Daratumumab for relapsed AL amyloidosis—When cumulative real‐world data precedes clinical trials: A multisite study and systematic literature review. (9th November 2020)

• Record Type: Journal Article
• Authors: Shragai, Tamir; Gatt, Moshe; Lavie, Noa; Vaxman, Iuliana; Tadmor, Tamar; Rouvio, Ory; Zektser, Miri; Horowitz, Nethanel; Magen, Hila; Ballan, Mouna; Suru, Celia; Luttwak, Efrat; Levi, Shai; Ziv‐Baran, Tomer; Avivi, Irit; Cohen, Yael C.
• Is Part Of: European journal of haematology. Volume 106:Number 2(2021)
• Page Start: 184
• Page End: 195
• Publication Date: 2020-11-09
• Subjects: cardiac amyloidosis -- daratumumab -- light‐chain amyloidosis; Hematology -- Periodicals; Blood -- Diseases -- Periodicals; Blood -- Periodicals; 616.15005
• DOI: 10.1111/ejh.13535
• Languages: English
• ISSNs: 0902-4441

Abstract

Abstract: Objectives: Patients with relapsed/refractory AL amyloidosis (RRAL) have poor prognosis, but emerging data shows promising results with the use daratumumab. We evaluated daratumumab treatment in RRAL in real‐world setting. Methods: A retrospective multisite study of RRAL patients treated with daratumumab alone and in combinations. Results: Forty‐nine patients, diagnosed between 1.1.2008 and 1.2.2018 were included; 27% also had multiple myeloma (MM). Revised Mayo score was ≥ 3 in 67%. Hematologic overall response rate was 81%, 64% achieved very good partial response (VGPR) or better. Concurrent active MM was associated with lower rates of VGPR (OR 0.19, 95% CI 0.04‐0.81; P = .03) in a multi‐variate analysis. Cardiac and renal responses were 74% and 73%, respectively. Median progression‐free survival (PFS) was 28.4 months and median overall survival (OS) was not reached; 2‐year PFS and OS were 68.6 ± 7.5% and 90.4 ± 4.6%, respectively. Hematologic response correlated with prolonged PFS and OS. Daratumumab was safe and well tolerated, no patients discontinued therapy due to toxicity. Our data was aligned with outcomes from a systematic literature review, which identified 10 case series (n = 517) and 2 clinical trials (n = 62) meeting prespecified criteria. Conclusions: Our data support favorable safety tolerability and efficacy of daratumumab among non‐selective RRAL patients in a real‐world setting.