The Sarcoma Assessment Measure (SAM): Preliminary Psychometric Validation of a Novel Patient-Reported Outcome Measure.

The Sarcoma Assessment Measure (SAM) was developed as a sarcoma-specific patient-reported outcome measure to be used in clinical practice. We have reported in detail how SAM has been developed in collaboration with patients and healthcare professionals. The aim of this paper is to report the preliminary validation of SAM.

The Impact of Specialist Care on Teenage and Young Adult Patient-Reported Outcomes in England: A BRIGHTLIGHT Study.

In England, health care policy promotes specialized age-appropriate cancer services for teenagers and young adults (TYA), for those aged 13-24 years at diagnosis. Specialist Principal Treatment Centers (PTCs) provide enhanced age-specific care for TYA, although many still receive all or some of their care in adult or children's cancer services. Our aim was to determine the patient-reported outcomes associated with TYA-PTC based care.

Young people's opinions of cancer care in England: the BRIGHTLIGHT cohort.

Objectives: The BRIGHTLIGHT cohort study was the national evaluation of cancer services for teenager and young adults (TYA). This was analysis of free-text survey data to better understand their experiences of cancer care.

Design: Cohort study SETTING: National Health Service hospitals delivering cancer care in England PARTICIPANTS: 830 young people newly diagnosed with cancer.

Patients' Experiences of a Sarcoma Diagnosis: A Process Mapping Exercise of Diagnostic Pathways.

Unlabelled: Patients with sarcoma often report prolonged time to diagnosis, which is attributed to the rarity of sarcoma and the low awareness of pre-diagnostic signs and symptoms.

Aims: To describe patients' experiences of pre-diagnostic signs/symptoms and pathways to diagnosis, including where help was sought, and the processes involved.

Methods: Mixed methods involving quantitative, qualitative and inductive thematic analyses using novel process mapping of patient journey data, as reported by the patients.

Comparison of two chemotherapy regimens in patients with newly diagnosed Ewing sarcoma (EE2012): an open-label, randomised, phase 3 trial.

Background: Internationally, a single standard chemotherapy treatment for Ewing sarcoma is not defined. Because different chemotherapy regimens were standard in Europe and the USA for newly diagnosed Ewing sarcoma, and in the absence of novel agents to investigate, we aimed to compare these two strategies.

Methods: EURO EWING 2012 was a European investigator-initiated, open-label, randomised, controlled phase 3 trial done in 10 countries.

A Qualitative Study of the Factors Influencing Patients' Experience of Soft Tissue Sarcoma in the United Kingdom.

Background: Treatment of soft tissue sarcoma frequently involves extensive surgery, loss of mobility, and complex rehabilitation programs. Poorer patient-reported outcomes are reported in comparison to those from patients with other cancer types. Understanding patient experience is therefore important to support patients and improve care.

Linking EORTC QLQ-C-30 and PedsQL/PEDQOL physical functioning scores in patients with osteosarcoma.

Purpose: The available questionnaires for quality-of-life (QoL) assessments are age-group specific, limiting comparability and impeding longitudinal analyses. The comparability of measurements, however, is a necessary condition for gaining scientific evidence. To overcome this problem, we assessed the viability of harmonising data from paediatric and adult patient-reported outcome (PRO) measures.

Survival after high-dose chemotherapy for refractory and recurrent Ewing sarcoma.

Background: outcome of primary refractory or recurrent Ewing sarcoma (RRES) is poor and the role of high-dose therapy (HDT) remains uncertain. We retrospectively reviewed all patients treated for RRES in the London Sarcoma Service (LSS) over a 22-year period with the aim of adding to the current literature and developing a prognostic risk score to aid clinical decision-making.

Methods And Results: One hundred and ninety-six patients were included; 64 patients received HDT, 98 standard non-HDT chemotherapy and 34 no systemic therapy.

Disease progression in osteosarcoma: a multistate model for the EURAMOS-1 (European and American Osteosarcoma Study) randomised clinical trial.

Objectives: Investigating the effect of prognostic factors in a multistate framework on survival in a large population of patients with osteosarcoma. Of interest is how prognostic factors affect different disease stages after surgery, with stages of local recurrence (LR), new metastatic disease (NM), LR+NM, secondary malignancy, a second NM, and death.

Design: An open-label, international, phase 3 randomised controlled trial.

Associations between diagnostic time intervals and health-related quality of life, clinical anxiety and depression in adolescents and young adults with cancer: cross-sectional analysis of the BRIGHTLIGHT cohort.

Background: The association of diagnostic intervals and outcomes is poorly understood in adolescents and young adults with cancer (AYA). We investigated associations between diagnostic intervals and health-related quality of life (HRQoL), anxiety and depression in a large AYA cohort.

Methods: Participants aged 12-24 completed interviews post-diagnosis, providing data on diagnostic experiences and the patient-reported outcomes (PROs) HRQoL, anxiety and depression.

Current approaches to management of bone sarcoma in adolescent and young adult patients.

Bone tumors are a group of histologically diverse diseases that occur across all ages. Two of the commonest, osteosarcoma (OS) and Ewing sarcoma (ES), are regarded as characteristic adolescent and young adult (AYA) cancers with an incidence peak in AYAs. They are curable for some but associated with unacceptably high rates of treatment failure and morbidity.

Reporting the whole story: Analysis of the 'out-of-scope' questions from the James Lind Alliance Teenage and Young Adult Cancer Priority Setting Partnership Survey.

Objective: We conducted a UK-wide survey to identify the top 10 research questions for young people's cancer. We conducted secondary analysis of questions submitted, which were 'out-of-scope' of the original survey aim. We sought to disseminate these questions, to inform practice, policy and the development of potential interventions to support young people with cancer.

Multimodal analysis of cell-free DNA whole-genome sequencing for pediatric cancers with low mutational burden.

Sequencing of cell-free DNA in the blood of cancer patients (liquid biopsy) provides attractive opportunities for early diagnosis, assessment of treatment response, and minimally invasive disease monitoring. To unlock liquid biopsy analysis for pediatric tumors with few genetic aberrations, we introduce an integrated genetic/epigenetic analysis method and demonstrate its utility on 241 deep whole-genome sequencing profiles of 95 patients with Ewing sarcoma and 31 patients with other pediatric sarcomas. Our method achieves sensitive detection and classification of circulating tumor DNA in peripheral blood independent of any genetic alterations.

Processes of care and survival associated with treatment in specialist teenage and young adult cancer centres: results from the BRIGHTLIGHT cohort study.

Objective: Survival gains in teenagers and young adults (TYA) are reported to be lower than children and adults for some cancers. Place of care is implicated, influencing access to specialist TYA professionals and research.Consequently, age-appropriate specialist cancer care is advocated for TYA although systematic investigation of associated outcomes is lacking.

Seeking international consensus on approaches to primary tumour treatment in Ewing sarcoma.

Background: The local treatment of Ewing sarcoma of bone involves surgery, radiotherapy or both. The selection of treatment depends on the anatomical extent of the tumour, the effectiveness of the proposed treatment, its morbidity, and the expectation of cure. However, not only are there variations in the approach to local treatment between individual patients, but also between treatment centres and countries.

SARC025 arms 1 and 2: A phase 1 study of the poly(ADP-ribose) polymerase inhibitor niraparib with temozolomide or irinotecan in patients with advanced Ewing sarcoma.

Background: In preclinical Ewing sarcoma (ES) models, poly(adenosine diphosphate ribose) polymerase (PARP) inhibitors were identified as a potential therapeutic strategy with synergy in combination with cytotoxic agents. This study evaluated the safety and dosing of the PARP1/2 inhibitor niraparib (NIR) with temozolomide (TMZ; arm 1) or irinotecan (IRN; arm 2) in patients with pretreated ES.

Methods: Eligible patients in arm 1 received continuous NIR daily and escalating TMZ (days 2-6 [D2-6]) in cohort A.

Longitudinal cohort study of the impact of specialist cancer services for teenagers and young adults on quality of life: outcomes from the BRIGHTLIGHT study.

Objectives: In England, healthcare policy advocates specialised age-appropriate services for teenagers and young adults (TYA), those aged 13 to 24 years at diagnosis. Specialist Principal Treatment Centres (PTC) provide enhanced TYA age-specific care, although many still receive care in adult or children's cancer services. We present the first prospective structured analysis of quality of life (QOL) associated with the amount of care received in a TYA-PTC DESIGN: Longitudinal cohort study.

Continuous 14 Day Infusional Ifosfamide for Management of Soft-Tissue and Bone Sarcoma: A Single Centre Retrospective Cohort Analysis.

Ifosfamide is used to treat soft-tissue sarcoma (STS) and bone sarcoma (BS), with improved efficacy at doses above 9 g/m/cycle. To mitigate treatment-associated toxicity with higher doses, continuous infusional ifosfamide is increasingly used. However, clinical outcome data remain limited.

Association of Self-reported Presenting Symptoms With Timeliness of Help-Seeking Among Adolescents and Young Adults With Cancer in the BRIGHTLIGHT Study.

Importance: Evidence regarding the presenting symptoms of cancer in adolescents and young adults can support the development of early diagnosis interventions.

Objective: To examine common presenting symptoms in adolescents and young adults aged 12 to 24 years who subsequently received a diagnosis of cancer and potential variation in time to help-seeking by presenting symptom.

Design, Setting, And Participants: This multicenter study is a cross-sectional analysis of the BRIGHTLIGHT cohort study, which was conducted across hospitals in England.

BRIGHTLIGHT researchers as 'dramaturgs': creating from complex research data.

Background: BRIGHTLIGHT is a national evaluation of cancer services for young people aged 13-24 years in England. It is a mixed methods study with six interlinked studies aiming to answer the question: do specialist cancer services for teenagers and young adults add value? http://www.brightlightstudy.

Development of a patient-reported experience questionnaire for patients with sarcoma: the Sarcoma Assessment Measure (SAM).

Purpose: The aim of the study was to develop a patient-reported outcome measure for patients with sarcoma-the Sarcoma Assessment Measure (SAM).

Methods And Results: The systematic development of SAM included a three-stage, mixed-methods study using semi-structured interviews, focus groups and questionnaires, with all stages involving patients from across the United Kingdom. In-depth interviews were conducted with 121 patients (50% male; aged 13-82; with soft tissue sarcoma (62%), bone tumours (28%) and gastrointestinal stromal tumours (10%)).

International randomised controlled trial for the treatment of newly diagnosed EWING sarcoma family of tumours - EURO EWING 2012 Protocol.

Background: Although there have been multiple randomised trials in newly diagnosed Ewing sarcoma family of tumours (ESFT) and these have been conducted over many years and involved many international cooperative groups, the outcomes for all stages of disease have plateaued. Internationally, the standard treatment of ESFT is not defined, and there is a need to add new agents other than conventional chemotherapy to improve outcomes. This trial will compare two different induction/consolidation chemotherapy regimens: (1) vincristine, ifosfamide, doxorubicin and etoposide (VIDE) induction and vincristine, actinomycin D, ifosfamide or cyclophosphamide, or busulfan and mephalan (VAI/VAC/BuMel) consolidation and (2) vincristine, doxorubicin, cyclophosphamide, ifosfamide and etoposide (VDC/IE) induction and ifosfamide and etoposide, vincristine and cyclophosphamide, vincristine, actinomycin D and ifosfamide, or busulfan and mephalan (IE/VC/VAI/BuMel) consolidation (randomisation 1, or R1).

The BRIGHTLIGHT National Survey of the Impact of Specialist Teenage and Young Adult Cancer Care on Caregivers' Information and Support Needs.

Background: Teenage and young adult cancer services in England are centralized in 13 principal treatment centers (TYA-PTC). These "specialist services" are designed to support caregivers as well as young people.

Objectives: To evaluate whether caregivers of young people with cancer had fewer unmet information and support needs if they had all/some care in a TYA-PTC.