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Bone marrow transplantation (BMT) in Europe for primary immunodeficiencies
other than severe combined immunodeficiency: a report from the European
Group for BMT and the European Group for Immunodeficiency
A Fischer, P Landais, W Friedrich, B Gerritsen, A Fasth, F Porta, A Vellodi, M Benkerrou, JP Jais and M Cavazzana-Calvo
Departement de Pediatrie et INSERM U 132, Hopital des Enfants-Malades,
Paris, France.
Bone marrow (BM) transplantations performed between 1977 and 1991 at 13
European centers in 149 patients with 11 different primary immunodeficiency
(ID) diseases (excluding severe combined immunodeficiency) were analyzed
retrospectively. Overall survival among recipients of HLA genetically
identical BM (n = 56) was 66%. Since October 1985, the date of a previous
survey, a significant improvement in survival has been achieved in most ID
diseases (overall survival, 81.5% v 51.7%; P < .01), primarily because
of a decrease in the frequency of infectious complications. In long-term
survivors, disease correction is excellent, with minimal sequelae in most
patients. In 22 patients who received closely matched BM (ie, from
phenotypically identical related donors, matched unrelated donors, or one
HLA-ag- mismatched related donors), the survival rate (45.5%) was not
significantly better than among 71 recipients of BM with 2 or 3 mismatched
HLA antigens (38%). In the latter group, favorable outcome was associated
with younger age, with transplantation since October 1985 (47% v 25%; P
< .0001), and with a diagnosis of leukocyte adhesion deficiency. The
improvement in outcome was mainly because of a higher engraftment rate and
a decrease in the frequency of infections, although Epstein-Barr
virus-induced B-lymphocyte proliferative disorders occurred in 16 patients
(mainly those with Wiskott-Aldrich syndrome), 10 of whom died. The
improvement in engraftment corresponded to the introduction of treatment in
vivo with anti-LFA-1 antibody to prevent rejection of T-cell-depleted
grafts (74% engraftment and 45% survival in 38 treated patients versus
37.5% and 21%, respectively, in 24 untreated patients.
Volume 83,
Issue 4,
pp. 1149-1154,
02/15/1994
Copyright © 1994 by The American Society of Hematology
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