Unresolved issues in hematopoietic stem cell transplantation for severe combined immunodeficiency: Need for safer conditioning and reduced late effects - 29/04/13
, Morton J. Cowan, MDAbstract |
In this review we discuss recent outcomes of hematopoietic cell transplantation (HCT) for patients with severe combined immunodeficiency (SCID), including survival, T- and B-cell reconstitution, and late effects, particularly those related to genotype, use of conditioning regimen, and use of alternative donors. We identify the following issues that require additional data, which can be obtained through cooperative studies: outcomes of patients with SCID who did not receive conditioning before alternative donor HCT; outcomes of patients with SCID who did not receive graft-versus-host disease prophylaxis after T cell–replete HCT; late effects of HCT for patients with SCID, including neurocognitive outcomes, growth, and development; and their relationship to genotype and use of alkylating agents for conditioning. Careful follow-up of outcomes of all newborns receiving diagnoses based on newborn screening programs for SCID is essential because data are scarce on the effects of conditioning regimens in very young patients. A consensus on the definition of T- and B-cell recovery, criteria for additional “boosts,” pharmacokinetic data of chemotherapy agents used in young children, and uniformity of the use of various chemotherapy agents are needed to compare results among institutions. Finally, development of new nontoxic conditioning regimens for HCT that can be safely used in very young children is required.
Le texte complet de cet article est disponible en PDF.Key words : Severe combined immunodeficiency, T cell–depleted stem cell transplantation, alternative donors, immune reconstitution, late effects of transplantation
Abbreviations used : ADA, γc, GVHD, HCT, IL-7R⍺, Jak3, MMRD, MRD, MUD, NK, RAG, SCID, TCD, TREC, UCB
Plan
| Disclosure of potential conflict of interest: M. J. Cowan has received a grant from the National Institutes of Health/National Institute of Allergy and Infectious Disease. B. Horn declares no relevant conflicts of interest. |
Vol 131 - N° 5
P. 1306-1311 - mai 2013 Retour au numéroBienvenue sur EM-consulte, la référence des professionnels de santé.
L’accès au texte intégral de cet article nécessite un abonnement.
Déjà abonné à cette revue ?
