Targeted treatment in children, especially with sarcomas, has limited utility due to small numbers of patients with driving mutations. Multimodal combination treatment and patient-centered approach combine treatment modalities administered based on comprehensive biological and empirical factors. Together, they extend precision oncology beyond a single molecular target. This is a single-institution real-world study of children and young adults diagnosed between 2004 and 2021, with relapsed/refractory and/or primary metastasizing high-risk solid tumors who underwent molecular tumor board. Patients underwent comprehensive biological examination of the tumor. A treatment recommendation was made subsequently combining empirical and biological data. All progressive/recurrent events during disease follow-up were analyzed using a hierarchical survival modeling approach combining individual patient time series in a meta-analytic framework for comparative analysis of treatment effects. A total of 93 patients were included in the analysis who experienced 189 events. Overall survival was 7.5 years. A comparative analysis utilizing event-free survival (EFS) ratio was possible in an ultra-high-risk subset of 66 patients with relapsed/progressive disease. Effectiveness was demonstrated in the treatment of sarcomas by empirical treatment (HR 0.57, p = 0.005), which consisted mainly of orally administered cyclophosphamide, vinblastine, and celecoxib, and by sunitinib or pazopanib (HR = 0.66, p = 0.032) in biologically guided administration. This study demonstrates that individualized treatment offers effective treatment options to a wide range of patients with high-risk cancers even for tumors without known high-level evidence molecular targets. We have shown that an alternative analytical approach based on the n-of-1-inspired framework is needed and applicable to demonstrate the effectiveness.
This study aimed to examine utilization, testing sequences, and associated genomic testing costs of companion diagnostics (CDx) and comprehensive genomic profiling (CGP) among Japanese patients with nine representative solid tumors. This retrospective study used anonymized data, from Medical Data Vision Co., Ltd. (MDV; January 2015-March 2025) and JMDC Inc. (JMDC; January 2015-January 2025), for patients with solid tumors of nine cancer types who underwent CDx and/or CGP testing or received any cancer treatment. Patient demographics, distribution and testing sequences of CDx and CGP, and associated genomic testing costs per patient were evaluated. Proportions of CDx and CGP testing varied across nine cancer types. Most patients underwent CDx testing once or twice, although some cohorts, particularly with non-small cell lung cancer (NSCLC), were tested thrice or more. Biliary tract cancer demonstrated the highest proportions for CGP testing alone, and both CDx and CGP testing. For both CDx and CGP testing, the greatest median costs were observed for ovarian and breast cancers, with bimodal peaks near US dollars (USD) 4000 and USD 5000. Median CDx costs were equal to or higher for patients who underwent both CDx and CGP testing compared with those who had CDx testing alone, particularly in breast, pancreatic, prostate, and ovarian cancers. For CDx testing alone, NSCLC, ovarian cancer, and prostate cancer had the highest costs, with a small peak near USD 1333. These results were mostly consistent across databases. Multiple CDx testing followed by CGP testing increased genomic testing costs per patient. Early implementation of CGP testing could reduce redundant testing and associated delays in treatment, thereby contributing to lower overall healthcare costs and more efficient treatment selection amid rapid advances in targeted therapies.
Locally advanced head and neck squamous cell carcinoma (LA-HNSCC) has major physical and psychosocial burdens. Although treatment challenges are well recognized, few studies have explored the full LA-HNSCC experience from first symptoms through to post-treatment recovery and survivorship. This exploratory, qualitative study sought to understand patient and caregiver perspectives across the LA-HNSCC journey. US adults with lived experience of LA-HNSCC (seven patients; two caregivers) participated in Johnson Johnson’s Head Neck Cancer Patient Engagement Research Council via virtual focus groups and online bulletin board activities. Transcripts and written responses were thematically analyzed using an a priori framework of three key stages: diagnosis and treatment planning, treatment, and post-treatment recovery and survivorship. Diagnostic journeys varied, with delays to diagnosis commonly perceived by participants as being attributed to multiple factors, including misinterpreted symptoms, difficulty accessing specialists, and limited provider awareness. Participants reported fear, uncertainty, and information overload during early decision-making. Treatment was described as burdensome, with side effects from radiation and chemotherapy leading to profound functional impairments affecting eating, swallowing, speech, and energy levels. Post-treatment long-term effects such as dry mouth, hearing loss, peripheral neuropathy, and anxiety about recurrence persisted and were often unexpected. Throughout these challenges, participants found strength, encouragement, and hope in peer support networks and valued knowledgeable and empathic communication with providers, as well as access to clear and digestible information; in particular, participants recommended early mental health support, tailored communication, and access to peer mentorship services. These exploratory, qualitative insights highlight unmet informational, psychosocial, and support needs in LA-HNSCC. Patients and their caregivers reported experiencing substantial, long-lasting physical and mental health effects that providers should be aware of to better support and empower patients throughout the care experience. A graphical abstract and patient journey infographic are available for this article. Infographic Head and neck cancer and its treatments can affect important bodily functions such as eating, speaking, and swallowing. The journey of head and neck cancer diagnosis and treatment can be challenging for both patients and their caregivers, including physical side effects and mental health impacts. This study asked a small group of patients with locally advanced head and neck cancer and caregivers to describe their experiences from the first signs of illness through treatment, recovery, and survivorship. Seven patients and two caregivers participated in online focus groups and written activities to share their perspectives. Many participants said they experienced delays before getting a correct diagnosis. Their symptoms were sometimes mistaken for more common issues, and it could be difficult to access specialists. This uncertainty often led to fear and emotional stress. Participants also felt overwhelmed by the amount of information they were given and the tasks and appointments they had to complete before starting treatment. Radiation and chemotherapy treatments led to painful and exhausting side effects that made daily life challenging. After treatment finished, many participants continued to experience unexpected long-lasting effects, including swallowing problems, hearing changes, nerve issues, and anxiety about the cancer returning. Participants found support, encouragement, and hope for the future in talking to other patients, sharing their experiences, and putting energy into healthy lifestyle factors. They valued clear information and access to emotional support. Participants recommended using patient support and mentorship networks, including organizations such as Imerman Angels and the Head and Neck Cancer Alliance.
Androgen receptor pathway inhibitors (ARPIs) are a critical prostate cancer (PC) treatment option. Previous research has suggested that the physical attributes of ARPI medications (e.g., pill formulation, pill shape, pill size, dosing) may influence patients’ treatment satisfaction, adherence, and persistence, which may impact patient outcomes and quality of life. The objective of this study was to describe Mexican clinical experts’ perspectives on how different attributes of pill forms of ARPIs may affect treatment preferences and compliance in patients with PC. This study employed a mixed-methods sequential design. In the first phase, we conducted a targeted literature review (TLR) to frame discussions with clinical experts. In phase 2, we conducted a structured expert elicitation (SEE) comprised of a survey, semi-structured individual interviews, and two focus groups with oncologists and urologists to gather insights about the influence of different ARPI attributes on patient preferences and compliance. In the third phase, we constructed a series of probabilistic mathematical models to analyze the likelihood of patients’ treatment preferences and adherence. The TLR identified pill size, shape, number, frequency, and form as key attributes that influence patients’ experiences and treatment compliance. Simpler regimens and smaller, easier-to-swallow formulations were consistently linked to greater comfort and adherence. In the SEE, participants ranked dosing frequency and pill count as the factors with the greatest influence on patient adherence when efficacy was comparable. During the interviews and focus groups, participants highlighted the importance of ≤ 2 pills/day, once-daily dosing, and small pill size alternatives. Findings from the Monte Carlo models were consistent with the earlier phases, supporting the importance of patient-preferred formulations. Findings from this mixed-methods study indicate that patient-preferred formulations may support better treatment adherence and support consideration of patient preferences in treatment decision-making between clinicians and patients to optimize adherence and outcomes in PC management.
