HSCT

Question 1

Diagnosis of SAA

Answer 1

Bone marrow cellularity < 30% and 2 of the following:

• ANC < 0.5
• ARC < 60
• platelets < 20

Question 2

poor prognostic factors in JMML

Answer 2

age >= 2 yrs
platelets < 40
high hemoglobin F

Question 3

what signal transduction pathway is overactivated in JMML

Answer 3

RAS signalling

Question 4

what genes are commonly mutated in JMML

Answer 4

PTPN11
NRAS
KRAS
NF1
CBL

Question 5

what are the stages of CML?

Answer 5

chronic phase
accelerated phase
blast crisis

Question 6

examples of 2nd generation TKIs

Answer 6

dasatinib
nilotinib
bosutinib

Question 7

what are the three risk factors including in the Pesaro classification for children with thalassemia

Answer 7

• adequate iron chelation
• portal fibrosis
• hepatomegaly > 2cm

Question 8

what is alemtuzumab

Answer 8

anti-CD52 (t-cells) for treatment of GVHD

Question 9

what is brentuximab

Answer 9

anti-CD30 conjugated to MMAE

Question 10

what are three metabolic conditions that can be cured by HSCT?

Answer 10

• hurler’s syndrome
• x-linked adrenoleukodystrophy
• metachromatic leukodystrophy

Question 11

what are the criteria for engraftment syndrome

Answer 11

Around the first day when ANC > 0.5:

• fever >= 38.3 C
• skin rash (> 25% of body surface)
• pulmonary edema (non cardiogenic) / hypoxia
• sudden increase in CRP

(note: there are minor criteria as well - increase in Cr, weight gain, hepatic dysfunction, diarrhea, encephalopathy)

Question 12

management of engraftment syndrome

Answer 12

Stop G-CSF

If fever persists after 48 hrs on antibiotics and cultures are negative, start methylpred 1mg/kg Q12hrs x 3 days then taper

Question 13

risk factors for engraftment syndrome

Answer 13

auto-HSCT, MAC, use of PBSC, G-CSF

Question 14

what are the HLA class I antigens

Answer 14

HLA A, B and C

Question 15

what are the HLA class II antigens

Answer 15

HLA DRB1, DQB1, DPB1

Question 16

3 goals of conditioning

Answer 16

• eradicate any malignant cells
• create a niche in the bone marrow for the donor HSC
• suppress recipient immune system

Question 17

two most common conditioning regimens

Answer 17

Cy/TBI (most common for ALL)

Bu/Cy (most common for myeloid malignancies)

Question 18

pros and cons of RIC

Answer 18

pros:
- less toxicity (end organ issues, infections etc)

cons:
- increased risk of graft failure, relapse

Question 19

name 5 factors to be considered when selecting a donor

Answer 19

• degree of matching (MHC)
• gender (male preferred > female)
• age (younger donor = better outcomes)
• blood type (minor factor)
• CMV status
• donor size (max allowed to take is 20mL/kg)
• HSC source

Question 20

where are class II MHC molecules located and what cells do they interact with?

Answer 20

on hematopoietic cells (APCs, B cells), interact with CD4 T cells

Question 21

where are class I MHC molecules located and what cells do they interact with?

Answer 21

on almost all nucleated cells (except some neurons) and interact with CD8 T cells

Question 22

which HLA antigens are included in 8/8 testing?

Answer 22

HLA-A, B, C

HLA-DR

Question 23

which type of cell product results in the fastest reconstitution?

Answer 23

PBSCs > BM > UC

Question 24

which cell product has the greatest risk of cGVHD

Answer 24

PBSCs > BM > UC

Question 25

what two medications can you use for mobilizing HSC?

Answer 25

G-CSF

Plerixafor (CXCR4 receptor antagonist)

Question 26

what are the chances of finding a matched unrelated donor?

Answer 26

depends on ethnicity - highest in people of european descent, lowest in african americans