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Circulation, Volume 151, Issue 13, Page e865-e865, April 1, 2025.
Currently, C5 inhibitors are administered in three-quarters of newly diagnosed patients with aHUS in Europe, with varied utilisation and discontinuation practices. Treatment withdrawal is common and safe, although relapses may occur, particularly in genetic aHUS. However, baseline disease severity, selective use in expert centres, and indication bias affect outcome comparability. Findings must be considered in the context of patient-specific factors and disease severity at the time of treatment decisions.
Reliable epidemiological data are crucial for organisation, management planning, organisation of care, and treatment development in medicine, including heart failure.1 Global burden estimate measures may vary as per specific needs, but prevalence is usually considered the best single determinant, with estimates of 1–3% within the population at large.2 However, this number, as well as other epidemiology measures frequently mentioned in the literature, is based on few methodologically well designed consecutive and contemporary studies.
Introduction
Precision oncology aims to provide individualised treatment recommendations based on patient-specific characteristics. In this rapidly evolving field with increasing numbers of biomarkers and potential therapeutic targets, there is a growing unmet need for evidence guiding these individualised treatment recommendations. The Precision Oncology Program (POP) harnesses real-world data (RWD) and imaging mass cytometry (IMC) to evaluate the feasibility and utility of integrating different data modalities to inform personalised treatment recommendations. This program uses patient-matched clinicogenomic data and spatial single-cell proteomics analysis to support profiling-driven decision-making for patients with cancer at the Molecular Tumor Board.
Methods and analysis
The collaborative POP project recruits patients across all tumour entities and stages at the Comprehensive Cancer Center Zurich (CCCZ). For patients in the POP, a clinically and molecularly matched cohort is identified within the nationwide (US-based) de-identified Flatiron Health-Foundation Medicine clinicogenomic database (CGDB). It assesses whether clinical, genomic and outcome data of the CGDB cohort can inform treatment recommendations. In addition, multiplexed imaging mass cytometry (IMC) is performed in formalin-fixed paraffin-embedded tissue to assess the potential impact of spatial proteomics on personalised treatment decisions. RWD and IMC information is reviewed in the Molecular Tumor Board to assess the potential impact of this information on therapy decisions. However, since this is an observational study, these additional recommendations remain nonprescriptive and will not be forwarded to the treating physician.
Ethics and dissemination
The study is registered at ClinicalTrials.gov (NCT06680726) and approved by the Canton of Zurich Ethics Committee (Project ID: 2022-02289). Project-specific informed consent is obtained from all participants. Deceased patients may also be included. In this case, a signed general consent form must be available. Data privacy is ensured by unique patient numbers for pseudo-anonymised data. Study findings will be disseminated through international peer-reviewed journals, conferences, and direct communication with participants and relevant organisations.
Trial registration number
NCT06680726.
Annals of Internal Medicine, Ahead of Print.
This Viewpoint explores concerns surrounding possible US withdrawal from the World Health Organization and the effect this would have on US global health leadership and health security.
Preliminary data show that both vaccines protected against infection and hospitalization.
Stroke, Ahead of Print. BACKGROUND:As the impact of stroke remains, primary healthcare will continue to be a critical platform managing the poststroke journey. We aimed to identify stroke survivors assisted by community health worker in Brazil and how they relate to the location of rehabilitation facilities locations.METHODS:We developed a cross-sectional study using deidentified data from a real-world database generated by a free data collection app used by community health workers from May 2015 to January 2021 in Brazil to identify stroke survivors and to assess demographics and clinical characteristics. We used data from a public database, Cadastro Nacional de Estabelecimentos de Saúde, for identifying rehabilitation facilities. Locations were obtained by a geocoding application programming interface (Google Maps Platform), distances were measured in kilometers, and travel time in minutes.RESULTS:Among 2 397 764 individuals assisted by community health workers, 21 785 were stroke survivors, representing a 0.9% prevalence. Among this subgroup, the majority were in the Northeast region (n=10 951; 50.3%) and 16 922 (77.7%) in urban areas. Most individuals (n=11 504; n=142; 52.8%) were women, the mean age was 66.5 (SD, 14.7), and 4313 reported physical disability. In total, 348 rehabilitation facilities were identified, mostly located in the Southeast region (40.8%). The mean distance from stroke survivor to facility was 79.13 km (SD, 97.73; median [1Q, 3Q], 47.64 km [12.19, 107.80 km]), and mean travel time was 81.18 minutes (SD, 85.85). The Southern region recorded the largest mean and median distance (mean 175.58 km; SD, 163.18; median [1Q, 3Q] 88.47 [59.38, 425.38]) to rehabilitation center and the longest mean travel time (144.48 minutes; SD, 112.57; median [1Q, 3Q] 92.34 [60.59, 305.12]).CONCLUSIONS:Despite the availability of rehabilitation centers in Brazil, geographic access as represented by the distances and travel times observed access is still suboptimal. As a means of improving the clinical pathway and resource allocation, the use of large real-world databases and adequate analysis may become a key component for real needs assessments.
Circulation, Volume 151, Issue 10, Page 653-655, March 11, 2025.
Circulation, Ahead of Print. BACKGROUND:A preservation system, the Organ Care System (OCS; TransMedics) uses normothermic pulsatile perfusion during organ transport for heart transplantation. This system has demonstrated favorable outcomes in hearts recovered from extended-criteria donors after brain death (DBD) and donors after circulatory death (DCD).METHODS:The OCS Heart Perfusion Registry collects data on US heart transplantations using the OCS, static cold storage (SCS), or thoracoabdominal normothermic regional perfusion (NRP) recovered from DBD or DCD donors. We analyzed donor and recipient characteristics and posttransplantation outcomes in patients transplanted with OCS donor hearts (either DBD or DCD) compared with SCS hearts and OCS hearts from DCD donors compared with those recovered with NRP followed by SCS. Propensity score matching was used in survival analyses to adjust for differences among recipient characteristics.RESULTS:Between 2021 and 2023, 3225 consecutive heart transplantations enrolled from 56 centers were analyzed in the Heart Perfusion Registry. The OCS was used in 854 of 3225 heart transplantations (26.4%), among which 340 (39.8%) were DBD and 514 (60.2%) were DCD donors, whereas 2174 DBD donors were recovered with SCS and another 197 DCD donors with NRP techniques. The OCS-DBD group experienced a greater number of organ offer refusals before final acceptance (13 versus 6; Wilcoxon rank sum,P
The ATR-FTIR (Attenuated Total Reflectance-Fourier Transform Infrared) plasma spectra features can identify AD-related pathological changes. These spectral features serve as digital biomarkers, providing valuable support in the early screening and diagnosis of AD.
This Review describes why comprehensive molecular biomarker testing is now required for the accurate diagnosis and grading and prognostication of central nervous system tumors.
Circulation, Ahead of Print. Aim:The “2025 ACC/AHA/ACEP/NAEMSP/SCAI Guideline for the Management of Patients With Acute Coronary Syndromes” incorporates new evidence since the “2013 ACCF/AHA Guideline for the Management of ST-Elevation Myocardial Infarction” and the corresponding “2014 AHA/ACC Guideline for the Management of Patients With Non–ST-Elevation Acute Coronary Syndromes” and the “2015 ACC/AHA/SCAI Focused Update on Primary Percutaneous Coronary Intervention for Patients With ST-Elevation Myocardial Infarction.” The “2025 ACC/AHA/ACEP/NAEMSP/SCAI Guideline for the Management of Patients With Acute Coronary Syndromes” and the “2021 ACC/AHA/SCAI Guideline for Coronary Artery Revascularization” retire and replace, respectively, the “2016 ACC/AHA Guideline Focused Update on Duration of Dual Antiplatelet Therapy in Patients With Coronary Artery Disease.”Methods:A comprehensive literature search was conducted from July 2023 to April 2024. Clinical studies, systematic reviews and meta-analyses, and other evidence conducted on human participants were identified that were published in English from MEDLINE (through PubMed), EMBASE, the Cochrane Library, Agency for Healthcare Research and Quality, and other selected databases relevant to this guideline.Structure:Many recommendations from previously published guidelines have been updated with new evidence, and new recommendations have been created when supported by published data.
Claessens Z, Fieuws S, Daems J, et al. Do European regulatory measures accelerate national market access in Belgium? A retrospective analysis of medicines centrally authorised between 2015 and 2020. BMJ Open 2025;15:e091361. doi:10.1136/bmjopen-2024–0 91 361
This article has been corrected since it was published online. Figure 4 of the paper has been updated from (lacking number of medicines included in conditional marketing authorisation)
to (including the number of medicines with conditional marketing authorisation)
Background
The introduction of immune checkpoint inhibitors (ICIs) has modified treatment modalities for patients with lung cancer, offering new alternatives for treatment. Despite improved survival benefits, ICIs may cause side effects impacting patients’ quality of life (QoL). We aim to study the changes in global QoL (gQoL) of patients with advanced-stage lung cancer up to 18 months after treatment with ICIs between 2015 and 2021.
Methods and analysis
A longitudinal cohort study was conducted using the Oncological Life Study: Living well as a cancer survivor data-biobank from the University Medical Center Groningen. Participants completed the European Organisation for Research and Treatment of Cancer QoL 30-item questionnaire, at the beginning of their ICI treatment (baseline) and then at 6, 12 and 18 months. Using joint modelling, changes in predicted mean gQoL were studied by treatment regimens from baseline to 18 months, while accounting for the competing risk of death and adjusting for prespecified covariates.
Results
Of the 418 participants with median age of 66 years, 39% were women. Patients receiving first-line immuno-monotherapy with palliative intent had a small improvement in their gQoL within 6 months and no clinically significant change thereafter. Patients receiving first-line immune-chemotherapy with palliative intent had a small improvement in their gQoL within 12 months and no clinically significant change thereafter. Patients with second/further line immunotherapy with palliative intent or first-line chemoradiotherapy followed by durvalumab with curative intent had no clinically significant change in their gQoL over 18 months.
Conclusion
The changes in gQoL over time among patients with advanced-stage lung cancer may vary by treatment regimens based on drug intensity, line and intent of treatment, which will help clinicians and patients understand the potential dynamic of treatments on QoL. It may further influence treatment decisions and patient management strategies, reflecting the practical implications of different treatment regimens.
In Reply We appreciate the opportunity to respond to Dr Mintzker’s thoughtful comments regarding our study on the diagnostic accuracy of blood biomarkers for the detection of Alzheimer disease.
