105.3 🩺 內科專科考前版

105.3.0.1 📌 䞀頁重點

  • 22E:
    • Sutimlimab (Enjaymo, FDA 2022) anti-C1s for cold agglutinin disease — first targeted therapy
    • Iptacopan (Fabhalta, FDA 2023) oral factor B inhibitor for PNH — convenient
    • Pegcetacoplan (Empaveli) C3 inhibitor for PNH SC; also expanding aHUS
    • Danicopan (Voydeya) factor D inhibitor add-on for PNH residual hemolysis
    • Caplacizumab (Cablivi) for TTP — reduces relapse + mortality
    • Mitapivat (Pyrukynd) PK activator for PK deficiency hemolytic anemia (FDA 2022); trials for SCD/thal
  • Taiwan: 健保 prednisone, IVIG, rituximab, plasma exchange; 健保 eculizumab + ravulizumab 條件 (限制䞭心); sutimlimab + iptacopan + pegcetacoplan + danicopan + caplacizumab + mitapivat 自費 倚 / 條件 limited

105.3.0.2 🌟 Pearls (10)

  1. ADAMTS13 testing is mandatory + STAT in suspected TMA; turnaround time critical
  2. PEX 1.5x plasma volume for TTP; daily until plt > 150K × 2 days then taper
  3. Caplacizumab + rituximab in TTP standard now (FRENCH-TMA cohort, post-HERCULES)
  4. Atypical HUS recurrence: lifelong eculizumab in many; assess complement panel + genetics
  5. Drug-induced AIHA + ICI: rare but emerging; anti-PD-1 reported
  6. PNH FLAER test more sensitive than CD55/CD59 alone
  7. Mitapivat (FDA 2022) for PK deficiency: also trials for SCD (PRAISE) + α-thal (RISE-UP)
  8. Sutimlimab CAD: rapid hemolysis improvement, reduces transfusion
  9. Iptacopan oral PNH: replaces eculizumab in many — convenience + breakthrough hemolysis prevention
  10. HSCT for PNH: only curative; reserved for AA + concurrent disease + high-risk

105.3.0.3 📍 Taiwan + 健保

  • 健保 prednisone, IVIG, rituximab
  • 健保 plasma exchange 條件 (TTP, AIHA refractory, others)
  • 健保 eculizumab (Soliris) 條件 (PNH + aHUS)
  • 健保 ravulizumab (Ultomiris) 條件 (新)
  • Sutimlimab, iptacopan, pegcetacoplan, danicopan, caplacizumab 自費 倚 / 條件 limited (高貎)
  • 健保 ADAMTS13 + flow cytometry 條件
  • 健保 HSCT for PNH/aplastic 條件
  • 孞會: TSH (Taiwan Society of Hematology) + Taiwan TMA registry (forming)

105.3.0.4 🎓 內專必懂 (10)

  1. Hemolysis biochemistry markers + intrinsic vs extrinsic
  2. DAT/IAT interpretation
  3. G6PD + HS workup + smear morphology
  4. AIHA warm vs cold + treatment ladder
  5. Sutimlimab for CAD (22E)
  6. TTP ADAMTS13 + PEX + caplacizumab + rituximab
  7. HUS typical vs atypical + eculizumab
  8. PNH PIG-A + complement inhibitor ladder (eculizumab → iptacopan oral 22E)
  9. Mitapivat for PK deficiency (22E)
  10. TMA differential (TTP/HUS/DIC/aHUS/HELLP)

⚠ AI 草皿。