Transfusing children with hemoglobinopathies.

Allografts Anemia, Sickle Cell / drug therapy Child Emergencies Erythrocyte Transfusion Exchange Transfusion, Whole Blood Genetic Therapy Hematopoietic Stem Cell Transplantation Hemoglobinopathies / therapy Humans Hydroxyurea / therapeutic use Multiple Organ Failure / therapy Thalassemia / therapy
Drépanocytose Gene therapy Greffe de cellules souches hématopoïétiques Hematopoietic stem cell transplantation Hydroxyurea Hydroxyurée Red blood cell transfusion Sickle cell disease Thalassemia Thalassémie Thérapie génique Transfusion érythrocytaire

Journal

Transfusion clinique et biologique : journal de la Societe francaise de transfusion sanguine
ISSN: 1953-8022
Titre abrégé: Transfus Clin Biol
Pays: France
ID NLM: 9423846

Informations de publication

Date de publication:
Sep 2019
Historique:
received: 25 06 2019
accepted: 25 06 2019
pubmed: 14 7 2019
medline: 18 2 2020
entrez: 14 7 2019
Statut: ppublish

Résumé

Thalassemia and sickle cell disease (SCD) are among the most common inherited diseases worldwide. Red blood cell transfusion is a cornerstone of their treatment, but its indications have significantly changed over the past years. New therapies are emerging in both syndromes: among them, hematopoietic stem cell transplantation is now routinely proposed, and gene therapy has shown promising preliminary results.

Identifiants

DOI: 10.1016/j.tracli.2019.06.323 PMID: 31300265
pubmed: 31300265
pii: S1246-7820(19)30388-X
doi: 10.1016/j.tracli.2019.06.323
pii:
doi:

Substances chimiques

Hydroxyurea X6Q56QN5QC

Types de publication

Journal Article Review

Langues

eng

Sous-ensembles de citation

IM

Pagination

147-149

Informations de copyright

Copyright © 2019. Published by Elsevier Masson SAS.

Auteurs

S Allali (S)

Department of general pediatrics and pediatric infectious diseases, Paris Descartes university, Necker-Enfants malades hospital, AP-HP, 149, rue de Sèvres, 75015 Paris, France; Laboratory of excellence, GR-Ex, 75015 Paris, France; Pediatric reference center for sickle cell disease, 75015 Paris, France.

M Taylor (M)

Department of general pediatrics and pediatric infectious diseases, Paris Descartes university, Necker-Enfants malades hospital, AP-HP, 149, rue de Sèvres, 75015 Paris, France; Laboratory of excellence, GR-Ex, 75015 Paris, France; Pediatric reference center for sickle cell disease, 75015 Paris, France.

S Albinni (S)

Établissement français du sang, Necker-Enfants malades hospital, 149, rue de Sèvres, 75015 Paris, France.

D Amiranoff (D)

Établissement français du sang, Necker-Enfants malades hospital, 149, rue de Sèvres, 75015 Paris, France.

M de Montalembert (M)

Department of general pediatrics and pediatric infectious diseases, Paris Descartes university, Necker-Enfants malades hospital, AP-HP, 149, rue de Sèvres, 75015 Paris, France; Laboratory of excellence, GR-Ex, 75015 Paris, France; Pediatric reference center for sickle cell disease, 75015 Paris, France. Electronic address: mariane.demontal@aphp.fr.

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Classifications MeSH

questionsmedicales.fr › Régions du corps › Transplants › Allogreffes › Transfusing children with hemoglobinopathies.
questionsmedicales.fr › Malformations et maladies congénitales, héréditaires et néonatales › Maladies génétiques congénitales › Hémoglobinopathies › Drépanocytose › Transfusing children with hemoglobinopathies.
questionsmedicales.fr › Personnes › Tranches d'âge › Enfant › Transfusing children with hemoglobinopathies.
questionsmedicales.fr › Environnement et santé publique › Santé publique › Urgences › Transfusing children with hemoglobinopathies.
questionsmedicales.fr › Thérapeutique › Biothérapie › Transfusion sanguine › Transfusion de composants du sang › Transfusion d'érythrocytes › Transfusing children with hemoglobinopathies.
questionsmedicales.fr › Thérapeutique › Biothérapie › Transfusion sanguine › Exsanguinotransfusion › Transfusing children with hemoglobinopathies.
questionsmedicales.fr › Techniques d'investigation › Techniques génétiques › Génie génétique › Thérapie génétique › Transfusing children with hemoglobinopathies.
questionsmedicales.fr › Procédures de chirurgie opératoire › Transplantation › Transplantation cellulaire › Transplantation de cellules souches › Transplantation de cellules souches hématopoïétiques › Transfusing children with hemoglobinopathies.
questionsmedicales.fr › Malformations et maladies congénitales, héréditaires et néonatales › Maladies génétiques congénitales › Hémoglobinopathies › Transfusing children with hemoglobinopathies.
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Transfusing children with hemoglobinopathies.
questionsmedicales.fr › Composés chimiques organiques › Amides › Urée › Hydroxy-urée › Transfusing children with hemoglobinopathies.
questionsmedicales.fr › États, signes et symptômes pathologiques › Processus pathologiques › Choc › Défaillance multiviscérale › Transfusing children with hemoglobinopathies.
questionsmedicales.fr › Malformations et maladies congénitales, héréditaires et néonatales › Maladies génétiques congénitales › Hémoglobinopathies › Thalassémie › Transfusing children with hemoglobinopathies.