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Gene therapy of rare diseases as a milestone in medicine - overview of the field and report on initial experiences in Slovenia.

Gene therapy has transitioned from a long-awaited promise to a clinical reality, offering transformative treatments for rare congenital diseases and certain cancers, which have a significant impact on patients' lives. Current approaches focus on gene replacement therapy, either in vivo or ex vivo, mostly utilizing viral vectors to deliver therapeutic genes into target cells. However, refining these techniques is essential to overcome challenges and complications associated with gene therapy to ensure long-term safety and efficacy. Slovenia has witnessed significant advancements in this field s

Preclinical B cell depletion and safety profile of a brain-shuttled crystallizable fragment-silenced CD20 antibody.

BACKGROUND: The blood-brain barrier (BBB) presents a major challenge for the development of monoclonal antibody (mAb)-based therapies for brain disorders. To improve the likelihood of success of such therapies, Roche Brainshuttle technology utilizes a single anti-transferrin receptor 1 (TfR1)-antigen-binding antibody fragment linked to a therapeutic antibody, allowing engagement with TfR1 to transport the therapeutic antibody into the brain via receptor-mediated transcytosis. METHODS: We compared Fc-silenced and Fc-competent variants of the Brainshuttle and the parental (non-shuttled) type II

Mindfulness-Based Group Medical Visits: Strategies to Improve Equitable Access and Inclusion for Diverse Patients in Cancer Treatment.

BackgroundMindfulness-based interventions (MBIs) are supported by clinical practice guidelines as effective non-pharmacologic interventions for common symptoms experienced by cancer patients, including anxiety, depression, and fatigue. However, the evidence predominately derives from White breast cancer survivors. Racial and ethnic minority patients have less access to integrative oncology care and worse cancer outcomes. To address these gaps, we designed and piloted a series of mindfulness-based group medical visits (MB-GMVs), embedded into comprehensive cancer care, for racially and ethnical

Antitumor Activity of Cannabinoids and Their Interaction with Chemotherapy: A Systematic Review and Meta-Analysis of Preclinical Evidence.

Background: Cannabinoids are studied as anticancer agents, but their effects vary across tumors, compounds, and experimental settings, underscoring the need to define consistent patterns. Our objective was to map cannabinoid efficacy across cancer preclinical models and identify tumor settings with the greatest translational promise. Methods: The protocol was registered on PROSPERO (CRD42025543744); PubMed, Embase, and CENTRAL were searched on 4 April 2024 for in vitro and in vivo studies assessing cannabinoid antitumor effects alone or with chemotherapy versus vehicle or chemotherapy only. Ra

Validation of the Malayalam Version of Leeds Assessment of Neuropathic Symptoms and Signs Pain Scale in Cancer Patients in the Regional Cancer Centre, Thiruvananthapuram, Kerala, India.

ObjectiveThe Self-administered Leeds Assessment of Neuropathic Symptoms and Signs (S-LANSS) is a 7-item self-report scale developed to identify pain which is of predominantly neuropathic origin. The aim of this study was to develop a Malayalam version of the LANSS and to test its validity and reliability in chronic pain patients.MethodologyWe enrolled 101 Malayalam-speaking chronic pain patients who visited the Division of Palliative Medicine, Regional Cancer Centre, Thiruvananthapuram, Kerala, India. The translated version of S- LANSS was constructed by standard means. Fifty-one neuropathic p

A case report of fludarabine associated ectopic atrial bradycardia and literature review of fludarabine induced bradycardia.

BACKGROUND: Fludarabine is a chemotherapeutic agent with lymphodepleting effects that is increasingly used as part of a conditioning regimen prior to allogeneic stem cell transplantation. Fludarabine is generally considered a relatively safe medication with only rare cases of cardiotoxic side effects. CASE PRESENTATION: Here, we present a case of a 30-year-old woman who was undergoing conditioning for a haploidentical cell transplantation for treatment of Fanconi anemia with a 5-day course of daily fludarabine infusion. After her second fludarabine infusion, she was noted to have ectopic atri

Epidemiology, Clinical Data, and Management of Aseptic Abscess Syndrome: Review of Published Cases Outside France.

Aseptic abscess syndrome is a clinical entity that is being increasingly documented. Unfortunately, apart from the French registry, there are no other studies presenting collective data. In this review, we sought to analyze clinical and laboratory data from case reports published from the rest of the world. A total of 107 articles were found through our literature search in PubMed, Scopus, and Google, which contained 108 patients who met our eligibility criteria, including pediatric cases. The mean age at diagnosis was 39.1 years, and 54.6% of the patients were female. Cases were found affecti

Повышение уровня информированности и знаний о раке в Йоханнесбурге и округах iLembe с помощью целенаправленных просветительских мероприятий на уровне общин: экспериментальное исследование.

В Южной Африке рак по-прежнему вызывает все большую озабоченность в области общественного здравоохранения, особенно в недостаточно обслуживаемых общинах, где различия в уровне информированности и доступе к медицинскому обслуживанию приводят к задержкам в диагностике. В рамках этого исследования была проведена оценка воздействия ориентированной на культурные особенности просветительской деятельности на основе адаптированного модуля исследований и просвещения по вопросам доступа к информации о раковых заболеваниях (c-CARE) в районах Йоханнесбурга и iLembe. Было проведено экспериментальное исслед

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… Christianity was the most commonly reported religion among both men and women, reported by 72.9% and 88.7% of participants, respectively. Most participants had attained at least secondary education. … Female ( n = 151) Age-Group | Number | Percent | Number | Percent 20–29 | 2 | 3.4 | 5 | 3.3 30–39 | 10 | 17.0 | 39 | 25.8 40–49 | 16 | 27.1 | 49 | 32.5 50–59 | 17 | 28.8 | 38 | 25.2 60–69 | 8 | 13.6 | 17 | 11.3 70+ | 6 | 10.2 | 3 | 2.0 Median (Range) | 51 (24–76) | | 48 (23–73) | Religion | | | | Christian …

Recognition and management of persistent chylomicronemia: A joint expert clinical consensus by the National Lipid Association and the American Society for Preventive Cardiology.

Extreme hypertriglyceridemia, defined as triglyceride (TG) levels ≥1000 mg/dL, is almost always indicative of chylomicronemia. The current diagnostic approach categorizes individuals with chylomicronemia into familial chylomicronemia syndrome (FCS; prevalence 1-10 per million), caused by the biallelic combination of pathogenic variants that impair the lipolytic action of lipoprotein lipase (LPL), or multifactorial chylomicronemia syndrome (MCS, 1 in 500). A pragmatic framework should emphasize the severity of the phenotype and the risk of complications. Therefore, we endorse the term "persiste

POSTER PRESENTATIONS

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Clinical Administration Characteristics of Subcutaneous and Intravenous Administration of Daratumumab in Patients With Multiple Myeloma at Mayo Clinic Infusion Centers.

PurposeMedian duration of daratumumab (DARA) administration for treatment of multiple myeloma is 3-7 hours for the intravenous formulation (DARA IV) and 3-5 minutes for the subcutaneous formulation (DARA SC). Here, we describe clinical administration characteristics of DARA using a novel method for data extraction from electronic health records.MethodsTime-based measurements were extracted using a scheduling/pharmacy software program that tracked patient movement through appointments for patients initiating DARA in Mayo Clinic infusion centers from April 5, 2017, to October 14, 2021. Cohorts i

The impact of COVID-19 on patients with hematological malignancies: the mixed-method analysis of an Israeli national survey.

BackgroundThe COVID-19 pandemic required reevaluation of the therapeutic approach and added emotional stress for patients with hematological malignancies at high risk of contracting the virus. We aimed to evaluate how it affected such patients during the second lockdown in Israel.MethodsThis national survey included Hebrew-speaking patients with hematological malignancy. This included three tools with 28 items of sociodemographic and medical baseline characteristics, management of hematological disease, and evaluation of emotional coping during COVID-19 pandemic; the Hebrew version of the Pati

A nationwide survey of knowledge of and compliance with cancer pain management guidelines by korean physicians.

PurposeAlthough cancer pain is prevalent, under-treatment still remains a problem. Knowledge of and compliance with guidelines for management of cancer pain were analyzed for exploration of physician-related barriers to cancer pain management. In addition, physicians' knowledge and its correlation with cancer pain control were audited.Materials and methodsFrom July 8 to December 2, 2010, a nationwide survey of house staff enquired about their knowledge of cancer pain control guidelines, and the medical records of patients under their care were analyzed.ResultsIn total, 180 physicians participa

Caregiver strain among patients of palliative care in Sri Lanka: validation of modified caregiver strain index - Sinhala version.

BackgroundCare givers of Palliated patients are at risk of adverse physical, psychosocial and emotional sequelae in varied nature. Efficient and valid assessment tools facilitate early detection to take corrective measures. The Modified Caregiver Strain Index (MCSI), composed of domains associated with caregiver strain is a simple and brief tool that can be used in both clinical and field settings. This study aimed to adapt and validate this in order to cater effective palliative care services in Sri Lanka.MethodsAfter cross-cultural adaptation, 200 primary caregivers in 3 teaching hospitals w

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… The main religions practised in Sri Lanka; Buddhism, Christianity, Islam and Hinduism highlight the importance of love and care, particularly for those who are sick. …

Acute pancreatitis with hypercalcemia caused by primary hyperparathyroidism associated with paraneoplastic syndrome: A case report and review of literature.

BACKGROUND: Although acute pancreatitis associated with hyperparathyroidism has occasionally been reported, acute pancreatitis with metabolic encephalopathy caused by hyperparathyroidism combined with paraneoplastic syndrome is an extremely rare entity and poorly described in the literature. CASE SUMMARY: We present a case of a 56-year-old female with upper abdominal discomfort and intermittent nausea and vomiting for 1 wk, without apparent abdominal pain or bloating, no jaundice and decreased blood pressure at the outset. The patient was ultimately diagnosed with moderately severe acute panc

EMA Review of Panobinostat (Farydak) for the Treatment of Adult Patients with Relapsed and/or Refractory Multiple Myeloma.

On August 28, 2015, a marketing authorization valid through the European Union was issued for panobinostat, in combination with bortezomib and dexamethasone, for the treatment of adult patients with relapsed and/or refractory multiple myeloma who have received at least two prior regimens including bortezomib and an immunomodulatory agent (IMiD).Panobinostat is an orally available histone deacetylase (HDAC) inhibitor that inhibits the enzymatic activity of HDAC proteins at nanomolar concentrations. HDAC proteins catalyze the removal of acetyl groups from the lysine residues of histones and some

Response Evaluation of Chemotherapy for Lung Cancer.

Assessing response to therapy allows for prospective end point evaluation in clinical trials and serves as a guide to clinicians for making decisions. Recent prospective and randomized trials suggest the development of imaging techniques and introduction of new anti-cancer drugs. However, the revision of methods, or proposal of new methods to evaluate chemotherapeutic response, is not enough. This paper discusses the characteristics of the Response Evaluation Criteria In Solid Tumor (RECIST) version 1.1 suggested in 2009 and used widely by experts. It also contains information about possible d

Critically ill cancer patient's resuscitation: a Belgian/French societies' consensus conference.

To respond to the legitimate questions raised by the application of invasive methods of monitoring and life-support techniques in cancer patients admitted in the ICU, the European Lung Cancer Working Party and the Groupe de Recherche Respiratoire en Réanimation Onco-Hématologique, set up a consensus conference. The methodology involved a systematic literature review, experts' opinion and a final consensus conference about nine predefined questions1. Which triage criteria, in terms of complications and considering the underlying neoplastic disease and possible therapeutic limitations, should be

A Review of the Regulatory Challenges of Personalized Medicine.

Personalized medicine integrates genomics with clinical and familial histories, revolutionizing healthcare by tailoring treatments to individual patient characteristics. At its core, pharmacogenomics enables the customization of medication prescriptions based on genetic profiles, enhancing drug efficacy and safety. This precision medicine approach addresses disease diagnosis, prevention, and treatment, offering targeted therapies for conditions like autoimmune disorders, rheumatoid arthritis, and neoplastic conditions. Examples of pharmacogenomics and personalized medicine include treatment fo

Using QIP-MS to Guide the Timing of MRD Testing in Patients With Multiple Myeloma: A Budget Impact Analysis From the French Payer Perspective.

PurposeSerum or urine protein electrophoresis (SPEP or UPEP) and immunofixation electrophoresis (SIFE or UIFE) are routinely used to detect M-proteins in MM patients. However, SPEP and SIFE are not sensitive enough to measure M-protein levels that are low but still clinically significant. This study aimed to evaluate the potential cost savings associated with using the EXENT GAM Assay, a serum-based quantitative-immunoprecipitation mass spectrometry (QIP-MS) diagnostic test instead of SIFE to guide the timing of minimal residual disease (MRD) testing for patients with multiple myeloma (MM).Pat