Sickle Cell Anemia Treatment

Question 1

SIckle Cell disease

1) characterized
2) when does it present?

Answer 1

1) genetic condition with clinical hallmark sxs of vaso-occlusive phenomena and hemolysis caused by an autosomal recessive hemoglobinopathy
2) characterized by presence of a particular hemoglobin structure that is sickled and poorly soluble when oxygen saturation is low
3) presents usually at a young age (hand/foot syndrome, hemolytic anemia, painful vaso-occlusive episodes)

Question 2

Sickle cell treatment modalities:

disease modifying

Answer 2

1) hydroxyurea
2) vaxelotor
3) crizanlizumab
4) L-glutamine

CHVL

Question 3

Sickle cell treatment modalities: infection prevention

Answer 3

these are highly susceptible to bacterial and viral infections - functional asplenia

1) immunizations
2) penicillin prophylaxis

Question 4

Sickle cell treatment modalities:

Chronic pain management

Answer 4

1) multi-modal approach- pharmacologic and non-pharmacologic

2) multiple pharmacologic classes used

Question 5

Hydroxyurea indications

Answer 5

sickle cell anemia, AML, head/neck CA

first line agent for sickle cell disease

Question 6

US Box warnings Hydroxyurea

Answer 6

1) bone marrow suppression- may cause severe myelosuppresion- monitor blood counts at baseline and throughout treatment
2) secondary malignancy- carcinogenic and may lead to secondary malignancies- advise sun protection

Question 7

MOA of Hydroxyurea

Answer 7

1) in SCD- increases RBC hgb F levels, RBC water content, deformability of sickled cells
2) oncology - ANTIMETABOLITE that prevents conversion of ribonucleotides to deoxynucleotides, halting cell cycle

Question 8

ADR of Hydroxyurea

Answer 8

1) eczema
2) macrocytosis (almost half of patients)
4) infections (can be severe)

Question 9

Voxelotor indications

Answer 9

sickle cell disease

Question 10

Voxelotor MOA

Answer 10

HbS polymerization inhibitor- reversibly binds to Hgb and stabilizes the oxygenated Hgb state- inhibit RBC sickling

Question 11

Voxelotor ADR

Answer 11

1) rash
2) GI disturbances
3) HA

Question 12

Voxelotor metabolism/excretion

Answer 12

Reduce dose in SEVERE liver impairment (pugh class c)

Question 13

Crizanlizumab Indications

Answer 13

Sickle cell disease- reduces frequency of vaso-occlusive crises- reduced pain crises by neaRly 45% vs palcebo

Question 14

MOA Crizanlizumab

Answer 14

Binds to P-selectin causing inhibition between endothelial cells, platelets, RBCs, and WBCS, resulting in decreased platelet aggregation, maintenance of blood flow and minimized sickle cell related pain

Question 15

Route Crizanlizumab

Answer 15

IV- monthly

Question 16

ADR Crizanlizumab

Answer 16

1) abdominal pain and nausea

2) back pain and arthralgia

Question 17

Prophylactic PCN recommendation for SCD

Answer 17

1) recommended until age 5 for all children with SCD
2) ages 3 mo- 3 year - PCN v
3) Ages 3-5 - PCN V (increased dose)
4) decreases pneumococcal infections and decreased risk of death with minimal AE short or long term
4) whether taking PCN or not, fever remains a LIFE THREATENING CONDITIONING IN THOSE WITH SCD

Question 18

Immunizations with SCD

Answer 18

1. all childhood vaccines according to normal schedule
2. pneumococcal vaccines- current recommendation is both PCV13 and PPSV23
3. meningococcal vaccine
4. influenza vaccine yearly