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Successful haematopoietic stem cell transplantation in 44 children from healthy siblings conceived after preimplantation HLA matching.
[wiskott-aldrich syndrome]
Haematopoietic
stem
cell
transplantation
(
HSCT
)
remains
the
best
therapeutic
option
for
many
acquired
and
inherited
paediatric
haematological
disorders
.
Unfortunately
,
the
probability
of
finding
an
HLA
matched
donor
is
limited
.
An
alternative
technique
is
PGD
combined
with
HLA
matching
,
which
offers
the
possibility
of
selecting
unaffected
embryos
that
are
HLA
compatible
with
the
sick
child
,
with
the
aim
of
possible
use
of
stem
cells
from
the
resulting
baby
in
future
.
Since
the
first
successful
report
for
Fanconi
anaemia
a
decade
ago
,
the
therapeutic
success
of
this
technique
was
reported
in
a
few
cases
and
for
a
limited
number
of
disorders
.
Here
,
we
report
full
recovery
of
44
sick
children
who
received
HSCT
from
healthy
infants
conceived
after
pre-implantation
HLA
matching
for
the
following
10
indications
;
beta
-thalassaemia
,
Wiskott-
Aldrich
syndrome
,
Fanconi
anaemia
,
sickle
cell
anaemia
,
acute
myeloid
leukaemia
,
acute
lymphoblastic
leukaemia
,
Glanzmann
's
thrombasthaenia
,
Diamond-
Blackfan
anaemia
,
X-
linked
adrenoleukodystrophy
and
mucopolysaccharidosis
type
I
.
No
serious
complications
were
observed
among
recipients
and
donors
.
Graft
failure
occurred
in
four
children
with
beta
-thalassaemia
where
a
second
HSCT
was
planned
.
Preimplantation
HLA
matching
is
a
reliable
technique
and
provides
a
realistic
option
for
couples
seeking
treatment
for
an
affected
child
when
no
HLA-matched
donor
is
available
.
Diseases
Validation
Diseases presenting
"paediatric haematological disorders"
symptom
wiskott-aldrich syndrome
x-linked adrenoleukodystrophy
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