Patient-derived data can increase breadth of knowledge in rare cancers like Waldenström's Macroglobulinemia (WM), including Patient-reported outcomes (PROs).
This study utilised www.cart-wheel.org, an ethically-approved online rare cancer database for patient-derived data, and harnessed the digital connectedness of WM patients globally, to develop a continuously expanding dataset and platform for hypothesis generation around WM.
An ethics committee-approved WM-specific extension to the www.cart-wheel.org questionnaire, developed by clinician and patient investigators, was released June 2016. Participants complete consent online and enter their symptom, pathology and treatment data. Recruitment strategies driven by the International Waldenström's Macroglobulinemia Foundation investigators utilised multiple social media platforms. Inclusion of the EORTC-QLQ-C30 quality of life (QoL) questionnaire went live in October 2018, with targeted recruitment messaging sent out in February 2019.
355 patients from 17 countries have been recruited, predominantly from USA (48%) and Australia (22%), with male predominance (62%). Median age at diagnosis was 61 (range 24–83), median IgM 27.4 g/L (IQR 13.1–40.3 g/L, n = 130) and median hemoglobin 112 g/L (IQR 97–128 g/L, n = 134). Of the 279 (79%) patients providing symptoms at diagnosis, fatigue/muscle weakness was most common (45%) and 32% were asymptomatic. Using the Impact of Event Scale for symptoms of post-traumatic stress disorder (PTSD) resulting from a cancer diagnosis, at median 50 months post-diagnosis, the mean score among 305 patients was 5.8 (no stress = 0, maximal stress = 24), with 29/305 (10%) scoring ≥13 (PPV 94% for PTSD, Thoresen et al, 2010). 41 different first-line therapeutic combinations were documented by 238 patients. Median time from diagnosis to first treatment for USA patients was 50 days (IQR 15–423, n = 104) vs Rest of World (ROW) 170 days (IQR 22–775, n = 120), (p = 0.06). Of the 213 therapies listed by 108 USA patients, 59 (28%) were reported as accessed by government or public program and 8 (4%) via clinical trial participation, whereas among ROW patients (n = 113), 122/225 (54%) were government/public program accessed and 31/225 (14%) through clinical trials (p = 0.0001 and p = 0.0002, respectively). In the three weeks following targeted messaging, 156 patients responded to the QoL questions. Early analysis of raw scores for overall health (Q29) and overall quality of life (Q30), ranging from 1: very poor to 7: excellent, demonstrated means of 5.1 and 5.8, respectively. Respondents who received BTK inhibitors (BTKi, n = 33) had comparable Q29 and Q30 scores compared to those who received non-BTKi treatment (n = 84): Q29 means 5.8 vs 5.5, Q30 6.1 vs 5.7 (p = 0.2 and p = 0.13, respectively). This was despite median 2 (IQR 1–4) lines of treatment in the BTKi group compared to median 1 (IQR 1–2) in the non-BTKi group.
The WhiMSICAL study is a robust global patient-derived data platform, providing insight into patient symptoms, including WM-related stress responses, diversity of therapies and QoL. Further recruitment (Project 1000) and encouragement of more complete and continuous data entry will increase the utility of WhiMSICAL. As an expanding and increasingly reliable body of data, WhiMSICAL has the potential to map real-world PROs, and provide a scientific and ethically-approved portal for patients’ voices globally.