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Vandetanib successfully controls medullary thyroid cancer-related Cushing syndrome in an adolescent patient.
[cushing syndrome]
Ectopic
Cushing
syndrome
due
to
ACTH
secretion
from
metastatic
medullary
thyroid
cancer
(
MTC
)
is
associated
with
significant
morbidity
and
mortality
.
The
aim
of
the
study
was
to
describe
the
first
case
of
Cushing
syndrome
associated
with
MTC
in
a
pediatric
patient
and
the
successful
reversal
of
Cushing
syndrome
with
tyrosine
kinase
inhibitor
(
vandetanib
)
therapy
.
A
17
-
year
-old
Brazilian
adolescent
presented
with
metastatic
MTC
and
associated
ACTH-dependent
ectopic
Cushing
syndrome
in
the
context
of
multiple
endocrine
neoplasia
type
2
B
.
When
the
patient
was
treated
with
the
tyrosine
kinase
inhibitor
vandetanib
,
rapid
decrease
in
serum
cortisol
and
improvement
of
clinical
symptoms
were
observed
.
We
describe
the
first
pediatric
case
of
clinical
and
biochemical
improvement
of
paraneoplastic
MTC
-related
Cushing
syndrome
after
treatment
with
vandetanib
.
Vandetanib
and
possibly
other
tyrosine
kinase
inhibitors
may
be
a
novel
beneficial
option
in
patients
with
neuroendocrine
tumor
-related
ectopic
Cushing
syndrome
.
Diseases
Validation
Diseases presenting
"cancer"
symptom
achondroplasia
acute rheumatic fever
adrenal incidentaloma
alpha-thalassemia
benign recurrent intrahepatic cholestasis
cadasil
canavan disease
carcinoma of the gallbladder
cholangiocarcinoma
coats disease
congenital adrenal hyperplasia
congenital diaphragmatic hernia
cowden syndrome
cushing syndrome
cutaneous mastocytosis
dedifferentiated liposarcoma
dystrophic epidermolysis bullosa
epidermolysis bullosa simplex
erdheim-chester disease
erythropoietic protoporphyria
esophageal adenocarcinoma
esophageal carcinoma
esophageal squamous cell carcinoma
familial hypocalciuric hypercalcemia
familial mediterranean fever
gm1 gangliosidosis
heparin-induced thrombocytopenia
hereditary cerebral hemorrhage with amyloidosis
hirschsprung disease
hodgkin lymphoma, classical
inclusion body myositis
junctional epidermolysis bullosa
kabuki syndrome
kallmann syndrome
kindler syndrome
lamellar ichthyosis
liposarcoma
locked-in syndrome
lymphangioleiomyomatosis
monosomy 21
neuralgic amyotrophy
oculocutaneous albinism
oligodontia
oral submucous fibrosis
papillon-lefèvre syndrome
pendred syndrome
pleomorphic liposarcoma
primary effusion lymphoma
proteus syndrome
pyomyositis
pyruvate dehydrogenase deficiency
severe combined immunodeficiency
sneddon syndrome
systemic capillary leak syndrome
triple a syndrome
von hippel-lindau disease
waldenström macroglobulinemia
well-differentiated liposarcoma
werner syndrome
wiskott-aldrich syndrome
wolf-hirschhorn syndrome
x-linked adrenoleukodystrophy
This symptom has already been validated