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1

Barakat, Mohammed M. A., Kadir Ertem, and Hüseyin Utku Özdeş. "An Uncommon Mass in The Distal Ulna; A Case of Atypical Osteoid Osteoma." European Journal of Therapeutics 31, no. 1 (2025): 67–70. https://doi.org/10.58600/eurjther2595.

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Osteoid osteoma (OO) is a benign osteoblastic tumor that commonly occurs in the diaphysis of long bones but is rarely found in peri-articular locations, particularly the wrist. This case report describes a 19-year-old female who presented with chronic wrist pain lasting two years, accompanied by nocturnal exacerbation and tenderness over the distal ulna. Imaging revealed a radiolucent lesion with a nidus, leading to a diagnosis of OO, later confirmed histologically following surgical excision. Differential diagnoses such as ulnar impingement syndrome, triangular fibrocartilage complex (TFCC) i
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2

Lee, Jong Wook, Jun Ho Jang, Je-Hwan Lee, et al. "Ineffective Corticosteroid Treatment for Hemolysis Management of Paroxysmal Nocturnal Hemoglobinuria." Blood 124, no. 21 (2014): 5151. http://dx.doi.org/10.1182/blood.v124.21.5151.5151.

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Abstract Background: The primary clinical manifestations of paroxysmal nocturnal hemoglobinuria (PNH) are hemolytic anemia, bone marrow failure (BMF), and thromboembolism (TE). For optimum management, the contribution of both hemolysis and BMF to the complex anemia of PNH should be determined. The treatment of a hemolytic episode should aim at diminishing hemolysis and preventing complications. Corticosteroids as treatment, for both chronic hemolysis and acute hemolytic exacerbations have been used with a variety of side effects of long term use. In the Korean PNH population, corticosteroid (7
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3

Kwon, Byung Jin, Hyung Wook Kim, Su Bum Park, et al. "Paroxysmal Nocturnal Hemoglobinuria Presenting with Chronic Abdominal Pain and Iron Deficiency Anemia." Korean Journal of Medicine 95, no. 1 (2020): 56–60. http://dx.doi.org/10.3904/kjm.2020.95.1.56.

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Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired disorder of hematopoietic stem cells characterized by chronic intravascular hemolysis, nocturnal hemoglobinuria, thromboembolic events and secondary bone marrow failure caused by uncontrolled complement activation. Generally, chronic abdominal pain and iron deficiency anemia are considered typical symptoms of gastrointestinal diseases, but are also common in PNH. We report a case of PNH presenting with chronic abdominal pain and iron deficiency anemia, along with a relevant literature review.
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4

Arnold, Louise M., Jill Stephenson, Richard Kelly, David Buchanan, Gareth Jones, and Peter Hillmen. "Home Infusion of Eculizumab: A Unique and Innovative Model of Drug Delivery to Reduce Treatment-Associated Burden and Enhance Quality of Life for Patients with PNH." Blood 112, no. 11 (2008): 4671. http://dx.doi.org/10.1182/blood.v112.11.4671.4671.

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Abstract Paroxysmal Nocturnal Hemoglobinuria (PNH) is an acquired clonal stem cell disease, characterised by intravascular hemolysis, bone marrow failure and lifethreatening thromboses. The median survival is 10–15 years, with the average age of presentation being in the 30’s. Symptoms include hemoglobinuria, fatigue, anemia, venous and arterial thromboses, recurrent pain, renal impairment, erectile dysfunction and pulmonary hypertension. The care of a patient with PNH is complex and challenging, as many experience chronic symptoms with periods of acute exacerbations. Historically the manageme
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5

Hussein, Naglaa. "Autoimmune hepatitis complicated with entrapment neuropathy & fibromyalgia: A case report." International Physical Medicine & Rehabilitation Journal 7, no. 2 (2022): 76–77. http://dx.doi.org/10.15406/ipmrj.2022.07.00308.

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46. y-old right-handed female with history of controlled autoimmune hepatitis since 2001 with negative liver biopsy on 2017 presented with chronic generalized pain affecting neck, upper back, buttocks, knee and chest associated with paresthesia and tingling of left hand with nocturnal exacerbation. Clinical exam revealed; Neurologically Positive Tinel sign over median nerve at wrist bilaterally, Positive Phalen test bilaterally, Musculoskeletal exam revealed multiple tender points bilaterally with limited neck Rom due to pain. Electrophysiological testing of both upper extremities documented b
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6

Amendola, A., G. Paternoster, S. P. Pascale, et al. "Coronary Artery By-Pass Grafting in Patient With Paroxysmal Nocturnal Hemoglobinuria (Case Report)." General Reanimatology 17, no. 2 (2021): 27–36. http://dx.doi.org/10.15360/1813-9779-2021-2-27-36.

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Paroxysmal nocturnal haemoglobinuria (PNH) is a clonal haematopoietic stem cell disease that presents with haemolytic anaemia, thrombosis and bone marrow failure. We report a case of a 51-year-old male with a history of PNH in treatment with Eculizumab admitted to our Hospital for acute chest pain and dyspnoea. The diagnosis was a triple vessel disease and patient was scheduled for coronary artery bypass grafting surgery. To balance the risk between thrombosis and bleeding in this particular clinical setting, we decided to use thromboelastography (TEG) as point of care solution and we used the
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7

Brodsky, Andres L., Brenner Sabando Velez, and Curutchet Ragusin. "Eculizumab Treatment of Paroxysmal Nocturnal Hemoglobinuria Relapsing After Bone Marrow Transplant and Subsequent Clonal: Case Report." Blood 118, no. 21 (2011): 5274. http://dx.doi.org/10.1182/blood.v118.21.5274.5274.

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Abstract Abstract 5274 Background: Paroxysmal nocturnal hemoglobinuria (PNH) is a chronic and life-threatening hematopoietic stem cell disorder characterized by deficiency of the GPI-anchored complement inhibitory proteins CD55 and CD59 on blood cells. The resulting uncontrolled complement activation is responsible for chronic hemolysis and can lead to serious clinical morbidities including thromboembolism (TE) and chronic kidney disease (CKD), which have been shown to increase risk of mortality. Patients may also experience debilitating quality-of-life (QoL) issues, including fatigue, shortne
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8

Easwar, V. Ra Rathina, R. Navaneethakrishnan, and Yogeshwar Agharkar. "A Case Report of Acute on Chronic Osteomyelitis of Distal Femur Managed with Sequestrectomy, Saucerization, and Stimulan Placement." Journal of Orthopaedic Case Reports 15, no. 7 (2025): 163–67. https://doi.org/10.13107/jocr.2025.v15.i07.5808.

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Introduction: Osteomyelitis is a bone infection that may present acutely or chronically. Acute on chronic osteomyelitis refers to the exacerbation of symptoms in a patient with an underlying chronic infection. This case report presents a 19-year-old male diagnosed with acute on chronic osteomyelitis of the right distal femur, with no history of a discharging sinus for the past 6 months. Osteomyelitis is a bone infection commonly caused by bacteria, with Staphylococcus aureus being the most frequent pathogen. Chronic osteomyelitis can occasionally experience acute flare-ups, referred to as “acu
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9

Takimot, Kayo. "Serratus Plane Block for Persistent Pain after Partial Mastectomy and Axillary Node Dissection." March 2016 3;19, no. 3;3 (2016): E481—E486. http://dx.doi.org/10.36076/ppj/2019.19.e481.

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Persistent pain after breast cancer surgery (PPBCS) is defined as chronic neuropathic pain that persists for more than 3 months after surgery. The pain can be sufficiently severe to cause long-term disabilities and interfere with sleep and daily life. Serratus plane block (SPB) is a novel, ultrasound-guided regional anesthetic technique that is suggested to achieve complete anesthesia of the anterolateral chest wall. Here, we demonstrate the efficacy of SPB as one of the treatment modalities for patients with PPBCS. A 73-year-old woman underwent a left partial mastectomy and axillary node diss
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10

Salar, O., B. Baker, T. Kurien, A. Taylor, and C. Moran. "Septic arthritis in the era of immunosuppressive treatments." Annals of The Royal College of Surgeons of England 96, no. 2 (2014): e11-e12. http://dx.doi.org/10.1308/003588414x13814021678196.

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Immunosuppressants have been the mainstay of treatment for certain inflammatory joint conditions for many years. Developments in this field, namely biological treatments, have led to a change in the classical presentation of acute bone, joint and soft tissue infections. The normal findings of severe pain and tenderness on examination may be absent or simply mimic a typical exacerbation of the chronic joint condition. A minimally raised white cell count and elevated C-reactive protein in the absence of systemic signs of infection may be interpreted as further evidence for the diagnosis of an ex
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11

Griesser, Christina, Michael Myskiw, and Werner Streif. "Paroxysmal Nocturnal Hemoglobinuria: An Underestimated Cause of Pediatric Thromboembolism." TH Open 04, no. 01 (2020): e36-e39. http://dx.doi.org/10.1055/s-0040-1702155.

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AbstractParoxysmal nocturnal hemoglobinuria (PNH) is a chronic disease caused by complement-mediated hemolysis. Clinical symptoms include intravascular hemolysis, nocturnal hemoglobinuria, thromboses, cytopenia, fatigue, abdominal pain, and a strong tendency toward bone marrow failure. It is a rare disease, especially in children, with high mortality rates without appropriate treatment.We here present the case of a 17-year-old girl with unprovoked muscle vein thrombosis. Flow cytometric analysis showed deficiency of glycosyl-phosphatidylinositol-anchored membrane proteins on all three hematopo
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12

Jarolia, Mansingh, Nitin Chauhan, Sai Krishna M L V, and Shivanand Gamangatti. "Great Toe Osteoid Osteoma with Atypical Presentation: A Case Report." Journal of Orthopaedic Case Reports 13, no. 12 (2023): 44–47. http://dx.doi.org/10.13107/jocr.2023.v13.i12.4072.

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Introduction: Osteoid osteoma is a common benign osteoblastic lesion of the bone. Although it commonly affects the cortex of long bones, it rarely involves the cancellous portion of the small bones such as phalanges. The occurrence of osteoid osteoma in phalanges poses a diagnostic dilemma. Case Report: In our case report, we presented a 26-year-old patient with long-standing pain over the great toe. The radiographs and magnetic resonance imaging (MRI) were unable to diagnose the lesion, and a single-photon emission computerized tomography scan picked up the nidus. She underwent radiofrequency
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13

Zhao, Yongdong, Fatma Dedeoglu, Polly J. Ferguson, et al. "Physicians’ Perspectives on the Diagnosis and Treatment of Chronic Nonbacterial Osteomyelitis." International Journal of Rheumatology 2017 (2017): 1–7. http://dx.doi.org/10.1155/2017/7694942.

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Background/Purpose. Understanding the practices of pediatric rheumatologists in diagnosing and treating chronic nonbacterial osteomyelitis (CNO) can provide important information to guide the development of consensus treatment plans. The objectives of this study were to determine physicians’ approaches to (1) diagnosing and monitoring CNO, (2) ordering a bone biopsy, and (3) making treatment decisions. Methods. A survey was distributed among members of the Childhood Arthritis and Rheumatology Research Alliance using a web-based questionnaire. Results. 121 of 277 (41%) attending physician membe
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14

Chazan, RN, Shoshana, Margaret P. Ekstein, MD, Nissim Marouani, MD, and Avi A. Weinbroum, MD. "Ketamine for acute and subacute pain in opioid-tolerant patients." Journal of Opioid Management 4, no. 3 (2018): 173. http://dx.doi.org/10.5055/jom.2008.0023.

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Prolonged acute pain, especially that of oncologic neurological origin, is at times difficult to control; it is seldom entirely alleviated by opioids. We report eight patients with severe pain, three of whom suffered from new onset oncologic metastatic bone pain, others had previous pain syndromes and presented with exacerbation of pain. Pain was associated with hyperalgesia and allodynia phenomena in two patients and with phantom pain in a third one. Tolerance to opioids had developed, and high IV doses of morphine, meperidine or fentanyl, and patient-controlled intravenous and epidural analg
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15

Kumar, Surender, Uma Garg, Naveen Sharma, Neha Salaria, and Deepak Verma. "Squamous cell carcinoma of tympano-mastoid region: a series of six cases." International Journal of Otorhinolaryngology and Head and Neck Surgery 4, no. 3 (2018): 874. http://dx.doi.org/10.18203/issn.2454-5929.ijohns20181891.

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<p class="abstract">The malignancies of tympanomastoid region are very rare entity. These tumours are more common in elderly persons in 6th and 7th decade. Males are more commonly affected than females. Chronically discharging ears are considered as risk factor which may be due to metaplasia in the middle ear mucosa following prolonged chronic infection. The most common symptoms are long standing blood tinged ear discharge, severe nocturnal pain, rapidly growing polypoidal or granulomatous mass in EAC or middle ear, peripheral facial palsy and painless ulceration over pinna or EAC. CT sc
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16

Valeri, Federica, Alessandra Borchiellini, Piercarla Schinco, and Mario Boccadoro. "Paroxysmal Nocturnal Hemoglobinuria With Budd-Chiari Syndrome Treated With Complement Inhibitor Eculizumab; A Case Report." Blood 122, no. 21 (2013): 4800. http://dx.doi.org/10.1182/blood.v122.21.4800.4800.

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Introduction Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired haemolytic anaemia caused by somatic mutation in the phosphatidylinositol glycan-complementation class A gene, resulting in absence of a key complement regulatory protein, CD59. Thrombosis occurs in up to 40% of PNH patients; it usually involves abdominal and cerebral veins and it is the leading cause of death disease related. Methods We describe the response to Eculizumab (Soliris, Alexion) in 28 years old male with PNH diagnosed as a consequence of Budd Chiari Syndrome, acute liver dysfunction, mild haemolytic anaemia
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17

Danilov, A. B., A. A. Pilipovich, and M. V. Pyastolova. "NOVEMA® NIGHT (diphenhydramine + naproxen) in patients with pain and insomnia: results of a multicenter non-interventional observational study." Neurology, Neuropsychiatry, Psychosomatics 15, no. 6 (2023): 56–63. http://dx.doi.org/10.14412/2074-2711-2023-6-56-63.

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Pain and sleep disorders are interrelated problems that significantly affect patients’ quality of life (QoL) and daily functioning.Objective: to evaluate the efficacy and safety of the use of the combination of diphenhydramine + naproxen (NOVEMA® NIGHT) in patients with acute pain syndrome or exacerbation of chronic pain syndrome and sleep disorders.Material and methods. The study included 4365 outpatients with acute pain (musculoskeletal pain, post-traumatic pain, headache) and sleep disorders who took naproxen 275 mg, 1 tablet in the morning and diphenhydramine 25 mg + naproxen 220 mg (NOVEM
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18

Stimpson, S. A., F. G. Dalldorf, I. G. Otterness, and J. H. Schwab. "Exacerbation of arthritis by IL-1 in rat joints previously injured by peptidoglycan-polysaccharide." Journal of Immunology 140, no. 9 (1988): 2964–69. http://dx.doi.org/10.4049/jimmunol.140.9.2964.

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Abstract The arthropathic activity of mouse recombinant IL-1 (mrIL-1) after intraarticular (i.a.) injection into rat ankles was investigated. Nanogram quantities of either mrIL-1 alpha or mrIL-1 beta induced an acute transient arthritis. Arthritis induced by i.a. mrIL-1 developed more rapidly and was more severe in ankles previously injured by i.a. injection of group A streptococcal peptidoglycan-polysaccharide (PG-APS) fragments. In addition, a protracted pain response, as judged by severe limping, occurred 60 to 90 min after mrIL-1 injection into joints previously injured by PG-APS or 4 to 6
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19

Cotorogea-Simion, Mihail, Sebastian Isac, Alina Tita, et al. "Mesenteric Ischemia in a Patient with Essential Thrombocythemia: Does COVID-19 Play Any Role? A Case Report and Overview of the Literature." Medicina 58, no. 9 (2022): 1147. http://dx.doi.org/10.3390/medicina58091147.

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Introduction: Chronic mesenteric ischemia is a rare entity with non-specific symptomatology; combined with rare etiologies, it could lead to unwarranted surgical indication. Case report: We report the case of an 85-year-old woman, with a history of hypertension, persistent thrombocytosis, atherosclerosis, and recent minor COVID-19 infection, presenting to the hospital with postprandial abdominal pain and nonspecific clinical examination findings; upon abdominal CT, superior mesenteric artery circumferential thrombosis was revealed. A bone marrow biopsy was performed due to suspected essential
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20

Bakirov, B. A., D. A. Kudlay, and V. N. Pavlov. "Eculizumab in the treatment of complement system disorders including paroxysmal nocturnal hemoglobinuria." Pediatric Hematology/Oncology and Immunopathology 19, no. 2 (2020): 193–99. http://dx.doi.org/10.24287/1726-1708-2020-19-2-193-199.

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The main function of the complement system is to provide humoral defence against foreign pathogens. It contributes to immune response and is a crucial component of innate immunity that provides immediate non-specific immune defence. Inherited or acquired deficiencies of the complement system associated with excessive activation or other impairments of complement activity have varied clinical manifestations. Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired clonal blood disorder that clinically manifests with anemia, thrombosis, chest and abdominal pain, chronic kidney disease and bo
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21

Maradiaga, Nidia C., Calvin Pohl, Emily Mackey, and Adam J. Moeser. "Early Life Adversity Programs Mast Cells Toward a Hyperactive Phenotype into Adulthood." Journal of Immunology 202, no. 1_Supplement (2019): 54.2. http://dx.doi.org/10.4049/jimmunol.202.supp.54.2.

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Abstract Early life adversity (ELA) is a risk factor in the onset and exacerbation of inflammatory disorders including allergy, asthma and chronic pain disorders such as irritable bowel syndrome (IBS). Our previous studies using murine and porcine models showed that mast cells (MCs), a key immune cell driving allergies and chronic pain disorders, display a hyperactive tissue phenotype in adult animals exposed to ELA. The mechanism by which ELA induces MC hyperactivity in adulthood remains unknown. The aim of this study was to determine if ELA programs bone marrow derived MC progenitors toward
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22

Murakami, Yoshiko, Michi Kawamoto, Norimitsu Inoue, et al. "Paroxysmal Nocturnal Hemoglobinuria Caused By Pigt Mutations; Atypical PNH." Blood 128, no. 22 (2016): 2450. http://dx.doi.org/10.1182/blood.v128.22.2450.2450.

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Abstract Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired GPI-anchor deficiency caused by the somatic mutation of PIGA gene in a hematopoietic stem cell (PIGA-PNH). Loss of function of PIGA causes loss of GPI anchored proteins including complement regulatory proteins. Main symptoms are hemolytic anemia and venous thrombosis caused by the uncontrolled complement activation, and bone marrow failure. For X-linked PIGA, one hit of somatic mutation causes GPI-deficiency. All other genes involved in GPI-anchor biosynthetic pathway are autosomal and hits to two alleles are required to cause G
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23

Oliveira, Simone, and Miguel Meira e. Cruz. "0832 ABOUT CLOCKS, SLEEP, PAIN AND THEIR INTERACTIVE ROLE IN TMJ OSTEOARTHIRIS - A CASE REPORT." SLEEP 46, Supplement_1 (2023): A366—A367. http://dx.doi.org/10.1093/sleep/zsad077.0832.

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Abstract Introduction As a major cause of disability among older people, Temporomandibular Joint Osteoarthritis (OA) remain a critical issue for both geriatric medicine and public health. While etiologic foundations of this condition are still unclear, it is well known that 24h-driven biological (circadian) rhythms are basic physiological requirements for the homeostatic control of peripheral tissues, such as skeletal muscle, bone and particularly cartilage. Sleep, as the most conspicuous circadian rhythm rooling health and welbeing, is also relevant for anabolic and catabolic processes within
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24

Yamakawa, Patricia Eiko, Ana Rita Da Fonseca, Iara Baldim Rabelo Gomes, et al. "Clinical Characteristics of Brazilian Patients with Paroxysmal Nocturnal Hemoglobinuria and Changing Prognosis with Eculizumab." Blood 134, Supplement_1 (2019): 2222. http://dx.doi.org/10.1182/blood-2019-132023.

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Introduction: Paroxysmal nocturnal hemoglobinuria (PNH) is a nonmalignant clonal disease of hematopoietic cells due to acquired mutations in the phosphatidylinositol glycan class A (PIG-A) gene, which is required for glycosylphosphatidylinositol (GPI) anchor biosynthesis. This leads to partial or complete absence of all GPI-linked proteins, who are complement regulatory proteins, resulting in an increased sensitivity of the red blood cells to the action of complement. PNH is characterized by signs and symptoms related to intravascular hemolysis, hypercoagulability state, and varying degrees of
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Alashkar, Ferras, Haemi Schemuth, Felix Nensa, Ulrich Dührsen, Thomas Wilfried Schlosser, and Alexander Röth. "The Role of Whole-Body Magnetic Resonance Imaging (WB-MRI) in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)." Blood 128, no. 22 (2016): 1271. http://dx.doi.org/10.1182/blood.v128.22.1271.1271.

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Abstract INTRODUCTION: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare clonal blood disorder of the hematopoietic stem cells characterized by chronic, uncontrolled complement-mediated intravascular hemolysis resulting in thrombophilia with thromboembolic events (TEs) and cytopenia due to bone marrow failure. PNH is the most vicious acquired thrombophilic state and TEs represent the leading cause of morbidity and mortality in PNH. PNH clone size correlates with the possibility to acquire a thrombosis but thromboses are also present in patients (pts) with small clones. Early detection of TEs
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Zadeh, M. Hossein, A. Sadeghipoor, A. Saadat, and M. Keyhani. "Marked Bone Marrow Necrosis Proceeding Acute Monoblasic Leukemia." Blood 106, no. 11 (2005): 4562. http://dx.doi.org/10.1182/blood.v106.11.4562.4562.

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Abstract Bone marrow necrosis (BMN) is a rare antemortem diagnosis with obscure ethiology. This entity is diagnosed mostly at postmortem examination, but it is also seen during the course of different diseases, mostly malignant.It is characterized morphologically by destruction of hemopoeitic tissue, including the stroma, with preservation of the bone.Conditions associated with BMN include infections, acute and chronic leukemia, Hodgkin’s and non-Hodgkin’s lymphoma, primary thrombocythemia, and metastatic carcinoma. It has also been reported in the presence of antiphospholipid antibodies, prev
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27

McNeil, Casey, Alma Habib, Hayrettin Okut, Sheryl Beard, Elizabeth Ablah, and Stephanie Hassouneh. "Administration of and Prescribing Opioids in Emergency Departments: A Retrospective Study." Kansas Journal of Medicine 14 (January 21, 2021): 1–4. http://dx.doi.org/10.17161/kjm.vol1413368.

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Opioid overdose was a cause of 42,249 deaths in the United States in 2016 (13.3 deaths per 100,000) and contributed to 67.8% of all drug overdose deaths in the USA in 2017.1,2 The rate of drug overdose resulting in death in Kansas in 2016 was 11.8 per 100,000, (333 total drug overdose deaths).2 Emergency departments (EDs) are a key intermediary in opioid prescriptions. In 2010, 31% of ED visits nationally resulted in an opioid prescription.3
 The number of opioid prescriptions from an ED varies greatly even for a single medical indication. For example, states varied from 40% to 2.8% of pa
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Shammo, Jamile, Sushupta M. Vijapur, Josefin Snellman, and Jilles M. Fermont. "Disease Management and Outcomes in Patients with Paroxysmal Nocturnal Hemoglobinuria: A Retrospective Analysis of Observational Data from the United States." Blood 142, Supplement 1 (2023): 5639. http://dx.doi.org/10.1182/blood-2023-179896.

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Background: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hemolytic disease characterized by complement-mediated hemolysis, thrombophilia, bone marrow failure (BMF), and renal disease. PNH is clinically heterogeneous, and hospitalization and regular blood transfusions may be needed. Treatment with currently approved C5 inhibitors (C5i), eculizumab and ravulizumab, have shown efficacy in intravascular hemolysis control and reduction in thrombosis risk. Yet, in routine clinical practice, a limited number of patients receive C5i in the United States. Moreover, less than 70% of patients trea
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Muus, Petra, Antonio M. Risitano, H. R. Castro-Malaspina, C. Michael Jones, Stephen Fuller, and Gerard Socie. "Clinical Impact of Unregulated Terminal Complement Activity in Never-Transfused Patients with Paroxysmal Nocturnal Hemoglobinuria." Blood 114, no. 22 (2009): 4029. http://dx.doi.org/10.1182/blood.v114.22.4029.4029.

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Abstract Abstract 4029 Poster Board III-965 Paroxysmal nocturnal hemoglobinuria (PNH) is a debilitating and life-threatening hematopoietic stem cell disorder characterized by unregulated activation of the complement system leading to chronic intravascular hemolysis and an inflammatory prothrombotic state. PNH evolves from the clonal expansion of hematopoietic stem cells with complete or marked loss of terminal complement inhibitors CD55 and CD59 from the surface of hematopoietic cells, rendering red blood cells susceptible to terminal complement-mediated hemolysis, and white blood cells and pl
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30

Morozov, Artem, Sofia Lvovich, Yulia Minakova, Elizaveta Sobol, Sergey Zhukov, and Ani Varpetyan. "A NOVEL METHOD FOR SURGICALTREATMENT OF OSTEOMYELITIS OF DISTAL PHALANX: A CLINICAL CASE." Archiv Euromedica 11, no. 6 (2021): 77–80. http://dx.doi.org/10.35630/2199-885x/2021/11/6.18.

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— Background: Osteomyelitis is an infectious-inflammatory destructive bone disease caused by nonspecific and specific microflora, or microbial communities. Diabetes mellitus is the most common pathology associated with osteomyelitis and may be responsible for exacerbation of chronic osteomyelitis. Objective: to demonstrate a new patented technique for surgical treatment of osteomyelitis of distal phalanx (I finger) on the example of a clinical case. Methods: A female patient P. 34 years old, who has type 2 diabetes mellitus, decompensated. Daily glucosometry shows changes in capillary blood gl
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31

Hughes, I., J. May, and C. Carpenter. "CHRONIC RECURRENT MULTIFOCAL OSTEOMYELITIS IN CHILDREN: A NATIONAL EXPERIENCE AT A TERTIARY REFERRAL CENTRE OVER 20 YEARS." Orthopaedic Proceedings 105-B, SUPP_4 (2023): 7. http://dx.doi.org/10.1302/1358-992x.2023.4.007.

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IntroductionChronic recurrent multifocal osteomyelitis (CRMO) is a rare condition characterised by bony pain and swelling which may be initially mistaken for bacterial osteomyelitis. The episodic course of the disease may confound the diagnosis and potentially be mistaken for a partial response to antimicrobial therapy. It is an orphan disease and consequently results in many unclear aspects of diagnosis, treatment and follow up for patients. The aim of this study is to evaluate a national tertiary centre's experience with the clinical condition and present one of the largest cohorts to date,
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32

Bass, Alec, Mylène Aubertin-Leheudre, Claude Vincent, et al. "Effects of an Overground Walking Program With a Robotic Exoskeleton on Long-Term Manual Wheelchair Users With a Chronic Spinal Cord Injury: Protocol for a Self-Controlled Interventional Study." JMIR Research Protocols 9, no. 9 (2020): e19251. http://dx.doi.org/10.2196/19251.

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Background In wheelchair users with a chronic spinal cord injury (WUSCI), prolonged nonactive sitting time and reduced physical activity—typically linked to this mode of mobility—contribute to the development or exacerbation of cardiorespiratory, musculoskeletal, and endocrine-metabolic health complications that are often linked to increased risks of chronic pain or psychological morbidity. Limited evidence suggests that engaging in a walking program with a wearable robotic exoskeleton may be a promising physical activity intervention to counter these detrimental health effects. Objective This
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Hill, Anita, Richard J. Kelly, Austin G. Kulasekararaj, et al. "Eculizumab in Paroxysmal Nocturnal Hemoglobinuria (PNH): A Report of All 153 Patients Treated in the UK." Blood 120, no. 21 (2012): 3472. http://dx.doi.org/10.1182/blood.v120.21.3472.3472.

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Abstract Abstract 3472 Paroxysmal Nocturnal Hemoglobinuria (PNH) is an acquired clonal stem cell disorder arising on the background of bone marrow failure and resulting in hemolytic anemia, thrombosis, pulmonary hypertension (PHT) and chronic kidney disease (CKD) through uncontrolled complement activation. Approximately half of patients treated with supportive therapies alone die as a result of their PNH. Eculizumab blocks C5 and thereby terminal complement activation. Complications are therefore prevented with reduction in intravascular hemolysis, transfusion requirements, thromboses, pulmona
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Reiss, Ulrike M., Jeffrey Schwartz, Kathleen M. Sakamoto, Geetha Puthenveetil, Masayo Ogawa, and Russell E. Ware. "Efficacy and Safety of Eculizumab in Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria." Blood 118, no. 21 (2011): 1034. http://dx.doi.org/10.1182/blood.v118.21.1034.1034.

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Abstract Abstract 1034 Background: Paroxysmal nocturnal hemoglobinuria (PNH) is a progressive, life-threatening disease characterized by chronic intravascular hemolysis caused by uncontrolled complement activation. The cellular abnormality in PNH originates from a somatic mutation in the PIG-A gene resulting in a deficiency of the glycosylphosphatidyl-inositol (GPI) anchored complement regulatory proteins, CD55 and CD59. Featuring a complex pathophysiology, PNH is associated with hemolysis, cytopenia, thromboembolism (TE), multi-organ damage, bone marrow failure, and death. Patients with PNH a
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Urbano-Ispizua, Alvaro, Hubert Schrezenmeier, Petra Muus, et al. "Clinical Characteristics of Classic Paroxysmal Nocturnal Hemoglobinuria (PNH) in Pediatric Patients: A Comparison with Classic PNH in Adults. An International PNH Registry Study." Blood 118, no. 21 (2011): 2102. http://dx.doi.org/10.1182/blood.v118.21.2102.2102.

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Abstract Abstract 2102 Background: Paroxysmal nocturnal hemoglobinuria (PNH) is a chronic, life-threatening hematopoietic stem cell disorder characterized by deficiency of the GPI-anchored complement inhibitory proteins CD55 and CD59. Uncontrolled complement activation is responsible for chronic hemolysis that leads to the serious clinical morbidities including thromboembolism (TE) and chronic kidney disease, which increase risk of mortality. PNH is infrequent in children and as such, there is a paucity of data in the literature examining their burden of disease. This is the largest series of
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Socié, Gérard, Hubert Schrezenmeier, Petra Muus, et al. "Eculizumab Protects Against TE and Prolongs Survival in Patients with Paroxysmal Nocturnal Hemoglobinuria: An International PNH Registry Study." Blood 120, no. 21 (2012): 3480. http://dx.doi.org/10.1182/blood.v120.21.3480.3480.

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Abstract Abstract 3480 Background: Paroxysmal nocturnal hemoglobinuria (PNH) is a chronic and life-threatening hematopoietic stem cell disorder characterized by uncontrolled complement-mediated hemolysis. PNH, in large part due to chronic hemolysis and platelet hyperactivation, is associated with thromboembolism (TE), one of the leading causes of disease mortality. Eculizumab, a monoclonal antibody that inhibits terminal complement activation, has been shown in clinical trials to reduce hemolysis and the incidence of TE. The International PNH Registry provides the opportunity to understand fro
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Lee, Jong Wook, Jun Ho Jang, Jin Seok Kim, et al. "Uncontrolled Complement Activation and the Resulting Chronic Hemolysis As Measured by LDH Serum Level At Diagnosis As Predictor of Thrombotic Complications and Mortality in a Large Cohort of Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH),." Blood 118, no. 21 (2011): 3166. http://dx.doi.org/10.1182/blood.v118.21.3166.3166.

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Abstract Abstract 3166 Introduction: Paroxysmal nocturnal hemoglobinuria (PNH) is a progressive, systemic, and life-threatening disease characterised by chronic uncontrolled terminal complement activation and hemolysis. Uncontrolled complement activation leads to red blood cell (RBC) hemolysis, platelet activation and subsequently thromboembolism (TE), renal and other organ impairment, pain, severe fatigue, poor quality of life and early mortality. Lactate dehydrogenase (LDH) serum level of ≥1.5x the upper limit of normal (LDH ≥1.5x) is a marker of uncontrolled complement activation that has b
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Justino, Caio Cesar, Patricia Eiko Yamakawa, Ana Paula Azambuja, et al. "Terminal Complement Inhibition Exhibiting Higher Response in Patients with Paroxysmal Nocturnal Hemoglobinuria: A Multicentric Real-World Evidence in Brazil." Blood 144, Supplement 1 (2024): 5689. https://doi.org/10.1182/blood-2024-209210.

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Introduction: Paroxysmal nocturnal hemoglobinuria (PNH) is characterized by acquired abnormalities in the PIG-A gene, leading to hemolytic anemia, a hypercoagulable state, and cytopenias. Complement inhibitors targeting C5 have transformed PNH management improving survival and quality of life. Patients and Methods: Multiparametric flow cytometry identified 338 PNH patients in five Brazilian centers, categorized as classic PNH, PNH with another hematologic disease, or subclinical PNH. Clinical and laboratory data were collected pre- and post-treatment using standardized forms based on informati
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Lisukov, Igor, Alexander Kulagin, Alexey Maschan, et al. "Effect of Eculizumab on Physician-Reported Symptoms in the Russian Cohort of the Paroxysmal Nocturnal Hemoglobinuria (PNH) International Registry." Blood 124, no. 21 (2014): 5163. http://dx.doi.org/10.1182/blood.v124.21.5163.5163.

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Abstract Background: Paroxysmal nocturnal hemoglobinuria is a rare clonal hematopoietic stem cell disease that can lead to life-threatening complications including thrombotic events (TE), chronic kidney disease (CKD) and pulmonary hypertension. In 2011 the International PNH Registry was implemented in the Russian PNH cohort to assess the natural history of PNH and the effects of treatment with eculizumab. Aims:We aim to evaluate the change from baseline to last follow-up in physician-reported symptoms, LDH and hemoglobin concentration in treated and untreated PNH patients. Design and methods:
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Kulasekararaj, Austin, Jacques Le Roux Malherbe, Andrew McDonald, et al. "BCX9930, a Potent, Selective, Oral Factor D Inhibitor, Demonstrates Proof-of-Concept As Monotherapy in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)." Blood 136, Supplement 1 (2020): 14–15. http://dx.doi.org/10.1182/blood-2020-138838.

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INTRODUCTION: PNH, a rare, chronic, life-threatening disease, is characterized by hemolytic anemia due to uncontrolled activity of the complement alternative pathway (AP), bone marrow failure, and thrombosis. Inhibition of C5 by intravenously administered eculizumab and ravulizumab reduces intravascular hemolysis, but PNH red blood cells (RBCs) become opsonized and susceptible to extravascular hemolysis (Risitano et al, Blood 2009). Only approximately half of PNH patients become transfusion independent with eculizumab treatment (Hillmen et al, NEJM 2006). BCX9930 is a potent, selective, orally
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41

Shammo, Jamile M., Rachel L. Mitchell, Kylene Ogborn, Ellen Salkeld, and Stephanie Chisolm. "Path to Diagnosis of Paroxysmal Nocturnal Hemoglobinuria: The Results of an Exploratory Study Conducted By the Aplastic Anemia and Myelodysplastic Syndrome International Foundation and the National Organization for Rare Disorders Utilizing an Internet-Based Survey." Blood 126, no. 23 (2015): 3264. http://dx.doi.org/10.1182/blood.v126.23.3264.3264.

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Abstract Introduction: Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare bone marrow failure disorder characterized by thrombosis and chronic intravascular hemolysis. Symptoms commonly associated with PNH include headaches, fatigue, weakness and back and abdominal pain. PNH has an adverse impact on patient's mortality and quality of life. Little research has been performed to understand and delineate the patients' experiences from onset of symptoms to diagnosis and very few reputable websites are available as resources for both healthcare providers and patients. The purpose of this study was
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Verstovsek, Srdan, Hagop Kantarjian, Ruben A. Mesa, et al. "Long-Term Follow up and Optimized Dosing Regimen of INCB018424 in Patients with Myelofibrosis: Durable Clinical, Functional and Symptomatic Responses with Improved Hematological Safety." Blood 114, no. 22 (2009): 756. http://dx.doi.org/10.1182/blood.v114.22.756.756.

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Abstract Abstract 756 Background: Myelofibrosis (MF) is the most serious of chronic myeloproliferative neoplasms (MPNs) for which there is no approved therapy. Exaggerated JAK2 signaling, either by gain-of-function mutations (eg, JAK2V617F) or high circulating levels of JAK1/2 activating cytokines (eg, interleukin [IL]-6), is believed to play a key role in MPN pathogenesis. INCB018424 (424), a selective oral inhibitor of JAK1/2, has demonstrated clinical benefits including reduction of splenomegaly and improvement of symptoms in a Phase I/II trial in MF patients. Thrombocytopenia was identifie
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Walter, Jeanette, Andrea Petermann-Meyer, Hatice Soysal, et al. "Quality of Life Analysis in Patients with AA Using the QLQ-AA/PNH-54 at Initial Diagnosis of AA - a Registry Based Analysis." Blood 142, Supplement 1 (2023): 5635. http://dx.doi.org/10.1182/blood-2023-182810.

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Objectives: Aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH) are ultra-rare, acquired bone marrow failure syndromes (BMFS) with need for long-term care and high morbidity despite recent therapeutic advances. There is a lack of disease-specific assessment tools of quality of life (QoL) in AA/PNH patients. So far, QoL has mostly been estimated according to surrogate parameters (e.g. blood counts, transfusion dependence or toxicity of treatment in AA patients) or has been assessed with tools specifically designed for cancer patients (e.g. EORTC Quality of Life Questionnaire QLQ-
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44

Schrezenmeier, Hubert, Austin G. Kulasekararaj, Lindsay Mitchell, et al. "Predictors for Improvement in Patient-Reported Outcomes: Post-Hoc Analysis of a Phase 3 Randomized, Open-Label Study of Eculizumab and Ravulizumab in Complement Inhibitor-Naïve Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)." Blood 138, Supplement 1 (2021): 2196. http://dx.doi.org/10.1182/blood-2021-144554.

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Abstract Background : Patients with PNH have uncontrolled terminal complement activation that can lead to thrombosis, organ damage, intravascular hemolysis (IVH), and clinical sequelae. It is also associated with debilitating patient-reported outcomes (PROs), such as fatigue, dyspnea, and pain that contribute to a poor quality of life (QoL). Whilst it is known that improvements in clinical outcomes are associated with C5 inhibitor (C5i) therapy in patients with PNH, evidence characterizing the relationship between clinical outcomes and fatigue or QoL are limited. Understanding key clinical dri
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Roeth, Alexander, Christopher J. Patriquin, Jeff Szer, et al. "Ravulizumab Effectiveness in the Real World: Evidence from the International PNH Registry." Blood 142, Supplement 1 (2023): 2722. http://dx.doi.org/10.1182/blood-2023-189165.

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Background Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease of uncontrolled terminal complement activation on blood cells that can cause intravascular hemolysis (IVH), major adverse vascular events (MAVEs), including thromboembolic events (TEs), and organ damage resulting in early mortality. Complement C5 inhibitors eculizumab (Ecu) and ravulizumab (Rav) are standard-of-care treatments that inhibit terminal complement activity and reduce morbidity and mortality by controlling IVH (reduction of lactate dehydrogenase [LDH]) and preventing TEs. Rav provides the same benefits as Ecu but
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Francesco, Francesco Fiore, Somma Francesco, D'Angelo Roberto, and Vincenzo. "Imaging-guided radiofrequency ablation of osteoid osteoma in typical and atypical sites: Long term follow up." March 18, 2021. https://doi.org/10.1371/journal.pone.0248589.

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Francesco Somma, Vincenzo Stoia, Roberto D’Angelo, Francesco Fiore:To assess efficacy and safety of imaging-guided radiofrequency ablation (RFA) of Osteoid Osteoma (OO) in both typical and atypical sites. Osteoid osteoma (OO) is a benign bone tumor (10% of benign bone tumors), composed of a central nidus generally of less than 15 mm in diameter and surrounded by osteoblasts and peripheral reactive zone of thickened cortical or trabecular bone and loose fibro vascular tissue . It is more frequent in males, usually in young adults . Herein, we present our oncologic center experience to ass
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R, Cunha, Verraest X, Martins P, Madureira R, and Pereira A. "Clinical case of preiser disease, bilateral femoral head and humeral head osteonecrosis associated with chronic corticosteroid therapy." Journal of Clinical Images and Medical Case Reports 2, no. 4 (2021). http://dx.doi.org/10.52768/2766-7820/1269.

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Osteonecrosis is a pathologic process that is associated with numerous conditions and therapeutic interventions. Most commonly the hip is involved but almost any bone can develop osteonecrosis. It can occur in the femoral head, but also affect the femoral condyles, humeral heads, proximal tibia, vertebrae, and small bones of the hand and foot. The most frequent etiological factors are trauma, alcoholism, and chronic corticosteroid therapy; the latter causing the most devastating form of osteonecrosis. Glucocorticoid-induced osteonecrosis is a known toxicity in pediatric and young adult patient
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Jankovic, Jelena, Branislava Milenkovic, Ivana Buha, et al. "OSTEOPOROSIS IN PATIENTS WITH COPD AND IMPACT ON QUALITY OF LIFE." Interciencia, 2023. http://dx.doi.org/10.59671/p2n5m.

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Chronic obstructive pulmonary disease (COPD) is a progressive disease parenchyma destruction. Smoking is a major risk factor for this disease with frequent comorbidities. Osteoporosis is a common comorbidity in patients with COPD. Osteoporosis is characterized by lower bone density and changes in bone microarchitecture, leading to increased sensitivity and bone fractures occur. Fractures cause pain, further worsen already damaged pulmonary ventilation, reduced mobility and disability, and increased risk of thromboembolic disease, and even death. Osteoporosis in patients with COPD is probably e
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Wery, Alexandre-Raphael, Coline Mortier, Quentin Cabrera, Mohamadou Niang, Moumini Kone, and Sarah Permal. "One haemolytic anaemia may hide another: Paroxysmal nocturnal haemoglobinuria masquerading as Plasmodium falciparum infection." European Journal of Case Reports in Internal Medicine, August 23, 2024. http://dx.doi.org/10.12890/2024_004749.

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Background: Paroxysmal nocturnal haemoglobinuria (PNH) is a rare, genetic and acquired haematologic disease that causes complement-mediated intravascular haemolytic anaemia, thrombosis and bone marrow failure. Case description: A 27-year-old migrant patient attended the emergency department in a context of fever and chills over the previous few days as well as chronic fatigue, dyspnoea and chest pain. His medical history included chronic anaemia and erectile dysfunction. Initial biology showed a haemoglobin of 6.3 g/dl, platelets of 25,000/µl, total leucocytes of 3,500/µl with 1,500 neutrophil
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Gonzalez, Diego Ennes, Renato Demarchi Foresto, Ana Luiza Santos Maldonado, et al. "Multiple extremity necrosis in fatal calciphylaxis: Case report." Brazilian Journal of Nephrology, July 8, 2020. http://dx.doi.org/10.1590/2175-8239-jbn-2020-0025.

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ABSTRACT Introduction: The clinical impact of vascular calcification is well established in the context of cardiovascular morbidity and mortality, but other clinical syndromes, such as calciphylaxis, although less frequent, have a significant impact on chronic kidney disease. Methods: Case report of a 27-year-old woman, who had complained of bilateral pain in her toes for 3 days, with the presence of small necrotic areas in the referred sites. She had a history of type 1 diabetes (25 years ago), with chronic kidney disease, on peritoneal dialysis, in addition to rheumatoid arthritis. She was a
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