Low-risk patients can be treated with induction therapy followed by SCT. Appropriate selection criteria and induction have shown a hematologic response in 71% of cases, with a complete response in 37% of cases.
Intermediate- and high-risk patients who are ineligible for transplant should receive systemic chemotherapy with corticosteroids (dexamethasone, prednisone), alkylating agents (melphalan, cyclophosphamide), immunomodulatory drugs (thalidomide, lenalidomide), and proteasome inhibitors (bortezomib). Cyclophosphamide, bortezomib, and dexamethasone (CyBorD) is considered the standard of care in specialized centers, with a hematologic response rate of 60% and a 23% complete response. The humanized anti-CD38 monoclonal antibody, daratumumab, has been incorporated into frontline therapy since 2021 (DARA-CyBorD). A randomized, phase 3 trial, ANDROMEDA (NCT03201965), is investigating the safety and efficacy of DARA-CyBorD compared with CyBorD. Its completion date is expected in August 2024, but a safety run-in study showed a hematologic response in 96% of patients, with a complete response in 82%.
High-risk patients represent 15%-20% of all those with AL amyloidosis. Given their advanced cardiac dysfunction, the median survival of these patients has been estimated at 7 months.
The treatment response can be evaluated at 3 or 6 months after the initiation of therapy. Internationally validated criteria include changes in levels of dFLC, NT-proBNP, proteinuria, and/or eGFR. Organ response usually closely follows a hematologic response. Patients who fail to achieve a good response should be rapidly shifted to alternative rescue regimens.
The patient in this case-initiated DARA-CyBorD and showed a hematologic response within 3 months. Combined therapy was continued for 6 months, but because of severe fatigue, his regimen was adjusted to daratumumab monotherapy, which he has been tolerating well. His diarrhea greatly improved with opium tincture. He started total parenteral nutrition three times per week, which resulted in weight gain and increased energy. His orthostatic hypotension improved with the use of compression stockings and an abdominal binder, as well as pharmacologic interventions: midodrine (an alpha agonist) and droxidopa (a prodrug of norepinephrine).
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Cite this: C. Elena Cervantes. Nephrology Case Challenge: Man on Keto Diet Has Severe Diarrhea and Neurogenic Bladder - Medscape - Feb 06, 2023.