Individual Replacement Therapy (IRT) with a Pasteurized C1-Inhibitor Concentrate Compared to Prophylaxis with Danazol in Patients with Hereditary Angioedema (HAE) - a Prospective Study.

Blood ◽  
2004 ◽  
Vol 104 (11) ◽  
pp. 1028-1028 ◽  
Author(s):  
Wolfhart Kreuz ◽  
Inmarculada Martinez-Saguer ◽  
Emel Aygoeren-Puersuen ◽  
Eva Rusicke ◽  
Thomas Klingebiel
Keyword(s):  
Quality Of Life ◽  
Side Effects ◽  
Replacement Therapy ◽  
Hereditary Angioedema ◽  
Parvovirus B19 ◽  
Laryngeal Edema ◽  
C1 Inhibitor ◽  
Life Threatening

Abstract Attenuated androgens like danazol are the current treatment of choice for long-term prophylaxis in patients with hereditary angioedema (HAE), who may experience life-threatening acute attacks. Unfortunately, this group of drugs bears the risk of severe side effects (e.g. depression, weight gain, hirsutism, transaminase elevations, liver adenoma and carcinoma, headaches, hypertension, menstrual abnormalities). Objectives: We therefore explored the option to administer a pasteurized, plasma-derived C1-Inhibitor concentrate (C1-INH, Berinert®P) for individual replacement therapy (IRT) in patients with HAE in whom danazol induced severe side effects, was not effective or was contraindicated (e.g. children, pregnant women). Methods: Prospective, observational, intra-individual cross over study, comparing efficacy, safety and quality of life of danazol (prophylaxis) vs. C1-INH (IRT) in 23 patients suffering from severe HAE. Results: Mean annual attack frequency decreased from 48.8 ± 37.1 (danazol) to 8.1 ± 22.1 (C1-INH); p<0.001. All patients who received pasteurized C1-INH were free of life threatening attacks (laryngeal edema) or adverse events, which did occur under long-term prophylaxis with danazol (Fig.). In addition, all quality of life parameters improved significantly; p<0.001. During the entire 20-year observation period with C1-INH in all patients there was no transmission of HIV type 1/2, hepatitis A-, B-, C-, G-viruses, or parvovirus B19. Conclusions: Compared to danazol, IRT with pasteurized C1-INH in patients with severe HAE significantly reduces the frequency of HAE attacks, especially of life-threatening attacks, and significantly improves the quality of life.

Proton Pump Inhibitors in 2021: Pros, Cons, and Everything in Between

2021 ◽  
pp. 263451612110217
Author(s):  
Joshua A. Sloan ◽  
Philip O. Katz
Keyword(s):  
Quality Of Life ◽  
Side Effects ◽  
Adverse Events ◽  
Proton Pump Inhibitors ◽  
Proton Pump ◽  
Drug Class ◽  
Life Threatening

The medical and lay literature has exploded with reports of adverse events associated with proton pump inhibitors over the last 10 to 15 years. The dissemination of these reports to patients and clinicians have created substantial concerns regarding what has been an exceptionally valuable drug class, dramatically improving patient quality of life, and in many cases preventing life threatening side effects of other medication. Patients are more frequently seeking to avoid these medications, and practitioners are reducing or discontinuing them to the patient’s detriment due to a misunderstanding of the data. This review will discuss the data regarding the most commonly publicized adverse events and attempt to put them in perspective.

scholarly journals Assessment and management of disease burden and quality of life in patients with hereditary angioedema: a consensus report

2021 ◽  
Vol 17 (1) ◽  
Author(s):  
Konrad Bork ◽  
John T. Anderson ◽  
Teresa Caballero ◽  
Timothy Craig ◽  
Douglas T. Johnston ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Hereditary Angioedema ◽  
Clinical Management ◽  
Disease Burden ◽  
The United States ◽  
Consensus Meeting ◽  
Consensus Statements ◽  
Consensus Report

Abstract Background Hereditary angioedema (HAE) is a rare disease characterized by unpredictable, potentially life-threatening attacks, resulting in significant physical and emotional burdens for patients and families. To optimize care for patients with HAE, an individualized management plan should be considered in partnership with the physician, requiring comprehensive assessment of the patient’s frequency and severity of attacks, disease burden, and therapeutic control. Although several guidelines and consensus papers have been published concerning the diagnosis and treatment of HAE, there has been limited specific clinical guidance on the assessment of disease burden and quality of life (QoL) in this patient population. Practical guidance is critical in supporting effective long-term clinical management of HAE and improving patient outcomes. The objective of this review is to provide evidence-based guidelines for an individualized assessment of disease burden and QoL in patients with HAE. Methods A consensus meeting was held on February 29, 2020, consisting of 9 HAE experts from the United States and Europe with extensive clinical experience in the treatment of HAE. Consensus statements were developed based on a preliminary literature review and discussions from the consensus meeting. Results Final statements reflect the consensus of the expert panel and include the assessment of attack severity, evaluation of disease burden, and long-term clinical management of HAE caused by C1-esterase inhibitor deficiency. Patient-reported outcome measures for assessing HAE attack severity and frequency are available and valuable tools; however, attack frequency and severity are insufficient markers of disease severity unless they are evaluated in the broader context of the effect on an individual patient’s QoL. QoL assessments should be individualized for each patient and minimally, they should address the interference of HAE with work, school, social, family, and physical activity, along with access to and burden of HAE treatment. Advances in HAE therapies offer the opportunity for comprehensive, individualized treatment plans, allowing patients to achieve minimal attack burden with reduced disease and treatment burden. Conclusion This consensus report builds on existing guidelines by expanding the assessment of disease burden and QoL measures for patients with HAE.

Elucidating the Potential Side Effects of Current Anti- Seizure Drugs for Epilepsy

2021 ◽  
Vol 19 ◽  
Author(s):  
Enes Akyüz ◽  
Mohd. Farooq Shaikh ◽  
Betül Köklü ◽  
Cansu Ozenen ◽  
Alina Arulsamy
Keyword(s):  
Quality Of Life ◽  
Side Effects ◽  
Adverse Effects ◽  
Side Effect ◽  
First Line ◽  
Epileptic Patients ◽  
Life Threatening ◽  
Visual Problems ◽  
Gaba Transmission

: Over the decades, various interventions have been developed and utilized to treat epilepsy. However, majority of epileptic patients are often first prescribed with anti-epileptic drugs (AED), now known as anti-seizure drugs (ASD), as a first line of defense to suppress their seizures and regain their quality of life. ASDs exert their anti-convulsant effects through various mechanisms of action including regulation of ion channels, blocking of glutamate-mediated stimulating neurotransmitter interaction, and enhancing the inhibitory GABA transmission. About one third of epileptic patients are often resistant to anti-convulsant drugs, while others develop numerous side effects which may lead to treatment discontinuation and further deterioration of quality of life. Common side effects of ASDs include headache, nausea and dizziness. However, more adverse effects such as auditory and visual problems, skin problems, liver dysfunction, pancreatitis and kidney disorders may also be witnessed. Some ASDs may even result in life-threatening conditions as well as serious abnormalities, especially in patients with comorbidities and in pregnant women. Nevertheless, some clinicians had observed a reduction in the development of side effects post individualized ASD treatment. This suggest that a careful and well-informed ASD recommendation to patients may be crucial for an effective and side-effect free control of their seizures. Therefore, this review aimed to elucidate the anticonvulsant effects of ASDs as well as their side effect profile, by discussing their mechanism of action and reported adverse effects based on clinical and preclinical studies, thereby providing clinicians with a greater understanding of the safety of current ASDs.

scholarly journals Adrenal Insufficiency - Aetiology, Diagnosis and Treatment

2015 ◽  
Vol 3 (1) ◽  
pp. 36-47
Author(s):  
Nazma Akter ◽  
Nazmul Kabir Qureshi
Keyword(s):  
Quality Of Life ◽  
Adrenal Insufficiency ◽  
Presenting Symptoms ◽  
Life Saving ◽  
Life Threatening ◽  
Diagnostic Work ◽  
Work Up ◽  
Term Management

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

scholarly journals Opioid Analgesia: Perspectives on Right Use and Utility

2007 ◽  
Vol 3;10 (5;3) ◽  
pp. 479-491 ◽  
Author(s):  
Jane C. Ballantyne
Keyword(s):  
Quality Of Life ◽  
Chronic Pain ◽  
Side Effects ◽  
Clinical Decision Making ◽  
Analgesic Efficacy ◽  
Clinical Decision ◽  
Opioid Analgesia ◽  
Opioid Treatment

The ability of opioids to effectively and safely control acute and cancer pain has been one of several arguments used to support extending opioid treatment to patients with chronic pain, against a backdrop of considerable caution that has been based upon fears of addiction. Of course, opioids may cause addiction, but the “principle of balance” may justify that “…efforts to address abuse should not interfere with legitimate medical practice and patient care.” Yet, situations are increasingly encountered in which opioid-maintained patients are refractory to analgesia during periods of pain, or even during the course of chronic treatment. The real question is whether analgesic efficacy of opioids can be maintained over time. Overall, the evidence supporting long-term analgesic efficacy is weak. The putative mechanisms for failed opioid analgesia may be related to tolerance or opioid-induced hyperalgesia. Advances in basic sciences may help in understanding these phenomena, but the question of whether long-term opioid treatment can improve patients’ function or quality of life remains a broader issue. Opioid side effects are well known, but with chronic use, most (except constipation) subside. Still, side effects can negatively affect the outcomes and continuity of therapy. This paper addresses 1) what evidence supports the long-term utility of opioids for chronic pain; 2) how side effects may alter quality of life; 3) the nature of addiction and why it is different in pain patients, and 4) on what grounds could pain medication be denied? These questions are discussed in light of patients’ rights, and warrant balancing particular responsibilities with risks. These are framed within the Hippocratic tradition of “producing good for the patient and protecting from harm,” so as to enable 1) more informed clinical decision making, and 2) progress towards right use and utility of opioid treatment for chronic pain. Key Words: Opioids, chronic pain, addiction, side effects, utility, ethics

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