Hepatoblastoma and Beckwith-Wiedemann Syndrome: curable

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Supplemental Data Table. Patient characteristics
Pt
Age at
HBL
pres.
Sex
Gest
BW
AFP at
HBL
diagnosis
Histology
1
Birth
F
35
weeks
NR
III/NR
No
(birth)
NR
NR
NR
Biopsy
2
Birth
F
36.5
weeks
3325 III/NR
grams
No
(birth)
171,533
ng/mL
Fetal
VCR, doxo
Unres
3
Birth
M
Term
4100 III/NR
grams
No
(birth)
226,029
ng/mL
Fetal
GTR
M
NR
4621 I/NR
grams
No
(birth)
NR
Fetal
Carbo, 5FU,
doxo, VCR,
cisplatin
None
4
1 day
5
23 days
M
38
weeks
NR
4290 III/NR
grams
NR
NR/I
AFP
and U/S
U/S
228,889
ng/mL
99,360
ng/mL
Fetal
GTR
6 weeks M
31
weeks
NR
NR
NR
NR
Doxo, carbo,
cisplatin
Cisplatin,
carbo, doxo,
and docetax at
pulm relapse
post initial
surgery; Pulm
RT
None
6
4 weeks M
7
8
11
weeks
M
Term
3800 III/I
grams
MRI
21,860
ng/mL
(1:50)
Embryonal
Cisplatin
CR
9
3 mos
M
31
1900
No
>70,000
Fetal and
None
Biopsy
age
N.A. Stage/
Screen
PRETEXT
NR
III/NR
Mixed fetal/
epithelial
Chemotherapy
Surgery
Outcome
Ref &
date
CR
CR with
negative
margins
None
Died at 5
mos from
diagnosis
Died of
MOF at 2
weeks
from
diagnosis
NED at 15
mos off
therapy
NED at
10+ years
off therapy
NED at 3.5
years old
NED at 10
years off
therapy
42007
Diagnosis
at autopsy;
Died of
lung
disease of
premat
AFP 98
ng/mL 2
mos off
therapy
DOD 17
91994
51991
61999
CHRCO
2013
72011
82012
102007
111996
weeks
grams
38
weeks
32
weeks
3754 NR
grams
LGA III/NR
NR
10
3 mos
F
11
3 mos
M
12
4 mos
M
38
weeks
3920 NR/II
grams
U/S
31,533
ng/mL
Poorly diff
VCR, CTX,
doxo, cisplatin
CR
13
14
4 mos
5 mos
NR
F
NR
Term
NR
LGA
NR
I/NR
NR
NR
NR
CR
5 mos
M
Term
LGA
I/NR
NR
NR
CR
NR
16
5 mos
F
NR
NR
NR/IV
NR
30,000
ng/mL
70,000
ng/mL
238,044
ng/mL
NR
NR
15
NR
AFP
and U/S
AFP
and U/S
NR
days post
diagnosis
NED at 6
years
Died at 2
mos from
diagnosis
NED 2
years from
diagnosis
NR
NR
NR
Doxo, cisplatin
17
18
19
5 mos
5 mos
5 mos
M
NR
M
NR
NR
NR
NR/II
NR
III/NR
NR
NR
U/S
6 mos
F
No
21
22
6 mos
6 mos
NR
M
865
II/NR
grams
NR
NR
NR
NR
Fetal and
embryonal
NR
NR
Cisplatin
NR
VCR, 5FU,
carbo, cisplatin
Doxo,
5-FU, VCR
NR
Chemotherapy
23
6 mos
F
NR
NR
III/NR
NR
NR
NR
183,000
ng/mL
18,000
ng/mL
NR
84,000
ng/mL
Elevated
Fetal
NR
NR
20
Premat
NR
26
weeks
27-28
weeks
NR
NR
LT at 17
mos of
age
Biopsy
NR
LT
Fetal and
embryonal
24
7 mos
F
Term
AGA
I/NR
25
26
7 mos
8 mos
NR
F
27
10 mos
F
NR
27-28
weeks
NR
NR
NR
1340 II/NR
grams
NR
NR
AFP
and U/S
NR
NR
5,361
ng/mL
NR
20,612
ng/mL
NR
28-34
2-10
mos
5M
2F
NR
NR
70,652 –
837,305
PRETEXT I
(2), II (2),
NR
NR
U/S
NR
NR
ng/mL
embryonal
609,471
ng/mL
NR
Fetal and
embryonal
NR
Chemotherapy
CR
None
Biopsy
122003
42007
132003
21998
142003
142003
Alive at 36
mos post
LT
NR
NR
NED at 33
mos
GTR
NED at 14
mos
NR
NR
Resection NR
151999
CCG
chemotherapy
CR
31995
NR
NR
CR
NR
Embryonal
NR
NR
NR
NR
Resection NR
21998
NR
NR
NR
191983
NR
Cisplatin,
pirarubicin; (2
Resection NED at 2in all 7
10 years
NED at 21
mos from
diagnosis
NR
NR
162000
21998
CHLA
2013
CHLA
2013
21998
172009
142003
181992
202012
(median
6 mos)
III (3), all
localized
35
11 mos
F
36
weeks
37
weeks
3856
grams
3610
grams
36
11 mos
F
37
11 mos
M
Term
4950 I/NR
grams
38
12 mos
F
32 to
33
weeks
39
12 mos
M
40
41
1 year
13 mos
42
14 mos
ng/mL
(median
211,820)
NR/I
NR
NR
No
also received
ifosfamide,
etoposide,
carboplatin; 1
high dose
acetaminophen)
180,000
ng/mL
550,000
ng/mL
Embryonal
Chemotherapy
Fetal and
embryonal
Pirarubicin,
etoposide,
carbo, cisplatin
U/S
10,760
ng/mL
Epithelial
NR
2839 IV/IV+M
grams
No
1,510,
000
ng/mL
NR
38
weeks
4400 III/NR
grams
No
365,000
(missed) ng/mL
Mixed
Doxo,
ifosfamide,
carbo, cisplatin,
irinotecan
Chemotherapy
NR
F
NR
NR
NR
NR
NR
435.7
ng/mL
NR
Embryonal
and fetal
M
38
weeks
2511 NR
grams
NR
MRI,
U/S and
AFP
No
NR
NR
NR
NR/II
(median 6
years) with
1 NED
post
relapse
treatment
and 1 NED
post
therapy
related
acute
leukemia
treatment
Resection NED at 9
years
CR
Died of
recurrent
AML/
MDS post
BMT
CR with
Alive at 17
negative months
margins
LT
NED 18
months
post LT
122003
211996
221988
232010
241996
NR
NR
Died 6 mos
post
surgery
(hepatic
failure),
NED
NR
NR
Resection NR
None
Unres
261970
CR
DOD at 1
month
from
252005
172009
43
15 mos
M
Term
44
15 mos
F
29 to
LGA
30 wks
45
1½
years
M
32
wks
46
47
19 mos
25 mos
NR
F
48
30 mos
49
<5
years
old
NR
50
4450 IV /NR
grams
diagnosis
DOD at
diagnosis
(autopsy)
NED at 4
years old
271993
NR
NR
Embryonal
None
None
AFP
and U/S
2,083
ng/mL
Chemotherapy
Biopsy
1350 IV /NR
grams
No
1:65,536
Fetal and
cholangioblastic
Epithelial,
mesen
CCG treatment
291986
NR
NR
NR
NR
NR
III/NR
NR
NR
NR
Elevated
NR
Fetal and
embryonal
NR
CCG treatment
F
NR
NR
III/NR
U/S
>33,000
ng/mL
NR
NR
NR
NR
NR
U/S
NR
NR
Doxo, 5FU,
VCR, cisplatin
and high dose
acetaminophen
with cisplatin
NR
Resection NED 21
mos from
diagnosis
NR
NR
CR
NED at 12
mos post
dx
CR
NED at 7
years post
surgery
NR
NED at > 9
years
312012
NR
NR
NR
II /NR
NR
NR
NR
Resection NED at 9.3
years
NR
NR
NR
NR
322009
I or II/NR
Epirubicin,
cisplatin
NR
NR
282008
21998
31995
302005
331995
51&52 NR
M
NR
NR
NR
NR
NR
NR
342002
53 to
NR
NR
NR
NR
NR
NR
NR
NR
56
Pt, patient; HBL, hepatoblastoma; pres., presentation; gest, gestational; BW, birthweight; N.A., North American; AFP, alphafetoprotein; ref, reference; F;
female; NR, not reported; mos, months; VCR, vincristine; doxo, doxorubicin; unres, unresectable; MOF, multi-organ failure; M, male; carbo, carboplatin;
5FU, 5-fluorouracil; GTR, gross total resection; NED, no evidence of disease; CR, complete resection; CHRCO, Children’s Hospital & Research Center
Oakland; U/S, ultrasound; docetax, docetaxel; pulm, pulmonary; RT, radiation therapy; premat, prematurity; DOD, died of disease; LGA, large for
gestational age; LGA, large for gestational age; diff’d, differentiated; CTX, cyclophosphamide; LT, liver transplant; CHLA, Children’s Hospital Los
Angeles; CCG, Children’s Cancer Group; AGA, appropriate for gestational age; AML, acute myeloblastic leukemia; MDS, myelodysplastic syndrome;
BMT, bone marrow transplant; mesen, mesenchymal.
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