WhiMSICAL (WALDENSTRÖM'S MACROGLOBULINEMIA STUDY INVOLVING CArt‐wheeL): A GLOBAL PATIENT‐DERIVED DATA REGISTRY MAPPING TREATMENT AND QUALITY OF LIFE

Introduction: Patient-derived data and reported outcomes (PROs) can increase breadth of knowledge in rare cancers like Waldenström's Macroglobulinemia (WM). This study utilised www.cart-wheel.org, an ethically approved online rare cancer database for patient-derived data, to develop a continuously expanding dataset for hypothesis generation around WM. Methods: An ethically approved WM-specific extension to the www.cart-wheel.org questionnaire was developed by clinician and patient investigators. Patients 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. Results: 355 patients from 17 countries have been recruited, predominantly from USA (48%) and male (62%). Median age at diagnosis was 61 years (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). Fatigue was the most common symptom listed at diagnosis (44%). Using the Impact of Event Scale for symptoms of posttraumatic stress disorder (PTSD) related to cancer, the mean score was 5.8 (no stress = 0, maximal stress = 24, n = 305), with 29 (10%) scoring >13 (PPV 94% for PTSD, Thoresen et al, 2010). 41 unique 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 USA patients (n = 108), 59 (28%) were reported as accessed by government or public program and 8 (4%) via clinical trials, whereas among ROW patients (n = 113), 122/225 (54%) were government/public program accessed and 31/225 (14%) via clinical trials (p = 0.0001 and p = 0.0002, respectively). 156 patients responded to the QoL questions within 3 weeks from targeted messaging. 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. Conclusions: WhiMSICAL is a robust global patient-derived data platform providing insight into patient symptoms, diversity of therapies and QoL. Further recruitment (Project 1000) and ongoing data entry will increase its utility. As it grows, WhiMSICAL has the potential to map real-world PROs to treatments and provide a scientific and ethically approved portal for patients’ voices globally. Keywords: BTK inhibitors; Waldenström's macroglobulinemia (WM). Disclosures: D'Sa, S: Consultant Advisory Role: Janssen Cilag, Amgen; Honoraria: Janssen Cilag, Amgen; Research Funding: Janssen Cilag, Amgen; Other Remuneration: Janssen Cilag (Education grant). Kersten, M: Honoraria: Kite Pharma, Novartis Pharmaceuticals Corporation, MSD, BMS, Gilead Sciences, Mundipharma, Millennium/Takeda, Celgene, Roche, Amgen; Research Funding: Millennium/Takeda, Celgene, Roche. Harrington, C: Stock Ownership: AbbVie, Gilead.