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Benefit and danger from immunotherapy in myasthenia gravis.

In the last years, significant advances have improved the knowledge of myasthenia gravis (MG) immunopathogenesis and have enabled to realize new molecules with a selective action targeting compounds of the immunological system. This review discusses emerging treatments for MG, including complement inhibitors, neonatal Fc receptor targeting agents, and B cell interfering drugs, focusing on benefit and danger. In the second section of the review, several related adverse events of immunotherapy, including MGonset, are debated.

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… FDA report: eculizumab (Soliris) for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Oncologist. 2008;13(9):993–1000. doi: 10.1634/theoncologist.2008-0086. 4. Legendre CM, Licht C, Muus P, Greenbaum LA, Babu S, Bedrosian C, et al. … De Latour RP, Brodsky RA, Ortiz S et al (2018) Ravulizumab (ALXN1210) versus eculizumab in adults with paroxysmal nocturnal hemoglobinuria: pharmacokinetics and pharmacodynamics observed in two phase 3 randomized, multicenter studies [abstract no. 626]. Blood. 132 19. …

A year in pharmacology: new drugs approved by the US Food and Drug Administration in 2023.

With 54 new drugs and seven cellular and gene therapy products, the approvals by the US Food and Drug Administration (FDA) recovered 2023 from the 2022 dent back to the levels of 2020-2021. As in previous years of this annual review, we assign these new drugs to one of three levels of innovation: first drug against a condition ("first-in-indication"), first drug using a novel molecular mechanism ("first-in-class"), and "next-in-class," i.e., a drug using an already exploited molecular mechanism. We identify four (7%) "first-in-indication," 22 (36%) "first-in-class," and 35 (57%) "next-in-class

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… Paroxysmal nocturnal hemoglobinuria is a non-malignant, rare blood disorder manifesting as hemolytic anemia, bone marrow failure, thrombosis, and complement deficiency (Brodsky 2014 ). … Paroxysmal nocturnal hemoglobinuria. Blood. 2014;124:2804–2811. doi: 10.1182/blood-2014-02-522128. …

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Aptamers: Potential Diagnostic and Therapeutic Agents for Blood Diseases.

Aptamers are RNA/DNA oligonucleotide molecules that specifically bind to a targeted complementary molecule. As potential recognition elements with promising diagnostic and therapeutic applications, aptamers, such as monoclonal antibodies, could provide many treatment and diagnostic options for blood diseases. Aptamers present several superior features over antibodies, including a simple in vitro selection and production, ease of modification and conjugation, high stability, and low immunogenicity. Emerging as promising alternatives to antibodies, aptamers could overcome the present limitations

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… Effect of eculizumab on hemolysis and transfusion requirements in patients with paroxysmal nocturnal hemoglobinuria. N. Engl. J. Med. 2004;350:552–559. doi: 10.1056/NEJMoa031688. 110. Meyers G., Weitz I., Lamy T., Cahn J.-Y., Kroon H. … Disease-Related Symptoms Reported across a Broad Population of Patients with Paroxysmal Nocturnal Hemoglobinuria. American Society of Hematology; Washington, DC, USA: 2007. 111. …

Beyond Numbers: Uses and Value of In-Trial Interview Data for Regulatory and Health Technology Assessment (HTA) Decision Making.

This research explores the growing importance of qualitative in-trial research (ITR) in regulatory and health technology assessment (HTA) decision making. Since 2020, the Food and Drug Administration (FDA) and European Medicines Agency (EMA) have emphasized incorporating patient experience data into drug development and approval. We reviewed regulatory guidance documents, drug approval records, and HTA reports from January 2017 to March 2025. This included labels and reviews for new drug applications from the FDA and EMA, the Patient-Focused Drug Development guidance series, guidance on rare d

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… EMA European Medicines Agency, FDA Food and Drug Administration. ^ FDA-reviewed drugs and indications with in-trial interview data: Aimovig (migraine), Blenrep (multiple myeloma), Bylvay (pruritus in progressive familial intrahepatic cholestasis), Fabhalta (paroxysmal nocturnal hemoglobinuria), Romvimza … EMA European Medicines Agency, FDA Food and Drug Administration. ^ FDA-reviewed drugs and indications with in-trial interview data: Aimovig (migraine), Blenrep (multiple myeloma), Bylvay (pruritus in progressive familial intrahepatic cholestasis), Fabhalta (paroxysmal nocturnal hemoglobinuria), Romvimza …

Biologics in Kidney Transplantation: Why and How Should We Personalize Their Dosage?

The therapeutic arsenal of immunosuppressive drugs in kidney transplantation has recently expanded with the introduction of biologics, approved or used off-label, that specifically target the immune system. Although therapeutic drug monitoring is well established for calcineurin inhibitors, its utility for biological immunosuppressants remains unclear. The objective of this narrative review was to evaluate whether biologics used to prevent or treat renal allograft rejection could be suitable candidates for therapeutic drug monitoring. This review provides (1) a comprehensive and up-to-date syn

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… Ravulizumab is a long-lasting complement C5 inhibitor also approved for the treatment of paroxysmal nocturnal hemoglobinuria. … Pharmacokinetics, pharmacodynamics, efficacy, and safety of ravulizumab in pediatric paroxysmal nocturnal hemoglobinuria. Blood Adv. 2024;8(11):2813–24. 10.1182/bloodadvances.2023012267. 38551806 10.1182/bloodadvances.2023012267 PMC11176942 61. …

Lenalidomide-associated pure red cell aplasia during the treatment of POEMS syndrome: A case report and brief review of the literature.

Lenalidomide is widely used in the treatment of hematological malignancies, with bone marrow suppression being one of the most common hematological adverse effects associated with its use. However, lenalidomide-associated pure red cell aplasia (PRCA) remains exceptionally rare. The present study describes a case of PRCA secondary to lenalidomide therapy in a patient with POEMS syndrome. A 61-year-old woman diagnosed with POEMS syndrome received lenalidomide (25 mg/day) in combination with dexamethasone. Following 3 months of treatment, she developed severe anemia accompanied by a markedly redu

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… Figure 1 Flow cytometric analysis for paroxysmal nocturnal hemoglobinuria. Representative histograms illustrating the normal expression of CD55 and CD59 in red blood cells. No paroxysmal nocturnal hemoglobinuria clone was detected. Figure 2 (A) Bone marrow smear findings. …

COVID-19 in 96 Patients With Hematologic Disease: The First Single-center Experience From the Czech Republic.

BackgroundCoronavirus disease 2019 (COVID-19) represents an important infectious complication associated with high mortality rates in patients with hematologic diseases. There have not been published any epidemiologic studies from Czech Republic so far.Patients and methodsThis study is the first analysis of patients with hematologic malignancies and bone marrow failure syndromes treated at single hematology center in the Czech Republic between March 1 and December 31, 2020, in whom COVID-19 infection was confirmed.ResultsThe sample comprised 96 patients aged 26 to 84 years (median, 66.0 years)

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… Two patients were observed for bone marrow failure syndromes, namely, aplastic anemia and paroxysmal nocturnal hemoglobinuria. … / 1 / 1 Primary myelofibrosis | 1 | 0 / 0 / 1 / 0 | 0 | 0 | 0 / 1 / 0 Chronic myelomonocytic leukemia | 1 | 0 / 0 / 1 / 0 | 0 | 1 | 0 / 1 / 0 Bone marrow failure syndromes | 2 | 0 / 0 / 1 / 1 | 1 (50.0) | 0 (0) | 0 / 2 / 0 Aplastic anemia | 1 | 0 / 0 / 0 / 1 | 0 | 0 | 0 / 1 / 0 Paroxysmal nocturnal hemoglobinuria …

Criteria for referring pediatric and adult patients with hematological diseases to palliative care: Consensus of the Brazilian Association of Hematology, hemotherapy and cell therapy (2025).

Palliative care is, according to the World Health Organization (WHO), "a global ethical responsibility" and crucial to person-centered healthcare, for the relief of physical and psychological symptoms and social and spiritual suffering, impacting the improvement of the quality of life not only of pediatric and adult patients dealing with life-threatening illnesses, but also of their families. Despite the growing recognition of palliative care in the hematological setting and the increase in the number of publications on the subject, the available data are still scarce and limited. The objectiv

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… Acquired AA and paroxysmal nocturnal hemoglobinuria (PNH) are diseases belonging to the group of bone marrow failure syndromes and should be considered as two distinct manifestations of a common pathophysiology, but that are interrelated. … Recommendations Referral criteria for patients with congenital bone marrow failure, acquired aplastic anemia, and paroxysmal nocturnal hemoglobinuria to palliative care 1. …

Microangiopathy in multiple myeloma: a case of carfilzomib-induced secondary thrombotic microangiopathy successfully treated with plasma exchange and complement inhibition.

BACKGROUND: Thrombotic microangiopathy (TMA) is a potentially organ and life-threatening condition affecting patients with multiple myeloma (MM). Cases of proteasome inhibitor-induced TMA and specifically carfilzomib-induced TMA have been rarely reported and standards for diagnostic workup and treatment are not available. CASE PRESENTATION: We describe a case of a male MM patient under salvage therapy including proteasome inhibitor carfilzomib following chemotherapy and autologous stem cell transplantation. The patient then developed acute kidney injury with clinical and laboratory signs of T

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… As urinary dip test for hemoglobin was highly positive and only few erythrocytes were present in sediment analysis, we hypothesized this being due to hemoglobinuria. Furthermore, the urine sediment was suggestive of acute tubular necrosis. … Proteinuria and hemoglobinuria were not detectable after four weeks of treatment. A graphic overview showing amelioration of kidney function throughout the treatment is given in Fig. 1 . The patient is alive and well to the current moment (9 months after TMA). …

Кассетная нефропатия в СИС: Раннее признание и экстракорпоральные стратегии улучшения результатов.

Острая почечная травма (АКИ), вызванная кастами, является частой, но недостаточно признанной причиной дисфункции почек в отделении интенсивной терапии. Она возникает, когда фильтрованные белки или пигменты - свободные световые цепи (ФЛК) в множестве миеломы, миоглобин в хабдомиолизе, билирубин в тяжелом холестазе или гемоглобин в внутриваскулярном гемолизе - возникают внутри почечных трубок, образуя обструктивные касты и вызывая окислительные и подстрекательские травмы. Раннее распознавание имеет важное значение, поскольку традиционные маркеры (кротинин, мочи) задерживаются. Формирующиеся биом

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… The PubMed database was searched using the following MeSH terms and keywords: (“cast nephropathy” OR “light chain nephropathy” OR “multiple myeloma” OR “rhabdomyolysis” OR “bile cast nephropathy” OR “hemoglobinuria” OR “hemolysis”) AND (“acute kidney injury” OR “kidney injury” OR “acute renal failure … Efficacy of complement inhibitors for patients with paroxysmal nocturnal hemoglobinuria: a systematic review and meta-analysis. Ther Adv Hematol. 2023;14 doi: 10.1177/20406207231216080. 64. Baeg S.I., Lee K., Jeon J., Jang H.R. …

Long-Term Results of Hematopoietic Cell Transplantation (HCT) for Adults with Blood Disorders- A Systematic Review.

Both autologous and allogeneic stem cell transplantations are widely used as a standard of care or significant clinical option in treating various malignant and non-malignant diseases of the hematopoietic system. The value of these treatment methods would be better assessed through the review of long-term progression-free and overall survival (PFS and OS) rates, which are highly dependent on the primary diagnosis and stage of disease at the time of transplantation as well as on the specific type of used transplantation procedure. While the best (over 90%) 5-, 10- and 15-year survival rates are

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… Outcome of allogeneic hematopoietic stem cell transplantation in adult patients with paroxysmal nocturnal hemoglobinuria. International Journal of Hematology, 113 (1), 122–127. 10.1007/s12185-020-02982-y 73. Parker, C. J. (2011). … Management of paroxysmal nocturnal hemoglobinuria in the era of complement inhibitory therapy. Hematology American Society of Hematology. Education Program, 2011 , 21–29. 10.1182/asheducation-2011.1.21 74. Henig, I., & Zuckerman, T. (2014). …

Characteristics and prognosis of patients with COVID-19 and hematological diseases in Japan: a cross-sectional study.

The Japanese Society of Hematology performed an observational cross-sectional study to clarify the morbidity, prognosis, and prognostic factors in patients with COVID-19 with hematological diseases (HDs) in Japan. The study included patients with HDs who enrolled in our epidemiological survey and had a COVID-19 diagnosis and a verified outcome of up to 2 months. The primary endpoints were characteristics and short-term prognosis of COVID-19 in patients with HDs. A total of 367 patients from 68 institutes were enrolled over 1 year, and the collected data were analyzed. The median follow-up amon

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… Table 2 Background hematological diseases and history of cellular therapies HD hematological disease, AIHA autoimmune hemolytic anemia, CAD cold agglutinin disease, PNH paroxysmal nocturnal hemoglobinuria, ITP idiopathic thrombocytopenic purpura, PRCA pure red cell aplasia, TTP thrombotic thrombocytopenic … purpura; LPL, lymphoplasmacytic lymphoma; MCL, mantle cell lymphoma; MDS, myelodysplastic syndrome; MF, myelofibrosis; MM, multiple myeloma; MPAL, mixed-phenotype acute leukemia; MPN, myeloproliferative neoplasm; PCNSL, primary central nervous system lymphoma; Ph, Philadelphia; PNH, paroxysmal nocturnal hemoglobinuria …

Updates on C3 Glomerulopathy in Kidney Transplantation: Pathogenesis and Treatment Options.

C3 glomerulopathy is a rare disease, characterized by an abnormal activation of the complement's alternative pathway that leads to the accumulation of the C3 component in the kidney. The disease recurs in more than half of kidney transplant recipients, with a significant impact on graft survival. Recurrence of the primary disease represents the second cause of graft loss after organ rejection. In C3 glomerulopathy, there are several risk factors which can promote a recurrence during transplantation, such as delayed graft function, infection and monoclonal gammopathy. All these events can trigg

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… It was first introduced for the treatment of paroxysmal nocturnal hemoglobinuria [ 49 ] and, subsequently, it was used for treatment of aHUS, with important results in improving renal function [ 50 ]. … The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria N. Engl. J. Med. 2006 355 1233 1243 10.1056/NEJMoa061648 16990386 50. Fakhouri F. Zuber J. Frémeaux-Bacchi V. Loirat C. Haemolytic uraemic syndrome Lancet Lond. …

Global perspectives on lupus nephritis: a review of clinical trials and therapeutic innovations.

BACKGROUND AND OBJECTIVES: Lupus nephritis (LN) remains a leading cause of morbidity and mortality in systemic lupus erythematosus (SLE). Although recent years have witnessed the approval of several targeted therapies, achieving long-term, drug-free remission remains challenging. This commentary evaluates the global landscape of interventional LN clinical trials from 2001 to 2026 to identify emerging trends and strategic gaps in drug development. METHODS: We analyzed 200 interventional pharmacological trials retrieved from three major global registries: ClinicalTrials.gov, Chinadrugtrials.org

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… Ravulizumab: a novel C5 inhibitor for the treatment of paroxysmal nocturnal hemoglobinuria . Ther Adv Hematol . ( 2019 ) 10 : 2040620719874728 . doi: 10.1177/2040620719874728 . PMID: 31534662 PMC6737867 31 Panse J Han B Cermak J Gonzalez Fernandez FA Gotoh A Kulasekararaj AG . … Patient-reported outcomes in patients with paroxysmal nocturnal hemoglobinuria treated with crovalimab and approved C5 inhibitors in the phase III COMMODORE 2 and 1 studies . Ann Hematol . ( 2025 ) 104 : 3199–212 . doi: 10.1007/s00277-025-06449-0 . …

Hematology

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… Fever, ↓ BP, CP, lumbar pain, hemoglobinuria, and bleed Stop transfusion and check blood. Fluids, diuretics, FFP, dialysis Acute hemolytic reaction Recipient Ab against donor RBC minor antigen, 1/600,000 Acute/delay. Milder form of above Stop transfusion and check blood. … Fever, ↓ BP, CP, lumbar pain, hemoglobinuria, and bleed | Stop transfusion and check blood. Fluids, diuretics, FFP, dialysis Acute hemolytic reaction | Recipient Ab against donor RBC minor antigen, 1/600,000 | Acute/delay. Milder form of above | Stop transfusion and check blood. …

Hematology

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… Fever, ↓ BP, CP, lumbar pain, hemoglobinuria, and bleed Stop transfusion and check blood. Fluids, diuretics, FFP, dialysis Acute hemolytic reaction Recipient Ab against donor RBC minor antigen, 1/600,000 Acute/delay. Milder form of above Stop transfusion and check blood. … Fever, ↓ BP, CP, lumbar pain, hemoglobinuria, and bleed | Stop transfusion and check blood. Fluids, diuretics, FFP, dialysis Acute hemolytic reaction | Recipient Ab against donor RBC minor antigen, 1/600,000 | Acute/delay. Milder form of above | Stop transfusion and check blood. …

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Profile and Antibiotic Pattern of Blood Stream Infections of Patients Receiving Hematopoietic Stem Cell Transplants in Southwest China.

BackgroundBloodstream infection (BSI) is a serious medical issue causing non-relapsed mortality in patients receiving hematopoietic stem cell transplantations (HSCT).MethodsThe characteristics of all patients receiving HSCT (autologous and allogeneic HSCT) in our hospital from 2013 to 2019 were studied. Ratios, medians, and ranges were calculated to describe categorical variables. Chi-square tests were performed to compare the difference between ratios.ResultsA total of 741 patients receiving 746 HSCT procedures-including 376 allogeneic, 370 autologous, and four of both types-were included in

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… BSI, bloodstream infection; HSCT, hematopoietic stem cell transplant; AML, acute myeloid leukemia; ALL, acute lymphoblastic leukemia; AUL, Acute undifferentiated leukemia; HL, Hodgkin lymphoma; NHL, non-Hodgkin lymphoma; CML, chronic myeloid leukemia; AA, aplastic anemia; PNH, paroxysmal nocturnal hemoglobinuria … Prevention and Control; CDC, Centers for Disease Control and Prevention; AML, acute myeloid leukemia; NHL, non-Hodgkin’s lymphoma; ALL, acute lymphoblastic leukemia; AUL, acute undifferentiated leukemia; HL, Hodgkin lymphoma; CML, chronic myeloid leukemia; AA, aplastic anemia; PNH, paroxysmal nocturnal hemoglobinuria …

Assessing the Influence of Different Comorbidities Indexes on the Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation in a Developing Country.

Although the application of Hematopoietic Cell Transplantation-specific Comorbidity Index (HCT-CI) has enabled better prediction of transplant-related mortality (TRM) in allogeneic hematopoietic stem cell transplants (AHSCT), data from developing countries are scarce. This study prospectively evaluated the HCT-CI and the Adult Comorbidity Evaluation (ACE-27), in its original and in a modified version, as predictors of post-transplant complications in adults undergoing a first related or unrelated AHSCT in Brazil. Both bone marrow (BM) and peripheral blood stem cells (PBSC) as graft sources wer

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… Variables Primary diseases included hematologic malignancies (acute leukemia, chronic myeloid leukemia, multiple myeloma, primary myelofibrosis, lymphomas, myelodysplastic syndrome, and unspecified myeloproliferative disorders) and non-malignant diseases (aplastic anemia, paroxysmal nocturnal hemoglobinuria … Abbreviations: ACE-27, Adult Comorbidity Evaluation; HCT-CI , Hematopoietic Cell Transplantation-specific Comorbidity Index * Other : includes Fanconi anemia, congenital dyskeratosis, paroxysmal nocturnal hemoglobinuria, multiple myeloma ** Adapted from Armand et al [ 21 ] *** Excluding hematologic malignancies …