2. clotting factors

Question 1

describe the normal clotting process

Answer 1

stage 1: blood vessels are damaged and bleeding starts
stage 2: blood vessels contract to slow to flow of blood to the injured area
stage 3: platelets stick to, and spread on, the walls of the damaged blood vessels. this is called platelet adhesion. these spreading platelets release substances that activated other nearby platelets which clump at the site of injury to form a platelet plug. this is referred to as platelet aggregation
stage 4: the surface of these activated platelets then provides a site for blood clotting to occur. clotting proteins like factor VIII and IX circulating in the blood are activated on the surface of the platelets to form a mesh-like fibrin clot

Question 2

what is the differences in the normal clotting process vs the clotting process in someone with a bleeding disorder

Answer 2

normal process: bleeding starts -> vessels constrict -> platelet plug -> fibrin clot

bleeding disorder: bleeding starts -> vessels constrict -> incomplete platelet plug -> incomplete formation of fibrin clot

Question 3

what is the process of fibrin formation?

Answer 3

fibrinogen, a glycoprotein, get hydrolyzed into fibrin by thrombin

Question 4

this is a tough, insoluble protein formed after injury to the blood vessels, which is essential in the clotting process

Answer 4

fibrin

Question 5

usually an inherited bleeding disorder in which the blood does not clot properly.

Answer 5

hemophilia

Question 6

hemophilia is caused by a deficiency of blood clotting factors ____, ___ or ___ which are carried on genes on the X chromosome

Answer 6

8,9 or 11

Question 7

will the daughters and/or sons of a man with hemophilia be carriers of the disease?

Answer 7

sons would be normal and daughters would be obligatory carriers

Question 8

will the daughters and/or sons of a woman carrier be carriers of the disease or get the disease?

Answer 8

sons have 50% chance of being hemophilic
daughters have 50% chance of being a carrier

Question 9

this type of hemophilia is caused by low levels of factor VIII (8); 90% gene mutation

Answer 9

hemophilia A

Question 10

this type of hemophilia is caused by low levels of factor IX (9); 9% gene mutation

Answer 10

hemophilia B

Question 11

this type of hemophilia is caused by low levels of factor XI (11); 1% gene mutation

Answer 11

hemophilia C

Question 12

this drug is a recombinant human antihemophilic factor VIII. it is used for the treatment of classic hemophilia A. it is produced by baby hamster kidney cells into which the human factor VIII gene is introduced. it is a highly purified glycoprotein consisting of multiple peptides including an 80kD and various extensions of 90 kD subunit

Answer 12

Kogenate FS

Question 13

this is the final step of manufacturing a biopharmaceutical

Answer 13

freeze drying/lyopholization

Question 14

how long after reconstitution does the lyophilized form of Kogenate need to be used

Answer 14

3 hrs

Question 15

true/false: Kogenate is given by injection (i.v)

Answer 15

true

Question 16

what is the purpose of the amino acids and sucrose in the formulation of Kogenate

Answer 16

to stabilize the formulation

Question 17

how must Kogenate be stored

Answer 17

• refrigerated but not frozen
• protect from extreme exposure to light and store lyophilized powered in the carton prior to use

Question 18

Kogenate FS, Recombinate, Advate, ReFacto and Hexilate are all trade names of products used to treat hemophilia. which one contains human albumin

Answer 18

Recombinate

Question 19

what are some common side effects of Kogenate

Answer 19

headache, cough, nausea, fever

Question 20

this drug is a human blood coagulation factor indicated in adult and paediatric patients with hemophilia B (congenital factor IX deficiency or Christmas disease); it is used for control and prevention of bleeding episodes and peri-operative management

Answer 20

BeneFIX

Question 21

this drug is a recombinant DNA derived coagulation factor IX concentrate indicated in adults and children with hemophilia B for control and prevention of bleeding episodes, peri-operative management, prophylaxis to prevent or reduce the frequency of bleeding episodes; Fc portion is fused to the effector protein, therefore this drug has an increased half-life (approx. 86 hours)

Answer 21

ALPROLIX

Question 22

this drug is the first long lasting antihemophilic factor (recombinant) Factor VIII, Fc fusion protein for use in a adults and children with hemophilia A; 5 day option and is sed for control and prevention of bleeding episodes, peri-operative management and routine prophylaxis to prevent or reduce the frequency of bleeding episodes

Answer 22

Eloctate

Question 23

this drug is a recombinant human antithrombin; it is a serine protease inhibitor that targets clotting factors IIa (thrombin)
it is used for the prevention of peri-operative and peri-partum thromboembolic (blood clot) events in hereditary antithrombin deficient patients; it was the first FDA approved protein drug produced by animals (goat milk)

Answer 23

ATryn