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Pilot clinical trial of macimorelin to assess safety and efficacy in patients with cancer cachexia.

BACKGROUND: Cancer cachexia is associated with reduced body weight, appetite and quality of life (QOL) with no approved treatments. Growth hormone secretagogues like macimorelin have potential to mitigate these effects. METHODS: This pilot study assessed the safety and efficacy of macimorelin for 1 week. Efficacy was defined a priori as 1-week change in body weight (≥0.8 kg), plasma insulin-like growth factor (IGF)-1 (≥50 ng/mL) or QOL (≥15%). Secondary outcomes included food intake, appetite, functional performance, energy expenditure and safety laboratory parameters. Patients with cancer ca

Mass Spectrometry Approaches for Identification and Quantitation of Therapeutic Monoclonal Antibodies in the Clinical Laboratory.

Therapeutic monoclonal antibodies (MAbs) are an important class of drugs used to treat diseases ranging from autoimmune disorders to B cell lymphomas to other rare conditions thought to be untreatable in the past. Many advances have been made in the characterization of immunoglobulins as a result of pharmaceutical companies investing in technologies that allow them to better understand MAbs during the development phase. Mass spectrometry is one of the new advancements utilized extensively by pharma to analyze MAbs and is now beginning to be applied in the clinical laboratory setting. The rise

Ход работы сессии 29th Европейский конгресс педиатрии.

Аннотация не предоставлена источником. Откройте карточку для перехода к публикации.

Long-term patient-reported neurocognitive outcomes in adult survivors of hematopoietic cell transplant.

Survivors of hematopoietic cell transplant (HCT) are at risk for neurocognitive impairments, which can negatively affect quality of life. Given limited studies, we aimed to describe the neurocognitive outcomes in a cohort of long-term adult HCT survivors. Eligible survivors (age ≥21 years at HCT and alive ≥2 years following HCT) completed a 60-question survey of neurocognitive function and quality of life, which included the Neuro-Quality of Life Cognitive Function Short Form (Neuro-QoL) and the Childhood Cancer Survivor Study Neurocognitive Questionnaire (NCQ). Analyses of risk factors includ

Cellular immunotherapy for hematological malignancy: recent progress and future perspectives.

Advancements in the field of cellular immunotherapy have accelerated in recent years and have changed the treatment landscape for a variety of hematologic malignancies. Cellular immunotherapy strategies exploit the patient's immune system to kill cancer cells. The successful use of CD19 chimeric antigen receptor (CAR) T-cells in treating B-cell malignancies is the paradigm of this revolution, and numerous ongoing studies are investigating and extending this approach to other malignancies. However, resistance to CAR-T-cell therapy and non-durable efficacy have prevented CAR-T-cells from becomin

Extracellular Vesicles After Allogeneic Hematopoietic Cell Transplantation: Emerging Role in Post-Transplant Complications.

Extracellular vesicles (EVs) play an important role in the cellular crosstalk by transferring bioactive molecules through biological barriers from a cell to another, thus influencing recipient cell functions and phenotype. Therefore, EVs are increasingly being explored as biomarkers of disease progression or response to therapy and as potential therapeutic agents in different contexts including in hematological malignancies. Recently, an EV role has emerged in allogeneic hematopoietic cell transplantation (allo-HCT) as well. Allogeneic hematopoietic cell transplantation often represents the on

Risk factors and outcome of COVID-19 in patients with hematological malignancies.

BackgroundPrognostic factors of poor outcome in patients with hematological malignancies and COVID-19 are poorly defined.Patients and methodsThis was a Spanish transplant group and cell therapy (GETH) multicenter retrospective observational study, which included a large cohort of blood cancer patients with laboratory-confirmed SARS-CoV-2 infection through PCR assays from March 1st 2020 to May 15th 2020.ResultsWe included 367 pediatric and adult patients with hematological malignancies, including recipients of autologous (ASCT) (n = 58) or allogeneic stem cell transplantation (allo-SCT) (n = 65

Both comorbidity and worse performance status are associated with poorer overall survival after external beam radiotherapy for prostate cancer.

BackgroundIn this retrospective study, we evaluated the biochemical recurrence rate, metastatic disease progression, and prostate cancer-specific and overall survival in patients curatively treated with external beam radiotherapy (EBRT) for early prostate cancer (PC). We also examined the prognostic effect of comorbidity by Charlson Comorbidity Index (CCI) and overall performance status by Eastern Clinical Oncology Group (ECOG) score.MethodsA total of 665 men treated between 2008 and 2013 were enrolled from Tampere University Hospital, Finland. Prostate-specific antigen (PSA) tests and hospita

The 44th Annual Meeting of the European Society for Blood and Marrow Transplantation: Physicians Poster Sessions

Аннотация не предоставлена источником. Откройте карточку для перехода к публикации.

Chimeric antigen receptor-based cell therapy for treating urological tumors.

Urological tumors represent a significant global health challenge, with conventional therapies often proving insufficient to control disease progression. Recent breakthroughs in cellular immunotherapy, particularly in chimeric antigen receptor (CAR)-T cell, CAR-natural killer cell, and CAR-macrophage therapies, have demonstrated remarkable potential for treating these malignancies. Ongoing research is actively refining CAR-based strategies to enhance their precision in targeting tumor-associated antigens. This review comprehensively summarizes the applications of CAR cell therapy in the follow

Rituximab resistance in ITP and beyond.

The pathophysiology of immune thrombocytopenia (ITP) is complex and encompasses innate and adaptive immune responses, as well as megakaryocyte dysfunction. Rituximab is administered in relapsed cases and has the added benefit of inducing treatment-free remission in over 50% of patients. Nevertheless, the responses to this therapy are not long-lasting, and resistance development is frequent. B cells, T cells, and plasma cells play a role in developing resistance. To overcome this resistance, targeting these pathways through splenectomy and novel therapies that target FcγR pathway, FcRn, complem

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… Additionally, CRs were observed in 8 out of 12 patients with multilineage cytopenias associated with common variable immunodeficiency, Evans syndrome, or systemic lupus erythematosus ( 84 ). …

Recombinant Human Granulocyte-Macrophage Colony-Stimulating Factor (rhu GM-CSF) as Adjuvant Therapy for Invasive Fungal Diseases.

BackgroundSargramostim (yeast-derived, glycosylated recombinant human granulocyte-macrophage colony-stimulating factor [rhu GM-CSF]) augments innate and adaptive immune responses and accelerates hematopoietic recovery of chemotherapy-induced neutropenia. However, considerably less is known about its efficacy as adjunctive immunotherapy against invasive fungal diseases (IFDs).MethodsThe clinical courses of 15 patients with pediatric malignancies and IFDs treated adjunctively with sargramostim at a single institution were analyzed in a retrospective cohort review. Further, a systematic review of

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… Trachana 2001 (Greece) [ 25 ] | 13 yo M | Common variable immunodeficiency following pulmonary candidiasis | Hepatitis ( A. terreus ) | Yes | Yes (prior fluconazole, LAmB) | Rhu GM-CSF 5 µg/kg/d | LAmB, itraconazole | No remarkable changes seen in number and size of fungal lesions. …

Ixazomib, pomalidomide and dexamethasone in triple-class exposed multiple myeloma patients - a phase II study.

Multi-class upfront regimens for multiple myeloma (MM) have improved outcomes, yet many patients become either triple-class exposed (TCE) or refractory (TCR) early in their disease course. This is a phase II, investigator initiated, open-label, single-arm, prospective, multicenter study evaluating all-oral Ixazomib-pomalidomide- dexamethasone (IPd) regimen for TCE RRMM patients. Sixty-one patients were enrolled between March 1st 2021 and March 17th 2024. The median age was 74 (range: 53-89) years; 25 (41%) were ≥75 years old. Fiftyseven percent had high-risk cytogenetics (t(4;14), t(14;16), +1

Аутоиммунные заболевания и инфекция микобактерии.

Микобактиальные инфекции и аутоиммунные заболевания затрагивают многих людей во всем мире, и все больше данных свидетельствует о наличии двунаправленных связей. В настоящем обзоре рассматриваются механизмы, с помощью которых различные аутоиммунные заболевания предрасположены пациентов к микобактическим инфекциям, и наоборот. Мы провели поиск PubMed/MedLINE с использованием ключевых слов "mycobacterium" и названий аутоиммунных условий для идентификации соответствующих документов. РЕЗУЛЬТАТЫ: Ремуатоидальная артритная терапия, особенно ингибиторы TNF-α, повышает риск туберкулеза (ТБ) и нетуберку

Воздействие Medial Oivocochlear Efferents на дискриминацию речи в шуме при множественном склерозе.

Психиатр с множественным склерозом (МС) сталкивается с трудностями в понимании речи в шуме, несмотря на нормальный слух. АИМ: Цель этого исследования состояла в том, чтобы определить взаимосвязь между уровнем звуковой дискриминации в шуме (SDN) и средним уровнем рефлекса оливокохлеара, а также сравнить пациентов, страдающих MS, с контрольной группой. МАТЕРИАЛЫ И МЕТОДЫ: Sixty участников с обычными слушаниями, включая 30 Психиатрические заболевания и 30 Включены меры контроля за состоянием здоровья. Для обеих групп на частотах выбросов из продуктов, вызывающих отоакустическое искажение (DPAE),

The unusual first sign of presentation of hepatocellular carcinoma: a rare case report.

BACKGROUND: Hepatocellular carcinoma (HCC) is the most common primary liver malignancy, characterized by aggressive biological behavior and a high propensity for extrahepatic spread. The most common sites of metastasis are the lungs, regional lymph nodes, bones, adrenal glands, and peritoneum. In contrast, the paranasal sinuses are an unusual site for metastatic HCC, and involvement of the sphenoid sinus represents an even rarer event. CASE DESCRIPTION: We report the case of a 67-year-old male patient who presented with transient diplopia, left eyelid ptosis, and refractory epistaxis as the i

Clinical characteristics and prognostic factors of sepsis in patients with malignancy.

Sepsis is a severe complication in patients with malignant tumors, leading to high mortality and increased need for intensive care. This study aimed to investigate the clinical characteristics and prognostic factors influencing sepsis outcomes in patients with malignant tumors. We included 4,858 patients with cancer diagnosed with sepsis between September 2019 and February 2020 whose data were collected from the Korean Sepsis Alliance, a nationwide multicenter cohort study. Cox regression analysis was used to identify predictors of 30-day and in-hospital mortality. In total, 65% of the patient

Коэффициент успеха зубоврачебных имплантантов у пациентов с аутоиммунными расстройствами: систематический анализ и мета-анализ.

АймАутоиммунные расстройства могут повлиять на метаболизм костей и заживление тканей, что может повлиять на исход зубного имплантата. Цель этого систематического обзора и метаанализа состояла в том, чтобы оценить выживаемость и успех пациентов с аутоиммунными заболеваниями с уделением особого внимания предельным потерям костей и кровотечению в ходе обследования (ПБ). seven базы данных (PubMed, MedLINE, Embase, Scopus, Web of Science, Cochrane Bibrary и CINAHL) проводились в соответствии с предпочтительными руководящими принципами, касающимися представления докладов по темам для системных обзор

Cost-effectiveness evidence on approved cancer drugs in Ireland: the limits of data availability and implications for public accountability.

BackgroundWe surveyed evidence published by Ireland's National Centre for Pharmacoeconomics (NCPE) on the cost-effectiveness of cancer drugs approved for funding within the Irish public healthcare system. The purpose is threefold: to assess the completeness and clarity of publicly available cost-effectiveness data of such therapies; to provide summary estimates of that data; to consider the implications of constraints on data availability for accountability regarding healthcare resource allocation.MethodsThe National Cancer Control Programme lists 91 drug-indication pairs approved between June

B-cell targeted therapeutics in clinical development.

B lymphocytes are the source of humoral immunity and are thus a critical component of the adaptive immune system. However, B cells can also be pathogenic and the origin of disease. Deregulated B-cell function has been implicated in several autoimmune diseases, including systemic lupus erythematosus, rheumatoid arthritis, and multiple sclerosis. B cells contribute to pathological immune responses through the secretion of cytokines, costimulation of T cells, antigen presentation, and the production of autoantibodies. DNA-and RNA-containing immune complexes can also induce the production of type

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… In addition, mutations in CD40L have been identified in a subset of patients with common variable immunodeficiency, which is characterized by a defect in B-cell differentiation and hypogammaglobulinemia [ 193 ]. …