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Neutrophilic and eosinophilic dermatoses associated with hematological malignancy.

Cutaneous manifestations of hematologic malignancy represent both a clinical challenge for the treating physician and a pathophysiological model for advancing the knowledge on individual neoplasms. Indeed, a growing body of evidence supports the concept of recurrent molecular defects associating with specific clinical features, as best exemplified by VEXAS. Herein neutrophilic and eosinophilic dermatoses of potential interest for both hematologists and dermatologists will be reviewed, including subcorneal pustular dermatosis-type IgA pemphigus, neutrophilic eccrine hidradenitis, Sweet's syndro

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… In line with its Th2 polarization, L-HES commonly presents urticaria/angioedema, pruritus and eczematous lesions. Unusual manifestations include subcutaneous nodules and palmoplantar haemorrhagic blisters ( 131 ). … Other possible yet nonspecific skin changes include urticaria/angioedema, telangiectasia, palmar erythema or lichenification, cutaneous atrophy, superficial venous thrombophlebitis, hyperpigmentation ( 149 ), erythroderma ( 150 ) and eosinophilic cellulitis ( 117 , 119 ). …

Burden, Outcome, and Comorbidities of Extrahepatic Manifestations in Hepatitis B Virus Infections.

Hepatitis B virus (HBV) infections affect approximately 296 million people around the world, and the prevalence of any past or present HBV infection during the years 2015-2018 was as high as 4.3%. Acute HBV infection often presents with nonspecific symptoms and is usually self-limited, but 5% of patients can have persistent infections leading to chronic HBV infection and the risk of turning into chronic HBV infection is significantly higher in babies with vertical transmission (95%). Patients with chronic HBV infection are usually asymptomatic, but 15 to 40% of chronic HBV carriers develop cir

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… Chronic Urticaria and Angioedema Chronic spontaneous urticaria is a subtype of urticaria that lasts for more than 6 weeks. It occurs in 0.5–1% of the population. … Up to 4% of patients present with angioedema [ 146 ]. Urticaria with periorbital edema has been reported as prodromal symptoms of acute hepatitis B infection [ 145 ]. 8.4. …

The use of validated and nonvalidated surrogate endpoints in two European Medicines Agency expedited approval pathways: A cross-sectional study of products authorised 2011-2018.

BACKGROUND: In situations of unmet medical need or in the interests of public health, expedited approval pathways, including conditional marketing authorisation (CMA) and accelerated assessment (AA), speed up European Medicines Agency (EMA) marketing authorisation recommendations for medicinal products. CMAs are based on incomplete benefit-risk assessment data and authorisation remains conditional until regulator-imposed confirmatory postmarketing measures are fulfilled. For products undergoing AA, complete safety and efficacy data should be available, and postauthorisation measures may includ

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… Abbreviations: AA, accelerated assessment; ‘Ciani’, Ciani and colleagues [ 8 ]; CMA, conditional marketing authorisation; F&P, Fleming and Powers [ 7 ]; FEV1, forced expiratory volume in 1 second; FVC, forced vital capacity; HAE, hereditary angioedema; HCV, hepatitis C virus; MA, marketing authorisation … For 2 products, this was a single endpoint, overall survival—abiraterone (Zytiga), for prostate cancer, and lanadelumab (Takhzyro), for hereditary angioedema (Tables 3 and 4 ). …

A Rare Case of Aggressive Systemic Mastocytosis With Skeletal Osteosclerotic Lesions on Presentation: A Diagnostic Conundrum.

Systemic mastocytosis (SM) is a heterogeneous group of disorders caused by mast cell proliferation. SM often presents with non-specific symptoms making it a diagnostic challenge. Moreover, presentation with bone involvement is highly uncommon. Here, we report a rare case of SM in a 68-year-old female who initially presented with gastrointestinal symptoms and was later found to have sclerotic bone lesions on imaging. This case highlights an unusual presentation of SM, informing clinicians of the importance of keeping this disease process on the differential list of diagnostic conundrums.

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… Symptoms arising related to the release of MC mediators include urticaria, angioedema, anaphylactoid attacks, flushing and bronchospasm, digestive manifestations (nausea, vomiting, abdominal pain), and neuropsychiatric symptoms (depression, anxiety, memory disorders, sleeping disorders). …

Antibodies to watch in 2023.

In this 14th installment of the annual Antibodies to Watch article series, we discuss key events in commercial monoclonal antibody therapeutics development that occurred in 2022 and forecast events that might occur in 2023. As of mid-November, 12 antibody therapeutics had been granted first approvals in either the United States or European Union (tebentafusp (Kimmtrak), faricimab (Vabysmo), sutimlimab (Enjaymo), relatlimab (Opdualag), tixagevimab/cilgavimab (Evusheld), mosunetuzumab (Lunsumio), teclistamab (TECVAYLI), spesolimab (SPEVIGO), tremelimumab (Imjudo; combo with durvalumab), nirsevim

Immunosuppressive and Non-Immunosuppressive Drugs for Heart Xenotransplantation in Humans: Is Europe Ready?

Xenotransplantation is becoming an emerging field of interest for the treatment of end-stage heart disease. In fact, the shortage of human heart donors in European countries requires the increasing investigation of alternative strategies such as heart xenotransplantation. Among different limitations in this peculiar field, the experimental pharmacological management of patients who undergo heart xenotransplantation is primarily narrowed by the indications for which these drugs have been previously authorized by international medicines agencies. In fact, many of these medications have been neve

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… This drug is approved in the U.S. and in the E.U. for the prophylaxis and treatment of hereditary angioedema. Its use was first explored in kidney transplantation to prevent or reduce hyperacute rejection, with promising preliminary in vitro results [ 21 ]. … Syndrome, etc) | IV | Off‐label use for AMR therapy (antibodies removal, antibody reactivity reduction) | Used in the first Baltimore case to treat hypogammaglobulinemia and during the first plasma exchange C1 Esterase Inhibitor (Berinert) | FDA 2009 EMA 2013 | Prophylaxis and treatment of hereditary angioedema …

Clinical Treatment Guidelines for Tafasitamab Plus Lenalidomide in Patients with Relapsed or Refractory Diffuse Large B-Cell Lymphoma.

Diffuse large B-cell lymphoma (DLBCL) accounts for approximately 24% of new cases of B-cell non-Hodgkin lymphoma in the US each year. Up to 50% of patients relapse or are refractory (R/R) to the standard first-line treatment option, R-CHOP. The anti-CD19 monoclonal antibody tafasitamab, in combination with lenalidomide (LEN), is an NCCN preferred regimen for transplant-ineligible patients with R/R DLBCL and received accelerated approval in the US (July 2020) and conditional marketing authorization in Europe (August 2021) and other countries, based on data from the L-MIND study. The recommended

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… Hypersensitivity reactions such as angioedema, anaphylaxis, and anaphylactic reactions have been reported with LEN. 16 , 17 Pre-treatment with medications such as acetaminophen, histamine H1 or H2 receptor antagonists, and/or glucocorticosteroids is recommended 30-120 minutes prior to tafasitamab infusion … Restart at original dose at investigator’s discretion • Continue anticoagulation throughout the course of lenalidomide treatment Severe cutaneous reaction | Grades 2-3 | • Consider interruption or discontinuation Grade 4 | • Permanently discontinue treatment Allergic reaction or hypersensitivity | Any angioedema …

Health technology assessment of drugs for rare diseases: insights, trends, and reasons for negative recommendations from the CADTH common drug review.

BackgroundA shift in biochemical research towards drugs for rare diseases has created new challenges for the pharmaceutical industry, government regulators, health technology assessment agencies, and public and private payers. In this article, we aim to comprehensively review, characterize, identify possible trends, and explore reasons for negative reimbursement recommendations in submissions made to the Common Drug Review (CDR) for drugs for rare diseases (DRD) at the Canadian Agency for Drugs and Technologies in Health (CADTH), a publicly funded pan-Canadian health technology assessment agen

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… Chemical/Selective Bradykinin B2 Receptor Antagonist Hereditary angioedema 1 a List with clinical criteria and/or conditions 19/03/2014 Afinitor Everolimus Novartis Pharmaceuticals Canada Inc. Chemical. … condition 27/02/2014 | Signifor | pasireotide | Novartis Pharmaceuticals Canada Inc. | Chemical/Somatostatin Analog | Cushing's Disease | 4 a | Do not list 04/03/2014 | Firazyr | icatibant | Shire Human Genetic Therapies (Canada) Inc. | Chemical/Selective Bradykinin B2 Receptor Antagonist | Hereditary angioedema …

Acute and Chronic Respiratory Failure in Cancer Patients

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… pleuroparenchymal fibroelastosis (PPFE) Cyclophosphamide Organizing pneumonia, pulmonary fibrosis, PPFE, ARDS, DAH, bronchospasm, pleural effusion, pulmonary veno-occlusive disease Doxorubicin Organizing pneumonia, pulmonary fibrosis, ARDS, radiation recall pneumonitis Etoposide ARDS, DAH, bronchospasm, angioedema … fibrosis, radiation recall pneumonitis, pulmonary edema, hypoxia Melphalan | Pulmonary fibrosis, ARDS, bronchospasm, organizing pneumonia, NSIP, UIP Methotrexate | Organizing pneumonia, pulmonary fibrosis, lung nodules, ARDS, AIP, DAH, bronchospasm, eosinophilic pleural effusion, pulmonary embolism, angioedema …

Unmasking TRALI: a case report of multiple myeloma and transfusion-associated event.

INTRODUCTION AND IMPORTANCE: Transfusion-related acute lung injury (TRALI) is a relatively rare but severe respiratory complication that can occur following the transfusion of blood products. Despite its infrequent occurrence, TRALI is a leading cause of transfusion-related mortality, underscoring the need for rapid diagnosis and appropriate management. However, diagnosis remains challenging due to its clinical overlap with other causes of acute respiratory distress syndrome (ARDS). CASE PRESENTATION: This report describes the case of a 57-year-old woman with multiple myeloma (MM) who develop

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… present | Usually absent or unchanged | Common, prominent | Sometimes present (especially with anaphylaxis) Additional signs | Absence of volume overload signs such as JVD d | Jugular venous distension, peripheral edema | Positive blood cultures with same organism as transfused product | Urticarial rash, angioedema …

Expedited drug review process: Fast, but flawed.

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… DPP-IV inhibitors were implicated in severe mouth ulcers, renal impairment, and angioedema. SGLT2 inhibitors are known to precipitate ketoacidosis and urosepsis. …

Lenalidomide use in multiple myeloma (Review).

Lenalidomide is a second-generation new immunomodulatory medication used to treat multiple myeloma (MM). Its mechanism of action involves affecting the expression of vascular endothelial growth factor, interleukin-6, cytochrome c, caspase-8, as well as other factors including immunological modulation and the direct killing of cells, among others, rendering it a fundamental medication, useful for the treatment of MM. Combining lenalidomide with other medications such dexamethasone, bortezomib, ixazomib, carfilzomib and daratumumab can markedly alleviate MM. When autologous-hematopoietic stem ce

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… Severe skin reactions (including allergic reactions) Angioedema, hypersensitivity, and severe skin reactions, including Stevens-Johnson syndrome (SJS), toxic epidermal necrolysis (TEN), and drug reactions with eosinophilia and systemic symptoms (DRESS), have been reported with the use of lenalidomide … If angioedema, hypersensitivity, grade 4 rash, exfoliative or maculopapular rash, or suspected SJS, TEN, and DRESS occur, the drug must be discontinued and must not be restarted after these reactions have resolved. …

Drug Repurposing for the Management of Depression: Where Do We Stand Currently?

A slow rate of new drug discovery and higher costs of new drug development attracted the attention of scientists and physicians for the repurposing and repositioning of old medications. Experimental studies and off-label use of drugs have helped drive data for further studies of approving these medications. A deeper understanding of the pathogenesis of depression encourages novel discoveries through drug repurposing and drug repositioning to treat depression. In addition to reducing neurotransmitters like epinephrine and serotonin, other mechanisms such as inflammation, insufficient blood supp

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… also affect descending noradrenergic and serotonergic pain transmission pathways from the brainstem to the spinal cord/Anticonvulsant, GABA analog | NA | Peripheral edema, Dizziness, drowsiness, headache, fatigue, weight gain, xerostomia, visual field loss, blurred vision | Hypersensitivity (e.g., angioedema … Usual effective dose: 1 to 1.5 mg/day. | Activating/sedating effects, angioedema, dyslipidemia, extrapyramidal symptoms, hematologic abnormalities, hyperglycemia, weight gain, hyperprolactinemia, neuroleptic malignant syndrome, orthostatic hypotension, QT prolongation, sexual dysfunction, temperature …

Quantifying hope: an EU perspective of rare disease therapeutic space and market dynamics.

Rare diseases, affecting millions globally, pose a significant healthcare burden despite impacting a small population. While approximately 70% of all rare diseases are genetic and often begin in childhood, diagnosis remains slow and only 5% have approved treatments. The UN emphasizes improved access to primary care (diagnostic and potentially therapeutic) for these patients and their families. Next-generation sequencing (NGS) offers hope for earlier and more accurate diagnoses, potentially leading to preventative measures and targeted therapies. In here, we explore the therapeutic landscape fo

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… market shares The EMA has approved interventions against 113 indications with ODD and 201 indications without ODD status till 2021.13 of these indications (multiple myeloma, acute lymphoblastic leukemia, type 1 Gaucher disease, pulmonary arterial hypertension, idiopathic pulmonary fibrosis, hereditary angioedema … Some of the key indications targeted by ODD drugs are multiple myeloma, cystic fibrosis, spinal muscular atrophy, endogenous Cushing syndrome, hereditary angioedema and acute myeloid leukemia ( Figure 3F ). …

Recent advances in renal transplantation: antibody-mediated rejection takes center stage.

Overlooked for decades, antibodies have taken center stage in renal transplantation and are now widely recognized as the first cause of allograft failure. Diagnosis of antibody-mediated rejection has considerably improved with identification of antibody-mediated lesions in graft biopsies and advances made in the detection of circulating donor-specific antibodies. Unfortunately, this progress has not yet translated into better outcomes for patients. Indeed, in the absence of a drug able to suppress antibody generation by plasma cells, available therapies can only slow down graft destruction. Th

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… Although C1 inhibition has not been tested in clinical AMR yet, a C1 inhibitor for treatment of patients with hereditary angioedema is already available [ 77 ]. … Nanofiltered C1 inhibitor concentrate for treatment of hereditary angioedema. The New England journal of medicine. 2010;363:513–22. doi: 10.1056/NEJMoa0805538. 78. …

Antibodies to watch in 2026.

The Antibodies to Watch article series provides annual updates on commercial late-stage clinical development, regulatory review, and marketing approvals of antibody therapeutics. Since the first article was published in 2010, the late-stage pipeline has grown from 26 antibody therapeutics to over 200, while during the same time numerous molecules in late-stage studies either transitioned to regulatory review and were approved or were terminated. In this installment of the series, we recap first marketing approvals granted to 19 antibody therapeutics in 2025, discuss 26 molecules currently in r

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… INN (Brand name) Target(s); Format Indication of first approval Country/region of first approval in 2025* Garadacimab (ANDEMBRY®) Factor XIIa; Human IgG4λ Prevention of hereditary angioedema attacks in adult and pediatric patients aged 12 years and older. … Garadacimab (garadacimab-gxii, ANDEMBRY®) is a human IgG4λ monoclonal antibody targeting Factor XIIa (FXIIa), developed for the treatment of hereditary angioedema (HAE). …

Antibodies to watch in 2025.

The commercial development of antibody therapeutics is a global enterprise involving thousands of biopharmaceutical firms and supporting service organizations. To date, their combined efforts have resulted in over 200 marketed antibody therapeutics and a pipeline of nearly 1,400 investigational product candidates that are undergoing evaluation in clinical studies as treatments for a wide variety of diseases. Here, we discuss key events in antibody therapeutics development that occurred during 2024 and forecast key events related to the late-stage clinical pipeline that may occur in 2025. In pa

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… INN or drug code | Target; Format | Indication(s) under review | Country/region of review Bentracimab | Ticagrelor; Human IgG1λ Fab | Reversal of the antiplatelet effects ofticagrelor | US Garadacimab | Factor XIIa; Human IgG4λ | Hereditary angioedema | EU, US, Japan, Canada Nipocalimab | FcRn; Human … Garadacimab (CSL312) is a hinge-stabilized, human anti-Factor XIIa IgG4λ antibody developed as a treatment for hereditary angioedema (HAE). …

Holistic Approach to the Diagnosis and Treatment of Patients with Tumor Metastases to the Spine.

The treatment of neoplastic spine metastases requires multi-faceted assessment and an interdisciplinary approach to patients. The metastases do not show specific symptoms but are often the first confirmation of the presence of a primary tumor in a patient. The diagnostic process includes imaging and invasive procedures, e.g., biopsy. It is essential to qualify the patient for an appropriate treatment using dedicated scales. Decompression of the spinal cord is a critical issue to save or restore neurological function in a patient with spine metastases. Surgical treatment ought to meet three cri

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… Their exact mechanism of action and dosing are a matter of debate, but they reduce angioedema and relieve inflammation in the area of metastasis. There are no precise guidelines for the dosage of corticosteroids, and they are selected individually to case [ 92 ]. … Using corticosteroids during therapy, which decrease angioedema in the area of the metastasis, alleviate pain and improve treatment results, is important [ 92 ]. Immunotherapy is also gaining more popularity, improving prognosis even in advanced metastatic disease [ 38 ]. …

Do payers value rarity? An analysis of the relationship between disease rarity and orphan drug prices in Europe.

Background and Objective: Orphan drugs have been a highlight of discussions due to their higher prices than non-orphan drugs. There is currently no European consensus on the method of value assessment for orphan drugs. This study assessed the relationship between the prevalence of rare diseases and the annual treatment cost of orphan drugs in France, Germany, Italy, Norway, Spain, Sweden, and UK. Methods: Approved orphan drugs and prevalence data were extracted from the European Medicines Agency website. Annual treatment costs were calculated using ex-factory price. Simple regression was used

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… Diacomit Severe myoclonic epilepsy in infancy 0.40 Elaprase Mucopolysaccharidosis II 0.02 Esbriet Idiopathic pulmonary fibrosis 3.00 Evoltra Precursor cell lymphoblastic leukaemia-lymphoma 0.40 Exjade Iron overload 2.70 Fabrazyme* Fabry disease 0.03 Farydak Multiple myeloma 3.20 Firazyr Hereditary angioedemas … epilepsy in infancy | 0.40 Elaprase | Mucopolysaccharidosis II | 0.02 Esbriet | Idiopathic pulmonary fibrosis | 3.00 Evoltra | Precursor cell lymphoblastic leukaemia-lymphoma | 0.40 Exjade | Iron overload | 2.70 Fabrazyme* | Fabry disease | 0.03 Farydak | Multiple myeloma | 3.20 Firazyr | Hereditary angioedemas …

Raised Serum Levels of Syndecan-1 (CD138), in a Case of Acute Idiopathic Systemic Capillary Leak Syndrome (SCLS) (Clarkson's Disease).

BACKGROUND Systemic capillary leak syndrome (SCLS) (Clarkson's disease) is a rare disorder of unknown etiology, characterized by transient episodes of hypotension, and the microvascular leak of fluids into the peripheral tissues, resulting in edema. Between 80-90% of patients with SCLS have a concomitant monoclonal gammopathy. Although translational in vitro studies have implicated vascular endothelial barrier dysfunction in the etiology of SCLS, the etiology and disease associations in clinical cases remain unknown. CASE REPORT We report a case of SCLS in a 49-year-old woman who initially pre

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… Based on clinical signs, symptoms, and laboratory tests, the differential diagnosis included sepsis, idiopathic anaphylaxis, hereditary angioedema, polycythemia vera, and cardiac insufficiency. …