UCL logo

UCL Discovery

UCL home » Library Services » Electronic resources » UCL Discovery

Real world outcomes of pomalidomide for treatment of relapsed light chain amyloidosis

Sharpley, FA; Manwani, R; Mahmood, S; Sachchithanantham, S; Lachmann, H; Gilmore, J; Whelan, C; ... Wechalekar, A; + view all (2018) Real world outcomes of pomalidomide for treatment of relapsed light chain amyloidosis. British Journal of Haematology 10.1111/bjh.15541. (In press).

[img] Text
Sharpley_Pom paper final modified version 13 06 18.pdf - ["content_typename_Accepted version" not defined]
Access restricted to UCL open access staff until 11 August 2019.

Download (509kB)

Abstract

Pomalidomide is a next-generation immunomodulatory agent with activity in relapsed light chain (AL) amyloidosis, but real world outcomes are lacking. We report the experience of the UK National Amyloidosis Centre. All patients with AL amyloidosis treated with pomalidomide between 2009 and 2017 were included. Data was collected on treatment toxicity and clonal response. Survival was calculated by the Kaplan-Meier method and outcomes reported on an intent-to-treat (ITT) basis. A total of 29 patients treated with pomalidomide were identified. Haematological responses at 3 months were: complete response (CR) nil, very good partial response (VGPR) 10 (35%), partial response (PR) 9 (31%), stable or progressive disease 7 (24%), unevaluable 3 (10%). On an ITT basis (n = 28), responses at 6 months were: CR- nil, VGPR-11 (39%), PR-2 (7%) and the remaining patients were non-responders 15 (53%). Median overall survival was 27 months (95% confidence interval 15·7-38·1 months). Median progression free survival (PFS) was 15 months (95% confidence interval 6·24-23·77). In conclusion, pomalidomide has activity in patients with relapsed AL amyloidosis. Responses are rapid and early responses may be predictive of a sustained overall response. Deep responses (VGPR or better) are seen in only a third of all patients and combination therapy needs to be explored.

Type: Article
Title: Real world outcomes of pomalidomide for treatment of relapsed light chain amyloidosis
Location: England
DOI: 10.1111/bjh.15541
Publisher version: https://doi.org/10.1111/bjh.15541
Language: English
Additional information: This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions.
Keywords: amyloidosis, chemotherapy, haemotoxicity, pomalidomide, survival
UCL classification: UCL > Provost and Vice Provost Offices
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine > Inflammation
URI: http://discovery.ucl.ac.uk/id/eprint/10058022
Downloads since deposit
1Download
Download activity - last month
Download activity - last 12 months
Downloads by country - last 12 months

Archive Staff Only

View Item View Item