Table 1 Updates on therapies targeting plasma cell clones in AL amyloidosis

Kastritis et al. [2]

III

NCT03201965

Dara-VCd

CD38

NDAL (195 pts in Dara-VCd arm)

HemCR 59.5%

≥ CarVGPR 64.4%,

carCR 40.7%

Median MOD-PFS not reached, 5-year survival 76.1%

Grade ≥ 3 AEs: lymphopenia 13%, pneumonia 8%

Kastritis et al. [3]

II

NCT04131309

Dara

CD38

Stage IIIB NDAL (n = 40)

HemORR 77.5%,

≥ hemVGPR 55%, hemCR 27.5%

CarORR 50%,

≥ carVGPR 40%,

carCR 10%

median OS 10.3 months

Cardiac SAEs 42.5%

Parker et al. [4]

II

NCT03499808

Isa

CD38

R/RAL (n = 43)

HemORR 77.1%,

≥ hemVGPR 57%, hemCR 6%

CarORR 56%, renORR 50%

2-year estimated survival 85%

Grade ≥ 3 AEs: lymphopenia 8.5%, infection 6%

Mollee et al. [5]

II

NCT05066607

Isa-Pd

CD38

Relapsed AL (n = 42)

≥ HemVGPR 80.0%, hemCR 51.0%

N/A

N/A

SAEs: 4 deaths, 26 infectious, 8 cardiac

AEs: hematological 15%, infectious 13.5%

Kastritis et al. [6]

II

NCT04617925

Belamaf

BCMA

R/RAL (n = 35)

HemORR 51.4%,

≥ hemVGPR 31.4%,

hemCR 2.9%

CarORR 11.4% at 3 months

Median MOD-PFS 9.99 months, median OS 21.54 months

Ocular AEs 97.1%

Lebel et al. [7]

I

NCT04720313

CART

HBI0101

BCMA

R/RAL (n = 16)

HemORR 94%, hemCR 75%

Organ response 50%

median OS 223 days

Grade 1–3 CRS 87.5%, grade 3–4 neutropenia 62.5%

Shen et al. [8]

II

NCT05996406

Ven-d

Bcl-2

NDAL with t(11;14)  (n = 36)

≥ HemVGPR 62.9%,

hemCR 37.1%

CarORR 35.0%,

renORR 90.9%

N/A

Grade ≥ 3 AEs: lymphopenia 5.6%, liver dysfunction 5.6%

Ailawadhi et al. [9]

Ib/II

NCT04942067

Lisa-Pd

Bcl-2

R/RAL (10 pts in AL arm)

HemORR 85.7%,

≥ hemVGPR 71.4%,

hemCR 14.3%

N/A

N/A

Neutropenia 20.4%, nausea 16.3%, diarrhea 12.2%¹