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Bone marrow transplantation in major histocompatibility complex class II
deficiency: a single-center study of 19 patients
C Klein, M Cavazzana-Calvo, F Le Deist, N Jabado, M Benkerrou, S Blanche, B Lisowska-Grospierre, C Griscelli and A Fischer
Unite d'Immunologie et d'Hematologie, Hopital Necker Enfants Malades,
Paris, France.
Major histocompatibility complex (MHC) class II deficiency (bare lymphocyte
syndrome) is a rare inborn error of the immune system characterized by
impaired antigen presentation and combined immunodeficiency. It causes
severe and unremitting infections leading to progressive liver and lung
dysfunctions and death during childhood. As in other combined
immunodeficiency disorders, bone marrow transplantation (BMT) is considered
the treatment of choice for MHC class II deficiency. We analyzed the files
of 19 patients who have undergone BMT in our center. Of the 7 patients who
underwent HLA- identical BMT, 3 died in the immediate posttransplant period
of severe viral infections, whereas the remaining 4 were cured, with
recovery of normal immune functions. Of the 12 patients who underwent
HLA-haplo- identical BMT, 3 were cured, 1 was improved by partial
engraftment, 7 died of infectious complications due to graft failure or
rejection, and 1 is still immunodeficient because of engraftment failure. A
favorable outcome in the HLA-non-identical BMT group was associated with an
age of less than 2 years at the time of transplantation. All the patients
with stable long-term engraftment had persistently low CD4 counts after
transplantation (105 to 650/microL at last follow up), but no clear
susceptibility to opportunistic infections despite persisting MHC class II
deficiency on thymic epithelium and other nonhematopoietic cells. We
conclude that HLA-identical and -haploidentical BMT can cure MHC class II
deficiency, although the success rate of haploidentical BMT is lower than
that in other combined immunodeficiency syndromes. HLA- haploidentical BMT
should preferably be performed in the first 2 years of life, before the
acquisition of chronic virus carriage and sequelae of infections.
Volume 85,
Issue 2,
pp. 580-587,
01/15/1995
Copyright © 1995 by The American Society of Hematology
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