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Response rates and survival in primary systemic amyloidosis
M A Gertz1, R A Kyle, P R Greipp
1Dysproteinemia Clinic, Mayo Clinic, Rochester, MN 55905.
Blood
|January 15, 1991
Summary
Alkylating agent-based chemotherapy shows benefit for a subset of patients with primary systemic amyloidosis (AL). A trial of chemotherapy is recommended, as responders demonstrate a significant survival benefit.
Area of Science:
- Hematology
- Oncology
- Nephrology
Background:
- Primary systemic amyloidosis (AL) is a plasma cell dyscrasia characterized by amyloid deposition.
- Treatment options for AL amyloidosis have historically been limited, with a focus on managing organ damage.
Purpose of the Study:
- To evaluate the response rate and survival outcomes of patients with AL amyloidosis treated with alkylating agent-based chemotherapy.
- To identify factors influencing treatment response and survival in AL amyloidosis.
Main Methods:
- Retrospective analysis of 153 patients with biopsy-proven AL amyloidosis.
- Assessment of response rates, survival, and prognostic factors, including serum creatinine and echocardiographic findings.
Main Results:
- Overall response rate to alkylating agent-based chemotherapy was 18% (27/153).
- Patients with nephrotic syndrome, normal creatinine, and no cardiac involvement had a 39% response rate.
- Median survival for responders was 89.4 months, compared to 14.7 months for non-responders.
- A subset of responders (78%) survived 5 years, while all non-responders died within this timeframe.
Conclusions:
- Alkylating agent-based chemotherapy offers a survival benefit for a subset of AL amyloidosis patients.
- Early identification of potential responders and risk stratification are crucial for optimizing treatment strategies.
- Further research into novel therapeutic approaches for AL amyloidosis is warranted.