Influence of severe combined immunodeficiency phenotype on the outcome of HLA non-identical, T-cell–depleted bone marrow transplantationA retrospective European survey from the European Group for Bone Marrow Transplantation and the European Society for Immunodeficiency☆,☆☆,★
Section snippets
PATIENTS AND METHODS
Between 1981 and March 1995, 178 patients with B– or B+ SCID received HLA non-identical T-cell–depleted BMT in 18 cooperating centers in Europe. Data on all BMTs performed during this period were collected from members of the EBMT/ESID. The analysis was performed on data available up to December 1, 1995, giving a minimum follow-up period of 6 months and a maximum of 14 years. B+ SCID was defined as blood T-cell counts (excluding maternal T cells) <250/μL and B-cell counts >50/μL. B– SCID was
Evidence of Better Prognosis for Patients with B+ SCID
As of December 1, 1995, 73 of 122 patients with B+ SCID and 20 of 56 patients with B– SCID were alive with T-cell engraftment, after T-cell–depleted non-identical BMT with a median follow-up of 57 months (range, 6 to 162 months) and 52 months (range, 6 to 161 months), respectively. Two additional patients with B+ SCID were alive and well after receiving a second BMT with a matched unrelated donor. The survival with T-cell engraftment/function was significantly better for patients with B+ SCID
DISCUSSION
In this retrospective study we found that the results of HLA non-identical T-cell–depleted BMT were significantly better for the group of patients with B+ SCID than for the group of patients with B– SCID. The 2 populations of patients were comparable for age at BMT, clinical status before BMT (including frequency of lung infection associated with poor prognosis), period during which BMT was performed, HLA incompatibility, conditioning regimen, modalities of T-cell depletion, donor/recipient sex
Acknowledgements
The following European centers have contributed to this study: Barcelona (Dr T. Espanol); Bruxelles (Dr A. Ferster); Belgrad (Dr M. Abinun); Brescia (Dr F. Porta); Gotebörg (Dr A. Fasth); Hannover (Dr W. Ebell); Leiden (Dr J. Vossen, Dr B. Gerritsen); London (Dr G. Morgan, Dr P. Veys); Lyon (Dr G. Souillet); Mainz (Dr O. Schofer); Nancy (Dr P. Bordigoni); Newcastle (Dr A. Cant); Paris (Dr A. Fischer); Pavia (Dr Locatelli); Ulm (Dr W. Friedrich); Utrecht (Dr B. Zegers); Zurich (Dr R. Seger).
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Supported by the European Group for Bone Marrow Transplanation, the European Society for Immunodeficiency, and the Biomed 2PL 96007 concerted action.
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Reprint requests: A. Fischer. Unité d’Immunologie et d’Hématologie Pédiatriques, Département de Pédiatrie, Hôpital Necker-Enfants Malades, 149 rue de Sèvres, 75743 Paris Cedex 15, France.
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