Thrombopoietin Receptor Agonists for Thrombocytopenia in Pediatric Hematologic Malignancies
Amanda E. Marinoff,
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Allyson Thrall,
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Kathryn Aaronson
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et al.
Pediatric Blood & Cancer,
Journal Year:
2025,
Volume and Issue:
unknown
Published: Jan. 8, 2025
ABSTRACT
Background
Thrombopoietin
receptor
agonists
(TPO‐RAs)
have
demonstrated
efficacy
in
treating
clinically
significant
thrombocytopenia,
including
chemotherapy‐induced
thrombocytopenia
adults.
However,
data
regarding
their
safety
and
pediatric,
adolescents,
young
adult
(AYA)
patients
with
hematologic
malignancies
are
limited.
Methods
We
retrospectively
identified
15
pediatric
AYA
aged
25
years
or
younger
treated
a
TPO‐RA
at
UCSF
Benioff
Children's
Hospitals
between
2015
2023.
Platelet
counts
transfusion
requirements
were
compared
before
after
therapy.
Results
The
median
age
initiation
was
16
(range:
7–25
years).
Nine
(60%)
had
history
of
bleeding
comorbidity
that
predisposed
to
severe
risk.
Eleven
received
romiplostim
four
eltrombopag.
platelet
count
significantly
increased
from
24
×
10
9
/L
baseline
54
3
weeks
any
therapy
(
p
=
0.029).
Monthly
decreased
two
units
0.007).
Fourteen
the
(93%)
achieved
sustained
>50,000/µL
within
8
weeks,
time
response
weeks.
No
TPO‐RA‐related
adverse
events
observed.
Conclusion
TPO‐RAs
effective
managing
refractory
being
for
malignancies,
favorable
profile,
even
among
multiple
comorbidities.
These
findings
warrant
further
investigation
through
prospective
clinical
trials
confirm
establish
guidelines
this
population.
Language: Английский
CD19 -targeted CAR T therapy treating hematologic malignancies: hidden danger is the next neighbor to security?
Xueshuai Ye,
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Min Ge,
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M. Tan
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et al.
Frontiers in Immunology,
Journal Year:
2025,
Volume and Issue:
16
Published: March 4, 2025
CD19-targeted
chimeric
antigen
receptor
(CAR)
T-cell
therapy
has
achieved
marvelous
results
in
the
treatment
of
patients
with
relapsed
and/or
refractory
B-cell
lymphomas,
acute
lymphoblastic
leukemia,
and
multiple
myeloma.
As
a
new
method
that
changed
existing
paradigm,
there
been
short
time
from
its
emergence
to
FDA
approval.
However,
increasing
number
cases
passage
time,
hidden
problems
have
gradually
exposed.
In
this
review,
we
summarize
short-
long-term
toxicity,
such
as
secondary
tumors
lethal
CAR
tumors,
hematologic
malignancies
treated
CD19-CAR-T
cells,
including
cytokine
release
syndrome
(CRS),
ICANS,
low
occurrence
rates
but
high
mortality,
T
cell
which
may
be
related
gene
modification
mechanism
viral
vectors
currently
approved
for
CAR-T
cells.
We
also
discuss
potential
investigational
strategies
designed
improve
safety
CAR-T-cell
therapy.
Language: Английский