scholarly journals Long-term prophylaxis in HAE patients

2019 ◽  
Vol 16 (3) ◽  
pp. 75-83
Author(s):  
T V Latysheva ◽  
E A Latysheva ◽  
I A Manto

Hereditary angioedema (HAE) due to of C1-inhibitor deficiency is a rare disease, which causes a strong physical and psychological impact on patients’ lives. HAE affects daily life, career opportunities, and social activity. In addition, fear of the edema development, as well as the associated with it pain and risk of death leads to depressive disorders specific for chronic (especially life-threatening) diseases. The emergence of modern pathogenetic drugs can minimize the effect of HAE and improve the patients’ quality of life. There are three different therapeutic strategies that are used for HAE treatment: long-term prophylaxis, short-term and on-demand therapy for acute attacks. This article focuses on long-term prophylaxis.

Author(s):  
T.V. Latysheva ◽  
E.A. Latysheva ◽  
I.A. Manto

Наследственный ангиоотек (НАО) с дефицитом С1ингибитора (С1ИНГ) представляет собой редкое заболевание, которое оказывает сильное воздействие на жизнь пациентов как физически, так и эмоционально. Непредсказуемость атак влияет на повседневную жизнь, выбор профессии, социальную активность. Кроме того, страх перед развитием отека, а также связанными с ним болью и риском смерти приводит к депрессивным расстройствам, характерным для хронических, в особенности жизнеугрожающих заболеваний. Появление современных патогенетических препаратов способно минимизировать влияние НАО и улучшить качество жизни больных. В основе терапии НАО лежат долгосрочная профилактика, краткосрочная профилактика и купирование острых атак. В данной статье речь пойдет о долгосрочной профилактике.Hereditary angioedema (HAE) due to of C1inhibitor deficiency is a rare disease, which causes a strong physical and psychological impact on patients lives. HAE affects daily life, career opportunities, and social activity. In addition, fear of the edema development, as well as the associated with it pain and risk of death leads to depressive disorders specific for chronic (especially lifethreatening) diseases. The emergence of modern pathogenetic drugs can minimize the effect of HAE and improve the patients quality of life. There are three different therapeutic strategies that are used for HAE treatment: longterm prophylaxis, shortterm and ondemand therapy for acute attacks. This article focuses on longterm prophylaxis.


2015 ◽  
Vol 3 (1) ◽  
pp. 36-47
Author(s):  
Nazma Akter ◽  
Nazmul Kabir Qureshi

Adrenal insufficiency is caused by either primary adrenal failure or by hypothalamic-pituitary impairment of the corticotropic axis. Adrenal insufficiency, first codified in 1855 by Thomas Addison, remains relevant in 2014 because of its lethal nature. Though, it is a rare disease but is life threatening when overlooked. Main presenting symptoms such as fatigue, anorexia and weight loss are nonspecific, thus diagnosis is often delayed. The diagnostic work-up is well established but some pitfalls remain. The diagnosis is adequately established by the 250 ?g ACTH (adrenocorticotropic hormone) stimulation test in most cases. Glucocorticoids provide life saving treatment but long-term quality of life is impaired, perhaps because therapy is not given in a physiologic way. Dehydroepiandrosterone-replacement therapy has been introduced that could help to restore quality of life. It may be useful in pubertal girls, but not in adults. Monitoring of glucocorticoid-replacement is difficult due to lack of objective methods of assessment and is therefore largely based on clinical grounds. Thus, long-term management of patients with adrenal insufficiency remains a challenge, requiring an experienced specialist. DOI: http://dx.doi.org/10.3329/dmcj.v3i1.22238 Delta Med Col J. Jan 2015; 3(1): 36-47


2018 ◽  
Vol 44 (1) ◽  
pp. 52-61
Author(s):  
Pritesh Ruparelia ◽  
Oshin Verma ◽  
Vrutti Shah ◽  
Krishna Shah

Juvenile Dermatomyositis is the most common inflammatory myositis in children, distinguished by proximal muscle weakness, a characteristic rash and Gottron’s papules. The oral lesions most commonly manifest as diffuse stomatitis and pharyngitis with halitosis. We report a case of an 8 year old male with proximal muscle weakness of all four limbs, rash, Gottron’s papules and oral manifestations. Oral health professionals must be aware of the extraoral and intraoral findings of this rare, but potentially life threatening autoimmune disease of childhood, for early diagnosis, treatment, prevention of long-term complications and to improve the prognosis and hence, the quality of life for the patient.


Author(s):  
Justyna Kałduńska ◽  
Karolina Skonieczna-Żydecka ◽  
Karina Ryterska ◽  
Joanna Palma ◽  
Wojciech Żwierełło ◽  
...  

Depressive disorders are common among young people and can decrease social competences and thus the quality of life. There is a relationship between the occurrence of depressive disorders and insomnia. The aim of the study was to determine the prevalence of insomnia and depressive behavior and assess the relationship between these among participants of the Pol’and’Rock Festival, Kostrzyn, Poland 2019. The study used the Athens Insomnia Scale (AIS) and the Beck Inventory II Scale (BDI-II). The study group consisted of 923 people, with the majority of women (n = 500; 54.2%). A total of 297 persons (32.2%) reported varying severity of depressive symptoms. Insomnia was observed in 261 (28.28%) respondents. Sleeping disturbances were observed more frequently in females. Persons with insomnia had a significantly higher BDI-II score. A strong positive correlation (r = 0.65) between the number of points obtained on the Beck and AIS scales was observed. Insomnia and depressive behavior are prevalent in the Polish population. Due to long-term social and economic consequences, special attention should be paid to the prevention, early detection and treatment of both disorders.


2020 ◽  
Vol 100 (1) ◽  
pp. 19-25
Author(s):  
Sarina K. Mueller ◽  
Maximilian Traxdorf ◽  
Konstantinos Mantsopoulos ◽  
Antoniu-Oreste Gostian ◽  
Matti Sievert ◽  
...  

Introduction: During the COVID-19 pandemic, worldwide over 600,000 human beings died due to the cause of the disease. In order to deescalate the transmission rate and to avoid crush loading the countries medical health systems social distancing, face masks, and lockdowns have been considered essential by the majority of governments. Whereas some countries have highly reduced or completely stopped otorhinolaryngological procedures, other countries have continued selected surgeries. The objective of this study was to analyze procedures and outcomes of continuing semielective and emergency surgeries during the COVID-19 pandemic. Methods: Retrospective analysis of n = 750 patients who received semi-elective or emergency surgery between March 26 and June 16, 2020, in the Otolaryngology Department of the Friedrich-Alexander-University of Erlangen-Nürnberg. All patients were screened for COVID symptoms and swabbed for SARS-CoV-2 prior to surgery. Results: Of the n = 750 patients, n = 699 patients received semielective surgery and n = 51 emergency surgery. For 27 patients, the swab result could not be awaited due to a life-threatening condition. In these cases, surgery was performed in full protective equipment. No patient was tested positive during or after the surgery (follow-up 45 to 127 days). No member of the medical personnel showed symptoms or was tested positive after contact with patients. Due to the continuation of surgeries, patients’ lives were saved and improvement of long-term quality-of-life and outcomes is anticipated. Conclusions: Continuing selected otorhinolaryngological surgeries is crucial for patients’ health, survival, and long-time quality of life, yet, the protection of the medical personnel has to be granted.


2019 ◽  
Vol 28 (6) ◽  
pp. 708-714
Author(s):  
N. L. Perel’man ◽  
V. P. Kolosov

The aim of this study was to investigate long-term change in health-related quality of life (HRQL) in patients with asthma in real clinical practice and in relation to emotional disorders, cooperativeness (cooperation between the patient and the physician, and adherence to treatment), and cold air-provoked bronchial hyperresponsiveness (BHR).Methods. This observational study involved 32 patients with mild to moderate asthma. The study duration was 7.5 to 10 years. HRQL was assessed using SF-36 questionnaire and Asthma Quality of Life Questionnaire (AQLQ); emotional disorders were diagnosed using Hospital Anxiety and Depression Scale (HADS). Patients’ cooperativeness assessment was based on number of a patient’s scheduled visits to a physician during the study. The asthma control level was evaluated using the Asthma Control Test (ACT).Results. A significant worsening was found in the social activity domain of HRQL in the total group of the patients. According to AQLQ, a significant improvement was seen in the activity and symptoms domains and in the total score of quality of life (QoL). Long-term pharmacological treatment of asthma decreased anxiety and depression levels. The asthma control (ACT score) increased from 13.2 ± 1.0 to 19.7 ± 1.1 (р = 0.00003). Anxiety and depression decreased in patients with high cooperativeness during the long-term follow-up, while the total QoL and the specific QoL were unchanged excluding the social activity domain. A significant worsening in the activity scale and in the emotional scale of specific QoL together with growing depression and decreasing control of asthma were found in patients with lower cooperativeness. The cold air-provoked BNR slowed down the long-term improvement in the social activity domain. The cold air-provoked BNR also decreased patient-reported evaluation of daily activity.Conclusion. The long-term basic therapy of asthma provides a positive trend in HRQL in the real clinical practice independently on worsening of the disease. Lower cooperativeness of a patient could decrease specific QoL, which, in turn, worsens the control of asthma and contributes to negative emotional background and destructive behavior.


2021 ◽  
Vol 22 (1) ◽  
Author(s):  
Michele Mondoni ◽  
Paolo Carlucci ◽  
Giuseppe Cipolla ◽  
Matteo Pagani ◽  
Francesco Tursi ◽  
...  

Abstract Background Haemoptysis is a challenging symptom that can be associated with potentially life-threatening medical conditions. Follow-up is key in these patients to promptly detect new or misdiagnosed pathologic findings. Few prospective studies have evaluated long-term prognostic outcomes in patients with haemoptysis. Furthermore, the role played by antiplatelet and anticoagulant drugs on mortality and recurrence rates is unclear. The aim of this study was to assess mortality after 18 months of follow-up. Furthermore, the incidence of recurrence and the risk factors for recurrence and death were evaluated (including the role played by anticoagulant and antiplatelet drugs). Methods Observational, prospective, multicentre, Italian study. Results 451/606 (74.4%) recruited patients with haemoptysis completed the 18 months follow-up. 22/604 (3.6%) diagnoses changed from baseline to the end of the follow-up. 83/604 (13.7%) patients died. In 52/83 (62.7%) patients, death was the outcome of the disease which caused haemoptysis at baseline. Only the diagnosis of lung neoplasm was associated with death (OR (95%CI): 38.2 (4.2–347.5); p-value: 0.0001). 166 recurrences were recorded in 103/604 (17%) patients. The diagnosis of bronchiectasis was significantly associated with the occurrence of a recurrence (OR (95% CI): 2.6 (1.5–4.3)); p-value < 0.0001). Anticoagulant, antiaggregant, and anticoagulant plus antiaggregant drugs were not associated with an increased risk of death and recurrence. Conclusions Our study showed a low mortality rate in patients with haemoptysis followed-up for 18 months. Pulmonary malignancy was the main aetiology and the main predictor of death, whereas bronchiectasis was the most frequent diagnosis associated with recurrence. Antiplatelet and/or anticoagulant therapy did not change the risk of death or recurrence. Follow-up is recommended in patients initially diagnosed with lower airways infections and idiopathic bleeding. Trial registration: NCT02045394


2021 ◽  
Vol 13 (2) ◽  
Author(s):  
Mariabeatrice Bertolani ◽  
Eleonora Rodighiero ◽  
Maria Beatrice De Felici del Giudice ◽  
Torello Lotti ◽  
Claudio Feliciani ◽  
...  

Vitiligo is an acquired pigmentary disorder afflicting 0.5-2% of the world population for both sexes and all races with a capricious and unpredictable course. It has a complex etiology and varies in its manifestation, progression and response to treatment. Even if the precise aetiology and pathobiology of the disease are complex and still debated, recent evidence supports that vitiligo is a T CD8+ cell-mediated autoimmune disease triggered by oxidative stress. To date no clinical, biological and histological criteria allow us to establish the prognosis with certainty. The choice of the best therapy for adult and childhood vitiligo is based on various factors, such as the patient’s age, psychological condition and expectations, distribution and extension of skin lesions, type of vitiligo (stable or not) and availability and cost of therapeutic options. Since vitiligo has a deep psychological impact on patients and their quality of life, treating the disease is very important. As dermatologists, we have important goals in the treatment of vitiligo patients: stabilization of the disease progression, repigmentation of the lesions and especially the persistence of the aforementioned repigmentation. Although several medical and surgical therapeutic options have been proposed, no definite cure has yet been developed and the long-term persistence of repigmentation is unpredictable. We review the different therapeutic options with particular attention on the recurrence rate.


Author(s):  
Matthew Baldwin ◽  
Hannah Wunsch

Many critically ill patients now survive what were previously fatal illnesses, but long-term mortality after critical illness remains high. While study populations vary by country, age, intervention, or specific diagnosis, investigations demonstrate that the majority of additional deaths occur in the first 6 to 12 months after hospital discharge. Patients with diagnoses of cancer, respiratory failure, and neurological disorders leading to the need for intensive care have the highest long-term mortality, while those with trauma and cardiovascular diseases have much lower long-term mortality. Use of mechanical ventilation, older age, and a need for care in a facility after the acute hospitalization are associated with particularly high 1-year mortality among survivors of critical illnesses. Due to challenges of follow-up, less is known about causes of delayed mortality following critical illness. Longitudinal studies of survivors of pneumonia, stroke, and patients who require prolonged mechanical ventilation suggest that most debilitated survivors die from recurrent infections and sepsis. Potential biologic mechanisms for increased risk of death after a critical illness include sepsis-induced immunoparalysis, intensive care unit-acquired weakness, neuroendocrine changes, poor nutrition, and genetic variance. Studies are needed to fully understand how the severity of the acute critical illness interacts with comorbid disease, pre-illness disability, and pre-existing and acquired frailty to affect long-term mortality. Such studies will be fundamental to improve targeting of rehabilitative, therapeutic, and palliative interventions to improve both survival and quality of life after critical illness.


2019 ◽  
Vol 5 (6) ◽  
pp. e330-e333 ◽  
Author(s):  
Pedro Iglesias ◽  
Alberto Martínez ◽  
Pablo Gajate ◽  
Teresa Alonso ◽  
Teresa Navarro ◽  
...  

Objective: Malignant insulinoma is an extremely uncommon tumor that is usually accompanied by severe hypoglycemia that is difficult to manage. At this time, the long-term effect of 177Lu-DOTATATE (lutetium [Lu-177]-DOTA-Tyr3-octreotate) on this tumor is not well known. Methods: We report a case of severe, life-threatening, and refractory hypoglycemia associated with malignant insulinoma treated with 177Lu-DOTATATE. Results: A 51-year-old woman was referred because of severe, life-threatening, and refractory hypoglycemia due to malignant insulinoma. The patient had been treated unsuccessfully with chemotherapy, targeted therapies, and symptomatic therapy with diazoxide, steroids, and somatostatin analogues without success. 177Lu-DOTATATE adequately controlled her hypoglycemia after the other conventional treatments failed. Conclusion: 177Lu-DOTATATE was effective in providing rapid and long-term symptomatic control of the hypoglycemia and significantly improved the quality of life of the patient.


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