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Ann Intern Med. 2004 Jan 20;140(2):85-93.

High-dose melphalan and autologous stem-cell transplantation in patients with AL amyloidosis: an 8-year study.

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  • 1Boston University School of Medicine, Boston, Massachusetts 02118, USA. mskinner@bu.edu

Abstract

BACKGROUND:

AL amyloidosis is a fatal disease resulting from tissue deposition of amyloid fibrils derived from monoclonal immunoglobulin light chains. Treatment with oral chemotherapy is minimally effective.

OBJECTIVE:

To test survival and organ response in a large sample of patients treated with high-dose intravenous melphalan (100 to 200 mg/m2) and autologous blood stem-cell transplantation.

DESIGN:

8-year longitudinal analysis of clinical effectiveness.

SETTING:

University-affiliated specialty referral clinic.

PATIENTS:

701 consecutive new patients with AL amyloidosis.

INTERVENTION:

High-dose chemotherapy and autologous stem-cell transplantation for patients who met eligibility requirements based on organ involvement and clinical status.

MEASUREMENTS:

Survival analysis of all patients evaluated and a detailed analysis of treatment outcome in the subgroup that received high-dose melphalan and stem-cell transplantation.

RESULTS:

Among 701 patients with AL amyloidosis, 394 (56%) were eligible for high-dose melphalan and stem-cell transplantation; 82 did not proceed with treatment because of patient choice or disease progression. Median survival of the 312 patients who initiated treatment was 4.6 years. A complete hematologic response, defined as no evidence of an underlying plasma cell dyscrasia 1 year after treatment, was achieved in 40% of patients and was associated with prolonged survival. Statistically significant improvements occurred in end-organ disease and were greater in patients with a complete hematologic response. Mortality rate within 100 days of treatment with high-dose melphalan and stem-cell transplantation was 13%; patients with cardiomyopathy had the highest mortality rates.

CONCLUSIONS:

Treatment of selected patients with AL amyloidosis by using high-dose melphalan and stem-cell transplantation resulted in hematologic remission, improved 5-year survival, and reversal of amyloid-related disease in a substantial proportion.

PMID:
14734330
[PubMed - indexed for MEDLINE]
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