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Cause-specific risks of childhood death in inherited epidermolysis bullosa.
[junctional epidermolysis bullosa]
To
determine
the
cause-
specific
risks
of
death
in
children
with
epidermolysis
bullosa
(
EB
)
.
Data
were
collected
throughout
the
continental
United
States
between
1986
and
2002
by
the
National
EB
Registry
.
The
study
design
is
cross-sectional
(
n
=
3280
)
,
containing
within
it
a
nested
randomly
sampled
longitudinal
subcohort
(
n
=
450
)
.
The
risk
of
death
during
infancy
and
childhood
was
greatest
in
junctional
EB
(
JEB
)
,
with
cumulative
and
conditional
risks
of
40
%
to
44
.
7
%
by
age
1
in
both
JEB
subtypes
,
rising
to
61
.
8
%
in
children
with
JEB
,
Herlitz
subtype
and
48
.
2
%
in
those
with
JEB
,
non-
Herlitz
subtype
(
JEB
-nH
)
by
age
15
.
In
decreasing
order
,
sepsis
,
failure
to
thrive
,
and
respiratory
failure
were
the
major
causes
of
death
in
children
with
JEB
,
plateauing
by
age
2
to
6
.
A
small
minority
of
children
with
epidermolysis
bullosa
simplex
,
Dowling-
Meara
subtype
was
at
risk
for
death
by
age
1
(
cumulative
risk
,
2
.
8
%
)
,
with
sepsis
and
respiratory
failure
accounting
for
cumulative
risks
of
1
.
9
%
and
0
.
9
%
.
Only
a
minority
of
children
with
recessive
dystrophic
epidermolysis
bullosa
,
Hallopeau-
Siemens
subtype
was
at
risk
of
death
(
cumulative
risk
=
8
%
by
age
15
)
.
Renal
failure
also
rarely
accounted
for
death
in
children
with
JEB
-nH
.
Infants
and
children
with
inherited
EB
,
particularly
those
with
JEB
,
are
at
significant
risk
of
death
as
a
result
of
disease
complications
.
Diseases
Validation
Diseases presenting
"respiratory failure"
symptom
achondroplasia
allergic bronchopulmonary aspergillosis
congenital diaphragmatic hernia
familial hypocalciuric hypercalcemia
gm1 gangliosidosis
harlequin ichthyosis
heparin-induced thrombocytopenia
inclusion body myositis
junctional epidermolysis bullosa
lamellar ichthyosis
legionellosis
lymphangioleiomyomatosis
proteus syndrome
pyruvate dehydrogenase deficiency
typhoid
von hippel-lindau disease
This symptom has already been validated