HSCT Flashcards
Diagnosis of SAA
Bone marrow cellularity < 30% and 2 of the following:
- ANC < 0.5
- ARC < 60
- platelets < 20
poor prognostic factors in JMML
age >= 2 yrs
platelets < 40
high hemoglobin F
what signal transduction pathway is overactivated in JMML
RAS signalling
what genes are commonly mutated in JMML
PTPN11 NRAS KRAS NF1 CBL
what are the stages of CML?
chronic phase
accelerated phase
blast crisis
examples of 2nd generation TKIs
dasatinib
nilotinib
bosutinib
what are the three risk factors including in the Pesaro classification for children with thalassemia
- adequate iron chelation
- portal fibrosis
- hepatomegaly > 2cm
what is alemtuzumab
anti-CD52 (t-cells) for treatment of GVHD
what is brentuximab
anti-CD30 conjugated to MMAE
what are three metabolic conditions that can be cured by HSCT?
- hurler’s syndrome
- x-linked adrenoleukodystrophy
- metachromatic leukodystrophy
what are the criteria for engraftment syndrome
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)
management of engraftment syndrome
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
risk factors for engraftment syndrome
auto-HSCT, MAC, use of PBSC, G-CSF
what are the HLA class I antigens
HLA A, B and C
what are the HLA class II antigens
HLA DRB1, DQB1, DPB1
3 goals of conditioning
- eradicate any malignant cells
- create a niche in the bone marrow for the donor HSC
- suppress recipient immune system
two most common conditioning regimens
Cy/TBI (most common for ALL)
Bu/Cy (most common for myeloid malignancies)
pros and cons of RIC
pros:
- less toxicity (end organ issues, infections etc)
cons:
- increased risk of graft failure, relapse
name 5 factors to be considered when selecting a donor
- 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
where are class II MHC molecules located and what cells do they interact with?
on hematopoietic cells (APCs, B cells), interact with CD4 T cells
where are class I MHC molecules located and what cells do they interact with?
on almost all nucleated cells (except some neurons) and interact with CD8 T cells
which HLA antigens are included in 8/8 testing?
HLA-A, B, C
HLA-DR
which type of cell product results in the fastest reconstitution?
PBSCs > BM > UC
which cell product has the greatest risk of cGVHD
PBSCs > BM > UC
what two medications can you use for mobilizing HSC?
G-CSF
Plerixafor (CXCR4 receptor antagonist)
what are the chances of finding a matched unrelated donor?
depends on ethnicity - highest in people of european descent, lowest in african americans
