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Medicaid Formulary Decisions and the Institute for Clinical and Economic Review: Abandoning Pseudoscience in Imaginary Pharmaceutical Pricing Claims.

Medicaid formulary committees and other gatekeepers face a difficult task. On the one hand they can utilize technical expertise in evaluating the real world evidence for clinical, quality of life and resource utilization claims for competing products while on the other hand they may be asked to assess claims built by simulation models for pricing and product access. A common option has been to take modeled claims from third parties such as the Institute for Clinical and Economic Review (ICER) at face value without challenging the model structure, its assumptions and its incremental cost-per-QA

Совпадение в полном тексте источника
… This lack of awareness of the I-QALY as an impossible mathematical construct extends to the use of the US National Health Interview Survey (NHIS) and the Behavioral Risk Factor Surveillance System (BRFSS) 25 . …

Future perspectives in melanoma research: meeting report from the "Melanoma Bridge", Napoli, December 5th-8th 2013.

The fourth "Melanoma Bridge Meeting" took place in Naples, December 5 to 8th, 2013. The four topics discussed at this meeting were: Diagnosis and New Procedures, Molecular Advances and Combination Therapies, News in Immunotherapy, and Tumor Microenvironment and Biomarkers.

Checkpoint inhibition and beyond: Precision immune engineering for the immune-privileged landscape of ocular malignancies.

Ocular malignancies, particularly uveal and conjunctival melanoma, exemplify tumors that evolve within one of the body's most immunologically constrained ecosystems, the eye's immune-privileged microenvironment. The limited success of PD-1/PD-L1 and CTLA-4 blockade in these cancers underscores the need to move beyond linear checkpoint inhibition toward multidimensional immune engineering. Through the confluence of synthetic bio-nanotechnology, AI-guided immunogenomics, and spatial immunomics, this review reframes ocular immunotherapy and redefines how tolerance and immunity might be programmat

Excess Body Weight and the Risk of Second Primary Cancers Among Cancer Survivors.

ImportanceLittle is known about the causes of second primary cancers among individuals with a history of cancer. Descriptive studies have suggested that lifestyle factors, including excess body weight, may be important.ObjectiveTo investigate whether excess body weight is associated with the risk of a second primary malignant neoplasm among cancer survivors.Design, setting, and participantsThis cohort study of adults in 21 states in the US used data from the Cancer Prevention Study II Nutrition cohort, a large prospective study that invited participants to respond to a survey in 1992 and bienn

Comprehensive Diagnosis and Management of POEMS Syndrome.

Polyneuropathy Organomegaly, Endocrinopathy, Monoclonal protein and Skin changes syndrome is a rare multisystem condition with a range of manifestations which are often overlooked as trivial comorbidities, until their whole triggers the possibility of the diagnosis. The diagnosis is typically delayed by 12-16 months, by which time patients can be severely disabled. There are no established consensus guidelines. We provide clinicians a comprehensive blueprint for managing POEMS from diagnostic suspicion through the work-up, selection of therapy, follow-up, and treatment of relapse based on publ

Improving management of patients with advanced cancer.

Development of bone metastases in patients with advanced cancer is associated with skeletal-related events (SREs) such as pathologic fractures, spinal cord compression, the requirement for surgery or palliative radiotherapy to bone, and hypercalcemia of malignancy. Skeletal morbidity may reduce patient mobility, limit functional independence, and impair quality of life (QOL). Proactive management of new or worsening bone pain or motor impairment is crucial because of the potential for rapid progression of symptoms. Administration of bisphosphonate therapy as a monthly infusion to patients with

The 11th NORDIC NUTRITION CONFERENCE NNC2016.

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Alcohol Misuse in Older Women: A Scoping Review of Correlates, Consequences, Treatment, and Prevention.

BackgroundAlcohol use and alcohol use disorder (AUD) among older women (defined as age 50 and over) have increased substantially in recent years. Compared to men and younger women, older women negotiate biopsychosocial transitions that put them at greater risk of alcohol-related health conditions as they age and are less reliably screened and treated for heavy alcohol use and AUD.ObjectivesThis scoping review represents a critical evaluation of research methodology and findings since 2004 on biopsychosocial correlates and consequences of alcohol misuse (i.e., heavy alcohol use, defined as four

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Genetic and Mechanistic Insights Inform Amyotrophic Lateral Sclerosis Treatment and Symptomatic Management: Current and Emerging Therapeutics and Clinical Trial Design Considerations.

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disorder affecting both upper and lower motor neurons. ALS is classically characterized by painless progressive weakness, causing impaired function of limbs, speech, swallowing, and respiratory function. The disease is fatal within 2-4 years, often the result of respiratory failure. The pathologic hallmark for a majority of ALS cases is aberrant cytoplasmic accumulations of the nuclear protein TAR-DNA binding protein (TDP-43). A total of 10-15% of ALS can be attributed to a single gene mutation, known as genetic or "familia

Унификация одиссеи: искусственный интеллект для диагностирования и лечения редких заболеваний.

Подытожить текущие проблемы в диагностике и лечении редких заболеваний (РД), осветить последние достижения в области искусственного интеллекта (АИ) для РД и предложить модель будущего состояния лечения пациентов РД. Многодисциплинарный экспертный обзор с кратким изложением современных практических задач и мер по ограничению ставок в области РД-клиентов с учетом ключевых клинических и исследовательских соображений в отношении нормативных и экономических ограничений. 10,000 известные РД оказывают коллективное воздействие 1 В 10 Американцы, в целом 30 million Люди. Ежегодно на РД приходится свыше

Устные бесплатные бумажные сессии.

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The Ups and Downs of STAT Inhibition in Acute Myeloid Leukemia.

Aberrant Janus kinase-signal transducer and activator of transcription (JAK-STAT) signaling is implicated in the pathogenesis of acute myeloid leukemia (AML), a highly heterogeneous hematopoietic malignancy. The management of AML is complex and despite impressive efforts into better understanding its underlying molecular mechanisms, survival rates in the elderly have not shown a substantial improvement over the past decades. This is particularly due to the heterogeneity of AML and the need for personalized approaches. Due to the crucial role of the deregulated JAK-STAT signaling in AML, select

Crosstalk between Circadian Rhythm Dysregulation and Tumorigenesis, Tumor Metabolism and Tumor Immune Response.

Circadian rhythm is a self-regulating 24-hour system that synchronizes with the day and night cycle in organisms. The regulation of this system is controlled by clock genes, which function to harmoniously express molecular levels that facilitate the orderly coordination of various cellular processes, such as sleep, metabolism, endocrine function, cell proliferation and immunity. The root cause of tumorigenesis is that the body loses its normal regulation of cell growth at the genetic level. Long-term disruptions in circadian rhythms caused by factors such as shift work, jet lag, and unstable s

Double jeopardy: how BRCA2, TP53, and DNMT3A redefine the link between infertility and cancer risk.

BackgroundGermline mutations in genes governing DNA repair, cell cycle regulation, and epigenetic modification are now recognized as common etiological factors for both cancer predisposition and reproductive dysfunction. This reveals a profound intersection between reproductive biology and oncogenesis.MethodA systematic narrative review was conducted. The literature search spanned PubMed/MEDLINE, Scopus, and Web of Science using keywords and MeSH terms related to infertility phenotypes, cancer predisposition syndromes, and shared molecular mechanisms (e.g., DNA repair, epigenetics).OutcomesThe

Insights into Cancer-Associated Thrombosis Leading Towards Ischemic Stroke.

Stroke leads to significant disability in most patients, whereas cancer elevates the occurrence of stroke. The incidence of cancer-associated stroke (CAS) is projected to rise as a result of improvements in cancer therapies. Various forms of cancer have been demonstrated to be linked to ischemic stroke. Cancer might influence stroke pathophysiology either directly or through coagulation that creates a hypercoagulative state, in addition to infections. Treatment methods for cancer, including chemotherapy, radiotherapy, and surgery, have all been demonstrated to increase the risk of stroke as we

Breaking Boundaries: Immunotherapy for Myeloid Malignancies.

Immunotherapy has revolutionized the treatment of myeloid oncologic diseases, particularly for patients resistant to chemotherapy or ineligible for allogeneic stem cell transplantation due to age or fitness constraints. As our understanding of the immunopathogenesis of myeloid malignancies expands, so too do the treatment options available to patients. Immunotherapy in myeloid malignancies, however, faces numerous challenges due to the dynamic nature of the disease, immune dysregulation, and the development of immune evasion mechanisms. This review outlines the progress made in the field of im

Bortezomib-induced peripheral neuropathy: from molecular mechanisms to clinical management.

Bortezomib (BTZ) is a first-generation proteasome inhibitor that has shown significant efficacy in the treatment of multiple myeloma (MM), providing significant therapeutic benefits without severely compromising overall patient health. However, one of the most common and debilitating side effects associated with BTZ therapy is bortezomib-induced peripheral neuropathy (BIPN). This condition is the leading complication observed in patients undergoing BTZ treatment and has a profound impact on treatment regimens, often necessitating dose reductions or alterations in the dosing schedule. Despite t

Are you "tilting at windmills" or undertaking a valid clinical trial?

In this review, several aspects surrounding the choice of a therapeutic intervention and the conduct of clinical trials are discussed. Some of the background for why human studies have evolved to their current state is also included. Specifically, the following questions have been addressed: 1) What criteria should be used to determine whether a scientific discovery or invention is worthy of translation to human application? 2) What recent scientific advance warrants a deeper understanding of clinical trials by everyone? 3) What are the different types and phases of a clinical trial? 4) What c

Inflammation in cancer: therapeutic opportunities from new insights.

As one part of the innate immune response to external stimuli, chronic inflammation increases the risk of various cancers, and tumor-promoting inflammation is considered one of the enabling characteristics of cancer development. Recently, there has been growing evidence on the role of anti-inflammation therapy in cancer prevention and treatment. And researchers have already achieved several noteworthy outcomes. In the review, we explored the underlying mechanisms by which inflammation affects the occurrence and development of cancer. The pro- or anti-tumor effects of these inflammatory factors