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Treatment of Chediak-Higashi syndrome by allogenic bone marrow
transplantation: report of 10 cases
E Haddad, F Le Deist, S Blanche, M Benkerrou, P Rohrlich, E Vilmer, C Griscelli and A Fischer
Unite d'Immuno-Hematologie Pediatrique, Hopital Necker-Enfants Malades,
Paris, France.
Chediak-Higashi syndrome is a rare condition characterized by
susceptibility to bacterial infections, defective natural killer activity,
and episodes of macrophage activation known as accelerated phases.
Chemotherapy can induce transient remission of the accelerated phase, but
relapses become less and less sensitive to treatment and ultimately lead to
death. Allogenic bone marrow transplantation (BMT) has been proposed as a
curative treatment for Chediak-Higashi syndrome. We report the outcome of
BMT in 10 such children. Seven received marrow from an HLA-identical
related donor and three from an HLA-nonidentical related donor. Three
patients died, two from a new accelerated phase after rejection of
transplanted bone marrow and one from cytomegalovirus (CMV) pneumonia. Six
of seven recipients of HLA- identical marrow and one of three recipients of
HLA-nonidentical marrow are alive and well without treatment 1.5 to 13
years after transplantation (median, 6.5 years). No manifestations of
accelerated phases have occurred in these seven patients, and significant
natural killer activity is detectable. Interestingly, BMT prevented
recurrence of accelerated phases in patients with limited numbers of
donor-type leukocytes after transplantation. Ocular and cutaneous albinism
were not corrected after transplantation. None of the patients developed
serious toxic reactions to the BMT conditioning regimen or have long- term
sequelae. These results show that HLA-identical BMT is an acceptable
curative treatment for Chediak-Higashi syndrome, whereas HLA- nonidentical
BMT remains an experimental approach.
Volume 85,
Issue 11,
pp. 3328-3333,
06/01/1995
Copyright © 1995 by The American Society of Hematology
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