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Gene therapy: principles, challenges and use in clinical practice.

INTRODUCTION: Gene therapy is an emerging topic in medicine. The first products have already been licensed in the European Union for the treatment of immune deficiency, spinal muscular atrophy, hemophilia, retinal dystrophy, a rare neurotransmitter disorder and some hematological cancers, while many more are being assessed in preclinical and clinical trials. OBJECTIVE: The purpose of this review is to provide an overview of the core principles of gene therapy along with information on challenges and risks. Benefits, adverse effects and potential risks are illustrated based on the examples of

Comparison of recognition of symptom burden in MPN between patient- and physician-reported assessment - an intraindividual analysis by the German Study Group for MPN (GSG-MPN).

Myeloproliferative neoplasms (MPN) are associated with a variety of symptoms that severely impact patients' quality of life and ability to perform daily activities. Recent studies showed differences in the perception of physician- versus patient-reported symptom burden. However, studies directly comparing patient- and physician-reported ratings are lacking. Here, a retrospective analysis on symptom burden of 3979 MPN patients of the Bioregistry of the German Study Group for MPN was conducted to intra-individually compare physician and patient reports collected at the same time. Cohen's kappa w

Search for novel biomarkers of febrile neutropenia in hematological patients: an overview.

A single-center prospective study was conducted at a tertiary care hospital in Kuopio Finland over the past two decades, searching for novel biomarker candidates to predict complicated course of febrile neutropenia (FN) in hematological patients. In Dec 2006‒Dec 2015, 85 patients with acute myeloid leukemia and 179 autologous hematopoietic stem cell transplant recipients were included. More than 20 biomarkers were evaluated in subsequent patient cohorts and compared to C-reactive protein (CRP) and procalcitonin (PCT). The samples were taken on d0-d2 from the beginning of FN. Here, we provide a

The Role of Natural Killer Cells as a Platform for Immunotherapy in Pediatric Cancers.

Purpose of reviewWe aim to review the most recent findings in the use of NK cells in childhood cancers.Recent findingsNatural killer cells are cytotoxic to tumor cells. In pediatric leukemias, adoptive transfer of NK cells can bridge children not in remission to transplant. Interleukins (IL2, IL15) can enhance NK cell function. NK cell-CAR therapy has advantages of shorter life span that lessens chronic toxicities, lower risk of graft versus host disease when using allogeneic cells, ability of NK cells to recognize tumor cells that have downregulated MHC to escape T cells, and possibly less li

Oncological emergencies associated with gastrointestinal tumors.

Oncological emergencies are defined as acute life-threatening conditions in cancer patients either as a result of the malignancy or as a result of its treatment. In this review, we focus on oncological emergencies associated with gastrointestinal tumors. They can be categorized by their system of origin as hematologic, neurologic or metabolic. Furthermore, we discuss mechanical emergencies such as intestinal obstruction and vena cava superior syndrome as well as acute gastrointestinal bleeding and pulmonary embolism. The patients' performance status as well as prognosis are essential during de

Профилактика и лечение венозного тромбоэмболия у пациентов с множественной миеломой: руководящие принципы клинической практики от имени Европейской сети миеломы.

Венозный тромбоэмболизм (VTE) является частым осложнением у пациентов с множественной миеломой (MM), что приводит к повышению заболеваемости, смертности, прерыванию лечения и снижению качества жизни. Основные рекомендации включают следующее: Информирование пациентов о риске ВТЕ и признаках/симптомах тромбоза глубокой вены и легочной эмболии. Специализированная помощь. - создание специализированного диагностического и лечебного механизма для профилактики и лечения ВТЕ в амбулаторных условиях. Оценка рисков. Регулярная оценка риска VTE с использованием подтвержденных оценок. Первичная тромбопроф

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… For patients with severe thrombocytopenia (<50 × 10 9 /L), if there is a high risk of thrombosis progression, full‐dose anticoagulation with platelet transfusion support can be administered, or for lower risk cases, intermediate or prophylactic dose LMWH is an option. …

A phase I dose-escalation study of lenalidomide in combination with gemcitabine in patients with advanced pancreatic cancer.

PURPOSE: Lenalidomide have both immunomodulatory and anti-angiogenic properties which could confer anti-cancer effects. The aim of this study was to assess the feasibility of combining lenalidomide with the standard treatment gemcitabine in pancreatic cancer patients with advanced disease. PATIENTS AND METHODS: Eligible patients had locally advanced or metastatic adenocarcinoma of the pancreas. Patients received lenalidomide days 1-21 orally and gemcitabine 1000 mg/m2 intravenously (days 1, 8 and 15), each 28 day cycle. Three cohorts of lenalidomide were examined (Cohort I = 15 mg, Cohort II

2023 Scientific Session of the Society of American Gastrointestinal and Endoscopic Surgeons (SAGES), Montréal, Canada, 19 March–April 1 2023: Posters

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The Mechanisms of Altered Blood-Brain Barrier Permeability in CD19 CAR T-Cell Recipients.

Cluster of differentiation 19 (CD19) chimeric antigen receptor (CAR) T cells are a highly effective immunotherapy for relapsed and refractory B-cell malignancies, but their utility can be limited by the development of immune effector cell-associated neurotoxicity syndrome (ICANS). The recent discovery of CD19 expression on the pericytes in the blood-brain barrier (BBB) suggests an important off-target mechanism for ICANS development. In addition, the release of systemic cytokines stimulated by the engagement of CD19 with the CAR T cells can cause endothelial activation and decreased expression

Impact of payment source, referral site, and place of residence on outcomes after allogeneic transplantation in Mexico.

BackgroundThe impact of social determinants of health in allogeneic transplant recipients in low- and middle-income countries is poorly described. This observational study analyzes the impact of place of residence, referring institution, and transplant cost coverage (out-of-pocket vs government-funded vs private insurance) on outcomes after allogeneic hematopoietic stem cell transplantation (alloHSCT) in two of Mexico's largest public and private institutions.AimTo evaluate the impact of social determinants of health and their relationship with outcomes among allogeneic transplant recipients i

Evaluation of the Incidence and Stage of Oral Mucositis in Patients Undergoing Hematopoietic Stem Cell Transplantation: A Retrospective Study.

AimThis study aims to retrospectively evaluate the incidence and stage of oral mucositis in patients undergoing hematopoietic stem cell transplantation.MethodsA total of 102 patient records of patients hospitalized between 2014 and 2019 in the adult hematopoietic stem cell transplantation clinic of a tertiary university hospital in Turkey were evaluated. Data were collected through a retrospective evaluation of patient records. Records made according to the WHO Oral Toxicity Scale included in the patient records during hospitalization in the adult hematopoietic stem cell transplantation clinic

Prognostic Factor Analysis of Overall Survival in Gastric Cancer from Two Phase III Studies of Second-line Ramucirumab (REGARD and RAINBOW) Using Pooled Patient Data.

PurposeTo identify baseline prognostic factors for survival in patients with disease progression, during or after chemotherapy for the treatment of advanced gastric or gastroesophageal junction (GEJ) cancer.Materials and methodsWe pooled data from patients randomized between 2009 and 2012 in 2 phase III, global double-blind studies of ramucirumab for the treatment of advanced gastric or GEJ adenocarcinoma following disease progression on first-line platinum- and/or fluoropyrimidine-containing therapy (REGARD and RAINBOW). Forty-one key baseline clinical and laboratory factors common in both st

Circulating biosignatures in multiple myeloma and their role in multidrug resistance.

A major obstacle to chemotherapeutic success in cancer treatment is the development of drug resistance. This occurs when a tumour fails to reduce in size after treatment or when there is clinical relapse after an initial positive response to treatment. A unique and serious type of resistance is multidrug resistance (MDR). MDR causes the simultaneous cross resistance to unrelated drugs used in chemotherapy. MDR can be acquired through genetic alterations following drug exposure, or as discovered by us, through alternative pathways mediated by the transfer of functional MDR proteins and nucleic

Bone Marrow Aplasia after CAR-T-Cell Therapy for Relapsed/Refractory Burkitt's Lymphoma.

Chimeric antigen receptor T-cells (CAR-T) are now a standard approach for treating relapsed/refractory B-cell lymphomas. Immune effector cell-associated hemophagocytic lymphohistiocytosis-like syndrome (IEC-HS) is a newly described entity that can manifest following CAR-T. Bone marrow (BM) aplasia is an uncommon manifestation of IEC-HS reported after CAR-T-cell therapy and is defined as the reduction or absence of hematopoietic progenitor cells resulting in severe pancytopenia. We describe the case of a 44-year-old female with relapsed/refractory Burkitt lymphoma (BL) who received treatment wi

Benefits of Chimeric Antigen Receptor T-Cell Therapy for B-Cell Lymphoma.

Objective: The aim was to study the benefits and risks of anti-CD19 chimeric antigen receptor (CAR) T-cells in adults with B-cell lymphoma. Methods: From October 2015 to October 2021, we treated five patients with B-cell lymphoma, comprising two with mantle cell lymphoma, one case of Burkitt lymphoma, one case of diffuse large B-cell lymphoma, and one case of chronic lymphocytic leukemia/small lymphocytic lymphoma. The patients were given the FC regimen 5 days before the infusion of anti-CD19 CAR T-cells. The median total number of CAR T-cells infusions was 350*10^6 (88*10^6-585*10^6). Results

Gaucher disease: the hematologist's perspective of a multisystemic disorder.

Gaucher disease (GD) exemplifies how a single genetic mutation can give rise to a complex multisystem disorder with profound hematological implications. Central to its pathophysiology is the lysosomal accumulation of glucosylceramide due to deficient glucocerebrosidase activity, together with abnormal folding and trafficking of the enzyme that induce endoplasmic reticulum stress and cellular dysfunction. These processes disrupt reticuloendothelial homeostasis and interfere with hematopoiesis. As a consequence, macrophage activation and chronic inflammation contribute to the cytopenias, splenom

Impact of anti-T-lymphocyte globulin dosing on graft versus host disease in matched sibling peripheral blood stem cell transplantation.

Anti-T-lymphocyte globulin (ATLG) is commonly administered to reduce graft-versus-host disease (GVHD) in allogeneic stem cell transplantation (allo-SCT). However, the optimal ATLG dose in matched-sibling-donor (MSD) peripheral blood stem-cell transplantation (PBSCT) remains uncertain. We compared two ATLG doses (15 mg/kg vs. 30 mg/kg) in the MSD-PBSCT setting to assess allo-SCT outcomes. In this single-center retrospective study, we included 165 consecutive patients with hematologic malignancies who underwent MSD-PBSCT. Of these, 71 received 15 mg/kg ATLG (ATLG-15), and 94 received 30 mg/kg AT

Управление неблагоприятными событиями, связанными с комбинациями на основе помалидомида у пациентов с повторной/редактируемой множественной миеломой.

Многоагентные режимы с иммуномодуляционными (IMID) агентами, такими, как палидомид, линалидомид и помалидомид, стали предпочтительным стандартом лечения пациентов с множественной миеломой (MM), что позволило улучшить показатели выживания. В настоящее время существуют three Агенты IMID, утвержденные для лечения MM: талидомид, линалидомид и помалидомид. Леналидомид обычно используется для лечения пациентов с недавно поставленным диагнозом MM, а также для лечения после пересадки стволовых клеток или после рецидива заболевания. Помалидомид, в центре внимания которого находится данный обзор, утверж

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… Management with granulocyte colony-stimulating factor (GCSF) and/or platelet transfusions may be used; given Patient 1’s ANC nadir of 0.7 × 10 9 /L, GCSF supportive care during the first 1–2 cycles may be of particular benefit until Patient 1 achieved disease control. …

Phase 1 study of selinexor plus carfilzomib and dexamethasone for the treatment of relapsed/refractory multiple myeloma.

Selinexor, an oral Selective Inhibitor of Nuclear Export, targets Exportin 1 (XPO1, also termed CRM1). Non-clinical studies support combining selinexor with proteasome inhibitors (PIs) and corticosteroids to overcome resistance in relapsed/refractory multiple myeloma (RRMM). We conducted a phase I dose-escalation trial of twice-weekly selinexor in combination with carfilzomib and dexamethasone (SKd) to determine maximum tolerated dose in patients with RRMM (N = 21), with an expansion cohort to assess activity in carfilzomib-refractory disease and identify a recommended phase II dose (RP2D). Du

Recent advances in biomimetic nanodelivery systems for cancer Immunotherapy.

Tumor immunotherapy is a developing and promising therapeutic method. However, the mechanism of tumor immune microenvironment and individual differences of patients make the clinical application of immunotherapy still very limited. The resulting targeting of the tumor environment and immune system is a suitable strategy for tumor therapy. Biomimetic nanodelivery systems (BNDS) coated with nanoparticles has brought new hope for tumor immunotherapy. Due to its high targeting, maximum drug delivery efficiency and immune escape, BNDS has become one of the options for tumor immunotherapy in the fut