Actionability of Synthetic Data in a Heterogeneous and Rare Health Care Demographic: Adolescents and Young Adults With Cancer.

Purpose: Research on rare diseases and atypical health care demographics is often slowed by high interparticipant heterogeneity and overall scarcity of data. Synthetic data (SD) have been proposed as means for data sharing, enlargement, and diversification, by artificially generating real phenomena while obscuring the real patient data. The utility of SD is actively scrutinized in health care research, but the role of sample size for actionability of SD is insufficiently explored.

Neoadjuvant chemotherapy for radiation associated angiosarcoma (RAAS) of the breast: A retrospective single center study.

Background: Radiation associated angiosarcoma (RAAS) of the breast is a rare malignancy with poor survival. Optimal treatment strategies remain uncertain due to a lack of data, and vary between surgery alone and a combination of surgery with (neo)adjuvant chemotherapy (NACT) and/or re-irradiation. The aim of this study was to evaluate the potential benefit of taxane based NACT.

Perspectives of patients, partners, primary and hospital-based health care professionals on living with advanced cancer and systemic treatment.

Purpose: An emerging group of patients lives longer with advanced cancer while receiving systemic treatment. This study aimed to investigate psychosocial aspects of living longer with advanced cancer, and experiences with psychosocial care, from the perspectives of patients, partners, and health care professionals (HCPs).

Methods: From May to December 2020, participants were purposively selected.

Identifying the informational needs and sources of support of Adolescent and Young Adult (AYA) cancer survivors to inform the development of a digital platform.

Purpose: This study aimed to examine the (age-specific) informational needs and support sources used by Adolescent and Young Adult (AYA) cancer survivors throughout their cancer trajectory and socio-demographic and clinical factors associated with most common AYA-related informational needs.

Methods: A cross-sectional questionnaire study was conducted among AYA cancer survivors (mean, 10.3 years after diagnosis, SD = 5.

Improving completion rates of patient-reported outcome measures in cancer clinical trials: Scoping review investigating the implications for trial designs.

Background: Patient-reported outcomes (PROs) play a crucial role in cancer clinical trials. Despite the availability of validated PRO measures (PROMs), challenges related to low completion rates and missing data remain, potentially affecting the trial results' validity. This review explored strategies to improve and maintain high PROM completion rates in cancer clinical trials.

Setting the international research agenda for sarcomas with patients and carers: results of phase II of the Sarcoma Patient Advocacy Global Network (SPAGN) priority setting partnership.

Background: Typically, researchers and clinicians determine the agenda in sarcoma research. However, patient involvement can have a meaningful impact on research. Therefore, the Patient-Powered Research Network (PPRN) of the Sarcoma Patient Advocacy Global Network (SPAGN) set up a Priority Setting Partnership (PSP).

Interleukin-6 in relation to early recurrence in primary, localized soft tissue sarcoma: An addition for existing risk classification systems?

Background: Several inflammatory markers have gained interest as prognostic factors for cancer. The aim of this study is to evaluate the inflammatory markers interleukin-6 (IL-6), C-reactive protein (CRP), neutrophil-to-lymphocyte ratio (NLR) and platelet-to-lymphocyte ratio (PLR) as predictive markers for aggressive behavior and early recurrences in primary, localized soft tissue sarcoma (STS).

Methods: 115 STS patients were retrospectively reviewed.

The survival disparity between children and adolescents and young adults (AYAs) with Ewing sarcoma in the Netherlands did not change since the 1990s despite improved survival: A population-based study.

Background: Adolescents and young adults (AYAs) with Ewing sarcoma have a worse prognosis than children. Population-based survival evaluations stratifying findings by important clinical factors are, however, limited. This Dutch population study comprehensively compared survival of children and AYAs with Ewing sarcoma over three decades considering diagnostic period, tissue of origin, tumor site, and disease stage.

Evaluating the Clinical Impact and Feasibility of Therapeutic Drug Monitoring of Pazopanib in a Real-World Soft-Tissue Sarcoma Cohort.

Introduction And Objective: Pazopanib is registered for metastatic renal cell carcinoma and soft-tissue sarcoma (STS). Its variable pharmacokinetic (PK) characteristics and narrow therapeutic range provide a strong rationale for therapeutic drug monitoring (TDM). Prior studies have defined target levels of drug exposure (≥ 20.

Improving Diagnosis and Care for Patients With Sarcoma: Do Real-World General Practitioners Data and Prospective Data Collections Have a Place Next to Clinical Trials?

There has been growing interest in the use of real-world data (RWD) to address clinically and policy-relevant (research) questions that cannot be answered with data from randomized controlled trials (RCTs) alone. This is, for example, the case in rare malignancies such as sarcomas as limited patient numbers pose challenges in conducting RCTs within feasible timeliness, a manageable number of collaborators, and statistical power. This narrative review explores the potential of RWD to generate real-world evidence (RWE) in sarcoma research, elucidating its application across different phases of the patient journey, from prediagnosis to the follow-up/survivorship phase.

The prediagnostic general practitioner care of sarcoma patients: A real-world data study.

Background: Limited understanding exists regarding early sarcoma symptoms presented during general practitioner (GP) consultations. The study explores GP visit patterns and recorded diagnoses in the 12 months preceding sarcoma diagnosis.

Methods: Sarcoma cases diagnosed from 2010 to 2020 were identified through the Netherlands Cancer Registry alongside general practice data.

Sunitinib for the treatment of metastatic gastrointestinal stromal tumors: the effect of TDM-guided dose optimization on clinical outcomes.

Background: Sunitinib is an oral anticancer drug approved for the treatment of among others gastrointestinal stromal tumor (GIST). Previous analyses demonstrated an exposure-response relationship at the standard dose, and minimum target levels of drug exposure have been defined above which better treatment outcomes are observed. Therapeutic drug monitoring (TDM) could be used as a tool to optimize the individual dose, aiming at sunitinib trough concentrations ≥37.

Financial difficulties experienced by patients with gastrointestinal stromal tumours (GIST) in the Netherlands: data from a cross-sectional multicentre study.

Purpose: This study aims to (1) explore the prevalence of patient-reported financial difficulties among GIST patients, differentiating between those currently undergoing tyrosine kinase inhibitor (TKI) treatment and those who are not; (2) investigate associations between financial difficulties and sociodemographic and clinical characteristics, work, cancer-related concerns, anxiety and depression and (3) study the impact of financial difficulties on health-related quality of life.

Methods: A cross-sectional study was conducted among Dutch GIST patients diagnosed between 2008 and 2018, who were invited to complete a one-time survey between September 2020 and June 2021. Patients completed nine items of the EORTC item bank regarding financial difficulties, seven work-related questions, the Hospital Anxiety and Depression Scale, Cancer Worry Scale and EORTC QLQ-C30.

Patient-reported outcomes in randomized clinical trials of systemic therapy for advanced soft tissue sarcomas in adults: A systematic review.

Background: This systematic review evaluates reporting of patient-reported outcomes (PROs) within randomized clinical trials (RCTs) for advanced soft tissue sarcoma (STS) patients.

Methods: A systematic literature search from January 2000 - August 2022 was conducted for phase II/III RCTs evaluating systemic treatments in adult patients with advanced STS. Quality of PRO reporting was assessed using the CONSORT PRO extension.

Elements of successful patient involvement in clinical cancer trials: a review of the literature.

Patient involvement in clinical cancer research has gained much ground in the past few years and studies demonstrated positive outcomes of such involvement. Yet, they also indicated a lack of evidence around best methods and practices to achieve successful patient involvement. The aim of this literature review was to provide a synthesis of elements contributing to successful and meaningful ways of involving patients in oncology trials across different stages of research.

Propranolol monotherapy in angiosarcoma - A window-of-opportunity study (PropAngio).

Background: Angiosarcoma is a rare and aggressive cancer of the endothelial cells. Propranolol, a non-selective β-blocker, was able to initiate apoptosis in angiosarcoma cell lines and its anti-tumor activity has been described in several case reports. The aim of this trial was to prospectively evaluate the anti-tumor activity of propranolol monotherapy in patients with angiosarcoma before proceeding to standard of care treatment.

Employment outcomes of adolescent and young adult cancer survivors and their partners: A Dutch population-based study.

Background: The aim of this population-based registry study was to examine the impact of cancer on employment outcomes in adolescent and young adult (AYA) survivors and their partners and associated sociodemographic and clinical characteristics.

Methods: A total of 2456 AYA cancer patients, diagnosed in 2013 and aged 18 through 39 years old, were selected from the Netherlands Cancer Registry and linked to employment data from Statistics Netherlands, from which 1252 partners of AYAs could be identified. For both patients and their partners, a control group with same age, migration background, and sex was selected.