Genetics and Clotting Meds

Recombinant Factor VIII (Hemophilia A)

• Use: First-line therapy for bleeding episodes or prophylaxis in children with Hemophilia A

• Route: IV infusion

• Key Points (MCH emphasis):

  • Provides clotting factor missing in Hemophilia A

  • Can be given at home by caregivers once trained

  • Must be given ASAP at first sign of joint bleeding (hemarthrosis)

  • Does not cure hemophilia → lifelong therapy

  • Risk: development of inhibitor antibodies that reduce drug effectiveness

  • Education: bleeding precautions (no IM injections, avoid aspirin/NSAIDs, soft toothbrush, avoid contact sports)

Morphine & Fentanyl (Sickle Cell Disease)

• Use: Gold-standard analgesia for vaso-occlusive pain crisis in children with SCD

• Route: IV opioids preferred (not IM → risk of muscle fibrosis and poor absorption)

• Key Points:

  • Regularly scheduled dosing or PCA (patient-controlled analgesia) is recommended

  • Avoid meperidine (Demerol) → neurotoxic metabolite can cause seizures

  • Always assess pain with age-appropriate scales

  • Side effects: respiratory depression, constipation, sedation (monitor closely)

  • Must balance pain control with safety (continuous pulse ox, naloxone available)

Hydroxyurea

• Use: Reduces frequency of sickle cell crises and acute chest syndrome

• MOA: Increases fetal hemoglobin (HbF), which decreases sickling of RBCs

• Key Points (MCH emphasis):

  • Indicated for children with frequent crises or severe disease

  • Requires regular CBC monitoring → can cause bone marrow suppression

  • Teratogenic → contraception required in adolescents of childbearing potential

  • Not a cure; improves quality of life and reduces hospitalizations

A child with hemophilia A develops joint swelling and refuses to bear weight. Which intervention is priority?
A. Apply warm compresses
B. Administer recombinant factor VIII
C. Encourage active range of motion
D. Elevate the leg and wait for improvement

B. Administer recombinant factor VIII
Rationale: Hemarthrosis requires immediate factor replacement. ROM and heat worsen bleeding; elevation may help but does not treat the underlying clotting deficit.

Parents of a 7-year-old with hemophilia ask about giving immunizations. Which response is best?
A. “Your child cannot receive any vaccines.”
B. “Vaccines should be given subcutaneously when possible.”
C. “All vaccines must be delayed until adulthood.”
D. “Vaccines should be given IM with a firm massage.”

B. “Vaccines should be given subcutaneously when possible.”
Rationale: IM injections increase bleeding risk; SQ administration is safer.

A 12-year-old with sickle cell crisis rates pain as 9/10. Which order should the nurse implement first?
A. Administer prescribed IV morphine
B. Apply cold compresses to painful areas
C. Offer acetaminophen PO
D. Encourage ambulation

A. Administer prescribed IV morphine
Rationale: IV opioids are the standard of care for acute vaso-occlusive pain crises.

Which statement by the parent of a child on a fentanyl PCA requires intervention?
A. “I will let my child push the button when in pain.”
B. “I understand naloxone may be used if breathing slows down.”
C. “I will press the PCA button while my child is asleep so they don’t wake in pain.”
D. “I know constipation is a common side effect.”

C. “I will press the PCA button while my child is asleep so they don’t wake in pain.”
Rationale: Only the patient should press the PCA button → prevents accidental overdose.

Which finding in a child on hydroxyurea requires immediate provider notification?
A. Hemoglobin 11 g/dL
B. White blood cell count 2,000/mm³
C. Mild nausea
D. Headache

B. White blood cell count 2,000/mm³
Rationale: Hydroxyurea can cause bone marrow suppression; leukopenia increases infection risk.