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Blood, 23 April 2009, Vol. 113, No. 17, pp. 4114-4124. Prepublished online as a Blood First Edition Paper on January 23, 2009; DOI 10.1182/blood-2008-09-177923. This Article Full Text Full Text (PDF) Supplemental Tables and Figure All Versions of this Article: blood-2008-09-177923v1 113/17/4114    most recent Alert me when this article is cited Alert me if a correction is posted Citation Map Services Email this article to a friend Similar articles in this journal Similar articles in PubMed Alert me to new issues of the journal Download to citation manager Rights and Permissions Citing Articles Citing Articles via HighWire Citing Articles via CrossRef Citing Articles via Google Scholar Google Scholar Articles by Neven, B. Articles by Fischer, A. Search for Related Content PubMed PubMed Citation Articles by Neven, B. Articles by Fischer, A. Related Collections Immunobiology Transplantation Clinical Trials and Observations Social Bookmarking                   What's this?  Previous Article  |  Table of Contents  |  Next Article  TRANSPLANTATION Long-term outcome after hematopoietic stem cell transplantation of a single-center cohort of 90 patients with severe combined immunodeficiency Bénédicte Neven13, Sandrine Leroy1,4, Hélène Decaluwe1, Francoise Le Deist5, Capucine Picard1,3,5,6, Despina Moshous13, Nizar Mahlaoui1,3, Marianne Debré1,3, Jean-Laurent Casanova1,3,6, Liliane Dal Cortivo2,3,7, Yoann Madec8, Salima Hacein-Bey-Abina2,3,7, Geneviève de Saint Basile2,3, Jean-Pierre de Villartay2,3, Stéphane Blanche1,3, Marina Cavazzana-Calvo2,3,7, and Alain Fischer13 1 Unité d'Immuno-Hématologie et Rhumatologie Pédiatrique, Hôpital Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris, Paris, France; 2 Développement Normal et Pathologique du Système Immunitaire, Inserm U768, Hôpital Necker-Enfants Malades, Paris, France; 3 Université Paris-Descartes, Paris, France; 4 Center of Statistics in Medicine, Wolfson College, University of Oxford, Oxford, United Kingdom; 5 Centre d'Étude des Déficits Immunitaires, Hôpital Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris, Paris, France; 6 Laboratoire de Génétique Humaine des Maladies Infectieuses, Inserm U550, Hôpital Necker-Enfants Malades, Université Paris Descartes, Paris, France; 7 Département de Biothérapie, Hôpital Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris, Paris, France; and 8 Unité d'Épidémiologie des Maladies Émergentes, Institut Pasteur, Paris, France Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative treatment for severe combined immunodeficiency (SCID). Detailed assessment of the long-term outcome of HSCT, ie, the occurrence of clinical events and the quality and stability of immune reconstitution, is now required. We performed a single-center retrospective analysis of the long-term outcome of HSCT in 90-patient cohort followed for between 2 and 34 years (median, 14 years). Clinical events and immune reconstitution data were collected. Almost half the patients have experienced one or more significant clinical events, including persistent chronic graft-versus-host disease (GVHD), autoimmune and inflammatory manifestations, opportunistic and nonopportunistic infections, chronic human papilloma virus (HPV) infections, and a requirement for nutritional support. With the notable exception of severe HPV infection, these complications tend to become less common 15 years later after HSCT. A multivariate analysis showed that the occurrence of these events correlated with non–genoidentical donors, diagnosis of Artemis SCID, and quality of immune reconstitution. In most cases, HSCT enables long-term survival with infrequent sequelae. However, the occurrence of relatively late-onset complications is a concern that requires specific means of prevention and justifies careful patient follow-up. CiteULike    Connotea    Del.icio.us    Digg    Reddit    Technorati    What's this? This article has been cited by other articles: F. T. Hakim TRECing long-term success in SCID Blood, August 13, 2009; 114(7): 1287 - 1288. [Full Text] [PDF] M. Sarzotti-Kelsoe, C. M. Win, R. E. Parrott, M. Cooney, B. K. Moser, J. L. Roberts, G. D. Sempowski, and R. H. Buckley Thymic output, T-cell diversity, and T-cell function in long-term human SCID chimeras Blood, August 13, 2009; 114(7): 1445 - 1453. [Abstract] [Full Text] [PDF]

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