POT
GVHD prevention and treatment
cyclosporine, mtx, abatacept
Steroids
Ruxolitinib
Tocilizumab
Calcineurin inhibitors
Risk Assessment in ET
Which mutation carries the most favorable prognosis?
What must be ruled out with plt >1 mil?
Age >65, Hx of VTE --> add on hydrea
MPL, JAK (V617F and exon 12/13), CALR
vWD
These are the mutations seen in CNL, CML, aCML, Hairy Cell
CSF3R, BCR-ABL, SETBP1, BRAF V600E
AML defining mutations regardless of blast count (name 3)
NPM1
CEBPA
TP53
t(8;21) / RUNX1::RUNX1T1
inv(16)/t(16;16) / CBFB::MYH11
t(15;17) / PML::RARA
KMT2A rearrangements (11q23)
MECOM rearrangements (inv3/t(3;3))
DEK::NUP214 t(6;9)
RBM15::MRTFA t(1;22)
NUP98 rearrangements
Chuvash polycythemia AND associated hepcidin level
Inheritance
Treatment
VHL mutation --> upregulation of hypoxic response with normal O2 --> incr EPO --> polycythemia
Low hepcidin (body wants to take in more iron)
AR
Phlebotomy/Aspirin/Benadryl
Drugs and mutations causing treatment related MDS
Alkylating agents -- del 5 and del 7 - 5-7 yrs after treatment
Topoisomerase inhibitors - 11q23 or 21q22
Favorable AML mutations and treatment pathway
Midostaurin mutation
Ivosidenib mutation
NPM1 w/o FLT3
CEBPA biallelic
IDH1/IDH2
FLT3-ITD
Anticoagulation reversal
DOACs - AXA
Vit K, PCC - warfarin
heparin - protamine suldate
Idarucizumab - dabigatran
Burkitts rearrangement
t(8,14), t(8,22), t(2,8)
cMYC activation
All patients need IT therapy! (ALL also)
deletion 5q is favorable in MDS (targeted therapy?)
deletion 7q is unfavorable
True
Lenalidomide
types of vWD
1 -- quantitative
1c -- increased clearance
2 - qualitative
2A --> Abnormal multimers
2B --> Binds Platelets
2M --> Has multimers
2N --> No Factor VIII
3 --> absence
Myelofibrosis IPSS score
Age > 65
Symptoms
Hb <10 (2 points) WBC >25k
Circulating blasts>1
0 - low
1-2 - intermediate 1
3-4 - intermediate 2
5-6 - high risk -- straight to allo
Unfavorable karyotype: 8, 7q, i17q, -5, 12q, inv3, 11q23
ASXL, EXH2, SRSF1, IDH1/2
T/F
PBSCT → Higher GvHD, lower graft failure
Bone Marrow → Higher graft failure, lower GvHD
True
Hypereosinophilic Syndrome diagnosis and treatment
>1500 x 6mos
organ involvement
FIPL1/PDGFRA mutation (5q33)
Hydrea/IFNa/Alemtuzumab/Jak inh/Imatinib
acquired vWD
Acquired vWD causes:
MGUS
Hypothyroidism (lack of synthesis, T1)
Myeloproliferative dx → sticky plt
Valvular → shearing – Heyde’s syndrome
LVAD
Wilms tumors
SOS/VOD criteria, ppx and treatment
T bili >2
hepatomegaly/RUQ pain
Sudden wt gain >2% from baseline
First 21days after HSCT
Ursodiol/defibrotide
Factor replacement for Hemophilia A and B
1 iu/kg increases fviii level by 2% --> goal 100%
Hemophilia B 1:1 ratio --> goal 50%
List side effects for:
Imatinib
Desatinib
Bosutinib
Ponatinib
Desatinib →pleural effusions, pancytopenia
Bosutinib → diarrhea
Ponatinib → high rate arterioocclusive events start at 45mg
Target Ferritin for hemochromocytosis
<50 with phlebotomy
<1000 with chelation
Myeloid Sarcoma treatment
AML
Chediak Higashi
LYST or CHS1 gene mut. Plt storage defect. Thrombocytopenia + anemia + neutropenia. BMbc MPO inclusions in neutrophils.
Oculocutaneous Albinism or immune deficiency – high mortality rate. Steroids, splenectomy, ASCT
Large, fused azurophilic granules on the smear in granulocytes
How to manage inhibitors in hemophilia A
If <5 bethesda units, can still be managed with factor replacement, if >5, cannot
Can use steroids to get rid of inhibitor – respond well if <20 units
If titers >20, need to add cyclophosphamide. Rituximab as 2nd line.
Who should get washed and who should get irradiated prbcs?
Washed: IgA deficiency, PNH, severe reactions
Irradiated: Severe immunodeficiencies, stem cell transplant, familial transfusions (HLA)
POEMS syndrome
Polyneuropathy, organomegaly, endocrinopathy, M spike, skin changes. dVRD +/- auto
Cryoprecipitate contains
Fibrinogen
Factor VIII
Factor XIII
Fibronectin
vWD