Amyloidosis

Amyloidosis is a disease in which specific precursor proteins misfold into beta-pleated sheets. Misfolded proteins adhere to form oligomers resulting in insoluble amyloid fibrils that deposit into tissue.

The FDA granted accelerated approval to daratumumab plus hyaluronidase-fihj in combination with bortezomib, cyclophosphamide and dexamethasone for treatment of newly diagnosed light chain amyloidosis.

Healio spoke with Taimur Sher, MBBS, MD, a hematologist, internist and oncologist at the Mayo Clinic Cancer and Multispecialty Amyloidosis Center, about the effects and potential intolerance issues of auto-hematopoietic stem cell transplantation in amyloid light chain (AL) amyloidosis patients.

Healio spoke with Taimur Sher, MBBS, MD, a hematologist, internist and oncologist at the Mayo Clinic Cancer and Multispecialty Amyloidosis Center, about advances in immunotherapy and improved approaches to AL amyloidosis.

Healio spoke with Taimur Sher, MBBS, MD, a hematologist, internist and oncologist at the Mayo Clinic Cancer and Multispecialty Amyloidosis Center, , on assessing the three most important factors in amyloid light chain (AL) amyloidosis patients and how relapses can guide treatment paths.

Healio spoke with Taimur Sher, MBBS, MD, a hematologist, internist and oncologist at the Mayo Clinic Cancer and Multispecialty Amyloidosis Center, about approaches to care in amyloid light chain (AL) amyloidosis and the future of monoclonal antibodies in treatment.

Utilization of pyrophosphate imaging to identify patients with cardiac amyloidosis has increased, although questions remain regarding its use for screening and to assess responses to therapy, according to a presentation.

The addition of bortezomib to oral melphalan and dexamethasone improved hematologic response rates and OS among patients with systemic light-chain amyloidosis, according to phase 3 study results published in Journal of Clinical Oncology.

While currently there is no cure for systemic amyloidosis, treatment options have recently expanded and have led to improved survival for both wild-type transthyretin and light-chain type amyloidosis that most commonly affect the heart.

Dhruv S. Kazi, MD, cardiologist and cardiovascular health economist at the Smith Center for Outcomes Research, Beth Israel Deaconess Medical Center, spoke with Healio about how tafamidis (Vyndamax, FoldRx/Pfizer) has changed the outlook of amyloidosis, but cost may remain a substantial barrier to access.