Premium
Successful Treatment With Ledipasvir/Sofosbuvir in an Infant With Severe Combined Immunodeficiency Caused by Adenosine Deaminase Deficiency With HCV Allowed Gene Therapy with Strimvelis
Author(s) -
Tucci Francesca,
Calbi Valeria,
Barzaghi Federica,
Migliavacca Maddalena,
Ferrua Francesca,
Bernardo Maria Ester,
Canarutto Daniele,
Consiglieri Giulia,
Recupero Salvatore,
Calzatini Francesco,
Gabaldo Michela,
Lucano Caterina,
Casiraghi Miriam,
Darin Silvia,
Dionisio Francesca,
Marktel Sarah,
Cestone Enza,
Finazzi Renato,
MieliVergani Giorgina,
Boeri Enzo,
Appleby Jonathan,
Abd Elaziz Dalia,
Ciceri Fabio,
Aiuti Alessandro,
Cicalese Maria Pia
Publication year - 2018
Publication title -
hepatology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 5.488
H-Index - 361
eISSN - 1527-3350
pISSN - 0270-9139
DOI - 10.1002/hep.30160
Subject(s) - ledipasvir , sofosbuvir , medicine , hepatitis c , hepatitis c virus , pediatrics , immunology , ribavirin , virology , virus
Patients with inborn error diseases can be candidates for autologous haematopoietic stem cells (HSC) gene therapies (GT) but may require negative viral screening, including Hepatitis C (HCV), to allow HSC manipulation in Good Manufacturing Practices areas. In case of HCV positivity, patients might be excluded from life-saving treatments. As HCV antibodies could be negative in young infant immunodeficient patients due to their immature/impaired immune system, or positive due to maternal-fetal antibody transmission, the risk is usually also evaluated on the basis of the HCV-RNA. This article is protected by copyright. All rights reserved.