vol.~107, No~6, p.~1483-1488. ISSN~0390-6078. Available from: https://dx.doi.org/10.3324/haematol.2020.265868.

Detailed Information on Publication Record