Risk-adapted autologous stem cell transplantation with adjuvant dexamethasone ± thalidomide for systemic light-chain amyloidosis: Results of a phase II trial

Adam D. Cohen, Ping Zhou, Joanne Chou, Julie Teruya-Feldstein, Lilian Reich, Hani Hassoun, Beth Levine, Daniel A. Filippa, Elyn Riedel, Tarun Kewalramani, Michael D. Stubblefield, Martin Fleisher, Stephen Nimer, Raymond L. Comenzo

Research output: Contribution to journalArticlepeer-review

112 Scopus citations

Abstract

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. We 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 (n = 45) 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). Organ involvement was kidney (67%), heart (24%), liver/GI (22%) and peripheral nervous system (18%), with 31% having two organs involved. 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 haematological response. By intention-to-treat, overall haematological 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 haematological and organ response rates in AL patients.

Original languageEnglish (US)
Pages (from-to)224-233
Number of pages10
JournalBritish Journal of Haematology
Volume139
Issue number2
DOIs
StatePublished - Oct 2007
Externally publishedYes

Keywords

  • Amyloidosis
  • Clinical trials
  • Risk-adapted melphalan
  • Stem cell transplantation
  • Thalidomide

ASJC Scopus subject areas

  • Hematology

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