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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.
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Submitted September 8, 2008
Accepted December 23, 2008
Long-term outcome after haematopoietic stem cell transplantation of a single-centre cohort of 90 patients with severe combined immunodeficiency: Long-term outcome of HSCT in SCID
Benedicte Neven, Sandrine Leroy, Helene Decaluwe, Francoise Le Deist, Capucine Picard, Despina Moshous, Nizar Mahlaoui, Marianne Debre, Jean-Laurent Casanova, Liliane Dal Cortivo, Yoann Madec, Salima Hacein-Bey-Abina, Genevieve de Saint Basile, Jean-Pierre de Villartay, Stephane Blanche, Marina Cavazzana-Calvo, and Alain Fischer*
Unite d'immuno-hematologie et rhumatologie pediatrique, Hopital Necker-Enfants Malades, Assistance publique-Hopitaux de Paris, Paris, France
Center of statistics in Medicine, Wolfson College, University of Oxford, Oxford, United Kingdom
Centre d'etude des déficits immunitaires, Hopital Necker-Enfants Malades, Assistance publique-Hopitaux de Paris, Paris, France
Developpement normal et pathologique du systeme immunitaire, Institut National de la Sante et de la Recherche Medicale, U768, Hopital Necker-Enfants Malades, Paris, France
Laboratoire de Genetique Humaine des Maladies Infectieuses, Institut National de la Sante et de la Recherche Medicale, U550, Hopital Necker-Enfants Malades, Paris, France
Departement de biotherapie, Hopital Necker-Enfants Malades, Assistance publique- Hopitaux de Paris, Paris, France
Unite d'epidemiologie des maladies emergentes, Institut Pasteur, Paris, France
* Corresponding author; email: alain.fischer{at}nck.aphp.fr.
Allogeneic haematopoietic stem cell transplantation (HSCT) is a curative treatment for severe combined immunodeficiency (SCID). Detailed assessment of the long-term outcome of HSCT, i.e. the occurrence of clinical events and the quality and stability of immune reconstitution is now required. We performed a single-centre retrospective analysis of the long-term outcome of HSCT in 90-patient cohort followed up 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 non-opportunistic 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-geno-identical donors, diagnosis of Artemis SCID and quality of immune reconstitution. In most cases, HSCT enables long-term survival with infrequent sequellae. However, the occurrence of relatively late-onset complications is a concern which requires specific means of prevention and justifies careful patient follow-up.
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