Referência de medicamentos
Etuvetidigene autotemcel
Etuvetidigene autotemcel is a medicine in the class miscellaneous.
- Classe do medicamento: Miscellaneous
Última atualização Fontes: US FDA label
Para profissionais de saúde — apenas referência. Esta página é um resumo breve, não é aconselhamento médico nem substitui a bula oficial ou o julgamento clínico. Verifique sempre antes de prescrever ou dispensar.
O conteúdo clínico desta página está disponível apenas em inglês.
Pontos-chave
- Classe do medicamento
- Miscellaneous
- Formas farmacêuticas comuns
- injeção
- Nomes comerciais
- 1
- Países e mercados
- 1
Como funciona
Adds full-length copies of the human WAS cDNA into the patient's hematopoietic stem cells by lentiviral vector transduction. After infusion the modified cells engraft in bone marrow and repopulate the hematopoietic compartment, and their progeny express WAS protein (WASP).
Dose habitual em adultos (resumo da bula)
Wiskott-Aldrich Syndrome: minimum recommended dose 7 × 10 6 CD34 + cells/kg based on body weight at infusion, single-dose intravenous infusion via central venous catheter; maximum volume must remain < 20% of estimated plasma volume
Gravidez e amamentação
- Gravidez
- Unsafe One-time gene therapy (own modified stem cells). No data in pregnancy. It must not be given during pregnancy because of the risks of the conditioning chemotherapy. Confirm a negative pregnancy test first; use contraception from stem-cell collection until at least 6 months after the infusion.
- Amamentação
- Unsafe No data on presence in milk. Nearly all patients are boys (X-linked disease). Because of the risks of conditioning chemotherapy, breastfeeding must stop during conditioning; afterwards follow the advice for the conditioning drugs.
Efeitos adversos relatados
- Catheter related infection
- Bacterial and viral infections
- Diarrhea
- Vomiting
- Stomatitis
- Liver injury
Contraindicações
- Hypersensitivity to the active substance or to any of the excipients
- Previous HSCT within 6 months prior to treatment, or HSCT with evidence of residual donor cells
- Previous treatment with hematopoietic stem cell gene therapy
Quem deve ter cuidado
- Hypersensitivity and infusion-related reactions, including anaphylaxis, may occur due to DMSO; monitor during and after infusion
- Engraftment failure may occur; if so, infuse non-transduced back-up stem cells
- Severe cytopenias occur for several weeks; monitor for at least 8 weeks
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Perguntas frequentes
What is Etuvetidigene autotemcel used for?
Etuvetidigene autotemcel is a medicine in the class miscellaneous.
What side effects have been reported with Etuvetidigene autotemcel?
Catheter related infection, Bacterial and viral infections, Diarrhea, Vomiting, Stomatitis, Liver injury.
Can Etuvetidigene autotemcel be taken in pregnancy or while breastfeeding?
Pregnancy: Unsafe. One-time gene therapy (own modified stem cells). No data in pregnancy. It must not be given during pregnancy because of the risks of the conditioning chemotherapy. Confirm a negative pregnancy test first; use contraception from stem-cell collection until at least 6 months after the infusion. Breastfeeding: Unsafe. No data on presence in milk. Nearly all patients are boys (X-linked disease). Because of the risks of conditioning chemotherapy, breastfeeding must stop during conditioning; afterwards follow the advice for the conditioning drugs.
What does Etuvetidigene autotemcel interact with?
MediHelp does not list a checked interaction for Etuvetidigene autotemcel yet. Always tell your pharmacist about every medicine you take.
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