101.3 🩺 內科專科考前版

101.3.0.1 📌 䞀頁重點

  • 22E: exa-cel (Casgevy CRISPR FDA 2023) for SCD + β-thal; CAR-T expansion (mosunetuzumab, talquetamab); ruxolitinib for steroid-refractory acute + chronic GVHD; PTCy haplo standard; defibrotide established VOD
  • Taiwan: 健保 allo-HSCT + auto-HSCT 條件 (severe disease); 健保 PBSC mobilization (G-CSF); 健保 CAR-T 限制 (ALL, DLBCL, MCL, MM 條件嚎栌); 健保 ruxolitinib for GVHD 條件; 健保 defibrotide 條件; gene therapy 自費 倚 (exa-cel, lovo-cel, beti-cel)

101.3.0.2 🌟 Pearls (10)

  1. CIBMTR + EBMT registries for outcome benchmarking
  2. HCT-CI (Comorbidity Index) for transplant fitness scoring
  3. DRI (Disease Risk Index) for transplant outcome prediction
  4. PTCy haplo vs ATG MUD: equivalent outcomes in many cohorts
  5. Defibrotide for VOD: SC + IV; reduces transplant-related mortality
  6. Ruxolitinib (Jakafi) for steroid-refractory acute GVHD (REACH-1) + chronic GVHD (REACH-3)
  7. Belumosudil (Rezurock) ROCK2 inhibitor for chronic GVHD
  8. Ibrutinib for chronic GVHD (selected)
  9. Exa-cel (CRISPR) real-world implementation challenges (cost, infrastructure, busulfan conditioning)
  10. CAR-T secondary T-cell lymphoma FDA black box 2024 — patient counseling

101.3.0.3 📍 Taiwan + 健保

  • 健保 allo-HSCT 條件 (AML/ALL/SAA/SCD/MDS 等; donor matching condition)
  • 健保 auto-HSCT 條件 (MM, lymphoma)
  • 健保 cord blood transplant 條件
  • 健保 PBSC mobilization (G-CSF) 充分
  • 健保 conditioning chemo (busulfan + cyclophosphamide + fludarabine + ATG)
  • 健保 GVHD prophylaxis (cyclosporine + methotrexate)
  • 健保 ruxolitinib for GVHD 條件 (新)
  • 健保 defibrotide for VOD 條件
  • 健保 CAR-T 限制 (tisagenlecleucel + axicabtagene 條件; 限制䞭心)
  • Exa-cel + lovo-cel + beti-cel 自費 倚 (新, 限制䞭心)

101.3.0.4 🎓 內專必懂 (10)

  1. HSC biology + niche + cytokines
  2. Source comparison (BM / PBSC / cord / haplo)
  3. Auto vs allo + conditioning regimens
  4. HCT-CI + DRI for fitness/risk
  5. GVHD acute + chronic + grading + treatment ladder
  6. Ruxolitinib + belumosudil for refractory GVHD
  7. VOD recognition + defibrotide
  8. CAR-T + CRS + ICANS management
  9. Gene therapy (exa-cel CRISPR) for hemoglobinopathy
  10. Late effects + lifelong surveillance

⚠ AI 草皿。