Showing 1 ideas for tag "modelling"

Goal 2: Reduce Human Disease

Understand the Impact of Thrombosis in Children with Cancer

CC: Despite the potential impact that venous thrombotic events (VTE) have on children with cancer, several unresolved issues remain. To date, we are yet to understand:
- incidence/prevalence of VTE according to cancer type/staging
- ideal imaging modalities to diagnose/follow VTE
- thromboprophylaxis according to thrombosis risk stratification (development of VTE predictors)
- efficacy/safety to anticoagulate children... more »

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? Critical Challenge (CC)

Details on the impact of addressing this CQ or CC

Venous thrombotic events (VTE) are now occurring in 1/200 children admitted to a tertiary pediatric facility. In around 70-90% of cases, VTE occurs in children with an underlying condition, amongst which cancer represents up to 1/3 of patients. Within this group of patients, the thrombotic complications are associated with a higher morbidity (e.g. higher recurrence rates, high rate of CNS events in acute leukemia) and mortality. Nevertheless, the clinical challenges highlighted in the itemized Critical Challenge Section illustrate the lack of basic science, translational and clinical research available, as well as the paucity of evidence-based medicine recommendations necessary to acoount for the increasing number of patients with this complication.
On the other hand, pediatric oncology is one of the areas of pediatric care where the medical progresses of the last decades have drastically changed the natural history of cancer in children. In light of much higher survival rates for almost all types of pediatric cancer, the focus has now shifted towards decreasing treatment-related, as well as disease-related morbidities, increasing the quality of life of the many survivors. Because VTE is now recognized as one of the significant remaining complications within this patient population, addressing the list summarized herein would contribute to further improve the care of children with cancer.

Feasibility and challenges of addressing this CQ or CC

The infrastructure that is already in place under the Children's Oncology Group (COG), where almost any new clinical and/or translational idea related to the care of children with cancer becomes part of a clinical trial, could be rolled over to explore many of the items listed under the CC Section.
As a principle, VTE in children with cancer develop due to: a) host-related factors; b) chemotherapy/treatment-related factors; and c) disease-related issues. Therefore, protocol- and disease-specific studies could address, under the auspices of COG, the prevalence of VTE according to cancer type in a prospective manner. Similarly, high risk groups for VTE could be submitted to standardized imaging and/or biomarker investigation prospectivelly, in addition to collection of outcome data related to VTE and to anticoagulation protocols. Furthermore, tumor specimens/genetic markers could be evaluated and correlated to the study outcomes. The challenges of reaching consensus during protocol development would allow identification of equipoise for certain clinical scenarios, obviating the need of trials, or the use of consensus techniques, before diagnostic/therapeutic protocols could be adopted.
In conclusion, the develoment of a multidisciplinary task force (i.e. pediatric radiologists, oncologists, hematologists, molecular biology experts), which, for the most part, is already in place (i.e. COG), would be instrumental to foster research on this extremely clinically relevant area.

Name of idea submitter and other team members who worked on this idea Leonardo R. Brandao, MD, MSc;

Voting

30 net votes
38 up votes
8 down votes
Active