Chapter 129: Paraneoplastic Syndromes
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View metadata, citation and similar papers at core.ac.uk brought to you by CORE provided by Frontiers - Publisher Connector MINI REVIEW published: 23 March 2017 doi: 10.3389/fimmu.2017.00336 Adaptive Immunity Is the Key to the Understanding of Autoimmune and Paraneoplastic inflammatory Central Nervous System Disorders Robert Weissert* Department of Neurology, Neuroimmunology, University of Regensburg, Regensburg, Germany There are common aspects and mechanisms between different types of autoimmune diseases such as multiple sclerosis (MS), neuromyelitis optica spectrum disorders (NMOSDs), and autoimmune encephalitis (AE) as well as paraneoplastic inflammatory disorders of the central nervous system. To our present knowledge, depending on the disease, T and B cells as well as antibodies contribute to various aspects of the pathogenesis. Possibly the events leading to the breaking of tolerance between the different diseases are of great similarity and so far, only partially understood. Beside Edited by: endogenous factors (genetics, genomics, epigenetics, malignancy) also exogenous fac- Björn Tackenberg, tors (vitamin D, sun light exposure, smoking, gut microbiome, viral infections) contribute Philipps University of Marburg, Germany to susceptibility in such diseases. What differs between these disorders are the target Reviewed by: molecules of the immune attack. For T cells, these target molecules are presented on Anne Kathrin Mausberg, major histocompatibility complex (MHC) molecules as MHC-bound ligands. B cells have Essen University Hospital, Germany Pavan Bhargava, an important role by amplifying the immune response of T cells by capturing antigen with Johns Hopkins School of Medicine, their surface immunoglobulin and presenting it to T cells. Antibodies secreted by plasma USA cells that have differentiated from B cells are highly structure specific and can have *Correspondence: important effector functions leading to functional impairment or/and lesion evolvement. -
214622Orig1s000
CENTER FOR DRUG EVALUATION AND RESEARCH APPLICATION NUMBER: 214622Orig1s000 MULTI-DISCIPLINE REVIEW Summary Review Office Director Cross Discipline Team Leader Review Clinical Review Non-Clinical Review Statistical Review Clinical Pharmacology Review NDA214622 Multi‐disciplinary Review and Evaluation Infigratinib (Truseltiq) NDA/BLA Multi‐disciplinary Review and Evaluation FDA review was conducted in conjunction with other regulatory authorities under a regular ORBIS. While the application review is completed by the FDA, the application is still under review at the other regulatory agencies (Health Canada and Therapeutic Goods Administration). Disclaimer: In this document, the sections labeled as “Data” and “The Applicant’s Position” are completed by the Applicant, which do not necessarily reflect the positions of the FDA. Application Type NDA Application Number(s) 214622 Priority or Standard Priority Submit Date(s) September 29, 2020 Received Date(s) September 29, 2020 PDUFA Goal Date May 29, 2021 Division/Office OOD/DO3 Review Completion Date Please check electronic date stamp Established Name Infigratinib Trade Name Truseltiq Pharmacologic Class FGFR Inhibitor Code name BGJ398 Applicant QED Therapeutics, Inc. Formulation(s) Oral capsule Dosing Regimen 125 mg orally once daily for 21 consecutive days followed by 7 days off therapy, in 28‐day cycles Applicant Proposed The treatment of adult patients with previously treated, Indication(s)/Population(s) unresectable locally advanced or metastatic cholangiocarcinoma with FGFR2 gene fusions or other rearrangement as detected by an FDA approved test. Recommendation on Approval Regulatory Action Recommended For the treatment of adults with previously treated, Indication(s)/Population(s) unresectable locally advanced or metastatic (if applicable) cholangiocarcinoma with an FGFR2 fusion or other rearrangement as detected by an FDA‐approved test. -
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REVIEW OF OPTOMETRY EARN 2 CE CREDITS: Positive Visual Phenomena—Etiologies Beyond the Eye, PAGE 58 ■ VOL. 155 NO. 1 January 15, 2018 www.reviewofoptometry.comwww.reviewofoptometry.com ■ ANNUAL CORNEA REPORT JANUARY 15, 2018 ■ CXL ■ EPITHELIAL DEFECTS How to Heal Persistent Epithelial Defects PAGE 38 ■ TRANSPLANTS Corneal Transplants: The OD’s Role PAGE 44 ■ INFILTRATES Diagnosing Corneal Infiltrative Disease PAGE 50 ■ POSITIVE VISUAL PHENOMENA CXL: Your Top 12 Questions —Answered! PAGE 30 001_ro0118_fc.indd 1 1/5/18 4:34 PM ĊčĞĉėĆęĊĉĆĒēĎĔęĎĈĒĊĒćėĆēĊċĔėĎēǦĔċċĎĈĊĕėĔĈĊĉĚėĊĘ ĊđĎĊċĎēĘĎČčę ċċĊĈęĎěĊ Ȉ 1 Ȉ 1 ĊđđǦęĔđĊėĆęĊĉ Ȉ Ȉ ĎĒĕđĊĎēǦĔċċĎĈĊĕėĔĈĊĉĚėĊ Ȉ Ȉ ĔēěĊēĎĊēę Ȉ͝ Ȉ Ȉ Ƭ 1 ǡ ǡǡǤ͚͙͘͜Ǥ Ȁ Ǥ ͚͙͘͜ǣ͘͘ǣ͘͘͘Ǧ͘͘͘ ĕĕđĎĈĆęĎĔēĘ Ȉ Ȉ Ȉ Ȉ Ȉ čĊĚėĎĔē̾ėĔĈĊĘĘ Ȉ Ȉ Katena — Your completecomplete resource forfor amniotic membrane pprocedurerocedure pproducts:roducts: Single use speculums Single use spears ͙͘͘ǡ͘͘͘ήĊĞĊĘęėĊĆęĊĉ Forceps ® ,#"EWB3FW XXXLBUFOBDPNr RO0118_Katena.indd 1 1/2/18 10:34 AM News Review VOL. 155 NO. 1 ■ JANUARY 15, 2018 IN THE NEWS Accelerated CXL Shows The FDA recently approved Luxturna (voretigene neparvovec-rzyl, Spark Promise—and Caution Therapeutics), a directly administered gene therapy that targets biallelic This new technology is already advancing, but not without RPE65 mutation-associated retinal dystrophy. The therapy is designed to some bumps in the road. deliver a normal copy of the gene to By Rebecca Hepp, Managing Editor retinal cells to restore vision loss. While the approval provides hope for patients, wo new studies highlight the resulted in infection—while tradi- the $425,000 per eye price tag stands as pros and cons of accelerated tional C-CXL has a reported inci- a signifi cant hurdle. -
Neuro-Ophthalmic Side Effects of Molecularly Targeted Cancer Drugs
Eye (2018) 32, 287–301 © 2018 Macmillan Publishers Limited, part of Springer Nature. All rights reserved 0950-222X/18 www.nature.com/eye 1,2,3 4 Neuro-ophthalmic side MT Bhatti and AKS Salama REVIEW effects of molecularly targeted cancer drugs Abstract The past two decades has been an amazing time culminated in indescribable violence and in the advancement of cancer treatment. Mole- unspeakable death. However, amazingly within cularly targeted therapy is a concept in which the confines of war have risen some of the specific cellular molecules (overexpressed, greatest advancements in medicine. It is within mutationally activated, or selectively expressed this setting—in particular World War II with the proteins) are manipulated in an advantageous study of mustard gas—that the annals of cancer manner to decrease the transformation, prolif- chemotherapy began touching the lives of eration, and/or survival of cancer cells. In millions of people. It is estimated that in 2016, addition, increased knowledge of the role of the over 1.6 million people in the United States will immune system in carcinogenesis has led to the be diagnosed with cancer and over a half a development of immune checkpoint inhibitors million will die.1 The amount of money being to restore and enhance cellular-mediated anti- spent on research and development of new tumor immunity. The United States Food and cancer therapies is staggering with a record $43 Drug Administration approval of the chimeric billion dollars spent in 2014. Nearly 30% of all monoclonal antibody (mAb) rituximab in 1997 registered clinical trials on the clinicaltrials.gov 1Department of for the treatment of B cell non-Hodgkin lym- website pertain to cancer drugs. -
Rituximab Monotherapy and Rituximab-Containing
J Clin Exp Hematop Vol. 55, No. 2, Nov. 2015 Case Study Rituximab Monotherapy and Rituximab-Containing Chemotherapy Were Effective for Paraneoplastic Pemphigus Accompanying Follicular Lymphoma, but not for Subsequent Bronchiolitis Obliterans Taichi Hirano,1) Yusuke Higuchi,1) Hiromichi Yuki,1) Shinya Hirata,1) Kisato Nosaka,1) Norito Ishii,2) Takashi Hashimoto,2) Hiroaki Mitsuya,1) and Yutaka Okuno1) A 60-year-old male patient suffered from mild exertional dyspnea, wheezing, and systemic blisters. He was diagnosed with paraneoplastic pemphigus (PNP) with follicular lymphoma in the pancreas head and pelvic cavity. He was first treated with eight cycles of rituximab; his blisters and erosions gradually improved and highly elevated levels of auto-antibodies related to PNP gradually decreased to normal levels. However, obstructive and restrictive respiratory failure still progressed. Computed tomography of the inspiratory and expiratory phases revealed obstructive pulmonary disorder, leading to a diagnosis of bronchiolitis obliterans (BO). The patient underwent plasma exchange and was repeatedly treated with rituximab monotherapy and rituximab-containing chemotherapies, but died 7 months after the diagnosis of BO. Early introduction of rituximab- containing regimens may be necessary to prevent the development of BO accompanying PNP. However, when a diagnosis of PNP-related BO is made, lung transplantation may also be considered for patients in whom rituximab-containing regimens are effective for PNP. 〔J Clin Exp Hematop 55(2) : 83-88, 2015〕 Keywords: follicular lymphoma, paraneoplastic pemphigus, bronchiolitis obliterans, rituximab Without control of the malignant neoplasms, resolution of the INTRODUCTION skin blisters is difficult and requires high doses of cortico- Paraneoplastic pemphigus (PNP) is a systemic autoim- steroids and immunosuppressive drugs. -
Eyelash-Enhancing Products: a Review Bishr Al Dabagh, MD; Julie Woodward, MD
Review Eyelash-Enhancing Products: A Review Bishr Al Dabagh, MD; Julie Woodward, MD Long prominent eyelashes draw attention to the eyes and are considered a sign of beauty. Women strive to achieve ideal lashes through various modalities, including cosmetics, cosmeceuticals, and pharmaceu- ticals. The booming beauty industry has introduced many cosmetic and cosmeceutical products with claims of enhancing eyelash growth; however, many of these products have not been tested for efficacy or safety and are promoted solely with company- and/or consumer-based claims. The only pharmaceu- tical approved by the US Food and Drug Administration for eyelash growth is bimatoprost ophthalmic solution 0.03% (Latisse, Allergan, Inc). In this article, eyelash physiology, causes of genetic and acquired trichomegaly, and pharmaceutical and cosmeceutical products that claim eyelash-enhancing effects are reviewed. COS DERMCosmet Dermatol. 2012;25:134-143. yelashes decorate the eyes and crystallize cosmeceuticals contain active ingredients that are intended the beauty of the face.1 Long and lush eye- to produce beneficial physiologic effects through medicinal Dolashes in particular Notare considered a sign properties. Copy3,4 Cosmeceuticals fall in the gray area between of beauty. Women strive to achieve more inert cosmetics and pharmaceuticals. Drugs are defined pronounced eyelashes using a variety of tech- by the FDA as “articles intended for use in the diagnosis, niques.E Cosmetics are the mainstay of eyelash enhance- cure, mitigation, treatment, or prevention -
Paraneoplastic Pemphigus: an Unusual Neoplasm Association
erimenta xp l D E e r & m l a a t c o i l n o i Journal of Clinical & Experimental Toosi et al. J Clin Exp Dermatol Res 2011, 2:4 l g y C f R DOI: 10.4172/2155-9554.1000124 o e l ISSN: 2155-9554 s a e n a r r u c o h J Dermatology Research Case Report Open Access Paraneoplastic Pemphigus: An Unusual Neoplasm Association Parviz Toosi1, Zahra Asadi Kani2, Mehdi Qeisari2 and Nastaran Namazi3* 1Professor of dermatology, Skin research center, Shohad-e-Tajrish hospital, Shahid Beheshti university of medical science, Tehran, Iran 2Dermatopathologist, Skin research center, Shohad-e-Tajrish hospital, Shahid Beheshti university of medical science, Tehran, Iran 3Resident of dermatology, Skin research center, Shohad-e-Tajrish hospital, Shahid Beheshti university of medical science, Tehran, Iran Abstract The occurrence of paraneoplastic pemphigus (PNP) in childhood has been reported infrequently. Association with benign mesentry tumors has not been reported in these patients before. Here, we report an unusual case of PNP in a child that was concomitant with benign mesentry fibroma and responded to medical treatment only after surgical excision of tumor. Keywords: Paraneoplastic pemphigus; Neoplasm association; Here we report an unusual case of childhood paraneoplastic Benign tumor; Mesentry fibroma pemphigus in association with benign mesocolon fibroma that responded to tumor excision dramatically. Introduction Case Report PNP, described by Anhalt et al. in 1990, is an IgG-mediated disease, characterized clinically by polymorphous eruption with prominent A 12 year old girl was referred to our dermatology clinic because mucosal and acral involvement in the presence of a known or occult of painful mucosal erosions from one month ago, made her unable neoplasm. -
Ocular Side Effects of Novel Anti-Cancer Biological Therapies
www.nature.com/scientificreports OPEN Ocular side efects of novel anti‑cancer biological therapies Vicktoria Vishnevskia‑Dai1*, Lihi Rozner1, Raanan Berger2,3, Ziv Jaron1, Sivan Elyashiv1, Gal Markel2,3 & Ofra Zloto1 To examine the ocular side efects of selected biological anti‑cancer therapies and the ocular and systemic prognosis of patients receiving them. We retrospectively reviewed all medical records of patients who received biological anti‑cancer treatment from 1/2012 to 12/2017 and who were treated at our ocular oncology service. The following data was retrieved: primary malignancy, metastasis, type of biological therapy, ocular side efects, ophthalmic treatment, non‑ocular side efects, and ocular and systemic disease prognoses. Twenty‑two patients received biological therapies and reported ocular side efects. Eighteen patients (81.8%) had bilateral ocular side efects, including uveitis (40.9%), dry eye (22.7%), and central serous retinopathy (22.7%). One patient (4.5%) had central retinal artery occlusion (CRAO), and one patient (4.5%) had branch retinal vein occlusion (BRVO). At the end of follow‑up, 6 patients (27.27%) had resolution of the ocular disease, 13 patients (59.09%) had stable ocular disease, and 3 patients (13.64%) had progression of the ocular disease. Visual acuity improved signifcantly at the end of follow‑up compared to initial values. Eighteen patients (81.8%) were alive at study closure. Biological therapies can cause a wide range of ocular side efects ranging from dry eye symptoms to severe pathologies that may cause ocular morbidity and vision loss, such as uveitis, CRAO and BRVO. All patients receiving biological treatments should be screened by ophthalmologists before treatment, re‑screened every 4–6 months during treatment, and again at the end of treatment. -
Case Report Follicular Lymphoma with Paraneoplastic Pemphigus As the First Symptom: a Case Report and Review of the Literature
Int J Clin Exp Pathol 2020;13(7):1915-1923 www.ijcep.com /ISSN:1936-2625/IJCEP0112784 Case Report Follicular lymphoma with paraneoplastic pemphigus as the first symptom: a case report and review of the literature Shishou Wu1,2, Dong Gao3, Yuanfeng Zhang4, Ping Yang2, Yunjun Wang1,2, Ning Wang2, Jianfeng Xu5, Guohua Yu1,2 1Department of Clinical Medicine, Binzhou Medical University, Yantai, China; Departments of 2Pathology, 3Dermo- tology, 4Hematology, 5Respiratory Medicine, Affiliated Yantai Yuhuangding Hospital, Qingdao University, Yantai, China Received April 18, 2020; Accepted June 3, 2020; Epub July 1, 2020; Published July 15, 2020 Abstract: Paraneoplastic pemphigus (PNP) is an autoimmune bullous dermatosis associated with tumors, first de- scribed by Anhalt et al. in 1990. Reports of paraneoplastic pemphigus complicated by follicular lymphoma (FL) are rare in the medical literature. Here, we retrospectively analyze a case of PNP accompanied by FL. The patient was a 54-year-old woman who suffered from PNP associated with FL at the beginning. She had received a pathological diagnosis and was treated with R-CHOP and other drugs. Her mucosal lesions and cutaneous lesions improved, and the FL was in remission. Eleven months later, she died of BO after receiving the diagnosis of PNP. We also review most of the studies and reports about PNP accompanied by FL. We list the clinicopathologic features, therapeutic schedule, and prognosis in order to improve hematologists’ understanding and treatment of the diseases. Keywords: Follicular lymphoma, paraneoplastic pemphigus, bronchiolitis obliterans Introduction Bronchiolitis obliterans (BO) is a life-threaten- ing form of irreversible, obstructive lung dis- PNP is a rare paraneoplastic, systemic autoim- ease. -
S2 Table. List of Syntax for 96 Diseases
S2 Table. List of syntax for 96 diseases 'autoimmune gastritis'/exp OR 'acantholysis'/exp OR 'acantholysis' OR 'acute disseminated encephalomyelitis'/exp OR 'adem (acute disseminated encephalomyelitis)' OR 'acute disseminated encephalitis' OR 'acute disseminated encephalomyelitis' OR 'encephalitis postvaccinalis' OR 'encephalitis, post-vaccinal' OR 'encephalomyelitis, acute disseminated' OR 'post vaccinal encephalitis' OR 'post vaccination encephalitis' OR 'post-infectious encephalitis' OR 'post-infectious encephalomyelitis' OR 'postinfection encephalitis' OR 'postinfectious encephalitis' OR 'postinfectious encephalomyelitis' OR 'postvaccinal encephalitis' OR 'postvaccinal encephalopathy' OR 'postvaccination encephalitis' OR 'postvaccine encephalitis' OR 'postvaccinial encephalitis' OR 'postvaccinial encephalomyelitis' OR 'smallpox vaccination encephalitis' OR 'vaccinal encephalitis' OR 'vaccination encephalopathy' OR 'vaccination post vaccinial encephalitis' OR 'vaccinia encephalitis' OR 'addison disease'/exp OR 'addison disease' OR 'addison`s disease' OR 'addisons disease' OR 'addison biermer disease' OR 'adult onset still disease'/exp OR 'adult onset still disease' OR 'still`s disease, adult- onset' OR 'allergic glomerulonephritis'/exp OR 'allergic glomerulonephritis' OR 'glomerulonephritis, allergic' OR 'glomerulonephritis, poststreptococcal' OR 'post streptococcal glomerulonephritis' OR 'poststreptococcal glomerulonephritis' OR 'anca associated vasculitis'/exp OR 'anca associated vasculitis' OR 'anca vasculitis' OR 'anca-associated -
Paraneoplastic Pemphigus Presenting Like Toxic Epidermal Necrolysis Huyenian Nguyen DO Lehigh Valley Health Network, [email protected]
Lehigh Valley Health Network LVHN Scholarly Works Department of Medicine Paraneoplastic Pemphigus presenting like Toxic Epidermal Necrolysis Huyenian Nguyen DO Lehigh Valley Health Network, [email protected] Kelly L. Reed DO Lehigh Valley Health Network, [email protected] Steven Oberlender MD, PhD Lehigh Valley Health Network, [email protected] Stephen M. Purcell DO Lehigh Valley Health Network, [email protected] Follow this and additional works at: http://scholarlyworks.lvhn.org/medicine Part of the Dermatology Commons, and the Medical Sciences Commons Published In/Presented At Nguyen, H., Reed, K., Oberlender, S., & Purcell, S. (2016, March 18). Paraneoplastic Pemphigus presenting like Toxic Epidermal Necrolysis. Poster presented at Philadelphia Dermatological Society, Philadelphia, PA. Nguyen, H., Reed, K., Oberlender, S., & Purcell, S. (2016, April 26). Paraneoplastic Pemphigus presenting like Toxic Epidermal Necrolysis. Poster presented at LVHN Department of Medicine Research Day, Lehigh Valley Health Network, Allentown, PA. This Poster is brought to you for free and open access by LVHN Scholarly Works. It has been accepted for inclusion in LVHN Scholarly Works by an authorized administrator. For more information, please contact [email protected]. Paraneoplastic Pemphigus presenting like Toxic Epidermal Necrolysis Huyenlan Nguyen, DO, Kelly Reed, DO, Steven Oberlender, MD, PhD, and Stephen M. Purcell, DO Lehigh Valley Health Network, Allentown, Pennsylvania Case Presentation: Discussion: Patient: -
Cutaneous Manifestations of Internal Malignancies in a Tertiary Health
736 ESPECIAL L Cutaneous manifestations of internal malignancies in a tertiary health care hospital of a developing country* Manifestações cutâneas de doenças malignas em um hospital terciário de um país em desenvolvimento Alex G. Ortega-Loayza 1 Willy Ramos 2 Ericson L. Gutierrez 3 Patricia Chavez de Paz 4 Lucia Bobbio 5 Carlos Galarza 6 Abstract: In a public hospital in Lima, Peru, 24 patients with 16 types of paraneoplastic dermatoses were iden- tified by data collection. The most frequent dermatosis was dermatomyositis (four patients). The other derma- toses were malignant acanthosis nigricans, palmoplantar keratoderma, bullous dermatoses, lymphomatoid papulosis, edematous scarring vasculitic panniculitis, Norwegian scabies, primary systemic amyloidosis, necrolytic migratory erythema, infective dermatitis, pancreatic panniculitis, generalized pruritus, Lesser-Trelat syndrome, and acquired ichthyosis. Most of these paraneoplastic dermatoses were diagnosed before (45.8%) or at the time of (38.5%) the diagnosis of the underlying malignancy. The most frequent underlying malignan- cies were lymphoma, adenocarcinomas of the upper digestive tract, and malignant neoplasms of the pancreas. The average age of the patients was 47.0 ± 16.9 years and the length of the disease since diagnosis was 13.7 months. The mortality rate was 75%. Paraneoplastic dermatoses are rare dermatologic entities that are difficult to diagnose. Surveillance is also hampered when patients do not have easy access to health care centers due to financial and geographical issues. However, when identified, they might facilitate the early diagnosis of an associated tumor and contribute to increase the surveillance of patients. Keywords: Dermatomyositis; Lymphoma; Paraneoplastic syndromes Resumo: Em um hospital público em Lima, Peru, 24 pacientes com 16 tipos de dermatoses paraneoplá- sicas foram identificados por meio de coleta de dados.