Inotersen and severe thrombocytopenia: Two case reports and review

被引:1
作者
Senin, Loreto Dominguez [1 ]
Garro, Cristina Borrachero [1 ]
Gomez, Ernesto Sanchez [1 ]
Santos-Rubio, Maria Dolores [1 ]
机构
[1] Hosp Juan Ramon Jimenez, Serv Farm Hosp, Ronda Exterior Norte S N, Huelva 21005, Spain
关键词
Key words inotersen; thrombocytopenia; amyloidosis; ANTISENSE OLIGONUCLEOTIDES; TRANSTHYRETIN; 2'-O-METHOXYETHYL;
D O I
10.5414/CP204190
中图分类号
R9 [药学];
学科分类号
1007 ;
摘要
Hereditary transthyretin amyloidosis (hATTR) is an ultra-rare illness. Inotersen is a 2???-O-methoxyethyl (2???MOE)modified antisense oligonucleotides (ASO) approved in 2018 as a polyneuropathy treatment for adults with hereditary transthyretin amyloidosis stages 1 or 2. Inotersen can produce grade 4 thrombocytopenia as a severe adverse reaction that can lead to potentially fatal hemorrhage complications. We describe our experience in the management of severe thrombocytopenia with inotersen. The onset of the thrombocytopenia and the incidences described in the cases in our hospital are different from that described in the literature. Also, recovery of platelet levels was faster in our patient who was administered human immunoglobulin G, which suggests that there is an immunological component.
引用
收藏
页码:311 / 316
页数:6
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