Blood

ATGAM

stimulation of hematopoietic stem cells with the release of IL-3 and GM-CSF

cyclosporine A

calcinerurin (phosphatase activator of T-cellls) inhibitor

Eltrombopag

TPO-R agonist - more megakaryocyte = more platelets

crizanlizumab

mAb against P-selectin (adhesion molecule on the surface of activated endothelial cells in blood vessels and platelets

decitabine

cytidine analog that inhibits nucleic acid synthesis —> increase gene expression and fetal Hb can carry oxygen better

folic acid

increases stored folate to enable more erythropoesisi

L-glutamine

decreases oxidative damage by impoving NAD redox potencial to alleviate pain

hydroxyurea

decreases binding of HbS to endothelia and decreases vaso-occulsion and resultant pain

deferasirox/deferiprone/defoxamine

iron chealtor

Luspatercept

fusion protein/antibody that blaocks TGF-beta singlining in blood - enhances RBC development and corrects erythropoeisis

betibeglogene autotemcel (zynteglo)

encode for B T87Q-globin gene

desmopressin

modified ADH nonapeptide with increased V2 selecitivity —> mkaes body release more VWF

aminocaproic acid

inhibits plasminogen activation - prevents clot dissolution

transexamic acid

inhibits plasminogen activation - prevents clot dissolution, 10x more potent

von-willwbrand factor

recombinant VWF, promotes hemostasis by helping platelets adhere to collagen and protects factor VIII from proteolysis

efanesoctocog alfa (altuviiio)

anti-hemophillic factor (longer acting VWF)

emicizumab

mAb binds activated factor IX and factor X and this mediates function of VIII (which is missing in hemA)

concizumab (alhemo) and marsticimab (hympavzi)

target tissue factor pathway inhibitor (TFPI) - natural soluable anticoagulant (when blocked = more coagulation)

valoctogene (Roctavian) and entranacogene (hemagenix)

gene therapy for hemA and hmB respectively

fitusiran (qfitlia)

RNAi therapy inhibits production of antithrombin