HEMATOLOGY CONFERENCE

Marimicah Angelique Valdez; Jayson So; Krisha Salazar; Charles

Rodriguez, Katrina Salvosa
University of Santo Tomas
Faculty of Medicine and Surgery
Department of Pediatrics

K.N.R.
2 YEARS AND 11 MONTHS OLD/MALE
Date of birth : 06/14/2012
Date of Admission : 04/15/2015
Home Address : Brgy. Angeles, Atimonan, Quezon
City
Religion : Roman Catholic
Informant/s : Mother
Reliability : Good

RIGHT UPPER
QUADRANT
ABDOMINAL MASS

Chief complaint

HISTORY

History of Present Illness

Review of Systems
Past Medical History
Personal History

Known case of Neurocutaneous

Melanosis
Seizure disorder - 2013

HISTORY OF PRESENT ILLNESS

10

months

PTA

striking epigastric area

Pain on the epigastric area
Treated as dyspepsia -> topical liniment
No fever, jaundice, diarrhea, anorexia, weight loss

Intermittent similar episodes

Interval No accompanying symptoms

History

HISTORY OF PRESENT ILLNESS

Patients mother palpated a

mass on the right upper
quadrant
Consult at Delos Santos
months Medical Center
History and PE were done
PTA
No abdominal mass

HISTORY OF PRESENT ILLNESS

2 months
PTA

Mother palpated a firm, nontender mass at the

RUQ
Occasional abdominal pain
Consult was done
Abdominal ultrasound
hepatomegaly of the right lobe of the liver
(11.2x6.8x8cm) with internal calcification in the
hilar region
Referral to a Pediatric Hematologist Oncologist

HISTORY OF PRESENT ILLNESS

1 month
PTA
ADMISSION

Persistence of RUQ abdominal mass

Consult was done with a private pedia
Hema-Onco at DLSMC
CT scan of the abdomen
CBC
ALT 47.53 U/L (normal value = 4-45)
(slightly elevated)
Advised surgery -> USTH admission

CT SCAN OF ABDOMEN

Hgb
RBC
Hct
Platelet
WBC
Differential Count
Neutrophils
Metamyelocyte
Bands
Segmenters
Lymphocytes
Monocytes
Eosinophils
Basophils

Reference
Range
115-125
4.5-5.1 x1012/L
0.34-0.37
150-450 x109/L
5-15.5 x109/L

0.50-0.70

0.00-0.05
0.50-0.70
0.20-0.40
0.00-0.07
0.00-0.05
0.00-0.01

04/13/15
102
4.92
0.32
352
11.1

0.48
0.57
0.38
0.05
-

REVIEW OF SYSTEMS
General Survey
Cutaneous:
HEENT:
Respiratory:
Cardiovascular:
Genitourinary
Endocrine:

(-) chills, (-) easy fatigability (-) weight change

(-) rashes, (-) dryness, (-) pallor
(-) eye redness/discharge, (-) epistaxis, (-) aural discharge;
(-) colds (-) dyspnea
(-) cyanosis, (-) syncope

(-) hematuria, (-) changes in urine color

(-) neck enlargement (-)profuse sweating

Nervous/Behavi
(-) loss of consciousness, (-) seizures; (-) mood changes
oral:
Musculoskeletal: (-) joint swelling (-) joint pain
Hematologic

(-) bleeding, no bruisability

PERSONAL HISTORY
Gestational
27 y/o G1P0
Prenatal checkup: started 6 weeks; total of 10 pre-natal check-ups
Unremarkable history of exposure to viral exanthems and radiation
Hepa B screening: non reactive
OGTT screening: normal
(-) HPN
Bacterial Vaginosis on 3rd trimester-> vaginal suppository for 1
week

PERSONAL HISTORY
Birth
Term via NSD at 39 weeks
Epidural anesthesia
Clear amniotic fluid
BW 3.47 kg, BL 48 cm

PERSONAL HISTORY
Neonatal
APGAR score unrecalled
Nursery for one week due to sepsis (jaundice
and vomiting)
Giant nevi at the back at birth
Newborn screening done unremarkable
Hearing screening done- unremarkable

FEEDING HISTORY
Exclusively breastfed for 7 months approximately every 2-3 hours and for 15
minutes per breast
Complementary feeding at 7 months

Started with pureed food and tablefood at

present

Food

Kcal

Daily

Breakfast
Milk (1/4 cup)
Snack
Milk (1/4 cup)
Skyflakes cracker (1 pc)
Lunch
Rice (3tbsp)
Fish soup (1/4 cup)
Fish, fried (1/4)
Milk (1/4 cup)
Afternoon Snack
Milk (1/4 cup)
Dinner
Rice (3tbsp)
Fish soup (1/4 cup)
Fish, fried (1/4)

113

113
40

34
115
53
113

113
56
151
53

ACI
RENI
Percent deficient

954
1070
10.84%

Month Gross Motor

s

Fine Motor

Receptive
Language

10

Pulls to stand
Crawls/ cruises

12

Walks with one

hand help

16

Walks alone
Creeps upstairs

18

Run stiffly
Walks backward

24

Runs well up and

down
Jumps

Pincer Grasp Understands

gestures
Responds to
sound of
name
Releases
Obeys simple
objects on
commands
request
with gesture
Makes tower Obeys simple
of 3 cubes
commands
without
gesture
Imitates

scribbling
and vertical
strokes

Expressive
Language
Discriminate
papa and
mama

Speaks few
words

PersonalSocial
Skills
Waves byebye

Kisses on
request

3 words other Indicates

than papa desires by
and mama pointing

Turns 2-3
pages at a
time
Feeds self
Removes
clothes
Listens to

RED FLAGS
Social Emotional Red Flags
18months lack of simple pretend play (18mo)

Receptive Language
Does not point to 3 body parts (18mo)

Expressive Language
Vocabulary of not more than 35-50words
(24mo)

PAST MEDICAL HISTORY

Yea
r
201
2
201
3

201
4

Congenital pigmented/melanocytic nevi

Seizure Disorder
Last attack: March 23, 2013
Last EEG and MRI: June 2013 (unremarkable)
Phenobarbital 60mg/pptab once a
Maintenance medicines:
day
Valproic Acid 250mg/5ml 2.5 ml TID
PTB
Diagnostics: Chest Xray, PPD (+), De Los Santos Medical

PAST MEDICAL HISTORY

Previous surgeries: None
Known allergies: unrecalled antibiotic
Previous blood transfusion: None

FAMILY HISTORY
(+) hypertension, (+) DM, (+) TBmaternal grandfather
(-) family history of malignancy
(-) liver or gastrointestinal disease
(-) asthma, allergy, thyroid disorders,
bleeding disorder

SOCIAL HISTORY
Source of funds: mother (househelper)
Lives in a well-lit, well-ventilated,
adequately spaced condominium (owned
by mothers employer)
No exposure to cigarette smoke
No pets at home
Water consumption: mineral water

IMMUNIZATION
Date Given

Route

Adverse
Reactions

BCG

6/15/12

IM

No reaction

Hep B 1

6/15/12

IM

No reaction

Hep B 2

2/27/13

IM

No reaction

Hep B 3

3/27/13

IM

No reaction

DTP1/OPV1

8/10/12

IM/PO

No reaction

Vaccine

Place
Given
De Los
Santos
Hospital
De Los
Santos
Hospital
Local health
center
Local Health
center
Local Health
center

IMMUNIZATION
DTP2/OPV2

9/12/12

IM/PO

No reaction

DTP3/OPV2

10/12/12

IM/PO

No reaction

Hib 1

2/9/13

IM

No reaction

Hib 2

7/12/13

IM

No reaction

Hib 3

8/20/13

IM

No reaction

Measles

3/27/13

IM

No reaction

MMR

8/16/13

SQ

No reaction

Varicella

10/25/13

SQ

No reaction

Rotavirus

Not Given

Local Health
center
Local Health
center
Private
Pediatrician
Private
Pediatrician
Private
Pediatrician
Private
Pediatrician
Private
Pediatrician
Private
Pediatrician
-

IMMUNIZATION UPDATE
Hepatitis B booster
Hepatitis A
Influenza
PPV

FAMILY PROFILE

Age

Educational
Attainment

Mother

31 y/o

3rd year
college

Father

Occupation Health Status

Househelper

Healthy

PHYSICAL
EXAMINATION

PHYSICAL EXAMINATION
General: awake, alert, well hydrated, well-nourished, carried
by mother, not in cardiorespiratory distress
Vital Signs: BP: 100/60mmHg CR 90 bpm regular, RR 21
cpm regular, Temp 36.5C
Anthropometrics:
Ht: 95.5 cm (z-score above 0);
Wt: 13 kg (z-score below 0),
WFH (z-score =-1)
Skin: warm, moist skin, good skin turgor, multiple nevus
distributed on face, trunk and extremities with tufts of hair,
largest patch measures: 27x 30 cm on the back extending to

PHYSICAL EXAMINATION
Head: normocephalic, no palpable masses, lesions, no facies
Eyes: eyelashes not matted eyelids not
conjunctiva, anicteric sclera, clear cornea

swollen,

pink

palpebral

Ears: no deformities, no aural discharge, non-hyperemic external auditory

canal, TM intact AU
Nose: nasal septum midline, no alar flaring, turbinates not congested, (-)
discharge
Mouth: moist lips and buccal mucosa, midline uvula and tongue, tonsils
not enlarged, non-hyperemic posterior pharyngeal wall, (-) lip and oral
lesions, (-) dental carries
Neck: supple neck, thyroid not enlarged, no cervical lymphadenopathy

PHYSICAL EXAMINATION
Lungs: symmetrical chest expansion, no retractions, equal vocal
fremiti, resonant on other lung fields, clear breath sounds
Heart: adynamic precordium, apex beat at the 4th LICS MCL, no
heaves, no thrills, no lifts, no murmurs
Abdomen: globular abdomen, umbilicus inverted, no visible
peristalsis, normoactive bowel sounds, Liver span 12cm, hard and
nodular liver edge
Genitourinary: ~4.5 penile length, no phimosis, testes descended
Extremities: pulses full and equal on all extremities, no cyanosis,
no edema, no limitation of movement, no clubbing of nails

PHYSICAL EXAMINATION
Neurolo
gic

Mental Status: awake, alert

Cranial Nerves:
CN I cannot be assessed
CN II pupils 2-3 mm equally reactive to light
CN III, IV, VI full and equal external ocular movement
CN V V1-V3 able to pinpoint sensation
CN VII no facial asymmetry
CN VIII gross hearing intact
CN IX, X uvula midline, (+) gag reflex
CN XI can turn head side to side against resistance
CN XII no tongue atrophy, tongue midline
Motor: spontaneous movement, able to push examiners hands
Sensory: intact, can identify touch and pain
Cerebellum: able to reach without tremors
Meningeal signs: (-) nuchal rigidity, (-) Brudzinski sign, (-) Kernigs sign
Deep Tendon Reflexes: 2+ on knees, biceps; (+) Babinski on Both
lower extremities

HISTORY OF PRESENT ILLNESS

Upon
admissio
n

LDH at 327 U/L (elevated)

alpha-fetoprotein at
60,500 ng/mL (elevated)
total protein at 8.59g/dl
(elevated)

SALIENT FEATURES

2 yrs 11mo old/Male

Palpable liver mass
(-) jaundice, diarrhea, vomiting, bleeding symptoms
BP: 100/60mmHg CR 130 bpm regular, RR 21 cpm
regular, Temp 36.5C
Ht: 95.5 cm (z-score= above 0); Wt: 13 kg (zscore= below 0), WFH z-score =-1
Globular abdomen, umbilicus inverted, no visible
peristalsis, normoactive bowel sounds,dull on right
upper quadrant and upper half of right lower

SALIENT FEATURES
UTZ: hepatomegaly of the right liver lobe
(11.2x6.8x8 cm) with some internal calcification in
the hilar region.
CT scan showed multilobulated solid and necrotic
tumor mass lesion with calcification in the liver
particularly in the right hepatic lobe
Elevated LFT, AFP, LDH
Low Hgb and Hct
Normal PT, PTT, Creatinine

INITIAL ASSESSMENT
Hepatoblastoma
Neurocutaneous Melanosis
Seizure disorder
Global Developmental Delay

APPROACH TO
DIAGNOSIS

look for a symptom, sign,

or laboratory finding
found in the least number
of diseases

PRESENTING
MANIFESTATION

Liver
Mass at
RUQ

DIFFERENTIAL DIAGNOSIS

Salient Features

Hepatoblastoma

HCC

3yo

Most common hepatic

tumor in children <3yo

5-18yo

Abdominal Mass

80%

Abdominal Pain

90%, usually in the right

lobe of the liver
None

Elevated LFT

15-30%

30-50%

Elevated AFP

60-70%

50%

Anemia

common

common

Abdominal distention

HEPATOBLASTOMA
SEIZURE DISORDER
NEUROCUTANEOUS
MELANOSIS
GLOBAL

Initial Assessment

COURSE IN THE WARD

Work-Ups
Management
Outcome

COURSE IN THE WARD

Upon admission, (4/15/15)
Neutropenic diet
Referred to Pedia-Surgery and Pedia
Neurology for further evaluation
Phenobarbital 60mg/pptab 1 pptab once a day (4.6mkday)
Valproic acid 250mg/5ml given 4 ml three times a day
Diazepam 5mg/SIVP for active seizures more than 3 minutes
Oxygen supplementation to be given via face mask at 10LPM

COURSE IN THE WARD

2nd HD (4/16/15)

3RD HD (4/17/15)

4TH HD (4/18/15)

3 glasses of milk per

day with daily review
of 24-hour food recall
as advised by the
Pedia-GIN

multivitamins
2.5ml
QD and zinc 10mg/ml
2ml QD
Pediasure 2.5 scoops
in 95 ml to make 110
ml

13.5 kg
Actual caloric intake
700
Pediasure
330
calories

COURSE IN THE WARD

6th HD (4/20/15)
Referred
interventional
radiology for
biopsy

7th HD (4/21/15)
to Referred
anesthesiologist
USG- sedation
prior
procedure

8th HD (4/22/25)
To NPO
for IVF hydration (D5NSS
to 1L)
Histopathologic study
Ibuprofen
200mg/5ml
2ml
Suggested to be started
on lactulose

FETAL PATTERN

FETAL PATTERN

COURSE IN THE WARD

9th HD (4/23/15) 10th (4/24/15)

11th-12th
HD 14th
(4/25-26/15)
(4/28/15)

Chest CT scan Tolerate feeding

->
no
lung
metastasis

Asymptomatic
Good activity

HD

2D-echo -> no
pericardial
effusion
Ondansetron
as
premedication to
chemotherapy

COURSE IN THE WARD

16th HD (4/30/15)
17th HD (5/1/15)
Doxorubicin + D5 Occasional rhonchi
water via slow IV Vomiting
postinfusion
Doxorubicin
Paracetamol
200mg/5mg, 4ml q4

18TH HD (5/2/15)
Clear nasal discharge
Increase
oral
fluid
intake
1 episode of vomiting
4 episodes of watery
loose stools
Fever,
chills
and
occasional
epigastric
pain
Cefuroxime
500mg/slow IV infusion
q8

COURSE IN THE WARD

19th
HD
(5/3/15)
Occasional
febrile episodes,
occasional
rhonchi
Pediasure

21st
HD
(5/5/15)
Febrile episodes,
vomiting,
cervical
lymphadenopath
y
Urine
culture:
gram-negative
bacilli
Acute
Pyelonephritis

22nd
HD
(5/6/15)
Urine
CS:
Klebsiella
pneumonia
Meropenem 260
mg/per slow IV
infueion

24th
HD
(5/8/15)
Pediasure
3.5
scoops
KUB ultrasound
->
bilaterally
enlarged
kidneys
Urinary
retention

COURSE IN THE WARD

26th HD (5/10/15) 27th HD (5/11/15) 28th HD (5/12/15) 29th HD (5/13/15)
Febrile
episodes,
chills, oral thrush,
CBC
was
compatible
with
febrile neutropenia
- Hgb 64, hct 0.19,
wbc 1.50, plt 25,
seg
0.02, lym
0.98, ANC 30
GCSF 150 mcg/vial

Febrile, poor oral Isolation room

intake, ill-looking
Renal DMSA scan
Meropenem
-> once stable
PiperacillinTazobactam
130 ml washed
pRBC transfusion
Pre-BT
medications:
Paracetamol
and
Diphenhydramine

Mupirocin
cream
for
left
foot
phlebitis
Less
frequent
febrile eppisodes
Semiformed stools
GCSF
Co-trimoxazole
and TMP+SMX

COURSE IN THE WARD

30th HD (5/14/15)

31st HD (5/15/15)

Cxr
Miconazole Cream
on diaper and
scrotal area

Febrile episodes
Six episodes of soft, mushy
stools

32nd hd
(5/16/15)
CXR->
pneumonia
Febrile
episodes
Vancomycin
TMP+SMX
-> Cotrimoxazole
Occassional
rhonchi
Nonprojectile
vomitus
No
meningeal
and
Babinski
sign noted

33rd HD (5/17/15)
Febrile episodes
Occasional cough
Vomited saliva once
Poor appetite
AC 1200 cm
Mushy yellow bowel
movement
Febrile at 41.3C ->
tepid sponge bath to
IV paracetamol due
to its persistence

COURSE IN THE WARD

35th HD (5/19/15)
patient had febrile
episodes
Repeat CBC with
platelet count (hgb
70, hct 0.21, plt
227, wbc 4.90, seg
0.50, lym 0.44,
bands 0.05, ANC
2,695)

36th HD (5/20/15)
patient was highly
febrile
41.2C,
blood culture and
sensitivity test and
urine culture and
sensitivity
were
requested
with
ARD
and
with
sensitivity
to
Cefuroxime,
Meropenem,
PiperacillinTazobactam,
Vancomycin,
Amikcain

37th HD (5/21/15)
blood and urine
culture
and
sensitivity testing
were
facilitated
with
ARD.
PediatricsInfectious service
advised that the
patient be started
on
Cefepime.
PiperacillinTazobactam
was
shifted
to
Cefepime (650 mg
per
slow
IV
infusion over 30

39th HD (5/23/15)
patient had febrile
episode
with
a
maximum
temperature
of
37.7C.
Tepid
Sponge bath was
done

DISCUSSION:
HEPATOBLASTOMA

Epidemiology
Pathophysiology
Clinical Presentation
Diagnosis
Management

HEPATIC TUMORS
Primary hepatic
tumors are rare
in children (12%)
1% of
malignancies in
children (annual
incidence of 1.6
cases per
million)
Lanzkowsky, Philip. Manual of Pediatric Hematology and Oncology. 5th Edition. 2011

EPIDEMIOLOGY
Predominantly in
children below 3 years
old (80%)
Etiology is unknown
associated with FAP,
Beckwith-Wiedermann
Syndrome

Alterations in the
antigen presenting
cell (APC) / B-catenin
pathway
Birth weight
increasing risk as birth
weight decreases

DISORDERS ASSOCIATED
WITH INCREASED RISK
OF HEPATOBLASTOMA
Low-birth-weight infant

Fetal alcohol Syndrome

Von Gierke Disease

Familial Adenomatous Polyposis

Congenital Cystathionuria and

Hemihypertrophy

Gardner Syndrome

Maternal Use of Hormonal therapy

Prader-Willi Syndrome

Exposure to Metals such as

welding and soldering fumes

Meckels Diverticulum

Type Glycogen Storage Disease

Beckwith-Wiedemann Syndrome

Congenital Absence of the

Adrenal Gland

Li-Fraumeni Syndrome

Congenital Absence of the Kidney

Trisomy 18

Umbilical Hernia
Lanzkowsky, Philip. Manual of Pediatric Hematology and Oncology. 5th Edition. 2011

DISORDERS ASSOCIATED
WITH INCREASED RISK
OF HEPATOBLASTOMA
Familial Adenomatous
Polyposis

Beckwith-Wiedemann
Syndrome

Lanzkowsky, Philip. Manual of Pediatric Hematology and Oncology. 5th Edition. 2011

HEPATOBLASTOMA
EPITHELIAL TYPE

Fetal malignant cells

Embryonal malignant cells

MIXED TYPE

Mesenchymal + Epithelial elements

Mixture

Pure more favorable outcome

CLINICAL PRESENTATION
Common Manifestations
Large, asymptomatic
abdominal mass
3x right lobe compared to the
left lobe
Unifocal

Disease Progression
Weight loss
Anorexia
Vomiting
Abdominal pain
Lanzkowsky, Philip. Manual of Pediatric Hematology and Oncology. 5th Edition. 2011

DIAGNOSIS: LABORATORY
ALPHA-FETOPROTEIN (AFP)
Diagnosis
Monitoring
Elevated in almost all
hepatoblastomas
ANEMIA
Common

BILIRUBIN, LIVER ENZYMES

Usually normal

HEPATITIS B AND C SEROLOGY

Should be obtained
Usually negative

THROMBOCYTOSIS
Occurs in 30% of patients

Nelson,M.R. (2011).Pediatrics. New York: Demos Medic

DIAGNOSIS: ANCILLARIES
PLAIN RADIOGRAPHS
Characterize the hepatic mass

CT / MRI OF THE ABDOMEN

Accurate
Defines the extent of the intrahepatic
tumor involvement
Defines the potential for surgical
resection

DIAGNOSIS: ANCILLARIES
ABDOMINAL ULTRASOUND
Characterize the hepatic mass

CT SCAN OF THE CHEST

Evaluation of metastasis

FETAL PATTERN

EMBRYONAL PATTERN

MIXED EMBRYONAL AND

FETAL PATTERN

MIXED EPITHELIAL AND

MESENCHYMAL PATTERN

PATIENTS SPECIMEN

PATIENTS SPECIMEN

MANAGEMENT
Only patients in
whom complete
resection can be
achieved have a
reasonable chance of
cure
Possible in 40-50% of
patients

Chemotherapy plays

Nelson,M.R. (2011).Pediatrics. New York: Demos Medic

MANAGEMENT: SURGERY
Patients who are candidate for complete
resection include those with:
Tumors confined to the right lobe
Tumors originating in the right lobe that do not
extend beyond the medial segment of the left lobe
Tumors confined to the left lobe

Biopsy should be performed in patients who

have tumor involvement of both lobes

Nelson,M.R. (2011).Pediatrics. New York: Demos

PRETEXT STAGE 1

PRETEXT STAGE 2

Childhood Liver Cancer Treatment - National Cancer Institute. (n.d.). Retrieved from
http://www.cancer.gov/types/liver/hp/child-liver-treatment-pdq#section/_373

PRETEXT STAGE 3

Childhood Liver Cancer Treatment - National Cancer Institute. (n.d.). Retrieved from
http://www.cancer.gov/types/liver/hp/child-liver-treatment-pdq#section/_373

PRETEXT STAGE 4

Childhood Liver Cancer Treatment - National Cancer Institute. (n.d.). Retrieved from
http://www.cancer.gov/types/liver/hp/child-liver-treatment-pdq#section/_373

MANAGEMENT
Stage Management
I and
II

Hepatoblastoma of pure fetal histology:

-complete surgical resection
-followed by watchful waiting
-or single-agent doxorubicin.
Hepatoblastoma with nonpure fetal histology:
-Gross surgical excision
-followed by four courses of combination chemotherapy
with cisplatin, vincristine, and fluorouracil or cisplatin and doxorubicin or cisplatin
alone.

III ad
IV

Chemotherapy followed by reassessment of surgical resectability followed by

complete surgical resection.
Chemotherapy followed by reassessment of surgical resectability. If the primary
tumor remains unresectable, an orthotopic liver transplantation may be
performed.
Childhood Liver Cancer Treatment - National Cancer Institute. (n.d.). Retrieved from
An alternative treatment approach ofhttp://www.cancer.gov/types/liver/hp/child-liver-treatment-pdq#section/_373
transarterial chemoembolization for

MANAGEMENT:
CHEMOTHERAPY
Drug

Route

Dosage

Days

Cisplatin

IV over 6
hours

100
1
mg/m^2 or

5Slow IV
600mg/m^ 2
Fluorouraci push over 2/dose
l
2-4minutes
Vincristine IV push
over 1
minute

1.5mg/m^ 2, 9, 16
2/dose

Doxorubici IV over
30mg/m^2 1, 2
n
15minutes /dose

Observations for possible AE

Hx, PEe, anthropometrics, VS
CBC
Electrolytes Ca, Mg, PO4,
creatinine, ALT/AST, bilirubin,
total protein/albumin, AFP
Tumor disease evaluation
Audiogram
Echocardiogram
A liver transplant consult
Pathology slides

MANAGEMENT:
CHEMOTHERAPY
Drug

Mechanism of Action

Adverse Effects

Cisplatin

Platinum coordination compound

that inhibits DNA synthesis

Nausea (76-100%)
Vomiting (76-100%)
Nephrotoxicity (28-36%)
Ototoxicity, especially in
children (31%)
Myelosuppression (2530%)
Anaphylaxis (1-20%)

5-Fluorouracil

inhibition of thymidylate synthase

(TS), the rate-limiting enzyme in the
pyrimidine nucleotide synthesis and
inhibition of DNA synthesis and
repair

Loss of appetite
Headache
Nausea Vomiting
Mucositis
Myelosuppression

MANAGEMENT:
CHEMOTHERAPY
Drug

Mechanism of Action

Adverse Effects

Vincristine

Vinca alkaloid; acts in M & S phases by

inhibiting microtubule formation,
inhibits DNA/RNA synthesis

Neurotoxicity
Cranial nerve palsies
Jaw pain
Autonomic neuropathy
(obstipation and ileus)

Doxorubicin

Anthracycline; intercalates between

DNA base pairs, impairs
topoisomerase II function and inhibits
replication & transcription

Neutropenia (52%)
Anemia (52%)
Leukopenia (42%)
Pruritus (37%)
Nausea (37%)
Stomatitis (37%)
Fatigue (33%)
CHF (30%)

MANAGEMENT:
RADIOTHERAPY
Not curative for intrahepatic disease
because hepatic tumor dose exceeds
hepatic tolerance
Useful for shrinking unresectable disease

PROGNOSIS
5 year Survival Rate
Stage

Percentage

I/II
III
IV

90
60
20
5 year Survival Rate

Pathology
Hepatoblastoma-fetal pattern
Fibrolamellar Carcinoma
Hepatoblastoma-embryonal
pattern
HCC

Prognosis
Favorable
Unfavaorable

CLINICA L E FFICA CY A ND P RO G NOSIS FA CTOR S FOR

ADVANCED HEPATO B LA STO MA IN CHILDREN: A 6YE AR RET ROSP ECTIV E ST UDY

JOURNAL APPRAISAL

CLINICAL QUESTION
Among pediatrics patients with
advanced hepatoblastoma treated with
multimodal combination therapy, what is
the mortality rate?

EDUCATIONAL
PRESCRIPTION

RELEVANCE

Is the objective of the article on prognosis

similar to your clinical diagnosis?

Yes.

PRIMARY VALIDITY
GUIDELINES
Was there a representative sample of patients
without the outcome at the start of the observation?

Yes

PRIMARY VALIDITY
GUIDELINES
Was follow up sufficiently long and
complete?

Yes.

PRIMARY VALIDITY
GUIDELINES
Were the criteria for determining the prognostic
factor and outcome explicit and credible (unbiased)?

Yes.

PRIMARY VALIDITY
GUIDELINES
Were there adjustments for other
prognostic factors?

None.

OVERALL, IS THE STUDY

VALID?
Was there a representative sample of patients

without the outcome at the start of the

observation?

Was follow-up sufficiently long and complete?

Were the criteria for determining the prognostic

factor and outcome explicit and credible
(unbiased)?
Were there adjustments for other prognostic
factors?
SINCE
ALL THE VALIDITY QUESTIONS WERE
FULFILLED, THE STUDY CAN BE CONSIDERED TO
BE VALID.

CAN THE RESULTS HELP ME IN

CARING
PATIENTS?
Are the FOR
study MY
patients
similar to my
own?

Yes.

CAN THE RESULTS HELP ME IN

CARING
FOR
MY PATIENTS?
Can I use
the results
to decide for an
intervention for my patient?

Yes.

FINAL RESOLUTION
Patients with epithelial type had poorer prognosis as
compared to patients with mixed type. Also, patients with
stage IV has a higher mortality rate (42.1%) as compared to
stage III (21.4%).
Survival rates of patients with HB treated with chemotherapy,
interventional therapy, surgery and APBSCT have been
significantly improved, with 5-year survival rate of about
70%. Hence, multimodality treatment effectively improved
remission rate and prolonged overall survival.

THANK