Info

🌱 來自: amyloidosis

treatment of amyloidosis

AL, poor prognosis

• Limited involvement:
  • high-dose melphalan → auto HSCT (NEJM 2007;357:1083)
• Not HSCT candidate:
  • VAD
  • dara-CyBorD :
    • daratumumab +
    • CYC + bortezomib + dexameth. (NEJM 2021;385:46).
  • [Low-dose melphalan + D] if Bor not tolerated.
• Relapse:
  • dara, ixazomib, Bor, or lenalidomide (Blood 2020;136:2620)

AA

• Rx underlying disease. Colchicine for FMF, esp. to ↓ renal dis. ? Anti-cytokine Rx (anakinra or tocilizumab) (Clin Exp Rheumatol 2015;33:46; Amyloid 2017;24:189).

ATTR

• Stabilize TTR tetramers: diflunisal slows neuropathy; tafamidis ↑ QoL, ↓ CV hosp/mortality (JAMA 2013;310:2658; NEJM 2018;379:1007)
• ↓ hepatic mut TTR production: siRNA (patisiran) or anti-sense oligo (inotersen) -improve neuropathy (NEJM 2018;379:11 & 22); CRISPR in trials (NEJM 2021;385;493)
• Liver transplant can benefit some mATTR forms (Muscle Nerve 2013;47:157)