Risk-adapted Therapy for Primary Systemic (AL) Amyloidosis
Status:
Completed
Trial end date:
2005-09-01
Target enrollment:
0
Participant gender:
Summary
High-dose melphalan (MEL) with autologous stem cell transplant (SCT) is an effective therapy
for systemic AL amyloidosis (AL), but treatment-related mortality (TRM) has historically been
high. The investigators performed a phase II trial of risk-adapted SCT followed by adjuvant
dexamethasone (dex) and thalidomide (thal) in an attempt to reduce TRM and improve response
rates. Patients with newly diagnosed AL involving £2 organ systems were assigned to MEL 100,
140, or 200 mg/m2 with SCT, based on age, renal function and cardiac involvement. Patients
with persistent clonal plasma cell disease 3 months post-SCT received 9 months of adjuvant
thal/dex (or dex if there was a history of deep vein thrombosis or neuropathy). TRM was 4.4%.
Thirty-one patients began adjuvant therapy, with 16 (52%) completing 9 months of treatment
and 13 (42%) achieving an improvement in hematological response. By intention-to-treat,
overall hematological response rate was 71% (36% complete response) with 44% having organ
responses. With a median follow-up of 31 months, 2-year survival was 84% (95% confidence
interval: 73%, 94%). Risk-adapted SCT with adjuvant thal/ dex is feasible and results in low
TRM and high hematological and organ response rates in AL patients.