Hemophilia

Question 1

inheritance pattern

Answer 1

x-linked recessive

Question 2

bleeding severity in hemophiliacs correlates to

Answer 2

degree of factor deficiency

Question 3

To watch for with bleeding in extremities in hemophiliacs

Answer 3

compartment syndrome

Question 4

Term for joint syndrome stemming from long term hemophilia

Answer 4

Hemophilic arthropathy

Question 5

Most important principle of management

Answer 5

Treat bleeding ASAP (will not stop)

Question 6

treatment options

Answer 6

1) *recombinant factor concentrates
2) AntiFibrinolytics
3) Hemlibra
4) DDAVP

Question 7

Breakthrough drug for hemophilia treatment + mechanism + indication

Answer 7

Emicizumab (factor VIII mimetic)

- prophylaxis

Question 8

Interpretation of mixing study in which aPTT doesn’t fully correct

Answer 8

• factor inhibitor (with a factor deficiency, aPTT will FULLY correct)
• This indicates acquire hemophilia A

Question 9

Treatment of acquired hemophilia A

Answer 9

Activated factor VII
OR
Activated prothrombin complex concentrate
OR
Recombinant porcine factor VIII

Question 10

Disease association of acquired hemophilia A

Answer 10

CLL

Question 11

Risk factors for acquired hemophilia

Answer 11

• Elderly adults

- Women

Question 12

Clotting factor deficient in hemophilia A

Answer 12

Factor VIII

Question 13

Clotting factor deficient in hemophilia B

Answer 13

Factor IX

Question 14

FDA approved recombinant factor IX concentrates

Answer 14

Benefix

Alprolix

Question 15

Hemlibra (emicizumab) mechanism

Answer 15

• replaces function of factor VIII

Question 16

Severe hemophilia A definition

Answer 16

Factor level less than 1%

Question 17

Factor level defining Moderate hemophilia A

Answer 17

Factor level between 1 and 5%

Question 18

Mild hemophilia A definition

Answer 18

Factor level between 5 and 50%

Question 19

initial factor dose required to raise factor VIII level to normal for severely deficient

Answer 19

50 units/kg

Question 20

Goal of factor replacement in a major bleed

Answer 20

Goal is to raise factor levels to 100% initially and then maintain factor levels above 50% by bolus q8-12h or continuous infusion.

Question 21

Management of mild bleed in patient with mild hemophilia A

Answer 21

Desmopression or Amikar if mucosal bleed

Question 22

Goal of factor replacement with major surgeries

Answer 22

Give Factor VIII to maintain factor level at 100%;

Taper postoperatively to maintain levels above 50%; do not let it drop below 50%

Question 23

Assay used to check for factor VIII inhibitor

Answer 23

Bethesda assay

Question 24

What is FEIBA? mechanism? use?

Answer 24

• activated PCC
• bypasses the Factor VIII/common pathway and provides factor II/prothrombin to form a clot
• used fro patients with factor VIII inhibitors

Question 25

problem with FEIBA

Answer 25

Can be highly thrombogenic

Question 26

Prophylaxis for bleeding for patients with an inhibitor or acquired hemophilia

Answer 26

FEIBA (activated prothrombin complex concentrate)

Question 27

What are the 2 management strategies for hemophiliacs?

Answer 27

On demand (factor replacement during bleeds) vs prophylactic

Question 28

Management of hemophiliac with hypersensitivity or anaphylaxis to recombinant factor

Answer 28

• can’t give them any of the factor (either plasma derived or recombinant)

Question 29

What is inhibitor development associated with?

Answer 29

• increased risk of anaphylactic reaction to factor administration

Question 30

Options for managing an inhibitor

Answer 30

• immune tolerance induction (ITI or desensitization)

- immunosuppressive drugs, including rituxan

Question 31

Management of hemophilia B patient with inhibitor

Answer 31

recombinant factor VIIa (Novo Seven)

Question 32

What is REBINYN?

Answer 32

• Pegylated recombinant factor IX

- indicated for on-demand treatment and control of bleeding episodes

Question 33

MOA of emicizumab

Answer 33

• bridges activated factor IX and factor X to restore the function of activated factor VIII