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.

Základní údaje

Originální název

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

Autoři

NISHIMURA, Jun-ichi (garant), Kiyoshi ANDO, Masayoshi MASUKO, Hideyoshi NOJI, Yoshikazu ITO, Jiří MAYER (203 Česká republika, domácí), 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 a Yuzuru KANAKURA

Vydání

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

Další údaje

Jazyk

angličtina

Typ výsledku

Článek v odborném periodiku

Obor

30205 Hematology

Stát vydavatele

Itálie

Utajení

není předmětem státního či obchodního tajemství

Odkazy

URL

Impakt faktor

Impact factor: 10.100

Kód RIV

RIV/00216224:14110/22:00128073

Organizační jednotka

Lékařská fakulta

DOI

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

UT WoS

000836816100029

Klíčová slova anglicky

Tesidolumab; paroxysmal nocturnal hemoglobinuria; C5-variant patients

Štítky

14110212, rivok

Příznaky

Mezinárodní význam, Recenzováno

Změněno: 18. 1. 2023 09:59, Mgr. Tereza Miškechová

Anotace

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 a Yuzuru KANAKURA. Tesidolumab (LFG316) for treatment of C5-variant patients with paroxysmal nocturnal hemoglobinuria. Haematologica. PAVIA: FERRATA STORTI FOUNDATION, 2022, roč. 107, č. 6, s. 1483-1488. ISSN 0390-6078. Dostupné z: 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 a Yuzuru KANAKURA. Tesidolumab (LFG316) for treatment of C5-variant patients with paroxysmal nocturnal hemoglobinuria. \textit{Haematologica}. PAVIA: FERRATA STORTI FOUNDATION, 2022, roč.~107, č.~6, s.~1483-1488. ISSN~0390-6078. Dostupné z: https://dx.doi.org/10.3324/haematol.2020.265868.

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