A 22-year-old woman with sickle cell disease (HbSS) develops sudden severe chest pain, dyspnea, fever (38.5°C), WBC 18,000, SpO2 88% on room air, and a new bilateral infiltrate on CXR. This is her third episode of acute chest syndrome (ACS) in 2 years. What is the most important long-term disease-modifying intervention to prevent recurrent ACS?
- A Hydroxyurea therapy ✓
- B Long-term prophylactic penicillin
- C Monthly exchange transfusion program
- D Voxelotor (HbS polymerization inhibitor)
Explanation
Hydroxyurea (hydroxycarbamide) is the cornerstone disease-modifying therapy for SCD, inducing fetal hemoglobin (HbF) production which inhibits HbS polymerization. The MSH (Multicenter Study of Hydroxyurea) trial showed significant reduction in ACS episodes (>50%), pain crises, hospitalizations, and transfusion requirements. Hydroxyurea is indicated for recurrent ACS (≥2 episodes/year), frequent pain crises, or severe disease. Monthly exchange transfusion prevents recurrent stroke but is not the first-line for recurrent ACS. Voxelotor and crizanlizumab are newer adjuncts but not first-line for recurrent ACS prevention.
Reference: Harrison's Principles of Internal Medicine, 21st ed.
High-yield for: NEET PGINI-CETNExTFMGEUSMLEPLABMRCP
Written and medically reviewed by the StethoPrep medical team.