Etuvetidigene autotemcel

Therapeutic indications

Etuvetidigene autotemcel is indicated for:

Wiskott-Aldrich Syndrome (WAS)

Population group: only infants (40 days - 1 year old) , children (1 year - 12 years old) , adolescents (12 years - 18 years old) , adults (18 - 65 years old)

Etuvetidigene autotemcel is indicated for the treatment of patients aged 6 months and older with Wiskott-Aldrich Syndrome (WAS) who have a mutation in the WAS gene for whom haematopoietic stem cell (HSC) transplantation is appropriate and no suitable human leukocyte antigen (HLA)-matched related haematopoietic stem cell donor is available.

For this indication, competent medicine agencies globally authorize below treatments (click for details):

7 × 10⁶ CD34+ cells/kg of body weight once, minimum recommended dose

Contraindications

Etuvetidigene autotemcel is contraindicated in the following cases:

Pregnancy

Lactation

Previous treatment with haematopoietic stem cell gene therapy or with allogeneic HSC transplant

at least one of

Gene therapy
Hemopoietic stem cell transplant

Anti-retrovirals

Antiretrovirals drugs

Blood, organ, tissue and cell donation

at least one of

Transplantation
Finding of eligibility to be a donor
Organ retrieval operation
Blood unit collection for directed donation, donor

Live vaccines

Live organism vaccines

< Previous: Overview

Next: Precautions >

© All content on this website, including data entry, data processing, decision support tools, "RxReasoner" logo and graphics, is the intellectual property of RxReasoner and is protected by copyright laws. Unauthorized reproduction or distribution of any part of this content without explicit written permission from RxReasoner is strictly prohibited. Any third-party content used on this site is acknowledged and utilized under fair use principles.