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Catching Them Early: Framework Parameters and Progress for Prenatal and Childhood Application of Advanced Therapies.

Advanced therapy medicinal products (ATMPs) are medicines for human use based on genes, cells or tissue engineering. After clear successes in adults, the nascent technology now sees increasing pediatric application. For many still untreatable disorders with pre- or perinatal onset, timely intervention is simply indispensable; thus, prenatal and pediatric applications of ATMPs hold great promise for curative treatments. Moreover, for most inherited disorders, early ATMP application may substantially improve efficiency, economy and accessibility compared with application in adults. Vindicating t

Septic shock in the immunocompromised cancer patient: a narrative review.

Immunosuppressed patients, particularly those with cancer, represent a momentous and increasing portion of the population, especially as cancer incidence rises with population growth and aging. These patients are at a heightened risk of developing severe infections, including sepsis and septic shock, due to multiple immunologic defects such as neutropenia, lymphopenia, and T and B-cell impairment. The diverse and complex nature of these immunologic profiles, compounded by the concomitant use of immunosuppressive therapies (e.g., corticosteroids, cytotoxic drugs, and immunotherapy), superimpose

Plasma Cell Disorders

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Idiotypic DNA vaccination for the treatment of multiple myeloma: safety and immunogenicity in a phase I clinical study.

We report on the safety and immunogenicity of idiotypic DNA vaccination in a phase I, non-randomised, open-label study in patients with multiple myeloma. The study used DNA fusion gene vaccines encoding patient-specific single chain variable fragment, or idiotype (Id), linked to fragment C (FrC) of tetanus toxin. Patients in complete or partial response following high-dose chemotherapy and autologous stem cell transplant were vaccinated intramuscularly with 1 mg DNA on six occasions, beginning at least 6 months post-transplant; follow-up was to week 52. Fourteen patients were enrolled on study

Acute kidney injury in patients with newly diagnosed high-grade hematological malignancies: impact on remission and survival.

BackgroundOptimal chemotherapy with minimal toxicity is the main determinant of complete remission in patients with newly diagnosed hematological malignancies. Acute organ dysfunctions may impair the patient's ability to receive optimal chemotherapy.Design and methodsTo compare 6-month complete remission rates in patients with and without acute kidney injury (AKI), we collected prospective data on 200 patients with newly diagnosed high-grade malignancies (non-Hodgkin lymphoma, 53.5%; acute myeloid leukemia, 29%; acute lymphoblastic leukemia, 11.5%; and Hodgkin disease, 6%).ResultsAccording to

Long-term treatment outcome of Castleman's disease: A real-world experience.

BACKGROUND: Castleman disease (CD), classified as unicentric CD (UCD) or multicentric CD (MCD), is a rare non-neoplastic lymphoproliferative disorder of unknown origin. Owing to its rarity, the clinical characteristics, therapeutic modalities, treatment outcomes, and prognostic factors related to UCD or MCD are not well defined. METHOD: We retrospectively analyzed 88 patients with CD, including those with hyaline-vascular, plasma-cell, mixed type, hypervascular, and plasmablastic subtypes, for presenting symptoms, physical, laboratory, and radiologic findings, and treatment response in the Ko

A cost-utility and budget impact analysis of allogeneic hematopoietic stem cell transplantation for severe thalassemic patients in Thailand.

BackgroundHematopoietic stem cell transplantation (HSCT) is the only curative treatment available to severe thalassemic patients. The treatment, however, is very costly, particularly in the context of low and middle income countries, and no studies have been carried out to explore its economic justifiability. This study aimed to estimate the cost-utility of HSCT compared with blood transfusions combined with iron chelating therapy (BT-ICT) for severe thalassemia in Thailand, and to investigate the affordability of HSCT using a budget impact analysis.MethodsA Markov model was used to estimate t

Ассоциация между диагностической историей и заболеваемостью раком 5 Годы: Реальный анализ наблюдений.

НЕЗАВИСИМЫЙ: Рак связан со многими ранее существовавшими заболеваниями, однако точное количественное определение этих связей по-прежнему сопряжено с трудностями. Хотя в некоторых исследованиях эти ассоциации были изучены, широкомасштабный анализ с использованием различных электронных данных о состоянии здоровья (ЭПЧ) по-прежнему носит ограниченный характер и не позволяет оценить риск заболевания раком в тех случаях, когда пациенты стратифицированы в результате межличностных различий. Использование реального набора данных по ЭПЧ из крупной системы здравоохранения Луизианы, включающей 8,283,236

Twenty-five years of type I interferon-based treatment: a critical analysis of its therapeutic use.

The clinical exploitation of type I interferon (IFN) as an antiviral and antineoplastic agent is based on the properties originally attributed to this cytokine family, with schedules reflecting only their antiviral and antiproliferative activities. Nevertheless, type I IFN has emerged as a central activator of the innate immunity. As current schedules of treatment for chronic hepatitis C and for hematological and solid tumors, based on the continuous administration of recombinant type I IFN or pegylated formulations, disregard viral resistance, host genetic variants predicting treatment outcom

Review Article: Immune Effector Cell-Mediated Enterocolitis Following CAR-T Cell Therapy-Clinical Features, Pathophysiology and Management.

BACKGROUND: Chimeric antigen receptor T-cell (CAR-T) therapy has revolutionised the treatment of hematologic malignancies, and its use is expanding rapidly into numerous other disease states including autoimmune diseases. However, CAR-T therapy is associated with a spectrum of immune-related toxicities. In addition to the already well characterized cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome, it has become apparent that rarely, CAR-T can cause gastrointestinal mucosal inflammation, termed immune effector cell-mediated enterocolitis (IEC-EC). AIMS: This

Совпадение в полном тексте источника
… This physiology is akin to common variable immunodeficiency‐associated enteropathy. The depletion of mucosal IgA may explain the high co‐occurrence of gastrointestinal infections in IEC‐EC which has been demonstrated [ 26 , 27 ]. … Histopathologic findings, while variable, include villous atrophy, intraepithelial lymphocytosis, and crypt apoptosis, a constellation that overlaps with several other conditions including coeliac disease, graft‐versus‐host disease, common variable immunodeficiency, medication‐induced enteropathy, and …

Spectrum of Pancytopenia in Adults Attending a Clinical Hematology Department: A Four-Year Experience From a Tertiary Care Center of Western India.

Background Pancytopenia is a common hematological condition encountered in clinical practice. Because there is a wide variation in causes of pancytopenia even in different populations of the same geographical region, identifying the pattern of underlying etiologies is crucial for proper management. This study was conducted to evaluate the clinico-hematological profile and different etiologies of pancytopenia among adults presenting to a clinical hematology department at a tertiary care hospital of Western India. Methodology This observational study was conducted over a period of four years on

Combating challenges in CAR-T cells with engineering immunology.

Chimeric antigen receptors (CAR) T cells (CAR-T) mark a significant step towards producing safe and effective personal anticancer treatments. CAR-T strategies engineers the T cells from the patients to allow specific binding to a tumour-specific antigen. CAR-Ts are a second-wave offensive strategy to clear out remaining chemotherapy-resistant tumour cells. Though showing practical antitumor abilities in multiple haematological malignancies and solid tumour cancers, the issues of antigen escape, tumour infiltration/penetration, and toxicity side effects limit the usage of prolonged CAR-T therap

KSHV and cancer: understanding the oncogenic machinery for next-generation diagnostic tools and therapies.

Kaposi's sarcoma-associated herpesvirus (KSHV), or human herpesvirus 8 (HHV-8), is an oncogenic virus responsible for Kaposi's sarcoma (KS) and lymphoproliferative disorders like primary effusion lymphoma (PEL) and multicentric Castleman disease (MCD). This review explores KSHV's oncogenic mechanisms, focusing on its ability to manipulate host cell signaling, evade immune detection, and promote tumorigenesis through latent and lytic viral proteins. Key oncoproteins, such as LANA, vCyc, vFLIP, and vGPCR, activate cancer hallmarks, as sustained proliferation, immune evasion, angiogenesis, and re

A Pathology Experience of Posttransplant Lymphoproliferative Disorder From One Tertiary Hospital: Pathology Concepts and Diagnostic Approach.

BACKGROUND: Solid organ transplantation and bone marrow/hematologic stem cell transplantation recipients face a heightened risk of developing malignancies or cancer as a result of immunosuppression. Posttransplant lymphoproliferative disorders (PTLD) are a range of disorders from benign lymphoid growth to lymphoma found post-transplant. Risk factors for PTLD include high immunosuppressive use and oncogenic effects of Epstein-Barr virus (EBV). There is a lack of comprehensive clinical and pathological documentation of PTLD cases among Saudi patients, and the available data are limited to a few

Advancing gene editing therapeutics: Clinical trials and innovative delivery systems across diverse diseases.

Gene editing is a groundbreaking therapeutic approach that can potentially treat a broad spectrum of genetic and acquired diseases. This review highlights recent clinical trials employing advanced gene editing technologies such as CRISPR-Cas9, zinc-finger nucleases (ZFNs), and base editors across multiple disease areas including metabolic disorders, autoimmune diseases, muscular dystrophies, and inherited eye disorders. Central to the success of these therapies is the development of efficient and safe delivery systems, including lipid nanoparticles (LNPs), viral vectors (adenoviral and lentivi

Extranodal diffuse large B-cell lymphoma with monoclonal gammopathy: an aggressive and primary refractory disease responding to an immunomodulatory agent.

BackgroundAlthough the clinical outcome of patients with diffuse large B cell lymphoma (DLBCL) has been improved by the addition of rituximab to standard chemotherapy, almost one-third fails or relapses after first line treatment. The presence of monoclonal gammopathy (MG) is a known adverse prognostic factor for DLBCL. Because this subset of patients does not benefit from R-CHOP, new therapeutic options are required. Herein, we report the first case of extranodal DBCL of the lung with a concomitant MG who achieved a long lasting complete remission with lenalidomide.Case presentationThe 73-yea

Смертность, связанная с инфекцией, после трансплантации стволовых клеток в условиях высокой антимикробной устойчивости.

: Инфекционные осложнения остаются одной из основных причин смертности после трансплантации стволовых клеток (ГХЦТ). Мы поставили перед собой цель описать инфекцию после ХСБТ и ее воздействие на выживание в центре региона с высоким антимикробным сопротивлением. МЕТОДЫ: В том числе 522 больные гематологическими расстройствами, проходящими через СХТ в университетском госпитале Коч, между 2016 и 2024. Кровоток и оппортунистические инфекции внутри 100 были проанализированы дни. Смертность, связанная с инфекцией, была определена как смерть, обусловленная главным образом инфекционными осложнениями.

Parvovirus B19-induced hemophagocytic lymphohistiocytosis: Case report and review of the literature.

HLH is a catastrophic and likely underdiagnosed pathology with multiple triggers including infection. PVB19 can cause persistent marrow infection leading to HLH despite negative acute serologic markers making timely diagnosis difficult. Increased awareness of PVB19-HLH is warranted given its potentially lethal nature and the careful interpretation required with serologic markers.

ImmunoPET in Multiple Myeloma-What? So What? Now What?

Despite constant progress over the past three decades, multiple myeloma (MM) is still an incurable disease, and the identification of new biomarkers to better select patients and adapt therapy is more relevant than ever. Recently, the introduction of therapeutic monoclonal antibodies (mAbs) (including direct-targeting mAbs and immune checkpoint inhibitors) appears to have changed the paradigm of MM management, emphasizing the opportunity to cure MM patients through an immunotherapeutic approach. In this context, immuno-positron emission tomography (immunoPET), combining the high sensitivity an

Monoclonal Gammopathy of Undetermined Significance (MGUS)-Not So Asymptomatic after All.

Monoclonal Gammopathy of Undetermined Significance (MGUS) is considered to be a benign precursor condition that may progress to a lymphoproliferative disease or multiple myeloma. Most patients do not progress to an overt condition, but nevertheless, MGUS is associated with a shortened life expectancy and, in a minority of cases, a number of co-morbid conditions that include an increased fracture risk, renal impairment, peripheral neuropathy, secondary immunodeficiency, and cardiovascular disease. This review aims to consolidate current evidence for the significance of these co-morbidities befo