The impact of the written request process on drug development in childhood cancer

2013 ◽  
Vol 60 (4) ◽  
pp. 531-537 ◽  
Author(s):  
Kristen M. Snyder ◽  
Gregory Reaman ◽  
Debbie Avant ◽  
Richard Pazdur
Author(s):  
Allyah Abbas‐Hanif ◽  
Homira Rezai ◽  
S. Faraz Ahmed ◽  
Asif Ahmed

BJS Open ◽  
2021 ◽  
Vol 5 (Supplement_1) ◽  
Author(s):  
◽  
Soham Bandyopadhyay

Abstract Introduction Childhood cancers are a leading cause of non-communicable disease deaths for paediatric patients around the world. The COVID-19 pandemic may have impacted on global children’s cancer services, which can have consequences for childhood cancer outcomes. The Global Health Research Group on Children’s Non-Communicable Diseases (Global Children’s NCDs) is currently undertaking the first international study to determine the variation in paediatric cancer management during the COVID-19 pandemic, and the short to medium term impacts on childhood cancer outcomes. Methods and analysis This is a multicentre, international, cohort study that will use routinely collected hospital data in a de-identified and anonymised form. Patients will be recruited consecutively into the study, with a 12 -month follow-up period. Patients will be included if they are below the age of 18 years and undergoing anti-cancer treatment for the following cancers: Acute lymphoblastic leukaemia, Burkitt’s Lymphoma, Hodgkin's lymphoma, Wilms Tumour, Sarcoma, Retinoblastoma, Gliomas, Medulloblastomas and Neuroblastomas. Patients must be newly presented or be undergoing active anti-cancer treatment from the 12th March 2020 to the 12th December 2020. The primary objective of the study is to determine 30- and 90-day all-cause mortality rates. This study will examine the factors that influenced these outcomes. Chi-squared analysis will be used to compare mortality between low and middle-income countries and high-income countries. Multilevel, multivariate logistic regression analysis will be undertaken to identify patient-level and hospital-level factors affecting outcomes with adjustment for confounding factors. Ethics and dissemination At the host centre, this study was deemed to be exempt from ethical committee approval due to the use of anonymised registry data. At other centres, participating collaborators have gained local approvals in accordance with their institutional ethical regulations. Collaborators will be encouraged to present the results locally, nationally, and internationally. The results will be submitted for publication in a peer reviewed journal.


2010 ◽  
Vol 28 (15) ◽  
pp. 2625-2634 ◽  
Author(s):  
Malcolm A. Smith ◽  
Nita L. Seibel ◽  
Sean F. Altekruse ◽  
Lynn A.G. Ries ◽  
Danielle L. Melbert ◽  
...  

Purpose This report provides an overview of current childhood cancer statistics to facilitate analysis of the impact of past research discoveries on outcome and provide essential information for prioritizing future research directions. Methods Incidence and survival data for childhood cancers came from the Surveillance, Epidemiology, and End Results 9 (SEER 9) registries, and mortality data were based on deaths in the United States that were reported by states to the Centers for Disease Control and Prevention by underlying cause. Results Childhood cancer incidence rates increased significantly from 1975 through 2006, with increasing rates for acute lymphoblastic leukemia being most notable. Childhood cancer mortality rates declined by more than 50% between 1975 and 2006. For leukemias and lymphomas, significantly decreasing mortality rates were observed throughout the 32-year period, though the rate of decline slowed somewhat after 1998. For remaining childhood cancers, significantly decreasing mortality rates were observed from 1975 to 1996, with stable rates from 1996 through 2006. Increased survival rates were observed for all categories of childhood cancers studied, with the extent and temporal pace of the increases varying by diagnosis. Conclusion When 1975 age-specific death rates for children are used as a baseline, approximately 38,000 childhood malignant cancer deaths were averted in the United States from 1975 through 2006 as a result of more effective treatments identified and applied during this period. Continued success in reducing childhood cancer mortality will require new treatment paradigms building on an increased understanding of the molecular processes that promote growth and survival of specific childhood cancers.


BMC Cancer ◽  
2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Rahel Kasteler ◽  
Christa Lichtensteiger ◽  
Christina Schindera ◽  
Marc Ansari ◽  
Claudia E. Kuehni ◽  
...  

Abstract Background Chest wall abnormalities are a poorly studied complication after treatment for childhood cancer. Chest wall abnormalities are not well-described in the literature, and little is known on the impact on daily life of survivors. Methods We investigated prevalence and risk factors of chest wall abnormalities in childhood cancer survivors in a nationwide, population-based cohort study (Swiss Childhood Cancer Survivor Study) with a questionnaire survey. We then interviewed a nested sample of survivors to validate types of chest wall abnormalities and understand their impact on the daily life of survivors. Results Forty-eight of 2382 (95%CI 2–3%) survivors reported a chest wall abnormality. Risk factors were older age at cancer diagnosis (16–20 years; OR 2.5, 95%CI 1.0–6.1), lymphoma (OR 3.8, 95%CI 1.2–11.4), and central nervous system tumors (OR 9.5, 95%CI 3.0–30.1) as underlying disease, and treatment with thoracic radiotherapy (OR 2.0, 95%CI 1.0–4.2), surgery to the chest (OR 4.5, 95%CI 1.8–11.5), or chemotherapy (OR 2.9, 95%CI 1.0–8.1). The nature of the chest wall abnormalities varied and included thoracic wall deformities (30%), deformations of the spine (5%) or both (55%), and scars (10%). Chest wall abnormalities affected daily life in two thirds (13/20) of those who reported these problems and necessitated medical attention for 15 (75%) survivors. Conclusion It is important that, during follow-up care, physicians pay attention to chest wall abnormalities, which are rare late effects of cancer treatment, but can considerably affect the well-being of cancer survivors.


2021 ◽  
pp. 107484072110014
Author(s):  
Nancy J. Moules ◽  
Catherine M. Laing ◽  
Wendy Pelletier ◽  
Gregory M. T. Guilcher ◽  
Jennifer A. Chan

While cure rates in pediatric oncology have improved over the past 30 years, childhood cancer remains the second leading cause of death in children aged 1 to 14. Developing therapies often require using cancerous tissues, which may come from deceased donors. Tumor banks collect, store, and distribute these donated samples. While tumor banking is more common, factors that contribute to parents’ decision and the impact of it on the family are not well understood. The purpose of this hermeneutic study was to understand the meaning and impact of tumor banking for parents of children who have died from cancer. Findings suggest that parents donating their child’s tumors unexpectedly found a sense of meaning in their loss. They also found a legacy of their child’s life; the living cells in some ways assisted the parents with grief. Aspects of this sensitive conversation and decision are discussed from the perspective of the parents’ experiences.


2021 ◽  
Vol 26 (4) ◽  
Author(s):  
Peter L. Molloy ◽  
Lester W. Johnson ◽  
Michael Gilding

A recent study assessed the investor performance of the Australian drug development biotech (DDB) sector over a 15-year period from 2003 to 2018. The current study builds on that research and extends the analysis to 2020, using a 10-year period starting 2010, to exclude the impact of the global financial crisis in 2008/09. Based on a value-weighted portfolio of all 41 DDB firms, the overall sector delivered a negative annualized return of -4.1%. Individual firm performance was also assessed using the compound annual growth rate (CAGR) in share price over the period as a measure of investor outcomes. On this basis 68% of firms produced negative CAGRs over the period, and of the 32% of firms that produced positive CAGRs, six firms produced CAGRs greater than 20% per annum and in three cases of recently-listed firms, the CAGR’s were greater than 50%. Overall however, the sector overall delivered very poor investor returns and despite a relatively large number of listed biotech firms, Australian biotechnology continues to be small and weak in terms of its contribution to global biotechnology industrialization. As such it lacks the critical mass to grow a robust bioeconomy based on drug development, which remains the standard-bearer of biotechnology industrialization.


Author(s):  
Keagan G. Lipak ◽  
Joseph R. Rausch ◽  
Rachel S. Fisher ◽  
Kemar V. Prussien ◽  
Olivia E. Clark ◽  
...  

2017 ◽  
Vol 34 (4) ◽  
pp. 345-361 ◽  
Author(s):  
Shaunna M. Burke ◽  
Jennifer Brunet ◽  
Amanda Wurz ◽  
Christina Butler ◽  
Andrea Utley

The benefits of informal physical activity during recovery from childhood cancer have rarely been investigated. This study adopted a multiple case study approach to explore the impact of recreational cycling on childhood cancer survivors’ experiences of well- and ill-being. Three semistructured interviews were conducted over a 3-month period with four survivors to explore their experiences of physical, psychological, and social well- and ill-being. Within-case analysis followed by cross-case analysis identified three themes that captured their well- and ill-being experiences with recreational cycling and cancer: (a) cultivating feelings and emotions, (b) experiencing physical changes, and (c) encountering positive and negative social interactions. The results from this study show that recreational cycling may be a useful adjunct to conventional treatments for the self-management of multiple domains of well- and ill-being during recovery from childhood cancer.


PLoS ONE ◽  
2021 ◽  
Vol 16 (1) ◽  
pp. e0245922
Author(s):  
Faye Lanni ◽  
Neil Burton ◽  
Debbie Harris ◽  
Susan Fotheringham ◽  
Simon Clark ◽  
...  

Optimised pre-clinical models are required for TB drug development to better predict the pharmacokinetics of anti-tuberculosis (anti-TB) drugs to shorten the time taken for novel drugs and combinations to be approved for clinical trial. Microdialysis can be used to measure unbound drug concentrations in awake freely moving animals in order to describe the pharmacokinetics of drugs in the organs as a continuous sampling technique. The aim of this work was to develop and optimise the microdialysis methodology in guinea pigs to better understand the pharmacokinetics of rifampicin in the lung. In vitro experiments were performed before progressing into in vivo studies because the recovery (concentration of the drug in the tissue fluid related to that in the collected dialysate) of rifampicin was dependent on a variety of experimental conditions. Mass spectrometry of the dialysate was used to determine the impact of flow rate, perfusion fluid and the molecular weight cut-off and membrane length of probes on the recovery of rifampicin at physiologically relevant concentrations. Following determination of probe efficiency and identification of a correlation between rifampicin concentrations in the lung and skeletal muscle, experiments were conducted to measure rifampicin in the sacrospinalis of guinea pigs using microdialysis. Lung concentrations of rifampicin were estimated from the rifampicin concentrations measured in the sacrospinalis. These studies suggest the potential usefulness of the microdialysis methodology to determine drug concentrations of selected anti-TB drugs to support new TB drug development.


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