Neonatal bone marrow transplantation for severe combined immunodeficiency
L Kane, A R Gennery, B N A Crooks, T J Flood, M Abinun, A J Cant
Department of
Paediatric Immunology, Newcastle General Hospital, Newcastle upon Tyne,
UK
Correspondence to: Dr Gennery, Department of Paediatric Immunology, Newcastle General Hospital, Westgate Road, Newcastle upon Tyne NE4 6BE, UK ARGennery{at}aol.com
Accepted 8 March 2001
AIMS
To evaluate
outcome following neonatal bone marrow transplantation (BMT) for severe
combined immunodeficiency (SCID) when there is a family history of a
previously affected sibling, and to compare results with those
published for in utero BMT.
METHODSA
retrospective review of cases referred and transplanted between 1987 and 1999, focusing on infectious and graft versus host disease (GvHD)
complications after BMT, and T and B lymphocyte function. Thirteen
patients received 18 stem cell transplants: four whole marrow, one cord
blood, 10 parental T cell depleted (TCD) haplo-identical, and three TCD
unrelated donor BMT. Nine were conditioned with busulphan and cyclophosphamide.
RESULTSAll are alive
and well (six months to 11.5 years after BMT). Six had grade I-II
acute GvHD and two chronic GvHD (now resolved). Three had a top up BMT
for poor T cell function, one had a third BMT for graft failure and
chronic GvHD, and one had a third BMT for graft failure. Twelve have
good in vitro proliferation to T cell mitogens, and all have normal
serum IgA levels. Three receive intravenous immunoglobulin; for one of
these, it is less than one year since BMT. Nine are above the 2nd
centile, and 10 of 12 old enough to be assessed have normal neurodevelopment.
CONCLUSIONThese
results are better than those published for in utero BMT for SCID.
Early postnatal BMT should be the preferred option in neonatal SCID.
Keywords: severe combined immunodeficiency; bone marrow transplantation; in utero transplantation
© 2001 by Archives of Disease in Childhood
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