Acta Haematologica Polonica,
Journal Year:
2021,
Volume and Issue:
52(4), P. 217 - 224
Published: Aug. 31, 2021
Hematopoietic
stem
cell
transplantation
(HSCT)
is
now
evolving
into
a
standard
treatment
in
some
autoimmune
diseases
(AD)
alongside
other
modern
therapy.
The
main
indications
are
multiple
sclerosis
and
systemic
for
which
HSCT
has
become
an
integral
standard-of-care
part
of
algorithms.
From
1994
to
the
beginning
2021,
data
from
EBMT
Registry
indicates
that
3,442
patients
(60%
females,
40%
males;
91%
adults,
9%
pediatric)
received
3,514
transplant
procedures
diseases,
with
over
90%
receiving
autologous
transplant.
Autoimmune
currently
fastest
growing
indication
European
Society
Blood
Marrow
Transplantation
(EBMT),
whilst
allogeneic
ADs
mainly
restricted
pediatrics,
especially
genetic
component.
Patient
selection
plays
key
role
providing
best
risk/benefit
ratio
procedure.
Intensity
conditioning
regimen
center
experience
also
important.
Ultimately,
future
depends
on
care
therapy,
influences
uptake
within
national/international
disease
specialist
communities.
Further
studies
necessary
order
establish
relative
benefit
current/future
each
disease,
define
mechanisms,
develop
clinical
biomarkers
help
select
monitor
patients,
health
economic
benefits
public
delivery.
We
present
current
perspective
summarizing
activity
across
EBMT,
including
centers
Poland.
Bone Marrow Transplantation,
Journal Year:
2022,
Volume and Issue:
57(8), P. 1217 - 1239
Published: May 19, 2022
For
over
two
decades,
the
EBMT
has
updated
recommendations
on
indications
for
haematopoietic
cell
transplantation
(HCT)
practice
based
clinical
and
scientific
developments
in
field.
This
is
eighth
special
report
HCT
haematological
diseases,
solid
tumours
immune
disorders.
Our
aim
to
provide
general
guidance
according
prevailing
countries
centres.
In
order
inform
patient
decisions,
these
must
be
considered
conjunction
with
risk
of
disease,
procedure
non-transplant
strategies,
including
evolving
cellular
therapies.
techniques
are
constantly
we
make
no
specific
recommendations,
but
encourage
harmonisation
practice,
where
possible,
ensure
experience
across
can
meaningfully
aggregated
via
registry
outputs.
We
also
recommend
working
JACIE
accreditation
standards
maintain
quality
laboratory
components
benchmarking
survival
outcomes.
Since
last
edition,
COVID-19
pandemic
affected
decision
making
activity
indications.
Although
full
impact
yet
determined,
that
delivered
ongoing
reference
national
guidance,
accordance
current
local
conditions.
Bone Marrow Transplantation,
Journal Year:
2023,
Volume and Issue:
58(6), P. 647 - 658
Published: March 6, 2023
Abstract
In
2021,
47,412
HCT
(19,806
(42%)
allogeneic
and
27,606
(58%)
autologous)
in
43,109
patients
were
reported
by
694
European
centers.
3494
received
advanced
cellular
therapies,
2524
of
which
CAR-T
treatments,
an
additional
3245
DLI.
Changes
compared
to
the
previous
year
treatment
(+35%),
+5.4%,
autologous
+3.9%,
more
pronounced
non-malignant
disorders.
Main
indications
for
myeloid
malignancies
10,745
(58%),
lymphoid
5127
(28%)
disorders
2501
(13%).
22,129
(90%)
solid
tumors
1635
(7%).
HCT,
use
haploidentical
donors
decreased
−0.9%
while
unrelated
sibling
increased
+4.3%
+9%.
Cord
blood
−5.8%.
Pediatric
overall
+5.6%
(+6.9%
+1.6%
autologous).
Increase
was
mainly
restricted
high-income
countries.
The
drop
activity
2020
partially
recovered
second
SARS-CoV-2
pandemic.
transplant
community
confronted
with
pandemic
challenge,
continued
providing
access
treatment.
This
annual
EBMT
report
reflects
current
activities
useful
health
care
resource
planning.
Bone Marrow Transplantation,
Journal Year:
2024,
Volume and Issue:
59(6), P. 803 - 812
Published: March 4, 2024
Abstract
In
2022,
46,143
HCT
(19,011
(41.2%)
allogeneic
and
27,132
(58.8%)
autologous)
in
41,854
patients
were
reported
by
689
European
centers.
4329
received
advanced
cellular
therapies,
3205
of
which
CAR-T.
An
additional
2854
DLI.
Changes
compared
to
the
previous
year
an
increase
CAR-T
treatments
(+27%)
decrease
(−4.0%)
autologous
(−1.7%).
Main
indications
for
myeloid
malignancies
(10,433;
58.4%),
lymphoid
(4,674;
26.2%)
non-malignant
disorders
(2572;
14.4%).
lymphomas
(7897;
32.9%),
PCD
(13,694;
57.1%)
solid
tumors
(1593;
6.6%).
HCT,
use
sibling
donors
decreased
−7.7%,
haploidentical
−6.3%
unrelated
−0.9%.
Overall
cord
blood
−16.0%.
Use
allogeneic,
a
lesser
degree
likely
reflecting
availability
new
treatment
modalities,
including
small
molecules,
bispecific
antibodies,
cells.
Pediatric
activity
remains
stable
(+0.3%)
with
differences
between
HCT.
continues
reached
cumulative
total
9039
treated
wide
across
countries.
After
many
years
continuous
growth,
application
seems
have
slowed
down.
EClinicalMedicine,
Journal Year:
2024,
Volume and Issue:
69, P. 102476 - 102476
Published: Feb. 10, 2024
Autoimmune
diseases
(ADs)
are
characterized
by
loss
of
immune
tolerance,
high
chronicity,
with
substantial
morbidity
and
mortality,
despite
conventional
immunosuppression
(IS)
or
targeted
disease
modifying
therapies
(DMTs),
which
usually
require
repeated
administration.
Recently,
novel
cellular
(CT),
including
mesenchymal
stromal
cells
(MSC),
Chimeric
Antigen
Receptors
T
(CART)
regulatory
(Tregs),
have
been
successfully
adopted
in
ADs.
An
international
expert
panel
the
European
Society
for
Blood
Marrow
Transplantation
International
Cell
Gene
Therapy,
reviewed
all
available
evidence,
based
on
current
literature
practices,
use
MSC,
CART
Tregs,
AD
patients
rheumatological,
neurological,
gastroenterological
indications.
Expert-based
consensus
recommendations
best
practice
quality
patient
care
were
developed
to
support
clinicians,
scientists,
their
multidisciplinary
teams,
as
well
providers
will
be
regularly
updated.
Bone Marrow Transplantation,
Journal Year:
2022,
Volume and Issue:
57(5), P. 742 - 752
Published: Feb. 22, 2022
Abstract
In
2020,
45,364
HCT
in
41,016
patients,
18,796
(41%)
allogeneic
and
26,568
(59%)
autologous
690
centers
were
reported.
Changes
observed
as
follows:
total
number
of
−6.5%,
−5.1%,
−7.5%,
more
pronounced
non-malignant
disorders
for
autoimmune
disease
HCT.
Main
indications
myeloid
malignancies
10,441
(25%),
lymphoid
26,120
(64%)
2532
(6%).
A
continued
growth
CAR-T
cellular
therapies
to
1874
(+65%)
patients
2020
was
observed.
HCT,
the
use
haploidentical
donors
increased
while
unrelated
sibling
decreased.
Cord
blood
by
11.7%
first
time
since
2012.
There
a
significant
increase
non-myeloablative
but
drop
myeloablative
conditioning
marrow
stem
cell
source.
We
interpreted
these
changes
being
due
SARS-CoV-2
pandemic
starting
early
Europe
provided
additional
data
reflecting
varying
impact
across
selected
countries
larger
cities.
The
transplant
community
confronted
with
challenge,
providing
access
treatment.
This
annual
report
EBMT
reflects
current
activities
useful
health
care
planning.
Bone Marrow Transplantation,
Journal Year:
2022,
Volume and Issue:
57(7), P. 1055 - 1062
Published: May 16, 2022
Abstract
Autoimmune
diseases
(ADs)
represent
a
heterogenous
group
of
complex
with
increasing
incidence
in
Western
countries
and
are
major
cause
morbidity.
Hematopoietic
stem
cell
transplantation
(HSCT)
has
evolved
over
the
last
25
years
as
specific
treatment
for
patients
severe
ADs,
through
eradication
pathogenic
immunologic
memory
profound
immune
renewal.
HSCT
ADs
is
recently
facing
unique
developmental
phase
across
transplant
centers.
This
review
provides
comprehensive
overview
recent
evidence
developments
area,
including
fundamentals
preclinical
research,
clinical
studies
neurologic,
rheumatologic
gastroenterologic
diseases,
which
indications
at
present,
along
rarer
indications.
Moreover,
we
describe
interwoven
challenges
delivering
more
advanced
cellular
therapies,
exploiting
mesenchymal
cells,
regulatory
T
cells
potentially
CAR-T
affected
by
ADs.
Overall,
discuss
past
current
indications,
efficacy,
associated
risks
benefits,
future
directions
therapies
severe/refractory
integrating
available
literature
European
Society
Blood
Marrow
Transplantation
(EBMT)
registry
data.
Frontiers in Immunology,
Journal Year:
2022,
Volume and Issue:
12
Published: Feb. 1, 2022
Multiple
sclerosis
(MS)
is
a
central
nervous
system
(CNS)
disorder,
which
mediated
by
an
abnormal
immune
response
coordinated
T
and
B
cells
resulting
in
areas
of
inflammation,
demyelination,
axonal
loss.
Disease-modifying
treatments
(DMTs)
are
available
to
dampen
the
inflammatory
aggression
but
ineffective
many
patients.
Autologous
hematopoietic
stem
cell
transplantation
(HSCT)
has
been
used
as
treatment
patients
with
highly
active
disease,
achieving
long-term
clinical
remission
most.
The
rationale
intervention
eradicate
autoreactive
lympho-ablative
regimens
restore
tolerance.
Immunological
studies
have
demonstrated
that
autologous
HSCT
induces
renewal
TCR
repertoires,
resurgence
regulatory
cells,
depletion
proinflammatory
subsets,
suggesting
“resetting”
immunological
memory.
Although
our
understanding
effects
progressed,
further
work
required
characterize
mechanisms
underlie
efficacy.
Considering
memory
disease-promoting
stem-like
multipotent
progenitors
involved
self-regeneration
effector
investigating
reconstitution
compartment
subsets
following
could
elucidate
those
mechanisms.
Since
all
subjects
need
be
optimally
protected
from
vaccine-preventable
diseases
(including
COVID-19),
there
ensure
vaccination
undergoing
effective
safe.
Additionally,
study
HSCT-treated
means
evaluating
responses
distinguish
broad
immunosuppression
resetting.
La Revue de Médecine Interne,
Journal Year:
2024,
Volume and Issue:
45(2), P. 79 - 99
Published: Jan. 13, 2024
Hematopoietic
stem
cell
transplantation
(HSCT)
for
severe
ADs
was
developed
over
the
past
25
years
and
is
now
validated
by
national
international
medical
societies
early
systemic
sclerosis
(SSc)
relapsing-remitting
multiple
(MS)
available
as
part
of
routine
care
in
accredited
center.
HSCT
also
recommended,
with
varying
levels
evidence,
an
alternative
treatment
several
ADs,
when
refractory
to
conventional
therapy,
including
specific
cases
connective
tissue
diseases
or
vasculitis,
inflammatory
neurological
diseases,
more
rarely
Crohn's
disease.
The
aim
this
document
provide
guidelines
current
indications,
procedures
follow-up
ADs.
Patient
safety
considerations
are
central
guidance
on
patient
selection
conditioning,
always
at
MATHEC
multidisciplinary
team
meeting
(MDTM)
based
recent
(less
than
3
months)
thorough
evaluation.
procedural
aspects
then
carried
out
within
appropriately
experienced
Joint
Accreditation
Committee
International
Society
Cellular
Therapy
SFGM-TC
centres
close
collaboration
specialist.
These
French
recommendations
were
performed
according
HAS/FAI2R
standard
operating
coordinated
Île-de-France
Reference
Centre
Rare
Systemic
Autoimmune
Diseases
(CRMR
MATHEC)
Filière
FAI2R
association
MaRIH.
task
force
consisted
patients
64
clinical
experts
from
various
specialties
centres.
data-derived
consensus-derived
will
help
clinicians
propose
their
evidence-based
way.
give
directions
future
research
area.
be
updated
newly
emerging
data.
Of
note,
other
therapies
that
have
not
yet
been
approved
practice
subject
ongoing
addressed
document.
La
greffe
de
cellules
souches
hématopoïétiques
(GCSH)
pour
les
maladies
auto-immunes
(MAI)
sévères
s'est
développée
au
cours
des
dernières
années
et
est
maintenant
validée
par
sociétés
savantes
nationales
internationales
la
sclérodermie
systémique
sévère
précoce
sclérose
en
plaques
récurrente-rémittente
disponible
dans
le
cadre
soins
courants
accrédités.
GCSH
également
recommandée,
avec
niveaux
preuve
variables,
comme
traitement
alternatif
plusieurs
MAI,
lorsqu'elles
sont
réfractaires
aux
thérapies
conventionnelles,
comprenant
cas
spécifiques
connectivites
ou
vascularites
;
neuro-inflammatoires
plus
rarement
Crohn
sévères.
L'objectif
ce
fournir
recommandations
sur
indications
actuelles,
procédures
suivi
MAI.
sécurité
reste
l'élément
majeur
guidant
sélection
conditionnement
patients,
toujours
validées
lors
réunion
concertation
pluridisciplinaire
nationale
base
d'une
évaluation
exhaustive
récente
(moins
mois)
du
patient.
ensuite
effectués
expérimentés
accrédités
SFGM-TC,
étroite
spécialiste
Ces
françaises
ont
été
réalisées
conformément
opérationnelles
coordonnées
référence
systémiques
rares
d'Île-de-France
sein
Le
groupe
travail
était
composé
cliniciens
issus
différentes
spécialités
différents
français.
issues
données
publiées
consensus
d'experts
aideront
à
proposer
leurs
atteints
MAI
sévère.
ouvrent
perspectives
recherche
clinique.
seront
régulièrement
mises
jour
fonction
nouvelles
publiées.
Il
convient
noter
que
autres
cellulaires
qui
n'ont
pas
encore
approuvées
autorités
santé
font
l'objet
d'essais
cliniques
ne
abordées