Evaluation of Self-Reported Progression and Correlation of Imatinib Dose to Survival in Patients with Metastatic Gastrointestinal Stromal Tumors: An Open Cohort Study

Abstract

ObjectivesSelf-reported progression was evaluated as a predictor of survival in patients with metastatic gastrointestinal stromal tumor (GIST).MethodsThis is a follow-up of an open cohort study of Life Raft Group (LRG) members with a diagnosis of KIT-positive metastatic GIST receiving imatinib from May 2000–December 2007 reporting their subjective response to therapy by completion of an internet-based questionnaire. Subjects received ≥ 1 year of imatinib and reported an initial positive response. Members reporting stable disease or progression were excluded. Self-reported progression-free survival (srPFS) was compared with overall survival (OS) and analyzed by starting and last reported dose.ResultsOne hundred sixty-nine subjects reported a mean starting dose of 527.8±177.9 mg/d at a mean age of 53.8±11.6 years at initial diagnosis. Of those reporting progression, 66% died versus 11% of those not reporting progression (P < 0.0001). When analyzed by last reported dose, a median srPFS benefit of 27.3 months was observed for the >400 mg/d group (P = 0.0017). Sixty-two percent of subjects who initiated therapy at >400 mg/d reported a dose reduction. When analyzed by last reported dose, a significant benefit in OS (P = 0.0229) and srPFS (P = 0.0069) was observed for subjects taking 600 over 400 mg/d.ConclusionssrPFS strongly correlated with OS. Significant advantages were observed when last reported dose was considered, as was higher daily dose. These observations suggest that careful escalation to intermediate daily doses should be investigated further for its potential to reduce the incidence and severity of adverse events, but also as a strategy against developing secondary resistance to imatinib.

Accelerated clinical discovery using self-reported patient data collected online and a patient-matching algorithm

“…In regards to the broader applicability of our approach, ALS may be a somewhat special case in that disease progression is relatively predictable, there are no effective treatments and patients are highly motivated to submit data. However, the same is true for many other rare and life-changing illnesses in need of effective treatments 27 . Preliminary attempts to replicate the matching techniques used in this study with multiple sclerosis patients suggest some benefit from using a matching algorithm to increase the accuracy of predictions in this episodic, treatable and slower-to-progress condition, although the benefits have been less substantial than for ALS.…”

Influence of Internet and Social Media in the Promotion of Alternative Oncology, Cancer Quackery, and the Predatory Publishing Phenomenon

“…Electronic consulting has facilitated faster enrollment in clinical trials and favored patients to travel to advanced oncologic treatment facilities [ 18 ]. Additionally, patients are now able to pool their own data (especially so-called patient-reported outcomes or PROs, considered extremely valuable endpoints in clinical trials) in international or multicenter aggregated databases that may help to corroborate clinical observations or generate new hypothesis for future trials [ 19 ].…”

Emerging Opportunities for Target Discovery in Rare Cancers

“…In other cases, patients have self-organized online and disseminated knowledge of their own disease (Call et al, 2010; Hauber et al, 2011). Illustrative examples include the role of patients with gastrointestinal stromal tumor (GIST) or chronic myeloid leukemia (CML) in the development and application of therapies targeting the KIT and Bcr-Abl oncoproteins (see Box 1).…”