Patient reported outcome measures in Waldenström macroglobulinaemia: A real‐world data analysis from the WMUK Rory Morrison Registry

Abstract

Waldenström macroglobulinaemia (WM) is an incurable chronic B-cell malignancy, but highly responsive to treatment. Treatments include fixed-duration chemotherapy and continuous oral chemoimmunotherapy. In this expanding field, it is important to have reliable information on the impact of the various therapies on patients' quality of life (QoL). Patient reported outcome measures (PROMs) are increasingly recognised as important to understand patient experience of disease beyond traditional clinical outcome measures. Four QoL questionnaires (EORTC QLQ-C30 [European Organisation for Research and Treatment of Cancer quality of life core questionnaire], BIPQ [Brief Illness Perception Questionnaire], HADS [Hospital Anxiety and Depression Scale], EQ-5D-5L [EuroQoL 5-dimensional descriptive system questionnaire]) are embedded in the UK national WM registry, the Rory Morrison Registry. We reviewed the results from a snapshot of PROMs. As of November 2021, 155 patients completed PROM datawith 98% completion rate across all 58 questions. Complete clinical information was available for 52 patients. The majority of QoL questions (69%) failed to elicit a notable median response. Only four questions elicited statistically significant responses when comparing groups, and these were exclusively found in the EuroQoL-5D-5L and HADS questionnaires. Our data suggest that widely used questionnaires may not be suitable for patients with WM. We advocate the development of WM-specific outcome measures to overcome this.

The Rory Morrison WMUK Registry for Waldenström macroglobulinaemia: The growth of a national registry for a rare disorder

“…As of March 2022, there were 234 patients registered for PROM, 152 of which engaged regularly with the four quarterly questionnaires. Provisional analysis of data is underway, 11 and development of WM‐specific PROMS is in progress.…”

“…Direct and sequential data entry into a web‐based registry was feasible, but these questionnaires were not representative enough in the WM setting. This is the subject of a separate piece of work by our group 11 and speaks to the need to develop WM‐specific outcome measures.…”

Born in the UK: A registry to improve and broadcast knowledge on Waldenström macroglobulinaemia

“…Since November 2017, the RMR has grown significantly, has successfully passed into adulthood and has already given birth to numerous analyses presented at international haematology congresses and/or recently published. 5,10 With such a broadcast, we have not finished hearing about it! ORC I D Eric Durot https://orcid.org/0000-0003-3463-0089 Alain Delmer https://orcid.org/0000-0002-1430-2574…”

“…In their paper, Uppal et al 1 briefly introduce their collection of PROMs within the RMR using four questionnaires. They recently published a provisional report with QoL data on 155 patients from the RMR 5 . They demonstrate the feasibility of digital PROMs in their registry with high completion rates and highlight the need to develop WM disease‐specific PROMs.…”

“…With >1300 patients registered from 22 centres; he would be impressed with how far the registry has come. Since November 2017, the RMR has grown significantly, has successfully passed into adulthood and has already given birth to numerous analyses presented at international haematology congresses and/or recently published 5,10 . With such a broadcast, we have not finished hearing about it!…”