Incidence of several cancers continues to increase in children and adolescents. Individual studies and international collaborations have made progresses in understanding risk factors of certain childhood cancers, yet gaps remain. The CCDI should also invest in supporting data sharing for etiologic research (population-based and mechanistic studies).
To further encourage sharing, the resource sharing plans submitted to the NIH should be reviewed for overall impact. NIH should also consider scoring them individually.
Facilitating interoperability across various data repositories as well as novel and harmonized data analytic tools that can be used across different systems: Many hematologic diseases, including those that impact the pediatric population are categorized as rare diseases. The rare, yet deleterious nature of these diseases (e.g., chronic myelomonocytic leukemia) make it challenging to build a critical mass of data, that ...more »
The American Society of Hematology is also committed to the understanding of inherited genomic profiles that predispose individuals to hematologic malignancies. As such, the Society has partnered with the Clinical Genome Resource to curate variants in genes (such as RUNX1 and GATA2) that are involved in predisposition to myeloid malignancies. Predisposition syndromes often manifest in children or young adults. Data collection ...more »
In addition to collecting genomic data on such patients, the following types of datasets and samples would be quite informative in disease diagnosis, therapy development, and treatment of pediatric patients with hematologic cancers: • Proteomic data with clinical correlation • Safety and toxicity data profiles at different time points • Treatment regimen • Outcomes data • Socioeconomic and demographic data • Biospecimens ...more »
The National Cancer Institute (NCI) has made a significant investment in the Genomic Data Commons (GDC) which already possesses robust analytical and visualization tools. There is a sizeable amount of genomic and phenotypic information relevant to pediatric malignancies currently residing in the database of Genotypes and Phenotypes (dbGaP). The NCI could play a role in ensuring that data in dbGaP is made available through ...more »
Proposals submitted to the NIH often require a Resource Sharing Plan document, and those proposing more than $500k in annual direct costs often require a Data Sharing Plan. These documents are not made public, making it onerous for investigators to understand what commitments have been made to the NIH as a condition of funding. For a use case, imagine a scenario where the reader of a manuscript identifies a described ...more »
Through the Childhood Cancer Data initiative, the National Cancer Institute could leverage existing national infrastructure such as the Center for International Blood and Marrow Transplant Research (CIBMTR), Pediatric Health Information System, and electronic health records to define optimal therapeutic strategies for pediatric patients with hematologic cancers. Such platforms could be useful because they contain comprehensive ...more »
Predictive disease models of pediatric hematologic cancers can inform efficient testing of possible novel therapeutic agents in the clinical setting. However, to ensure that these models are useful in prioritizing novel therapeutic targets, pre-clinical assessments must be conducted. Conducting such analysis efficiently and consistently will require investigators to share pre-clinical data generated from models (e.g., ...more »
Refining risk stratification protocols and enhancing risk prediction in the pediatric population to identify children at risk of developing hematologic cancers: While the treatment outcome of children with acute leukemias (e.g., acute myeloid leukemia and ALL) has improved over the years, there remains a population of these patients who are unable to respond favorably to available therapies. Predicting poor response remains ...more »
Understanding the biology of pediatric hematologic cancer development through: • Insights from epigenetics: In all hematologic malignancies, including acute and chronic leukemias, and lymphomas, there are both inherited and somatic genetic alterations that contribute to predisposition, transformation, and disease progression. While genomics plays a significant role in the onset and progression of such malignancies, ...more »
Dear Committee; - i would like to introduce you to my idea " relapse prediction using machine learning algorithms " - my topic is about turning the initial data collected from the patients when introduced for first time before starting the treatment into insights using data science. it depends on each disease type and according to the treatment protocol. - by using an Electronic Data Capture (EDC) system , we can turn ...more »
Dear committee, I would like to write a new plan for two date sets for cancer therapy of youth. One set of data is based on the pharmacological data set coming from the patient's cells embedded embryo of Zebrafish. Such PDX (Patient-Derived Xenograft Model; PDX) is now getting realistic and offer more precise medicine of cancer therapy. Because of the mechanism of Zebrafish based PDX, each embryo of Zebrafish needs ...more »
Cancer treatment outcomes depend on the hospital environment. Research should evaluate the effects of ambient lighting (color, intensity, timing), and the best time of day for chemotherapy and feeding and other interventions which can disrupt a patient's normal circadian rhythm. This initiative was chosen as representative of the recommendations from members of the Society for Research on Biological Rhythms (SRBR).
There is a pressing need for a large international database on childhood and adolescent cancer patients who are also infected with the Human Immunodeficiency Virus (HIV). I believe that existing data is too fragmented and scattered to be readily useful for some types of research. The data for this proposed database would be obtained from hospitals and research facilities, not just from this country, but from other ...more »