Autologous stem cell transplantation (ASCT) is considered the most effective therapy in AL, but treatment related mortality (TRM) is significant. Melphalan-dexamethasone (MDex) is well tolerated and grants a high response rate (

Blood 2004; 103: 2936-8
). In order to reduce TRM, after stem cell harvest, we treated AL patients eligible for ASCT with first-line MDex, offering second-line ASCT to non-responders (NR). Eligibility to ASCT was determined according to Comenzo and Gertz (
Blood 2002; 99: 4276-82
), including good and intermediate risk patients. Response to MDex was evaluated every 3 cycles. MDex was continued until either complete remission (CR) or partial hematologic response (PR) associated with organ response (PR+OR) was reached. MDex was discontinued after 3 cycles in NR or after 9 cycles in patients in PR without organ response (PRNOR). Patients in PRNOR or NR received second-line therapy: ASCT if they were still eligible, or thalidomide-dexamethasone (
TDex, Blood 2002; 105: 2949-51
) if they became ineligible for ASCT. Forty patients (median age 55 years, range 32–67 years, 20 males, 15 good-risk) were enrolled between March 2002 and June 2005. Twenty-five (63%) responded to MDex (11 CR, 14 PR). Hematologic response was accompanied by OR in 23 cases. There was no TRM. Seventeen patients (42%), 15 NR and 2 in PRNOR received second-line therapy. Seven were still eligible for ASCT. Although 2 of them were enrolled as good-risk, at the time of ASCT all were intermediate-risk and received conditioning with melphalan 140 mg/m2. Two patients died within 100 days and 2 (29%) responded. The 2 responders had obtained PRNOR after MDex. Ten patients received second-line TDex. One died within 100 days and none responded. Overall, 6 patients (15%), none of whom responded to MDex, died. Median follow-up of living patients is 27 months. Median survival was not reached. Response to MDex improved survival (p<.001). The efficacy and tolerability of MDex is confirmed. Approximately 60% of NR to MDex lose eligibility for ASCT. After MDex, second-line ASCT is associated with apparently increased TRM. Response to second-line ASCT was observed only in patients who obtained PR with MDex.

Topics:
autologous stem cell transplant, dexamethasone, immunoglobulin deposition disease, melphalan, primary amyloidosis, second line treatment, complete remission, follow-up, thalidomide, stem cells

Disclosure: No relevant conflicts of interest to declare.

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2006, The American Society of Hematology
2006
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