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… METHODS: This is a single-centre retrospective observational study of aHUS treated between August 2021 to August 2022 at a tertiary care public sector hospital in Northern India. It was a retrospective study based on hospital records. … METHODS: This is a single-centre retrospective observational study of multiple myeloma treated between January 2022 to August 2022 at a tertiary care public sector hospital. It was a retrospective study based on hospital records. …

Understanding Rare Anemias: Emerging Frontiers for Diagnosis and Treatment.

Background-This review provides a comprehensive overview of rare anemias, emphasizing their hereditary and acquired causes, diagnostic advancements, and evolving treatment strategies. It outlines the significance of rare anemias within public health, historical challenges in recognition and treatment, and the role of European initiatives like ENERCA and EuroBloodNet in advancing care. Content-This document discusses diagnostic technologies like next-generation sequencing and the impact of artificial intelligence, alongside the promising avenues of gene therapy, targeted drug treatments, and st

Инфекционные токсичные вещества, связанные с биконкретными антителами и клетками CAR-T в множестве миеломы: систематический обзор.

Лечение с перенацеливанием клеток изменило лечение повторной/охлажденной множественной миэломы (RRMM), но связано со значительным риском инфицирования. Мы систематически анализировали инфекционные заболевания после терапии Т-клеток CAR и биконкретных антител (BSAbs) с уделением особого внимания частотности, времени, патогенам, факторам риска и профилактике. Следуя руководящим принципам PRISMA, мы обыскали Паббмеда до мая. 22, 2026, и включает исследования, содержащие информацию о инфекционных результатах у пациентов, проходящих курс лечения с помощью клеток CAR-T или BsAbs. Всего 123 Был прове

The Psychometric Performance of Generic Preference-Based Measures in Informal Carers: A Systematic Review of Validation Studies.

BACKGROUND AND OBJECTIVE: A growing number of health technology assessment agencies recommend inclusion of informal carer outcomes in health economic evaluations. While generic preference-based measures (GPBMs) are favoured, the evidence regarding their performance in measuring the health-related quality of life of informal carers has not been synthesised. The aim of this systematic review was to synthesise the psychometric evidence of GPBMs in informal carers. METHODS: Following Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines, a literature search (index

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… However, international guidelines now support the inclusion of informal carer costs and benefits in economic evaluations, particularly when conducting an analysis using a societal or a public sector perspective [ 1 , 2 ]. …

Challenges in Implementing Comprehensive Precision Medicine Screening for Ovarian Cancer.

Precision medicine has revolutionised targeted cancer treatments; however, its implementation in ovarian cancer remains challenging. Diverse tumour biology and extensive heterogeneity in ovarian cancer can limit the translatability of genetic profiling and contribute to a lack of biomarkers of treatment response. This review addresses the barriers in precision medicine for ovarian cancer, including obtaining adequate and representative tissue samples for analysis, developing functional and standardised screening methods, and navigating data infrastructure and management. Ethical concerns relat

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… Partnerships between private and public sectors can also enhance accessibility, ensuring that advanced services are available to both researchers and patients. …

CAR-T State of the Art and Future Challenges, A Regulatory Perspective.

This review is an outlook on CAR-T development up to the beginning of 2023, with a special focus on the European landscape and its regulatory field, highlighting the main features and limitations affecting this innovative therapy in cancer treatment. We analysed the current state of the art in the EU and set out a showcase of the field's potential advancements in the coming years. For this analysis, the data used came from the available scientific literature as well as from the European Medicines Agency and from clinical trial databases. The latter were investigated to query the studies on CAR

Burden of cancer in the Eastern Mediterranean Region, 2005-2015: findings from the Global Burden of Disease 2015 Study.

ObjectivesTo estimate incidence, mortality, and disability-adjusted life years (DALYs) caused by cancer in the Eastern Mediterranean Region (EMR) between 2005 and 2015.MethodsVital registration system and cancer registry data from the EMR region were analyzed for 29 cancer groups in 22 EMR countries using the Global Burden of Disease Study 2015 methodology.ResultsIn 2015, cancer was responsible for 9.4% of all deaths and 5.1% of all DALYs. It accounted for 722,646 new cases, 379,093 deaths, and 11.7 million DALYs. Between 2005 and 2015, incident cases increased by 46%, deaths by 33%, and DALYs

Prediction of myelosuppression in cervical cancer after concurrent chemoradiotherapy by CT radiomics-based model.

BackgroundConcurrent chemoradiotherapy (CCRT) was highly effective in treating cervical cancer (CC) but raised the risk of bone marrow suppression. However, models to predict the risk of myelosuppression in CC patients after CCRT based on computed tomography (CT) imaging histology are immature.MethodsThe Region of interest (ROI) of the CT images was segmented, and radiomic features were extracted. Next, important features were further selected. The training and test sets were split 7:3, and 8 machine learning algorithms were applied to classify the features in the training set. The model's per

Medication Experience and Adherence to Oral Chemotherapy: A Qualitative Study of Patients' and Health Professionals' Perspectives.

Lack of adherence constitutes one of the most important challenges in patients undergoing treatment with oral antineoplastic drugs (ANEO). Understanding cancer patients' experiences with respect to their medication is key for optimizing adherence and therapeutic results. We aimed to assess the medication experience (ME) in patients with cancer in treatment with ANEO, to describe the barriers and facilitators related to the disease and its treatment and to compare them with the healthcare professionals' perspectives. We carried out an exploratory qualitative study in the University Hospital of

Recanalisation theraphy for acute ischemic stroke in cancer patients.

To date, very few studies focused their attention on efficacy and safety of recanalisation therapy in acute ischemic stroke (AIS) patients with cancer, reporting conflicting results. We retrospectively analysed data from our database of consecutive patients admitted to the Udine University Hospital with AIS that were treated with recanalisation therapy, i.e. intravenous thrombolysis (IVT), mechanical thrombectomy (MT), and bridging therapy, from January 2015 to December 2019. We compared 3-month dependency, 3-month mortality, and symptomatic intracranial haemorrhage (SICH) occurrence of patien

Ongoing clinical trials and treatment options for patients with systemic sclerosis-associated interstitial lung disease.

SSc is a rare CTD that affects multiple organ systems, resulting in substantial morbidity and mortality. Evidence of interstitial lung disease (ILD) is seen in ∼80% of patients with SSc. Currently there is no approved disease-modifying treatment for ILD and few effective treatment options are available. CYC is included in treatment guidelines, but it has limited efficacy and is associated with toxicity. MMF is becoming the most commonly used medication in clinical practice in North America and the UK, but its use is not universal. Newer agents targeting the pathogenic mechanisms underlying SSc

Возрастной состав Ассоциации по борьбе с диетическим протеином зависит от смертности всех пациентов с хроническими почечными заболеваниями.

Различается ли воздействие низкобелкового рациона питания на переход к конечной стадии почечных заболеваний (ЭСРД) и риск смертности среди молодых и пожилых людей с хроническими почечными заболеваниями (КХД) неясным. Мы провели ретроспективное когортное исследование CKD для изучения связи между потреблением белка и смертностью или почечными результатами, и влияет ли возраст на эту связь. В состав группы входили: 352 пациенты со стадией G3-5 CKD, за которым последовали на медиане 4.2 годы, прошли учебную госпитализацию и для которых исходное потребление белка оценивалось на основе 24-h Образцы

Leveraging patient experience data to guide medicines development, regulation, access decisions and clinical care in the EU.

Patient experience data (PED), provided by patients/their carers without interpretation by clinicians, directly capture what matters more to patients on their medical condition, treatment and impact of healthcare. PED can be collected through different methodologies and these need to be robust and validated for its intended use. Medicine regulators are increasingly encouraging stakeholders to generate, collect and submit PED to support both scientific advice in development programs and regulatory decisions on the approval and use of these medicines. This article reviews the existing definition

Artificial intelligence in cancer immunotherapy: current trends in predicting response and personalizing treatment.

Artificial intelligence (AI) can transform cancer immunotherapy by enabling more accurate prediction of treatment responses, the discovery of specific biomarkers, and the development of personalised treatment plans. Traditional single-marker biomarkers (PD-L1, TMB, MSI) lack consistency across tumour types and cannot be used to assess tumour heterogeneity or the dynamic tumour microenvironment (TME). This review synthesises developments in multimodal AI models that combine genomics, transcriptomics, radiomics, digital pathology (pathomics), circulating biomarkers, and clinical evidence to crea

Precision Medicine Through Network Language: Integrating Clinical Insight and Data Expertise.

Precision medicine is facing a critical transition driven by the growing complexity of biological data and the insufficient ability of current models to translate such data into clinically meaningful information. Linear, single-gene approaches are no longer adequate to explain the multifactorial nature of most modern diseases, whose phenotypes emerge from combinations of genetic, molecular, and environmental factors. Network-based precision medicine addresses this by providing a systemic framework capable of integrating heterogeneous omics data, interactomes, and clinical information to identi

Precision genomic profiling in Gaucher disease: insights from atypical presentations.

BackgroundGaucher disease (GD) is characterized by significant phenotypic heterogeneity, even among patients with identical GBA1 genotypes, suggesting the role of genetic and/or epigenetic modifiers. The enzymatic defect and pathological accumulation of glucosylceramide (GlcCer) lead to chronic metabolic inflammation, providing ample opportunities for interaction with other biological pathways to influence disease expression. Herein, we developed a model of precision medicine in this prototype single-gene disorder.MethodsThis study leveraged a well-characterized, longitudinally followed cohort

The challenge of chemotherapy-related cognitive impairment: assessing and managing cognitive decline after cancer treatment.

Chemotherapy-related cognitive impairment (CRCI) refers to a decline in cognitive function in patients during and after cancer treatment and is mainly associated with the use of cytotoxic chemotherapy (CT). As CT is still an essential component in the treatment of many cancers and taking into account the fact that cancer survival rates are increasing, CRCI may negatively impact the quality of life and working capacity of a growing number of individuals, especially those treated with curative intent in conditions such as breast cancer. There is still a need to address several issues related to

Novel humanized CD19-CAR-T (Now talicabtagene autoleucel, Tali-cel™) cells in relapsed/ refractory pediatric B-acute lymphoblastic leukemia- an open-label single-arm phase-I/Ib study.

Chimeric Antigen Receptor-T (CAR-T) cell therapy is effective for relapsed/refractory B-acute lymphoblastic leukemia (r/r B-ALL) but is not universally available. We developed a novel humanized CD19-directed CAR-T (HCAR19) approved for Phase 1/1b/2 trials. Patients aged 3-25 years were enrolled with r/r B-ALL and ineligible for allogeneic stem cell transplant. Lymphodepletion utilized standard-dose fludarabine and cyclophosphamide. A 3 + 3 design testing 3 dose-ranges was used to determine Phase-2 Dose (P2D): Dose-A, 1 × 106 HCAR19 cells/kg, Dose-B, 3-5 × 106/kg, and Dose-C, 10-15 × 106/kg. Pr

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… However, not all patients were able to undergo Allo-SCT due to various factors including financial constraints, long waiting lists at the public sector hospitals or reluctance to proceed with second transplant. …

Adverse drug reactions and drug-related problems with supportive care medications among the oncological population.

AimThe current study emphasizes the impact of adverse drug reactions (ADRs) and Drug-Related Problems (DRPs) caused by supportive care medications administered with chemotherapy.MethodThis is a longitudinal observational study carried out at the Ramaiah Medical College Hospital in Bengaluru, Karnataka, India, at the Department of Oncology. The data was recorded using a specifically created data collecting form. Based on the PCNE (Pharmaceutical Care Network Europe), DRPs are identified. The WHO probability scale, Modified Hartwig and Siegel for ADR severity assessment, Naranjo's algorithm for

Facilitators and barriers to implementing patient-reported outcomes in clinical oncology practice: a systematic review based on the consolidated framework for implementation research.

BackgroundIn clinical oncology practice, patient-reported outcomes (PROs) are essential for assessing the symptom burden, quality of life, and psychological status of patients. However, there remains a gap between the use of PROs in an oncologic setting and its implementation. Furthermore, numerous reviews in PRO implementation are often based on one particular technology, setting, or health condition, making it difficult to obtain a comprehensive and coherent summary of available evidence to help plan and undertake implementation. This systematic review aims to identify and integrate enablers