ABSTRACT
Estimating progression-free survival (PFS) and overall survival (OS) superiority during clinical trials of multiple myeloma (MM) has become increasingly challenging as novel therapeutics have improved patient outcomes. Thus, it is imperative to identify earlier endpoint surrogates that are predictive of long-term clinical benefit to expedite development of more effective therapies. Minimal residual disease (MRD)-negativity is a common intermediate endpoint that has shown prognostic value for clinical benefit in trials of patients with multiple myeloma (MM). This meta-analysis was based on the FDA guidance for considerations for a meta-analysis of MRD as a clinical endpoint and evaluates MRD-negativity as an early endpoint reasonably likely to predict long-term clinical benefit. Eligible studies were phase 2 or 3 randomized controlled clinical trials measuring MRD negativity as an endpoint in patients with MM, with follow-up of ≥6 months following an a priori defined time point of 12±3 months post-randomization. Eight newly diagnosed MM-(NDMM)-studies evaluating 4,907 patients were included. Trial-level associations between MRD-negativity and PFS were R2WLSiv (95% CI) 0.67 (0.43-0.91) and R2copula 0.84 (0.64->0.99) at the 12-month timepoint. The individual-level association between 12-month MRD negativity and PFS resulted in a global odds ratio of 4.02 (95% CI: 2.57-5.46). For relapse/refractory MM-(RRMM), there were four studies included, and the individual-level association between 12-month MRD negativity and PFS resulted in a global odds ratio of 7.67 (4.24-11.10). A clinical trial demonstrating a treatment effect on MRD is reasonably likely to eventually demonstrate a treatment effect on PFS, suggesting that MRD may be an early clinical endpoint reasonably likely to predict clinical benefit in MM, that may be used to support accelerated approval and thereby expedite the availability of new drugs to patients with MM.
ABSTRACT
The 21st Century Cures Act (Cures Act), signed into law in 2016, was designed to advance new therapies by modernizing clinical trials, funding research initiatives, and accelerating the development and use of health information technology. To analyze the current issues in cancer care related to the implementation and impact of the Cures Act, NCCN convened a multistakeholder working group. Participants discussed the legislation's impact on the oncology community since enactment and identified the remaining gaps and challenges as experienced by stakeholders. In June 2020, the policy recommendations of the working group were presented at the virtual NCCN Policy Summit: Accelerating Advances in Cancer Care Research: A Lookback at the 21st Century Cures Act in 2020. The summit consisted of informative discussions and a multistakeholder panel to explore the recommendations and the future of the Cures Act. This article explores identified policy recommendations from the NCCN Working Group and the NCCN Policy Summit, and analyzes opportunities to advance innovative cancer care and patient access to data.
Subject(s)
Biomedical Research/trends , Neoplasms , Humans , Neoplasms/therapyABSTRACT
Psoriasis is a skin condition that affects over 100 million people worldwide, while multiple myeloma (MM) accounts for 10% of all hematologic malignancies in the US. There has been limited research on the intersection of psoriasis and MM, and clinicians often face difficult decisions in treating patients diagnosed with both conditions. For instance, the management of psoriasis with systemic immunotherapies in MM patients can be challenging because of concern about immunosuppression and possible worsening of MM. Online crowdsourcing platforms have recently become innovative tools that can actively empower patients in scientific research by enabling the contribution of health data. One such platform, HealthTree®, helps MM patients find optimal myeloma treatments and has registered > 6000 patients, many of whom have uploaded medical records and genetic profiles. By taking advantage of patient health data available on HealthTree, researchers can gain a greater understanding of the clinical characteristics and treatment responses of patients diagnosed with psoriasis and MM. In this case series, we first report a psoriasis and MM patient treated with the IL-17 inhibitor ixekizumab who demonstrated a temporary, 2-month improvement in MM biomarkers (M-protein, kappa, and kappa:lambda ratio). We then report on the clinical characteristics of 18 patients with verified profiles on HealthTree indicating concurrent psoriasis and MM conditions. We surveyed gender, age, psoriasis type, psoriasis treatment history, myeloma type, myeloma genetic features, and myeloma association with bone damage, hypercalcemia, or osteopenia. Four patients were treated with systemic immunomodulators for psoriasis, with responses suggesting that these therapies did not worsen MM progression. Our results validate crowdsourcing as a way to assess patient demographics and treatment responses for use in dermatology research. We examine the demographics of patients diagnosed with psoriasis and MM and investigate the use of systemic immunomodulators for treatment of psoriasis in MM patients.
ABSTRACT
Social media utilizes specific media platforms to allow increased interactivity between participants. These platforms serve diverse groups and purposes including participation from patients, family caregivers, research scientists, physicians, and pharmaceutical companies. Utilization of these information outlets has increased with integration at conferences and between conferences with the use of hashtags and "chats". In the realm of the "e-Patient" it is key to not underestimate your audience. Highly technical information is just as useful as a basic post. With growing use, social media analytics help track the volume and impact of content. Additionally, platforms are leveraging each other for uses, including Twitter, blogs, web radio, and recorded video and images. We explore information on social media resources and applications from varying perspectives. While these platforms will evolve over time, or disappear with new platforms taking their place, it is apparent they are now a part of the everyday experience of oncology communication.
