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Browsing by Subject "Pediatric cancers"
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Item A Collaborative Model for Accelerating the Discovery and Translation of Cancer Therapies(American Association for Cancer Research, 2017-11-01) Maertens, Ophélia; McCurrach, Mila E.; Braun, Benjamin S.; De Raedt, Thomas; Epstein, Inbal; Huang, Tannie Q.; Lauchle, Jennifer O.; Lee, Hyerim; Wu, Jianqiang; Cripe, Timothy P.; Clapp, D. Wade; Ratner, Nancy; Shannon, Kevin; Cichowski, Karen; Pediatrics, School of MedicinePreclinical studies using genetically engineered mouse models (GEMM) have the potential to expedite the development of effective new therapies; however, they are not routinely integrated into drug development pipelines. GEMMs may be particularly valuable for investigating treatments for less common cancers, which frequently lack alternative faithful models. Here, we describe a multicenter cooperative group that has successfully leveraged the expertise and resources from philanthropic foundations, academia, and industry to advance therapeutic discovery and translation using GEMMs as a preclinical platform. This effort, known as the Neurofibromatosis Preclinical Consortium (NFPC), was established to accelerate new treatments for tumors associated with neurofibromatosis type 1 (NF1). At its inception, there were no effective treatments for NF1 and few promising approaches on the horizon. Since 2008, participating laboratories have conducted 95 preclinical trials of 38 drugs or combinations through collaborations with 18 pharmaceutical companies. Importantly, these studies have identified 13 therapeutic targets, which have inspired 16 clinical trials. This review outlines the opportunities and challenges of building this type of consortium and highlights how it can accelerate clinical translation. We believe that this strategy of foundation-academic-industry partnering is generally applicable to many diseases and has the potential to markedly improve the success of therapeutic development.Item Right on Schedule: Improving the Rate of Clinic Appointments Scheduled Prior to Hospital Hospital Discharge(Wolters Kluwer, 2022-01-21) Rahim, Mahvish Q.; Griffin, Jordyn; Hege, Kerry; Mueller, Emily L.; Kauffman, Kristine; Corman, Stacey; Anderson, Kari; Woodburn, Stayce; Drayton Jackson, Meghan; Pediatrics, School of MedicineIntroduction: Children with cancer and blood disorders have many healthcare needs that often require inpatient and outpatient management. There is potential for a lapse in care when patients frequently transition between these settings. We aimed to improve the process and increase the rate of scheduled outpatient follow-up appointments at the time of inpatient discharge for all pediatric hematology-oncology patients from a baseline of 68-80%. Methods: A multidisciplinary team developed several Plan-Do-Study-Act cycles to standardize and improve the process of scheduling follow-up appointments, communication to schedulers, and discussion of discharge planning. QI Macros for Excel Version 2019.06 was used for statistical analysis. Our primary outcome was displayed over time with a p-chart. Results: Plan-Do-Study-Act interventions had a statistically significant impact in increasing the percentage of patients with follow-up outpatient appointments scheduled at the time of inpatient discharge from a baseline of 68% to consistently over 80%. Conclusions: This study demonstrates that standardization of care processes and reminders and education of healthcare providers about the new approaches can improve the rates of outpatient follow-up appointments scheduled at the time of hospital discharge from inpatient care.