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Newer Therapies for Amyloid Cardiomyopathy

Rajshekhar Chakraborty1, Eli Muchtar1, Morie A Gertz2

  • 1Division of Hematology, Mayo Clinic, Rochester, MN, USA.

Current Heart Failure Reports
|August 8, 2016
PubMed

Insights

Systemic amyloidosis commonly affects the heart, impacting survival. Treatments for light chain (AL) and transthyretin (TTR) amyloidosis range from chemotherapy and transplantation to novel agents and supportive care.

Area of Science:

  • Cardiology
  • Hematology
  • Nephrology

Background:

  • Systemic amyloidosis frequently involves the heart and kidneys, leading to increased morbidity and reduced survival.
  • Light chain (AL) and transthyretin (TTR) amyloidosis are the primary types causing cardiac involvement.
  • Cardiac amyloidosis significantly impacts patient outcomes and treatment tolerance.

Purpose of the Study:

  • To review current and emerging treatments for cardiac amyloidosis.
  • To discuss management strategies for both AL and TTR amyloidosis.
  • To highlight the role of transplantation and pharmacologic agents.

Main Methods:

  • Review of established and novel therapeutic approaches for cardiac amyloidosis.
  • Analysis of treatment outcomes for different amyloidosis subtypes.
  • Discussion of supportive care and emerging drug therapies.

Main Results:

  • Traditional treatments for AL amyloidosis include chemotherapy and stem cell transplantation.
  • Novel agents show promise for AL amyloidosis in various settings.
  • Orthotopic heart transplantation (OHT) with autologous stem cell transplantation (ASCT) improves outcomes.
  • Orthotopic liver transplantation (OLT) is primary for TTR amyloidosis, but cardiac progression can occur.
  • Combined OLT and OHT offers benefits in select TTR amyloidosis patients.
  • Pharmacologic agents are under investigation to stabilize TTR.
  • Supportive care for heart failure is crucial for all cardiac amyloidosis types.

Conclusions:

  • Treatment strategies for cardiac amyloidosis vary based on type (AL vs. TTR).
  • Transplantation, novel agents, and supportive care are key components of management.
  • Further research is needed for pharmacologic stabilization of TTR and optimal use of supportive therapies.
Keywords:
Amyloid cardiomyopathyCardiac involvementLight chainSystemic amyloidosisTransthyretin

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