Ultra-rare sarcomas are a group of mesenchymal malignancies with an incidence ≤1 case/million people per year. While ultra-rare sarcomas have low incidence, they comprise more than 80 different entities, including bone and soft tissue tumours, that collectively constitute around 20% of all sarcoma cases. This means that the challenges inherent to ultra-rare sarcomas apply to many patients overall and are representative of the challenges that all patients with ultra-rare cancers face. While there is a wide degree of heterogeneity between the different ultra-rare sarcoma types, their very low incidence leads to a common set of challenges:
A broad group of representatives from the global sarcoma community, including clinicians, scientists, patient advocates and non-profit organisations have come together to establish a consortium to address these issues. The consortium is developing a platform to overcome these obstacles and to progress the treatment of these diseases. The central aim of the PUSH platform is to maximise the knowledge that we gain from interacting with and treating every single patient affected by an ultra-rare sarcoma and to support developing new treatments to improve outcomes and quality of life.
The platform will collect data, prospective and retrospective, on all patients from around the world regardless of their treatment, whether it is standard of care, an off-label treatment, or a new investigative agent. PUSH will incorporate: patient-curated data from patient advocacy registries, structured surveys, patient reported outcomes, and quality of life questionnaires; academic datasets collected and curated at sarcoma centres of excellence; extensive and comprehensive real world data; prospectively collected data of patients receiving standard of care treatment; data extracted from prospective clinical trials, including historical trials. Translational data will be incorporated from laboratories and biobanks. All available data will be used to support the design of new prospective studies and to create external control groups when randomization is not feasible, developing and employing innovative methodological and biostatistical approaches, disease-appropriate clinical trials, and outcomes.
While preserving the accuracy of the pathological diagnosis and data quality, the platform will be decentralized and open to participating sites of all types. It will have a 'master platform protocol' defining the structure, governance, ethics, data management and core principles. To this core, a set of additional protocols can be added as amendments, each defining a specific sub-study which participating parties can adopt as required. Inclusive disease-specific working groups will help direct these efforts. It will include consistent consultation with regulators to ensure that the collected data is of regulatory quality given the challenges of ultra-rare diseases. The data platform for PUSH will feature a hybrid architecture that is explicitly designed to ensure central access for reporting and data analysis, while retaining local hosting of data and minimising concerns about privacy, central control, and ownership.
PUSH is designed to include prospective clinical trials. Sites that do not join the interventional arm of these trials may still collect data on patients with these diseases — in effect contributing to virtual 'controls' to act as comparators. The branch of the PUSH consortium devoted to prospective clinical trials will be called PUSH IT (IT, International Trial). The first study will aim to support the prospective assessment of sirolimus as a treatment for progressive EHE.
PUSH are also be working on a Retrospective study of spindle cell and pleomorphic sarcoma.
And have just launched a Prospective Observational Study in LGFMS/SEF
If you wish to know more about PUSH, then please contact us at:
