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Drug Treatment of Neuromyelitis Optica Spectrum Disorders: Out with the Old, in with the New?

IntroductionNeuromyelitis optica spectrum disorders (NMOSD) are rare neuroinflammatory demyelinating diseases of the CNS, mainly affecting optic nerves, spinal cord and brainstem regions. The diagnosis depends on clinical symptoms, MRI findings and the detection of autoantibodies against the water channel aquaporin 4 (AQP4-Ab). This autoantibody is particularly important for diagnostic sensitivity and specificity and further sets the course for major therapeutic decisions. Due to a relapsing course with the accumulation of disability, relapse prevention by immunotherapy is crucial in NMOSD. Un

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… Non-inferiority to eculizumab has been proven by two large phase III trials in patients with paroxysmal nocturnal hemoglobinuria (PNH). 70 , 71 Based on these findings, ravulizumab was approved by the FDA and EMA for the use in adult PNH in 2018 and 2019. 72 In December 2019, a phase 3 open-label study …

Evans syndrome: clinical perspectives, biological insights and treatment modalities.

Evans syndrome (ES) is a rare and chronic autoimmune disease characterized by autoimmune hemolytic anemia and immune thrombocytopenic purpura with a positive direct anti-human globulin test. It is classified as primary and secondary, with the frequency in patients with autoimmune hemolytic anemia being 37%-73%. It predominates in children, mainly due to primary immunodeficiencies or autoimmune lymphoproliferative syndrome. ES during pregnancy is associated with high fetal morbidity, including severe hemolysis and intracranial bleeding with neurological sequelae and death. The clinical presenta

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… kidney impairment; 14 Kasabach–Merritt syndrome, a rare disease predominant in early infancy with a high mortality rate of about 30% characterized by microangiopathic hemolytic anemia, thrombocytopenia, low fibrinogen levels, increment of D-dimer and giant hemangioma; 15 , 16 and paroxysmal nocturnal hemoglobinuria …

FDA-Approved Drugs Containing Amide Functionality in the Last Five Years (2021-2025): Pharmaceutical Use, Trends and Synthetic Approaches.

The amide functional group remains a cornerstone of medicinal chemistry, serving as an indispensable scaffold in the design of modern therapeutics. This review presents an analysis of FDA-approved drugs (small molecules and peptides with MW ®), amides form the structural backbone of peptidomimetics, enabling high-affinity binding to a viral protease. In precision oncology, as seen with adagrasib (Krazati®), the amide acts as a critical, metabolically stable linker that positions a covalent warhead for selective inhibition of a mutant kinase. This analysis underscores that amide's unique combin

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… Danicopan Approved on 29 March 2024, danicopan ( 71 , brand name Voydeya ® , Figure 55 a) is an add-on treatment to C5 inhibitors for adults with paroxysmal nocturnal hemoglobinuria (PNH) who have persistent extravascular hemolysis. …

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

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… We previously identified a combination of the absence of minor paroxysmal nocturnal hemoglobinuria clones (PNH - ) and a short telomere length (sTL) as a strong predictor of poor IST response. … Clinical phenotype encompasses hemolytic anemia, bone marrow failure syndrome and thrombotic events, allowing for the recognition of two major entities: classical paroxysmal nocturnal hemoglobinuria (cPNH) and bone marrow failure associated paroxysmal nocturnal hemoglobinuria (BMF/PNH). …

Биология и болезни крыс

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Fate of hematopoietic stem cells determined by Notch1 signaling (Review).

Regulation of the fate of hematopoietic stem cells (HSCs), including silencing, self-renewal or differentiation into blood line cells, is crucial to maintain the homeostasis of the human blood system and prevent leukemia. Notch1, a key receptor in the Notch signaling pathway, plays an important regulatory role in these properties of HSCs, particularly in the maintenance of the stemness of HSCs. In recent decades, the ubiquitination modification of Notch1 has been gradually revealed, and also demonstrated to affect the proliferation and differentiation of HSCs. Therefore, a detailed elucidation

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… transplantation; MM, multiple myeloma; MDS, myeloproliferative disorders; NHL, non-Hodgkin's lymphoma; HL, Hodgkin's lymphoma; ALL, acute lymphoblastic leukemia; CLL, chronic lymphocytic leukemia; AML, acute myelogenous leukemia; CML, chronic myelogenous leukemia; AA, aplastic anemia; PNH, paroxysmal nocturnal hemoglobinuria …

Recent advances in use of fresh frozen plasma, cryoprecipitate, immunoglobulins, and clotting factors for transfusion support in patients with hematologic disease.

Hematologic diseases include a broad range of acquired and congenital disorders, many of which affect plasma proteins that control hemostasis and immune responses. Therapeutic interventions for these disorders include transfusion of plasma, cryoprecipitate, immunoglobulins, or convalescent plasma-containing therapeutic antibodies from patients recovering from infectious diseases, as well as concentrated pro- or anticoagulant factors. This review will focus on recent advances in the uses of plasma and its derivatives for patients with acquired and congenital hematologic disorders.

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… Hemoglobinuria and acute kidney injury requiring hemodialysis following intravenous immunoglobulin infusion. Am J Kidney Dis. 2010;55(1):148–151. doi: 10.1053/j.ajkd.2009.06.013. 86. Daw Z, Padmore R, Neurath D, et al. …

Искусственный интеллект, видоизменяющий парадигму гематологического диагноза и лечения злокачественных заболеваний: от статической оценки до динамического управления точностью.

Искусственный интеллект (ИИ) все чаще изучается в качестве инструмента содействия более точному и динамичному управлению диагностикой и лечением гематологов. В отличие от предыдущих обзоров, в которых основное внимание уделялось отдельным типам заболеваний или изолированным технологическим путям, в настоящем документе содержится всеобъемлющий обзор текущих прикладных программ АИШ и последних достижений в области диагностики, классификации, оценки прогнозов и принятия решений по лечению лейкемии, лимфомы, множественной миэломы и миэлодиспластических синдромов. Она охватывает ключевые технически

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… Its diagnosis should be distinguished from other bone marrow failure disorders with overlapping clinical manifestations, particularly aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH), which are distinct entities but may similarly present with peripheral cytopenias [ 94 ]. …

Экстренные ситуации в гематологии: Почему, когда и как я лечусь?

Гематологические чрезвычайные ситуации являются критическими медицинскими условиями, требующими неотложного внимания в силу их быстрого развития и опасного для жизни характера. В качестве примера можно привести гиперкальцемию, которая часто ассоциируется с раком, таким как множественная миелома, что может привести к серьезной неврологической и сердечной дисфункции. Гиперлейкоцитоз, распространенный в острой миэлоидной лейкемии, повышает риск лейкостаза и многоорганического отказа. Кислородный кризис, обычное осложнение серповидноклеточной болезни, является результатом вазооккуляции, приводящей

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… Laboratory features include a positive Coombs test, an increase in lactate dehydrogenase, indirect bilirubin, reduced haptoglobin, and hemoglobinuria. …

Aptamers and Antisense Oligonucleotides for Diagnosis and Treatment of Hematological Diseases.

Aptamers or chemical antibodies are single-stranded DNA or RNA oligonucleotides that bind proteins and small molecules with high affinity and specificity by recognizing tertiary or quaternary structures as antibodies. Aptamers can be easily produced in vitro through a process known as systemic evolution of ligands by exponential enrichment (SELEX) or a cell-based SELEX procedure. Aptamers and modified aptamers, such as slow, off-rate, modified aptamers (SOMAmers), can bind to target molecules with less polar and more hydrophobic interactions showing slower dissociation rates, higher stability,

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… malignancies such as leukemias, lymphomas, myeloma, and other myeloproliferative or lymphoproliferative disorders, as well as clonal and nonclonal benign diseases, such as some myelodysplastic syndromes, chronic granular lymphocyte disorders, monoclonal gammopathies, mastocytosis, paroxysmal nocturnal hemoglobinuria …

Unraveling the Role of Allo-Antibodies and Transplant Injury.

Alloimmunity driving rejection in the context of solid organ transplantation can be grossly divided into mechanisms predominantly driven by either T cell-mediated rejection (TCMR) and antibody-mediated rejection (ABMR), though the co-existence of both types of rejections can be seen in a variable number of sampled grafts. Acute TCMR can generally be well controlled by the establishment of effective immunosuppression (1, 2). Acute ABMR is a low frequency finding in the current era of blood group and HLA donor/recipient matching and the avoidance of engraftment in the context of high-titer, pref

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… Anticomplement C5 therapy with eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome . …

Anticoagulation for Cancer Patients in Special Situations: A Narrative Review of Guidelines and Literature.

Cancer-associated thrombosis (CAT) is a major cause of morbidity and mortality in patients with cancer. The management of special situations-including recurrent venous thromboembolism (VTE), thrombosis at unusual sites, and central venous catheter-associated thrombosis (CVC-AT)-remains particularly challenging because of the limited availability of high-quality evidence. This narrative review synthesizes recommendations from major international and Spanish clinical practice guidelines and expert consensus documents, including those from SEOM, ESMO, ASCO, NCCN, ITAC and SEMI, to provide a struc

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… In addition to cancer, risk factors for SPVT include recent abdominal surgery, abdominal mass, pancreatitis, cirrhosis, paroxysmal nocturnal hemoglobinuria, myeloproliferative neoplasms, and JAK2V617F mutations. The use of exogenous estrogens has also been implicated [ 4 ]. …

Targeting autoimmune mechanisms by precision medicine in Myasthenia Gravis.

Myasthenia Gravis (MG) is a chronic disabling autoimmune disease caused by autoantibodies to the neuromuscular junction (NMJ), characterized clinically by fluctuating weakness and early fatigability of ocular, skeletal and bulbar muscles. Despite being commonly considered a prototypic autoimmune disorder, MG is a complex and heterogeneous condition, presenting with variable clinical phenotypes, likely due to distinct pathophysiological settings related with different immunoreactivities, symptoms' distribution, disease severity, age at onset, thymic histopathology and response to therapies. Cur

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… Danicopan (ALXN2040) is an oral small molecule complement factor D (FD) inhibitor that showed improved benefit-risk profile as add-on therapy to ravulizumab or eculizumab in patients with complement-mediated paroxysmal nocturnal hemoglobinuria (PNH) compared to placebo ( 132 ). …

Structure and function of therapeutic antibodies approved by the US FDA in 2025.

In 2025, the 50th anniversary of the invention of hybridoma technology, the United States Food and Drug Administration (US FDA) approved a total of 44 new molecular entities (NMEs), of which 10 were therapeutic antibody-based molecules. Additionally, the US FDA approved 31 small molecules (of which one was a peptide), and 3 new nonantibody therapeutic proteins, but no new chimeric antigen receptor (CAR)-T-cell therapeutics. Of the new antibody-based biologics, two were antibody-drug conjugates, one was a T-cell engager-bispecific antibody, two were Fc-modified, half-life extended antibodies, a

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… GlaxoSmithKline; HBV, hepatitis B virus; HIV, human immunodeficiency virus; IBD, inflammatory bowel disease; IgG, immunoglobulin G; IL, interleukin; L/d, liters per day; LDL-C, low-density lipoprotein-cholesterol; NA, not applicable; P. falciparum, Plasmodium falciparum ; PNH, paroxysmal nocturnal hemoglobinuria …

Acute myeloid leukemia: from NGS, through scRNA-seq, to CAR-T. dissect cancer heterogeneity and tailor the treatment.

Acute myeloid leukemia (AML) is a malignant blood cancer with marked cellular heterogeneity due to altered maturation and differentiation of myeloid blasts, the possible causes of which are transcriptional or epigenetic alterations, impaired apoptosis, and excessive cell proliferation. This neoplasm has a high rate of resistance to anticancer therapies and thus a high risk of relapse and mortality because of both the biological diversity of the patient and intratumoral heterogeneity due to the acquisition of new somatic changes. For more than 40 years, the old gold standard "one size fits all"

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… Table 1 Risk factors concerning AML development Predisposing factors of AML Antecedent blood disorder | - Myelodysplastic syndromes - Chronic Myeloid Leukemia - Polycytemia Vera - Essential Thrombocytemia - Paroxismal Nocturnal - Hemoglobinuria - Aplastic Anemia - Myelofibrosis Genetic syndromes, family …

Прогресс в развитии лекарственных средств на основе пептида: платформы для доставки, терапевтические средства и вакцины.

Успешное утверждение лекарств на основе пептида можно отнести на счет совместных усилий по целому ряду дисциплин. Интеграция новых методов разработки и синтеза лекарственных средств, отображение библиотечных технологий, систем доставки, достижений в области биоинженерии и искусственного интеллекта в значительной степени ускорила разработку новейших лекарств на основе пептида, эффективно устраняя препятствия, связанные с их характером, такие, как быстрое обезвреживание и деградация, необходимость субколичественной инъекции, ведущей к усилению дискомфорта пациентов, и в конечном итоге продвигая

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… Zealand Pharma 30 Pegcetacoplan Empaveli ® 2021 C3 inhibitor C3 Treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) s.c . … | 2021 | GCGR agonists (glucagon receptor agonists) GCGR | The treatment of severe hypoglycemia in pediatric and adult patients with diabetes aged 6 years and above s.c . | Zealand Pharma 30 | Pegcetacoplan Empaveli ® | 2021 | C3 inhibitor C3 | Treatment of adult patients with paroxysmal nocturnal hemoglobinuria …

Research Progress in Pharmacological Targeted Therapy for Myasthenia Gravis.

Myasthenia gravis (MG) is a disorder of neuromuscular junction transmission that is mediated by autoantibodies and depends on T cell help, causing fatigable muscle weakness. Limitations of conventional first-line treatments, including long-term adverse effects and inadequate control of refractory disease, have driven targeted therapy to the forefront of MG research. This narrative review summarizes second-line and later-line targeted biological and cellular therapies, covering agents directedat B and T cells, complement inhibitors, neonatal Fc receptor (FcRn) antagonists and cytokine inhibitor

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… Specifically, Pozelimab exerts selective binding to C5 to block its terminal pathway activation, whereas Cemdisiran suppresses hepatic biosynthesis of C5. 91 This synergistic treatment modality is under investigation for managing paroxysmal nocturnal hemoglobinuria (PNH), 92 and a Phase III randomized …

Biopharmaceuticals for Cancer Treatment: An Update.

Cancer is one of the most debilitating diseases, causing profound physical and psychological impacts on patients. The global number of cancer patients has been steadily rising over the last few decades. The development of effective cancer therapies remains the primary focus in life science research, requiring a precise understanding of specific pathways and the regulation of tumor markers. The approved cancer therapies demonstrate effectiveness by acting on specific regulatory enzymes, components required for cell division, hormones, and cell-specific receptors, etc. While small-molecule drugs

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… . | A humanized IgG2/4 kappa antibody against terminal complement protein C5 | mAb | To treat atypical hemolytic uremic syndrome and paroxysmal nocturnal hemoglobinuria | Intravenous, Subcutaneous/ Nonspecific degradation into small peptides and individual amino acids/ N/A | June 7, 2028 | [ 189 ] Gamifant …

High-Dose Vitamin C in Advanced-Stage Cancer Patients.

High-dose intravenously administered vitamin C (IVC) is widely used in cancer patients by complementary and alternative medicine practitioners. The most frequent indications for IVC therapy result from the belief in its effectiveness as a potent anti-cancer agent which additionally enhances chemosensitivity of cancer cells and reduces chemotherapy-related toxicities and fatigue intensity. In this narrative review, we decided to deal with this issue, trying to answer the question whether there is any scientific evidence supporting the rationale for application of high-dose IVC therapy in advanc

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… However, even when administered orally, vitamin C might induce hemolysis in patients with paroxysmal nocturnal hemoglobinuria [ 152 ]. …

Ограниченные данные о преимуществах упражнений у пожилых людей с гематологическими отклонениями: систематический обзор и мета-анализ.

Пожилые пациенты, получающие антинеопластическое лечение, сталкиваются с такими проблемами, как слабость и снижение физической способности и функциональности. Цель этого систематического обзора и метаанализа заключалась в оценке воздействия практических занятий на результаты физической деятельности, качество жизни, связанное со здоровьем (QoL), и бремя симптомов у пожилых пациентов выше. 65 годы, когда гематологические заболевания проходят антинеопластическую терапию. Этот обзор соответствует руководящим принципам Кокрана, а поиск литературы последний раз обновлялся. 27 Март 2024, включая иссл

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… stimulation; Non-sec: Non-secretory; NR: Not reported; NYHA: New York Heart Association Functional Classification; Oligo-sec: Oligosecretory; PA: Physical activity; PBSCT: Peripheral blood stem cell transplantation; PID: Primary immune deficiency; PMF: Primary myelofibrosis; PNH: Paroxysmal nocturnal hemoglobinuria …