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Prioritization of therapy uncertainties in Dystrophic Epidermolysis Bullosa: where should research direct to? an example of priority setting partnership in very rare disorders.
[dystrophic epidermolysis bullosa]
Dystrophic
Epidermolysis
Bullosa
(
DEB
)
is
a
rare
genodermatosis
(
7
cases
per
million
)
that
causes
blisters
and
erosions
with
minor
trauma
in
skin
and
mucosa
,
and
other
systemic
complications
.
A
recently
updated
systematic
review
showed
that
the
research
evidence
about
DEB
therapies
is
poor
.
As
new
trials
in
DEB
are
difficult
and
expensive
,
it
is
important
to
prioritizise
research
that
patients
and
clinicians
consider
more
relevant
.
To
describe
and
prioritize
the
most
important
uncertainties
about
DEB
treatment
shared
by
patients
,
carers
and
health
care
professionals
(
HCPs
)
in
order
to
promote
research
in
those
areas
.
A
DEB
Priority
Setting
Partnership
(
PSP
)
was
established
,
including
patients
,
carers
and
HCPs
.
DBE
uncertainties
were
gathered
from
patients
and
clinicians
,
and
prioritized
in
a
transparent
process
,
using
the
methodology
advocated
by
the
James
Lind
Alliance
.
In
the
consultation
stage
,
323
uncertainties
were
submitted
by
58
participants
.
Once
the
duplicated
and
non-treatment
uncertainties
were
removed
,
the
remainder
were
reduced
to
a
list
of
24
most
voted
questions
.
These
24
uncertainties
were
prioritized
in
a
final
workshop
where
a
balanced
number
of
patients
,
carers
and
HCPs
selected
the
top
10
therapy
uncertainties
.
The
final
list
includes
interventions
in
wound
care
,
itch
and
pain
management
,
treatment
and
prevention
of
syndactyly
,
cancer
prevention
and
future
promising
therapies
.
The
final
list
of
the
top
10
treatment
uncertainties
on
the
management
of
DEB
provides
guidance
for
researchers
and
funding
bodies
,
to
ensure
that
future
research
answers
questions
that
are
important
to
both
clinicians
and
patients
.
The
method
proposed
by
the
James
Lind
Alliance
is
feasible
for
very
rare
disorders
.
Diseases
Validation
Diseases presenting
"updated systematic review showed that the research evidence"
symptom
dystrophic epidermolysis bullosa
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