Life Raft Group

Tenosynovial Giant Cell Tumor (TGCT) is a rare, locally aggressive neoplasm that adversely impact patients' physical function and quality of life (QoL). This cross-sectional analysis leverages real-world data from the TGCT Support Patient Registry to elucidate the patient experience with TGCT and the disease burden across healthcare systems. A total of 497 patients from 32 countries, 71.4% (n = 355) with diffuse-TGCT (D-TGCT), 18.9% (n = 94) with localized TGCT (L-TGCT), and 9.7% (n = 28) with unspecified TGCT were included in this cross-sectional analysis of the TGCT Support Registry. A majority of patients (61.2%, n = 304) were diagnosed by orthopedic/sports medicine surgeons, half (n = 248) were misdiagnosed prior to their TGCT diagnosis, and 32% (n = 278) of patients were diagnosed > 24 months following symptom onset. 79.1% (n = 393) of all patients had ≥ 1 resection and 63% of those patients reported ≥ 1 recurrence. Of those patients that had recurrence following resection, 59% had ≥ 2 recurrences. 23% of patients (n = 115) changed occupations or prematurely retired due to TGCT and the proportion of patients increased with > 2 surgeries. Greater awareness of TGCT among HCPs is needed to facilitate diagnosis and referral to multidisciplinary teams is warranted to reduce recurrence rates, number of surgical interventions, and improve QoL.

Mutational testing for Gastrointestinal Stromal Tumor (GIST) patients remains underutilized. In this retrospective analysis, the target population (n = 1556) reported: 904 had molecular testing (“Tested”) vs. 652 without testing (“Untested”). Overall survival (OS) was 14.7 vs. 12.7 years (p < 0.00001), in metastatic patients 1st line OS was 8.9 vs. 5.9 years in the Tested vs. Untested group (n = 416 vs. n = 254), respectively. From 1st – 3rd-line, no difference has been (self-)reported for progression-free survival (PFS). Dropout to/for further lines of treatment was 15% for patients with a Tested mutation vs. 47% in Untested patients:

Abstract Background Due to the low mutational testing rate in patients with Gastrointestinal Stromal Tumors (GIST), The Life Raft Group (LRG), a non-profit organization that provides support, advocacy, and conducts research for patients with GIST, analyzed various factors that may have an impact on patients’ ability to receive mutational testing. Methods A survey about mutational testing for patients with GIST, or their caregivers, was conducted in June 2020. The survey, sent to 1004 GIST patients and caregivers through email, was promoted through social media with instructions to contact the LRG to participate. The survey was designed by the LRG Patient Registry Department. Members of the LRG, regardless of Patient Registry status, were eligible to participate. Results A total of 295 patients/caregivers participated in this study (response rate: 29.4%). The percentage of patients who indicated they had received mutational testing was much higher in this survey (80%) than in the general GIST community (26.7%). Several reasons were cited for having a test, including: “My doctor ordered/suggested that I have it done” (54%); “The Life Raft Group advised/suggested I have it done” (25%); “I asked my doctor to have it done” (22%); “I had it done as part of a clinical trial” (5%); “I am not sure” (3%) and “Other” (14%). Mutational testing resulted in a treatment change in 25% of cases. Patients were able to select more than one option when completing this question resulting in a percentage greater than 100.Conclusions The LRG membership is voluntary and proactive; patients who join are more likely to participate in surveys and mutational testing, as well has more likely to have a GIST specialist. Mutational testing can influence understanding a patient’s GIST and the treatment best suited to each case. These are extremely important findings, as it helps ensure that patients are on the proper treatment, which should lead to better outcomes.