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Medicinal Chemistry > Hemophillia > Flashcards

Hemophillia Flashcards

(75 cards)

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1
Q

What is hemophilia?

A

Genetically inherited bleeding disorder

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2
Q

What genes are affected and where is it located?

A

Genes for factor VIll and IX located on the x chromosome

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3
Q

What is the root cause for hemophilia

A

Low or no production of particular clotting factors

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4
Q

What are the types of hemophilia

A

Hemophilia A: factor VIll deficiency

Hemophilia B: factor IX deficiency

Acquired hemophilia: autoantibody against VIII

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5
Q

What are hemophilia complications?

A

Joint damage/disease

Risk for viral infections

Inhibitor/ neutralizing antibodies

Heart disease, high blood pressure

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6
Q

Joint damage/ disease

A

Chronic bleeding at joints

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7
Q

Risk of viral infections

A

HIV

Hepatitis

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8
Q

Inhibitor/ neutralizing antibodies

A

30% gf hemophilia A patients develop abs

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9
Q

Heart disease/HBP

A

Caused partly by inability to be active

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10
Q

Hemophilia therapeutics

A

Plasma transfusion of low concentrations of clotting factors

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11
Q

How was plasma concentration of clotting factors improved

A

Cryopercipitation

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12
Q

What was the major concern for increased plasma concentration of clotting factors through cryopercipitation

A

Neutralizing antibodies produced in the body

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13
Q

Cryoprecipitate factor VIll

A

Treat hemophilia A

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14
Q

What is the major concern for the use of cryoprecipitate factor VIll

A

Caries risk of viral infection

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15
Q

How is cryoprecipitate factor VIll prepared

A

From a single donor and whole blood

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16
Q

Why was cryoprecipitate factor VIll preferred over lypholized

A

Lypholization disrupts the polymeric structure of factor VIll and it requires pooling from multiple homers

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17
Q

Lyophilized factor IX

A

Treatment of hemophilia B

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18
Q

How is lyophilized factor IX prepared

A

Extracted from plasma, most inactive protein but can still activate clotting factors

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19
Q

Why is heparin added for patients taking lyophilized factor IX

A

To limit thrombin overactivation

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20
Q

What is mononine?

A

Monoclonal antibody specific to factor IX to cleanly isolate the targeted factor IX which is separated from antibody and undergoes further purification

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21
Q

How is monomine prepared and administered?

A

Pooled from human plasma and administered via IV

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22
Q

Which patient is administered NovoSeven (rFactor VIIa)

A

Patient with VIl deficiency and treatment of bleeding episodes in hemophilia

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23
Q

What is the MOA of NovoSeven

A

Increases thrombin activity activity for both type A and B

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24
Q

How is NovoSeven prepared?

A

Human gene recombinantly expressed in baby hamster kidney cells

Secreted protein is cleaved to its VIIa active form

Packaged in lyophilized single use vial (IV)

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25
What is NoveSeven RT
Room temperature stable formulation of NovoSeven
26
Obizur (r porcine factor VIll): what type of hemophilia is it used for?
A
27
Obizur is what kind of therapeutic
Antibody with a 90 kD heavy chain and 80 kD light chain
28
How is obizur produced?
Expressed in hamster kidney cells and affinity for purified
29
Obizur half life
11 hours
30
How is obizur packaged and administered
Packaged lyophilized Administered IV
31
True or false: obizur has a comparable activity to human factor VIII
True
32
Eloctate (r Factor VIII- Fc fusion protein) is approved for the treatment of what type of hemophilia
A
33
What type of therapeutic is eloctate?
Analog of hFactor VIII covalently linked to human lgG
34
When the IgG region of eloctate binds to an IgG receptor, what occurs?
There is a delay in lysosomal degradation of IgG by returning them to circulation
35
Eloctate half life
16-20hrs
36
How is eloctate produced?
Expressed in HEK cells and purified
37
How is eloctate-packaged and administered
lyophilized and IV
38
Nuwiq (r human factor VIIII; truncated) is approved for the treatment of what type of hemophilia
A
39
What is the characteristic of nuwiq
Contains heavy and light chain that lacks a b-domain
40
True or false: Nuwiq heavy and light chain not are covalently attached
True
41
Nuwiq half life
17 hours
42
How is Nuwiq produced
HEK cells and purified
43
How's Nuwiq packaged and administered
lyophilized and IV
44
Kovaltry (r human factor VIll) is approved for what type of hemophilia?
A
45
What type of therapeutic is kovaltry?
Unmodified full length human factor VIII
46
How is kovaltry produced?
Express in hamster kidney cells and co-expressed with HSP70 proper folding
47
Kovaltry half life
12 hrs
48
How is kovaltry packaged and administered?
Lyophilized and IV
49
Aystyla (r single chain factor VIII) is approved for what type of hemophilia
A
50
How's afstyla designed
Single chain with heavy and light chain covalently linked
51
What clotting factor does afstyla increases its stability and has affinity for?
Von willebrand factor
52
What is the function of Von willebrand factor?
Prevents degradation of factor VIII
53
What other uses are there for afstyla?
Hemophilia A bleeding episodes Routine prophylaxis preop treatment
54
Afstyla half life
14 hours
55
How is afstyle prepared and administered
Lyophilized and IV
56
How is hemlibra (emicizumab) used in hemophilia theragry
It is a bispecific antibody that targets factor IXa and X and used as prophylaxis to reduce hemophilia A bleeding events, for patients with or without factor VIll inhibitors
57
What are other uses of hemlibra
Swelling and joint pain
58
What is the black box warning for hemlibra
Thrombotic microangiopathy and thrombotic events
59
What is esperoct (anti-hemophilic factor, glyopegylated- exei) used for
Hemophilia A
60
What is esperoct
Factor 8 analog linked to PEG (40 kD), used to prevent or reduce bleeding episodes of hemophilia A
61
Why does esperoct have PEG
It increases half life and reduces clearance
62
Esperoct half life
21.7 hrs
63
Aprolix (Factor IX-Fc fusion protein) use?
Hemophilia B
64
What type of therapeutic is alprolix
Recombinant factor IX fused to Fc fusion protein (IgG) Delays degradation and increases half-life
65
How is alprolix produced?
Expressed in HEK cells and purified
66
Alprolix half life
86 hours
67
Idelvion (r Factor IX fused to albumin) hemophilia type
B
68
Idelvion therapeutic type
Genetic fusion of human factor IX and human | albumin; expressed as a single protein
69
Idelvion mechanisms action
Circulates as fusion protein until factor IX is activated Albumin is cleaved from factor IX only when it is needed for clotting
70
Idelvion half life
90 hours
71
Rebinyn (Factor IX, GlycoPEGylated) Hemophilia type
B
72
Rebinyn (Factor IX, GlycoPEGylated) therapeutic type
Factor IX is glycoPEGylated to increase | circulating half-life of Factor IX
73
Rebinyn (Factor IX, GlycoPEGylated) Half life
118 hours
74
Rebinyn (Factor IX, GlycoPEGylated) Modification causes
Lower bleeding frequency and less frequent IV dosing
75
What is the only reconstituted IV
Idelvion (r Factor IX fused to albumin)

Medicinal Chemistry (2 decks)

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