Improving cellular therapy for primary immune deficiency diseases: recognition, diagnosis, and management

Détails

ID Serval
serval:BIB_19A9087F9D9C
Type
Article: article d'un périodique ou d'un magazine.
Collection
Publications
Titre
Improving cellular therapy for primary immune deficiency diseases: recognition, diagnosis, and management
Périodique
J Allergy Clin Immunol
Auteur(s)
Griffith L. M., Cowan M. J., Notarangelo L. D., Puck J. M., Buckley R. H., Candotti F., Conley M. E., Fleisher T. A., Gaspar H. B., Kohn D. B., Ochs H. D., O'Reilly R. J., Rizzo J. D., Roifman C. M., Small T. N., Shearer W. T., Workshop Participants
ISSN
1097-6825 (Electronic)
ISSN-L
0091-6749
Statut éditorial
Publié
Date de publication
12/2009
Volume
124
Numéro
6
Pages
1152-60 e12
Langue
anglais
Notes
Griffith, Linda M
Cowan, Morton J
Notarangelo, Luigi D
Puck, Jennifer M
Buckley, Rebecca H
Candotti, Fabio
Conley, Mary Ellen
Fleisher, Thomas A
Gaspar, H Bobby
Kohn, Donald B
Ochs, Hans D
O'Reilly, Richard J
Rizzo, J Douglas
Roifman, Chaim M
Small, Trudy N
Shearer, William T
eng
R01 AI071163/AI/NIAID NIH HHS/
U54 AI082973/AI/NIAID NIH HHS/
Z01 HG000122-11/Intramural NIH HHS/
Z99 HG999999/Intramural NIH HHS/
J Allergy Clin Immunol. 2009 Dec;124(6):1152-60.e12. doi: 10.1016/j.jaci.2009.10.022.
Résumé
More than 20 North American academic centers account for the majority of hematopoietic stem cell transplantation (HCT) procedures for primary immunodeficiency diseases (PIDs), with smaller numbers performed at additional sites. Given the importance of a timely diagnosis of these rare diseases and the diversity of practice sites, there is a need for guidance as to best practices in management of patients with PIDs before, during, and in follow-up for definitive treatment. In this conference report of immune deficiency experts and HCT physicians who care for patients with PIDs, we present expert guidance for (1) PID diagnoses that are indications for HCT, including severe combined immunodeficiency disease (SCID), combined immunodeficiency disease, and other non-SCID diseases; (2) the critical importance of a high degree of suspicion of the primary care physician and timeliness of diagnosis for PIDs; (3) the need for rapid referral to an immune deficiency expert, center with experience in HCT, or both for patients with PIDs; (4) medical management of a child with suspicion of SCID/combined immunodeficiency disease while confirming the diagnosis, including infectious disease management and workup; (5) the posttransplantation follow-up visit schedule; (6) antimicrobial prophylaxis after transplantation, including gamma globulin administration; and (7) important indications for return to the transplantation center after discharge. Finally, we discuss the role of high-quality databases in treatment of PIDs and HCT as an element of the infrastructure that will be needed for productive multicenter clinical trials in these rare diseases.
Mots-clé
Child, *Hematopoietic Stem Cell Transplantation, Humans, Immunologic Deficiency Syndromes/*diagnosis/*surgery, *Practice Guidelines as Topic, Severe Combined Immunodeficiency/diagnosis/surgery
Pubmed
Création de la notice
01/11/2017 11:29
Dernière modification de la notice
20/08/2019 13:50
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