Hepatoblastoma and Beckwith-Wiedemann Syndrome

(1)

Supplemental Data Table. Patient characteristics Pt Age at

HBL pres.

Sex Gest

age

BW N.A. Stage/

PRETEXT

Screen AFP at diagnosis

HBL Histology

Chemotherapy Surgery Outcome Ref &

date

1 Birth F 35

weeks

NR III/NR No (birth)

NR NR NR Biopsy Died at 5

mos from diagnosis

42007

2 Birth F 36.5

weeks

3325 grams

III/NR No

(birth)

171,533 ng/mL

Fetal VCR, doxo Unres Died of

MOF at 2 weeks from diagnosis

51991

3 Birth M Term 4100

grams III/NR No

(birth) 226,029

ng/mL Fetal Carbo, 5FU, doxo, VCR, cisplatin

GTR NED at 15 mos off therapy

61999

4 1 day M NR 4621

grams

I/NR No

(birth)

NR Fetal None CR NED at

10+ years off therapy

CHRCO 2013

5 23 days M 38

weeks

4290 grams

III/NR AFP

and U/S

228,889 ng/mL

Fetal Doxo, carbo, cisplatin

GTR NED at 3.5 years old

72011

6 4 weeks M NR NR NR/I U/S 99,360

ng/mL

Mixed fetal/

epithelial

Cisplatin, carbo, doxo, and docetax at pulm relapse post initial surgery; Pulm RT

CR with negative margins

NED at 10 years off therapy

82012

7 6 weeks M 31

weeks

NR NR NR NR NR None None Diagnosis

at autopsy;

Died of lung disease of premat

91994

8 11

weeks

M Term 3800

grams

III/I MRI 21,860

ng/mL (1:50)

Embryonal Cisplatin CR AFP 98 ng/mL 2 mos off therapy

102007

9 3 mos M 31 1900 III/NR No >70,000 Fetal and None Biopsy DOD 17 ¹¹1996

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weeks grams ng/mL embryonal days post diagnosis

10 3 mos F 38

weeks 3754

grams NR NR 609,471

ng/mL Fetal and

embryonal Chemotherapy CR NED at 6 years

122003

11 3 mos M 32

weeks

LGA III/NR NR NR NR None Biopsy Died at 2

mos from diagnosis

42007

12 4 mos M 38

weeks 3920

grams NR/II U/S 31,533

ng/mL Poorly diff VCR, CTX,

doxo, cisplatin CR NED 2 years from diagnosis

132003

13 4 mos NR NR NR NR NR NR NR NR NR NR ²1998

14 5 mos F Term LGA I/NR AFP

and U/S

30,000 ng/mL

NR NR CR NR ¹⁴2003

15 5 mos M Term LGA I/NR AFP

and U/S

70,000 ng/mL

16 5 mos F NR NR NR/IV NR 238,044

ng/mL

NR Doxo, cisplatin LT at 17 mos of age

Alive at 36 mos post LT

151999

17 5 mos M Premat NR NR/II NR NR Fetal Cisplatin Biopsy NR ¹⁶2000

19 5 mos M 26

weeks

NR III/NR U/S 183,000

ng/mL

NR VCR, 5FU,

carbo, cisplatin

LT NED at 33 mos

CHLA 2013

20 6 mos F 27-28

weeks

865 grams

II/NR No 18,000

ng/mL

Fetal and embryonal

Doxo, 5-FU, VCR

GTR NED at 14 mos

CHLA 2013

22 6 mos M NR NR NR U/S 84,000

ng/mL

NR Chemotherapy Resectio n

NR ¹⁷2009

23 6 mos F NR NR III/NR NR Elevated Fetal and

embryonal

CCG

chemotherapy

CR NED at 21 mos from diagnosis

31995

24 7 mos F Term AGA I/NR AFP

and U/S

5,361 ng/mL

26 8 mos F 27-28

weeks

1340 grams

II/NR NR 20,612

ng/mL

Embryonal NR Resectio

n

NR ¹⁸1992

27 10 mos F NR NR NR NR NR NR NR NR NR ¹⁹1983

28-34 2-10 mos

5M 2F

NR NR PRETEXT I

(2), II (2),

NR 70,652 – 837,305

NR Cisplatin,

pirarubicin; (2

Resectio n in all 7

NED at 2- 10 years

202012

(3)

(media n 6 mos)

III (3), all

localized ng/mL

(median 211,820)

also received ifosfamide, etoposide, carboplatin; 1 high dose acetaminophen)

(median 6 years) with 1 NED post relapse treatment and 1 NED post therapy related acute leukemia treatment

35 11 mos F 36

weeks

3856 grams

NR/I NR 180,000

ng/mL

Embryonal Chemotherapy Resectio n

NED at 9 years

122003

36 11 mos F 37

weeks 3610

grams NR No 550,000

ng/mL Fetal and

embryonal Pirarubicin, etoposide, carbo, cisplatin

CR Died of

recurrent AML/

MDS post BMT

211996

37 11 mos M Term 4950

grams I/NR U/S 10,760

ng/mL Epithelial NR CR with

negative margins

Alive at 17 months

221988

38 12 mos F 32 to

33 weeks

2839

grams IV/IV+M No 1,510, 000 ng/mL

NR Doxo,

ifosfamide, carbo, cisplatin, irinotecan

LT NED 18

months post LT

232010

39 12 mos M 38

weeks

4400 grams

III/NR No

(missed )

365,000 ng/mL

Mixed Chemotherapy CR Died 6 mos

post surgery (hepatic failure), NED

241996

40 1 year NR NR NR NR NR NR NR NR NR NR ²⁵2005

41 13 mos F NR NR NR/II MRI,

U/S and AFP

435.7

ng/mL Embryonal

and fetal NR Resectio

n NR ¹⁷2009

42 14 mos M 38

weeks 2511

grams NR No NR NR None Unres DOD at 1

month from

261970

(4)

diagnosis

43 15 mos M Term 4450

grams

IV /NR NR NR Embryonal None None DOD at

diagnosis (autopsy)

271993

44 15 mos F 29 to

30 wks

LGA I or II/NR AFP and U/S

2,083 ng/mL

Fetal and cholangio- blastic

Chemotherapy Biopsy NED at 4 years old

282008

45 1 ½

years M 32

wks 1350

grams IV /NR No 1:65,536 Epithelial,

mesen CCG treatment Resectio

n NED 21

mos from diagnosis

291986

47 25 mos F NR NR III/NR NR Elevated Fetal and

embryonal

CCG treatment CR NED at 12 mos post dx

31995

48 30 mos F NR NR III/NR U/S >33,000

ng/mL

NR Doxo, 5FU,

VCR, cisplatin and high dose acetaminophen with cisplatin

CR NED at 7 years post surgery

302005

49 < 5 years old

NR NR NR NR U/S NR NR NR NR NED at > 9

years

312012

50 NR NR NR NR II /NR NR NR NR Epirubicin,

cisplatin

Resectio n

NED at 9.3 years

322009

51&52 NR M NR NR NR NR NR NR NR NR NR ³³1995

53 to

56 NR NR NR NR NR NR NR NR NR NR NR ³⁴2002

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

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Angeles; CCG, Children’s Cancer Group; AGA, appropriate for gestational age; AML, acute myeloblastic leukemia; MDS, myelodysplastic syndrome;

BMT, bone marrow transplant; mesen, mesenchymal.