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Long-term outcomes of nonconditioned patients with severe combined immunodeficiency transplanted with HLA-identical or haploidentical bone marrow depleted of T cells with anti-CD6 mAb.
[omenn syndrome]
Between
1981
and
1995
,
20
children
with
severe
combined
immunodeficiency
(
SCID
;
median
age
at
transplant
,
6
.
5
[
range
,
0
.
5
-
145
]
mo
,
12
with
serious
infection
)
were
treated
with
haploidentical
T
cell-depleted
(
anti-
CD
6
antibody
)
bone
marrow
(
median
number
of
5
.
7
[
0
.
8
-
18
.
8
]
x
10
(
8
)
nucleated
cells
/
kg
)
from
mismatched
related
donors
(
MMRDs
)
,
and
5
children
with
SCID
(
median
age
at
transplant
,
1
.
8
[
0
.
5
-
5
.
0
]
mo
,
1
with
serious
infection
)
were
given
unmanipulated
bone
marrow
from
matched
related
donors
(
MRDs
)
.
No
conditioning
or
graft-versus-host
disease
(
GvHD
)
prophylaxis
was
used
.
To
assess
the
outcomes
of
patients
with
SCID
who
received
bone
marrow
from
MMRDs
or
MRDs
.
We
reviewed
the
medical
records
of
these
25
consecutive
patients
with
SCID
(
4
with
Omenn
syndrome
)
.
Of
the
20
patients
who
received
bone
marrow
from
MMRDs
,
12
engrafted
,
10
survived
at
a
median
age
of
15
.
2
[
10
.
0
-
19
.
1
]
years
,
4
had
chronic
GvHD
(
lung
,
intestine
,
skin
)
,
5
required
intravenous
immunoglobulin
,
and
8
attended
school
or
college
.
Two
of
5
patients
who
died
had
chronic
GvHD
,
and
2
developed
lymphoproliferative
disease
.
Of
the
5
patients
who
received
bone
marrow
from
MRDs
,
5
engrafted
,
5
survived
at
a
median
age
of
23
.
3
[
18
.
5
-
26
]
years
,
1
had
chronic
GvHD
(
lung
,
skin
)
,
2
required
intravenous
immunoglobulin
,
and
4
attended
school
or
college
.
Treatment
of
critically
ill
patients
with
SCID
with
anti-
CD
6
antibody
T
cell-depleted
MMRD
marrow
resulted
in
an
overall
50
%
long
-term
survival
of
patients
(
83
%
survival
of
those
engrafted
)
.
The
principal
barriers
to
long
-term
survival
were
delay
in
diagnosis
,
life-threatening
infection
,
failure
to
engraft
,
and
chronic
GvHD
.
Educational
goals
were
achieved
in
most
of
the
survivors
.
Diseases
Validation
Diseases presenting
"developed lymphoproliferative disease. of the 5 patients"
symptom
omenn syndrome
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