Light-chain (AL) amyloidosis - null regimens

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The purpose of this page is to provide references to "null therapy" such as placebo and observation. While not clinically relevant, these references provide further insight into the historical development of the treatment landscape. See the main ligh-chain (AL) amyloidosis page for current regimens.


First-line therapy (including transplant ineligible)

No induction

Regimen

Study Years of enrollment Evidence Comparator Comparative Efficacy
Huang et al. 2014 (NJCT-0703) 2009-2012 Randomized Phase 2 (C) BD Seems to have inferior OS

No induction prior to transplant.

Subsequent treatment

References

  1. NJCT-0703: Huang X, Wang Q, Chen W, Zeng C, Chen Z, Gong D, Zhang H, Liu Z. Induction therapy with bortezomib and dexamethasone followed by autologous stem cell transplantation versus autologous stem cell transplantation alone in the treatment of renal AL amyloidosis: a randomized controlled trial. BMC Med. 2014 Jan 6;12:2. link to original article contains dosing details in manuscript link to PMC article PubMed NCT01998503

Placebo

Regimen

Study Years of enrollment Evidence Comparator Comparative Efficacy
Kyle et al. 1978 NR Randomized (E-esc) MP Shorter time on treatment

No active antineoplastic treatment.

References

  1. Kyle RA, Greipp PR. Primary systemic amyloidosis: comparison of melphalan and prednisone versus placebo. Blood. 1978 Oct;52(4):818-27. link to original article PubMed