To explore how parents of children with de novo retinoblastoma (RB) experience the diagnostic process and acute treatment phase, and to identify factors that may support parental coping and adaptation.

A qualitative interview study using reflexive thematic analysis.

National Retinoblastoma Unit at Aarhus University Hospital, Denmark.

Thirty-one parents (21 mothers, 10 fathers) of 21 children diagnosed with de novo RB were recruited via hospital follow-up clinics and a support group day.

For most parents, the diagnostic process was short. In cases of diagnostic delay, parents described frustration and guilt due to missed symptoms. Receiving the RB diagnosis was described as a surreal experience, accompanied by feelings of shock, grief and loss of control. Parents faced challenges in adapting to rapid medical decisions and the unfamiliar demands of hospital protocols. However, meeting the clinical experts was a relief, as parents felt they were in capable hands, experiencing empathetic communication and a clearly framed treatment plan. Parents emphasised the importance of support systems, including family, healthcare professionals and the child’s resilience, as crucial for coping with and managing the diagnosis.

Parents faced a sudden and disruptive transition from symptom recognition to life-altering diagnosis and treatment. While professional care and communication were experienced as supportive, they did not eliminate the emotional impact. Clinical pathways should prioritise early validation of parental concerns and provide transparent communication, both prior to referral and throughout treatment. Future research should examine longer-term parental adjustment and identify interventions that support emotional resilience beyond the acute phase.

The standard treatment of oral cavity cancers (OCC) relies on surgery and postoperative radiotherapy (poRT) for advanced stages or poor factors. In more than 75% of cases, reconstructive surgery with a flap aims to restore the function lost with tumour resection. Current poRT planning and delineation guidelines omit the presence of a flap. It may be assumed that poRT with flap sparing may allow for reducing radio-induced toxicities and improving functional outcomes, without impairing local primary control. The OPTIFLAP trial assesses non-inferior locoregional control using flap sparing compared with conventional flap-agnostic radiotherapy in patients with OCC, while reducing treatment-related toxicity and improving functional outcomes.

The OPTIFLAP study is a French, multicentre, 1:1 randomised, phase III, controlled trial. It will recruit 348 patients with OCC with a flap. Recruitment is active with the first enrolment on 2 July 2025 and is planned over 48 months. The primary outcome is non-inferior 2-year locoregional control rate using flap sparing compared with flap-agnostic radiotherapy (as per standard routine practice) in completely resected OCCs undergoing poRT. Key secondary outcomes include rates of toxicities, locoregional relapse-free survival, progression-free survival, overall survival, quality of life, functional outcomes (assessed by the Performance Status Scales for Head and Neck Cancer, the MD Anderson Dysphagia Inventory (self-questionnaire) and the Phonation Handicap Index (self-questionnaire)), flap doses and outcomes between arms depending on dosimetric parameters. The trial incorporates translational ancillary studies addressing individual radiosensitivity, salivary microbiome evolution, radiomics and dosiomics of flap changes, as well as medico-economic evaluation.

The study protocol has been approved by the Medical Ethics Committee East III (January 2025; Ref 24.05832.000442) and the French Agency for Medical and Health Products Safety (December 2024; ID-RCB: 2024-A01764-43) and was validated by review boards of all participating centres. Written informed consent will be obtained from all participants. Study results will be published in international peer-reviewed scientific journals and presented at relevant scientific conferences.

NCT06798922.

Cardiovascular (CV) disease is the leading cause of morbidity and mortality globally. Low-density lipoprotein cholesterol (LDL-C) is an important modifiable risk factor of major adverse cardiovascular events. Patients without prior myocardial infarction (MI) or stroke but with established risk factors and elevated LDL-C may benefit from intensive lipid-lowering therapy (LLT); however, the size and potential healthcare burden of this population globally are not known. The benefits of evolocumab, a proprotein convertase subtilisin/kexin type 9 inhibitor, in these patients, are currently being studied in the phase 3 Effect of Evolocumab in Patients at High Cardiovascular Risk Without Prior Myocardial Infarction or Stroke (VESALIUS-CV) trial. To characterise the high-risk pre–CV-event (VESALIUS-CV–like) individuals in the real world, an observational study is being conducted across multiple countries.

This retrospective cohort study will use a common protocol and an analytical common data model approach to characterise VESALIUS-CV–like individuals in the real world across different geographical regions and healthcare settings. The study period will be from 2010 to 2022, subject to data availability in study sites. Patients aged 50 years and older at high risk of CV disease but without prior MI or stroke will be included in this study. VESALIUS-CV–like individuals are defined through a combination of the following: (1) one diagnosis of coronary artery disease, cerebrovascular disease, peripheral artery disease or diabetes with microvascular complications or chronic insulin use; (2) an elevated LDL-C measurement and (3) other high-risk factors. The objectives of this study are to estimate the prevalence of VESALIUS-CV–like individuals, describe their characteristics and care pathways and estimate their incidence rates of CV events and healthcare costs. The prevalence of VESALIUS-CV–like individuals will be expressed as annual prevalence; patient characteristics at index date will be presented using summary statistics; care pathways will be summarised as LLT prescription across time; and the incidence of defined CV events will be expressed as events per person-years as well as at certain time periods. Healthcare costs will be presented as CV-related costs in different time periods.

Approvals of the study protocol were obtained from relevant local ethics and regulatory frameworks for each participating database. The results of the study will be submitted to peer-reviewed scientific publications and presented at scientific conferences.