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. 2023 Oct;41(4):725-732.
doi: 10.1002/hon.3135. Epub 2023 Mar 27.

Outcomes for patients with systemic light chain amyloidosis and Mayo stage 3B disease

Foteini Theodorakakou  1 , Alexandros Briasoulis  1 , Despina Fotiou  1 , Ioannis Petropoulos  1 , Georgios Georgiopoulos  1 , Niki Lama  2 , Nikolaos Kelekis  2 , Evangelos Repasos  1 , Magdalini Migkou  1 , Kimon Stamatelopoulos  1 , Meletios A Dimopoulos  1 , Efstathios Kastritis  1
Affiliations

Outcomes for patients with systemic light chain amyloidosis and Mayo stage 3B disease

Foteini Theodorakakou et al. Hematol Oncol. 2023 Oct.

Abstract

Patients with cardiac light chain amyloidosis and Mayo stage 3b disease define a high-risk population with very poor prognosis. Here, we report treatment outcomes of 80 consecutive patients with newly diagnosed AL and Mayo 3b who received novel regimens. Early mortality (<1 month) rate was 12.5%. On intention-to-treat, overall hematologic response rate was 40%, with complete response (CR)/very good partial response (VGPR) in 25% and partial response (PR) in 15%. At 1- and 3- month landmark analysis CR or VGPR/PR rates were 25%/23.5% and 34%/25.5%, respectively. Among patients that were treated with daratumumab-based therapies, 52.6% and 85.7% achieved at least VGPR within one 1 and 3 months, respectively. Three-month cardiac response rate was 11.3% and 6-month was 18.8%. At least hemVGPR at 3 months was associated with cardiac response at 6 months (p = 0.034). Median overall survival (OS) was 6.3 months. At 1-month landmark at least hemPR was associated with better median OS (24.1 vs. 4.9 months, p = 0.017) and at 3-month landmark, at least hemVGPR was associated with a median OS of 40.7 versus 17 months for hemPR and 7.4 months for those without hematologic response (p = 0.028). Cardiac response at 3 months was associated with longer median OS (59.7 vs. 10.9 months, p = 0.044). Factors associated with poorer survival were κ-light chain amyloidosis (median OS 2.9 vs. 7.4 months, p = 0.028), peripheral nerve involvement (3.4 vs. 10.45 months, p = 0.024), systolic blood pressure <90 mmHg (2 vs. 8 months, p = 0.002), baseline LVEF <55% (median OS 3.4 vs. 32 months, p = 0.29) and New York Heart Association (NYHA) class (2.7 months for NYHA 3B-4 vs. 8 months for NYHA 2-3A, p = 0.02). Twenty-one patients (26.3%) received salvage therapy and ORR was 57.1%. Median OS for patients who received second line therapy was 24 months. In conclusion, patients with Mayo 3b disease benefit from early hematologic response but cardiac response rates remain low.

Keywords: NTproBNP; cardiac amyloidosis; free light chains; mayo stage.

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References

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