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Novel targeted treatments in hairy cell leukemia and other hairy cell-like disorders.

In the category of mature B-cell neoplasms, splenic B-cell lymphoma and leukemia were clearly identified and include four distinct entities: hairy cell leukemia (HCL), splenic marginal zone lymphoma (SMZL), splenic diffuse red pulp lymphoma (SDRPL) and the new entity named splenic B-cell lymphoma/leukemia with prominent nucleoli (SBLPN). The BRAFV600E mutation is detected in nearly all HCL cases and offers a possibility of targeted therapy. BRAF inhibitors (BRAFi) represent effective and promising therapeutic approaches in patients with relapsed/refractory HCL. Vemurafenib and dabrafenib were

Advances in Molecular Imaging and Radionuclide Therapy of Neuroendocrine Tumors.

Neuroendocrine neoplasms make up a heterogeneous group of tumors with inter-patient and intra-patient variabilities. Molecular imaging can h … he detectability of NET lesions via positron emission tomography (PET) imaging and improvements in pretreatment planning using dosimetry. Especially notable is PET imaging with the radionuclide Copper-64. Increasing interest is also being paid to theranostics of grade 3 and purely differentiated NETs, for example, via targeting of the C-X-C motif chemokine re …

Forgoing life-sustaining treatment - a comparative analysis of regulations in Japan, Korea, Taiwan, and England.

BackgroundRegulations on forgoing life-sustaining treatment (LST) have developed in Asian countries including Japan, Korea and Taiwan. However, other countries are relatively unaware of these due to the language barrier. This article aims to describe and compare the relevant regulatory frameworks, using the (more familiar) situation in England as a point of reference. We undertook literature reviews to ascertain the legal and regulatory positions on forgoing LST in Japan, Korea, Taiwan, and England.Main textFindings from a literature review are first presented to describe the development of th

Hematological Diseases and Osteoporosis.

Secondary osteoporosis is a common clinical problem faced by bone specialists, with a higher frequency in men than in women. One of several causes of secondary osteoporosis is hematological disease. There are numerous hematological diseases that can have a deleterious impact on bone health. In the literature, there is an abundance of evidence of bone involvement in patients affected by multiple myeloma, systemic mastocytosis, thalassemia, and hemophilia; some skeletal disorders are also reported in sickle cell disease. Recently, monoclonal gammopathy of undetermined significance appears to inc

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… Effects of teriparatide retreatment in a patient with β-thalassemia major Transfusion 2015 55 2905 2910 10.1111/trf.13237 26174236 113. Pinto V.M. Balocco M. Quintino S. Forni G.L. Sickle cell disease: A review for the internist Int. Emerg. …

Combination Strategies to Optimize Efficacy of Dendritic Cell-Based Immunotherapy.

Dendritic cells (DCs) are antigen-presenting cells (APCs) that are essential for the activation of immune responses. In various malignancies, these immunostimulatory properties are exploited by DC-therapy, aiming at the induction of effective anti-tumor immunity by vaccination with ex vivo antigen-loaded DCs. Depending on the type of DC-therapy used, long-term clinical efficacy upon DC-therapy remains restricted to a proportion of patients, likely due to lack of immunogenicity of tumor cells, presence of a stromal compartment, and the suppressive tumor microenvironment (TME), thereby leading t

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… Dendritic cell vaccination combined with temozolomide retreatment: results of a phase I trial in patients with recurrent glioblastoma multiforme. J Neurooncol. (2015) 121:319–29. 10.1007/s11060-014-1635-7 63. Pellegatta S, Eoli M, Cuccarini V, Anghileri E, Pollo B, Pessina S, et al. …

Light Chain Amyloidosis.

Light chain (AL) amyloidosis is caused by a usually small plasma-cell clone that is able to produce the amyloidogenic light chains. They are able to misfold and aggregate, deposit in tissues in the form of amyloid fibrils and lead to irreversible organ dysfunction and eventually death if treatment is late or ineffective. Cardiac damage is the most important prognostic determinant. The risk of dialysis is predicted by the severity of renal involvement, defined by the baseline proteinuria and glomerular filtration rate, and by the response to therapy. The specific treatment is chemotherapy targe

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… There is no consensus on criteria to start rescue therapy in relapsing subjects. 91 Cardiac progression should not be awaited, because it is associated with shorter survival. 90 Relapsing patients can be treated by repeating upfront therapy, if possible, although this is associated with shorter time to retreatment …

Предотвращение событий, связанных со скелетом, в новой диагностированной миеломе.

Несмотря на увеличение числа новых методов лечения недавно диагностированной множественной миеломы (НДММ), предотвращение событий, связанных со скелетом (СРП), по-прежнему является сложной задачей. В настоящем обзоре резюмируются механистические причины болезни костей миэломы, данные, подтверждающие использование бисфофонатов и ингибиторов РАНКЛ, а также оптимальное управление предотвращением НОС у пациентов НДММ. Как золедронная кислота (ZA), так и деносумаб являются приемлемыми вариантами обработки с сопоставимыми характеристиками безопасности и эффективности. Однако у пациентов, которые явл

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… . | Retreatment should be initiated at the time of disease relapse Figure 2 Treatment algorithm for bone-directed therapy in newly diagnosed multiple myeloma. Abbreviations: ASCT: autologous stem cell transplant; PI: proteosome inhibitor; VGPR: very good partial response; ZA: zoledronic acid. …

Targeting B Cell Maturation Antigen in Patients with Multiple Myeloma: Current Perspectives.

Relapsed/refractory multiple myeloma remains a challenging disease necessitating the development of more effective treatment options. In the past decade, myeloma therapies have made significant advancements with the introduction of new treatment modalities. One of the new major targets for these novel therapeutics has been B-cell maturation antigen (BCMA), which is expressed on mature B-lymphocytes and plasma cells. There are three main categories of BCMA-targeted therapies currently available, including bispecific antibodies (BsAbs), antibody drug conjugates (ADCs), and chimeric antigen recep

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… Effective anti-BCMA retreatment in multiple myeloma. Blood Adv . 2021;5(15):3016–3020. doi: 10.1182/bloodadvances.2021004176 110. Xu J, Chen LJ, Yang SS, et al. Exploratory trial of a biepitopic CAR T-targeting B cell maturation antigen in relapsed/refractory multiple myeloma. …

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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… The ORR was 51% (17.8% CR) at the end of therapy and 31% at 12 months with responses seen on retreatment ( 124 ). …

Monoclonal Antibodies: Leading Actors in the Relapsed/Refractory Multiple Myeloma Treatment.

Multiple myeloma is a complex hematologic malignancy, and despite a survival improvement related to the growing number of available therapeutic options since 2000s, it remains an incurable disease with most patients experiencing relapse. However, therapeutic options for this disease are constantly evolving and immunotherapy is becoming the mainstay of the therapeutic armamentarium of Multiple Myeloma (MM), starting with monoclonal antibodies (MoAbs) as elotuzumab, daratumumab and isatuximab. Elotuzumab, the first in class targeting SLAMF7, in combination with lenalidomide and dexamethasone and

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… An ongoing randomized phase II study ( NCT03871829 ; LYNX) will evaluate the efficacy and safety of retreatment with daratumumab sc in patients with RRMM previously exposed to daratumumab iv. In Table 2 we summarized the ongoing clinical studies with daratumumab in RRMM. …

Burden of Disease, Treatment Patterns, and Healthcare Resource Utilization in Multiple Myeloma in the Middle East and North Africa Region: A Targeted Literature Review.

Globally, the multiple myeloma (MM) landscape varies widely, with incidence, treatment patterns, outcomes, and healthcare resource utilization (HCRU) differing across countries and regions. Many factors contribute to these differences, including ethnicity, awareness, and socioeconomic influences. This is the first analysis aiming to explore factors associated with myeloma incidence and patient characteristics in the Middle East and North Africa (MENA) region. A literature search was conducted using PubMed and Embase from 1 January 2015 to 30 May 2025. Articles reporting outcomes of interest in

Нейропсихологические аспекты расстройств контроля импульсов в болезни Паркинсона.

Синдромы импульсного контроля (ИДД), такие, как чрезмерные азартные игры, навязчивое сексуальное поведение, поедание бингов, навязчивые покупки, а также синдром регулирования дофамина, могут возникать как изнурительное нервно-психическое осложнение болезни Паркинсона (PD). Хотя патофизиология не в полной мере понятна, она, вероятно, включает мезолимбичную допаминергическую чрезмерную оптимизацию в сочетании с факторами уязвимости, связанными с заболеваниями, в плане поощрения, мотивации и ингибиционных сетей контроля. В настоящем описательном обзоре резюмируются данные о нейропсихологических,

Пересмотр подходов к реабилитации и образу жизни для повышения качества жизни людей с множественным склерозом: обзор скопирования.

Фон: Множественный склероз (МС) связан со сложными физическими, когнитивными и поведенческими проблемами, которые в совокупности влияют на качество жизни. Включение таких компонентов образа жизни, как оптимизация сна, поведение в рационе питания, борьба со стрессом и стратегии саморегулирования, в процесс реабилитации может способствовать достижению результатов, выходящих за рамки традиционных подходов. Целью этого аналитического обзора было картирование реабилитационных мероприятий, которые сочетают в себе психомоторные, когнитивные, ориентированные на образ жизни или мультимодальные элементы

The influence of targeted therapies in the inner ear.

Scientific research has significantly propelled advancements in healthcare. One notable application is precision medicine, which seeks to analyze and comprehend disease pathology to offer personalized medical treatments to patients. Targeted oncology, a branch of precision medicine, focuses on identifying and targeting specific molecules that regulate cancer cells, thereby minimizing harm to healthy cells. Different types of targeted therapy against cancer include monoclonal antibodies and small molecules. This manuscript intends to provide an overview of the influence of these targeted oncolo

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… Velcade (Bortezomib) receives 2 new FDA indications: for retreatment of patients with multiple myeloma and for first-line treatment of patients with mantle-cell lymphoma. Am. health and drug benefits 8 (Spec Feature), 135–140. Rajapakse A., O'Leary C., Gundelach R., Deva R., O'Byrne K. (2020). …

Chimeric Antigen Receptor T-Cell Revolution Remodeling Immunity to Conquer Autoimmune Disease.

Chimeric antigen receptor T (CAR-T) cell therapy, a groundbreaking technology in tumor immunotherapy, has demonstrated unprecedented potential in the field of autoimmune diseases in recent years. This article provides a systematic review of the developmental trajectory and core concepts of CAR-T-cell therapy in autoimmune diseases, emphasizing its conceptual evolution from traditional "killing" strategies to "precision immune remodeling". Leveraging multitarget approaches (e.g., CD19 and B-cell maturation antigen) and chimeric autoantigen receptor technology, it achieves efficient elimination

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… In the study by Wang et al. [ 13 ], a patient who received a subtherapeutic dose of CAR-T cells experienced transient remission but failed to sustain control of autoantibodies, with recurrence even after full-dose retreatment, highlighting the importance of sufficient initial expansion. …

Novel Immunotherapies for Myasthenia Gravis.

Myasthenia gravis (MG), a prototype autoimmune neurological disease, had its therapy centred on corticosteroids, non-steroidal broad-spectrum immunotherapy and cholinesterase inhibitors for several decades. Treatment-refractory MG and long-term toxicities of the medications have been major concerns with the conventional therapies. Advances in the immunology and pathogenesis of MG have ushered in an era of newer therapies which are more specific and efficacious. Complement inhibitors and neonatal Fc receptor blockers target disease-specific pathogenic mechanisms linked to myasthenia and have pr

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… 2 months or 750 mg/m 2 every 2 weeks for 1 month | All patients showed marked improvement at the end of follow up of 22.6 months Hehir et al, 2017 74 | Prospective review, multicentric | 55 MuSK+ MG; 24 treated with Rituximab and 31 with no exposure to rituximab | 4 weekly doses of 375 mg/m 2 with retreatment …

Chinese expert consensus recommendations for the administration of immune checkpoint inhibitors to special cancer patient populations.

Immune checkpoint inhibitors (ICIs) targeting programmed cell death 1, programmed cell death ligand 1, and cytotoxic T lymphocyte-associated antigen-4 have shown significantly durable clinical benefits and tolerable toxicities and have improved the survival of patients with various types of cancer. Since 2018, the National Medical Products Administration of China has approved 17 ICIs as the standard treatment for certain advanced or metastatic solid tumors. As ICIs represent a broad-spectrum antitumor strategy, the populations eligible for cancer immunotherapy are rapidly expanding. However, t

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… Moreover, this consensus does not cover all special challenging patient populations, such as those with epidermal growth-factor receptor-positive NSCLC, those with ICI rechallenge or retreatment, and those with special organ metastases. …

Current status and updated recommendations for diagnosis and treatment of plasma cell myeloma in Switzerland.

The availability of drugs such as thalidomide, bortezomib and lenalidomide changed the landscape in myeloma treatment and has extended the median survival up to 10 years with a substantial improvement in quality of life. This development prompted a Swiss expert panel to re-evaluate the current status and formulate updated clinical recommendations for the diagnosis and treatment of plasma cell myeloma. These recommendations should help clinicians in their decision making to achieve the best outcome based on currently available data.

From toxicity to immunity: Evaluating CAR-T and conventional therapies in multiple myeloma through quality-adjusted life year (QALY) outcomes.

Multiple myeloma (MM) remains characterized by recurrent relapse and cumulative treatment burden despite major therapeutic advances. This study compared conventional stepwise regimens with CAR-T cell therapy in terms of QoL, toxicity, cost, and ethical considerations within a model-based comparative framework. This comparative pharmacoeconomic modeling analysis was based on published clinical trial data. Kaplan-Meier survival curves were digitized to estimate mean overall survival (OS) and progression-free survival (PFS) using area-under-the-curve integration. Treatment costs were calculated b

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… Approaches To Managing Relapsed Myeloma: Switching Drug Class or Retreatment With Same Drug Class? Indian J Hematol Blood Transfus 2025; 41: 478–493. 10.1007/s12288-025-02008-9 46. Medina-Herrera A, Sarasquete ME, Jiménez C, et al. …

Clinical impact of checkpoint inhibitors as novel cancer therapies.

Immune responses are tightly regulated via signaling through numerous co-stimulatory and co-inhibitory molecules. Exploitation of these immune checkpoint pathways is one of the mechanisms by which tumors evade and/or escape the immune system. A growing understanding of the biology of immune checkpoints and tumor immunology has led to the development of monoclonal antibodies designed to target co-stimulatory and co-inhibitory molecules in order to re-engage the immune system and restore antitumor immune responses. Anti-cytotoxic T-lymphocyte-associated antigen 4 (CTLA-4) antibodies were among t

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… 2015/Dec 2016 | NCT01285609 Melanoma Ipilimumab | I/II | Uveal melanoma | Recruiting ( n = 141) | Primary: MTD Secondary: OS | Nov 2017 | NCT01585194 Ipilimumab + temozolomide | II | Metastatic melanoma | Ongoing ( n = 64) | Primary: 6-month PFS | May 2015 | NCT01119508 Ipilimumab vs. chemotherapy (retreatment …