Autologous stem cell transplantation in light-chain amyloidosis
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memo https://doi.org/10.1007/s12254-020-00644-7
Autologous stem cell transplantation in light-chain amyloidosis Alexandra Böhm
Received: 1 July 2020 / Accepted: 1 August 2020 © Springer-Verlag GmbH Austria, part of Springer Nature 2020
Summary Treatment of patients with light-chain amyloidosis (AL amyloidosis) has changed over the last 20 years, and early mortality rates have decreased with prolongation of survival. However, many patients are not cured with conventional therapy. Therefore, all patients should be assessed at diagnosis to determine eligibility for autologous stem cell transplantation (ASCT) since high-dose melphalan and ASCT have been shown to induce long-term hematologic and clinical responses with treatment-related mortality (TRM) 15-year long-term OS in 30% (n = 159 patients between 1996 and 2003) of the patients [15]. These survivors were younger and were less likely to have lambda as the involved light chain and/or heart involvement. Median OS of patients with heart involvement was only 4 years, compared
Autologous stem cell transplantation in light-chain amyloidosis
to 11 years without. Receiving full-dose melphalan and achieving complete remission (CR) were independent predictors of OS. Day 100 TRM was 21%. A series of 629 patients at the Boston University School of Medicine [16, 17] reported a median survival of 7.6 years with a TRM in patients transplanted after 2005 of 3.4%. CR was obtained in 40% of patients with no difference for patients < or >65 years of age. The median OS for patients receiving 200 mg/m2 melphalan was 10.5 years compared with 5 years for those receiving 100 to 140 mg/m2 melphalan. Improvement in the function of at least one organ system occurred in almost 80% of those patients achieving CR, and in 39% of those who did not achieve CR. OS at 1, 5, 10, and 15 years for those with hematologic CR is 100%, 88%, 72%, and 57%, respectively, compared with those without CR of 94%, 60%, 34%, and 18%, respectively. In a prospective trial by Gertz et al. from the Mayo Clinic Rochester patients were allowed to choose between ASCT and standard therapy with oral melphalan plus dexamethasone [18]. Patients receiving conventional treatment had a 3-year progression-free survival (PFS) of 29% and an OS of 58.8%. Patients receiving ASCT had a 3-year PFS of 51.7% and an OS of 83.6%, hence, clearly favoring high-dose melphalan. The overall organ response rate with ASCT was 35.2%. A recent retrospective study analyzed “early” vs “deferred” ASCT, depending on whether ASCT was performed within 90 days after stem cell collection. In all, 527 patients underwent early and 124 deferred ASCT with no difference in OS [19].
Induction therapy pre and consolidation post ASCT Induction therapy is a standard component of multiple myeloma (MM) treatment and offered to AL amyloidosis patients with concurrent MM or with ≥10% bone marrow plasma cells [20]. The main goal of initial treatment is reducing the plasma cell clone and amyloid production and limit further organ damage. Patients not responding t
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