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Influence of severe combined immunodeficiency phenotype on the outcome of HLA non-identical, T-cell-depleted bone marrow transplantation: a retrospective European survey from the European group for bone marrow transplantation and the european society for immunodeficiency.
J Pediatr. 1999 Jun; 134(6):740-8.JPed

Abstract

We analyzed the outcomes of 214 HLA non-identical T-cell-depleted bone marrow transplantations (BMTs), performed in 178 consecutive patients for treatment of severe combined immunodeficiencies (SCID). Patients were treated in 18 European centers between 1981 and March 1995. SCID variants, that is, absence of T and B lymphocytes (B-) or absence of T cells with presence of B lymphocytes (B+) were found to have a major influence on outcome. The disease-free survival was significantly better for patients with B+ SCID (60%) as compared with patients with B- SCID (35%) (P =.002), with a median follow-up of 57 months and 52 months, respectively. Other factors associated with a poor prognosis were the presence of a lung infection before BMT (odds ratio = 2.47 [1.99-2.94]) and the use of monoclonal antibodies for T-cell depletion of the graft (odds ratio = 1.67 [1. 18-2.15]). Additional factors influencing outcome were age at BMT (<6 months) and period during which BMT was performed. Better results were achieved after 1991. Reduced survival of patients with B- SCID was associated with a higher incidence of early deaths from infection, a diminished rate of marrow engraftment, a trend to a higher incidence of chronic graft-versus-host disease, and slower kinetics of T/B immune function development. In both groups of patients, the use of busulfan (8 mg/kg total dose) and cyclophosphamide (200 mg/kg total dose) as a conditioning regimen provided the best cure rate (74% for patients with B+ SCID and 43% for patients with B- SCID, respectively), although results were not statistically significantly different from other regimens. This retrospective analysis should lead to the design of adapted measures to the performance of HLA non-identical BMT in patients with distinct SCID conditions.

Authors+Show Affiliations

Unité d'Immunologie et d'Hématologie Pédiatriques, Département de Pédiatrie, Hôpital Necker-Enfants Malades; Paris, France.No affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Clinical Trial
Comparative Study
Journal Article
Multicenter Study
Research Support, Non-U.S. Gov't

Language

eng

PubMed ID

10356144

Citation

Bertrand, Y, et al. "Influence of Severe Combined Immunodeficiency Phenotype On the Outcome of HLA Non-identical, T-cell-depleted Bone Marrow Transplantation: a Retrospective European Survey From the European Group for Bone Marrow Transplantation and the European Society for Immunodeficiency." The Journal of Pediatrics, vol. 134, no. 6, 1999, pp. 740-8.
Bertrand Y, Landais P, Friedrich W, et al. Influence of severe combined immunodeficiency phenotype on the outcome of HLA non-identical, T-cell-depleted bone marrow transplantation: a retrospective European survey from the European group for bone marrow transplantation and the european society for immunodeficiency. J Pediatr. 1999;134(6):740-8.
Bertrand, Y., Landais, P., Friedrich, W., Gerritsen, B., Morgan, G., Fasth, A., Cavazzana-Calvo, M., Porta, F., Cant, A., Espanol, T., Müller, S., Veys, P., Vossen, J., Haddad, E., & Fischer, A. (1999). Influence of severe combined immunodeficiency phenotype on the outcome of HLA non-identical, T-cell-depleted bone marrow transplantation: a retrospective European survey from the European group for bone marrow transplantation and the european society for immunodeficiency. The Journal of Pediatrics, 134(6), 740-8.
Bertrand Y, et al. Influence of Severe Combined Immunodeficiency Phenotype On the Outcome of HLA Non-identical, T-cell-depleted Bone Marrow Transplantation: a Retrospective European Survey From the European Group for Bone Marrow Transplantation and the European Society for Immunodeficiency. J Pediatr. 1999;134(6):740-8. PubMed PMID: 10356144.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Influence of severe combined immunodeficiency phenotype on the outcome of HLA non-identical, T-cell-depleted bone marrow transplantation: a retrospective European survey from the European group for bone marrow transplantation and the european society for immunodeficiency. AU - Bertrand,Y, AU - Landais,P, AU - Friedrich,W, AU - Gerritsen,B, AU - Morgan,G, AU - Fasth,A, AU - Cavazzana-Calvo,M, AU - Porta,F, AU - Cant,A, AU - Espanol,T, AU - Müller,S, AU - Veys,P, AU - Vossen,J, AU - Haddad,E, AU - Fischer,A, PY - 1999/6/4/pubmed PY - 1999/6/4/medline PY - 1999/6/4/entrez SP - 740 EP - 8 JF - The Journal of pediatrics JO - J Pediatr VL - 134 IS - 6 N2 - We analyzed the outcomes of 214 HLA non-identical T-cell-depleted bone marrow transplantations (BMTs), performed in 178 consecutive patients for treatment of severe combined immunodeficiencies (SCID). Patients were treated in 18 European centers between 1981 and March 1995. SCID variants, that is, absence of T and B lymphocytes (B-) or absence of T cells with presence of B lymphocytes (B+) were found to have a major influence on outcome. The disease-free survival was significantly better for patients with B+ SCID (60%) as compared with patients with B- SCID (35%) (P =.002), with a median follow-up of 57 months and 52 months, respectively. Other factors associated with a poor prognosis were the presence of a lung infection before BMT (odds ratio = 2.47 [1.99-2.94]) and the use of monoclonal antibodies for T-cell depletion of the graft (odds ratio = 1.67 [1. 18-2.15]). Additional factors influencing outcome were age at BMT (<6 months) and period during which BMT was performed. Better results were achieved after 1991. Reduced survival of patients with B- SCID was associated with a higher incidence of early deaths from infection, a diminished rate of marrow engraftment, a trend to a higher incidence of chronic graft-versus-host disease, and slower kinetics of T/B immune function development. In both groups of patients, the use of busulfan (8 mg/kg total dose) and cyclophosphamide (200 mg/kg total dose) as a conditioning regimen provided the best cure rate (74% for patients with B+ SCID and 43% for patients with B- SCID, respectively), although results were not statistically significantly different from other regimens. This retrospective analysis should lead to the design of adapted measures to the performance of HLA non-identical BMT in patients with distinct SCID conditions. SN - 0022-3476 UR - https://www.unboundmedicine.com/medline/citation/10356144/Influence_of_severe_combined_immunodeficiency_phenotype_on_the_outcome_of_HLA_non_identical_T_cell_depleted_bone_marrow_transplantation:_a_retrospective_European_survey_from_the_European_group_for_bone_marrow_transplantation_and_the_european_society_for_immunodeficiency_ L2 - https://linkinghub.elsevier.com/retrieve/pii/S0022-3476(99)70291-X DB - PRIME DP - Unbound Medicine ER -