scholarly journals Considerations for transition from subcutaneous to oral prophylaxis in the treatment of hereditary angioedema

2021 ◽  
Vol 17 (1) ◽  
Author(s):  
Richard G. Gower ◽  
Mary Wilber
Keyword(s):  
Quality Of Life ◽  
Hereditary Angioedema ◽  
Genetic Disorder ◽  
Subcutaneous Administration ◽  
Case Series ◽  
Case Presentation ◽  
Burden Of Treatment ◽  
Management Plans

Abstract Background Hereditary angioedema (HAE) is a rare genetic disorder characterized by unpredictable localized episodes of edema, which is frequently managed with long-term prophylactic medications. Until recently, long-term prophylaxis has predominantly required regular intravenous or subcutaneous administration, however the recent approval of berotralstat (Orladeyo™) offers an orally administered prophylactic which may be associated with a lower burden of treatment compared to injectable options for some patients. Case presentation This report describes four participants in the APeX-S trial who transitioned from subcutaneously administered lanadelumab (Takhzyro®) to daily oral berotralstat for long-term HAE prophylaxis. Lanadelumab dosing continued after berotralstat commencement in all patients and was tapered before discontinuation in three of the four patients. No substantial increases in HAE attack rates were observed after the transition to berotralstat monotherapy. One patient experienced a treatment-related adverse event (dyspepsia), which was mild and self-resolving. Conclusions All four patients described in this case series successfully transitioned from lanadelumab to berotralstat monotherapy for long-term prophylaxis without significant complications and without the use of a complex transition protocol. The decision to transition to berotralstat monotherapy and how the transition should be achieved was discussed between patient and physician, ensuring that the comfort and perspectives of the patients were considered during the treatment transition. This report highlights the importance of individualization of HAE management plans to address both the disease and treatment burdens of HAE, and thus to provide the best possible quality of life for each patient.

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.

scholarly journals Hereditary angioedema: an update on causes, manifestations and treatment

2019 ◽  
Vol 80 (7) ◽  
pp. 391-398 ◽  
Author(s):  
Hilary J Longhurst ◽  
Konrad Bork
Keyword(s):  
Quality Of Life ◽  
Hereditary Angioedema ◽  
Genetic Disorder ◽  
Treatment Options ◽  
Correct Diagnosis ◽  
Self Administration ◽  
Diagnosis And Management ◽  
Life Threatening ◽  
Short And Long Term

Hereditary angioedema is a rare genetic disorder caused by deficiency of C1 esterase inhibitor (C1-INH) and characterized by recurrent episodes of severe swelling that affect the limbs, face, intestinal tract and airway. Since laryngeal oedema can be life-threatening as a result of asphyxiation, correct diagnosis and management of hereditary angioedema is vital. Hereditary angioedema attacks are mediated by bradykinin, the production of which is regulated by C1-INH. Hereditary angioedema therapy relies on treatment of acute attacks, and short- and long-term prophylaxis. Acute treatment options include C1-INH concentrate, icatibant and ecallantide. Self-administration of treatment is recommended and is associated with increased quality of life of patients with hereditary angioedema. Advances in diagnosis and management have improved the outcomes and quality of life of patients with hereditary angioedema.

Long-term Outcomes of Deep Brain Stimulation for Neuropathic Pain

Neurosurgery ◽  
2012 ◽  
Vol 72 (2) ◽  
pp. 221-231 ◽  
Author(s):  
Sandra G.J. Boccard ◽  
Erlick A.C. Pereira ◽  
Liz Moir ◽  
Tipu Z. Aziz ◽  
Alexander L. Green
Keyword(s):  
Quality Of Life ◽  
Neuropathic Pain ◽  
Short Form ◽  
Case Series ◽  
Mcgill Pain Questionnaire ◽  
Euroqol 5D ◽  
Deep Brain

Abstract BACKGROUND: Deep brain stimulation (DBS) to treat neuropathic pain refractory to pharmacotherapy has reported variable outcomes and has gained United Kingdom but not USA regulatory approval. OBJECTIVE: To prospectively assess long-term efficacy of DBS for chronic neuropathic pain in a single-center case series. METHODS: Patient reported outcome measures were collated before and after surgery, using a visual analog score, short-form 36-question quality-of-life survey, McGill pain questionnaire, and EuroQol-5D questionnaires (EQ-5D and health state). RESULTS: One hundred ninety-seven patients were referred over 12 years, of whom 85 received DBS for various etiologies: 9 amputees, 7 brachial plexus injuries, 31 after stroke, 13 with spinal pathology, 15 with head and face pain, and 10 miscellaneous. Mean age at surgery was 52 years, and mean follow-up was 19.6 months. Contralateral DBS targeted the periventricular gray area (n = 33), the ventral posterior nuclei of the thalamus (n = 15), or both targets (n = 37). Almost 70% (69.4%) of patients retained implants 6 months after surgery. Thirty-nine of 59 (66%) of those implanted gained benefit and efficacy varied by etiology, improving outcomes in 89% after amputation and 70% after stroke. In this cohort, >30% improvements sustained in visual analog score, McGill pain questionnaire, short-form 36-question quality-of-life survey, and EuroQol-5D questionnaire were observed in 15 patients with >42 months of follow-up, with several outcome measures improving from those assessed at 1 year. CONCLUSION: DBS for pain has long-term efficacy for select etiologies. Clinical trials retaining patients in long-term follow-up are desirable to confirm findings from prospectively assessed case series.

Welfare of cats 5–29 months after perineal urethrostomy: 74 cases (2015–2017)

2019 ◽  
Vol 22 (6) ◽  
pp. 582-588 ◽  
Author(s):  
Margaret R Slater ◽  
Sharon Pailler ◽  
J’mai M Gayle ◽  
Illoneete Cohen ◽  
Erin L Galloway ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Case Series ◽  
Life Outcomes ◽  
Retrospective Case ◽  
Quality Of Life Scale ◽  
Life Scale ◽  
Potential Factors ◽  
American Society

Objectives The objective of this study was to evaluate the long-term quality of life in cats following perineal urethrostomy. Methods This study comprised a retrospective case series of 74 cats with urinary obstructions that received perineal urethrostomies at the American Society for the Prevention of Cruelty to Animals (ASPCA) Animal Hospital between September 2015 and July 2017. Medical records were reviewed for information on the number of obstructions prior to surgery, urinary tract problems at the time of surgery and other potential factors influencing long-term welfare. Owners were surveyed by telephone and text 5–29 months after their cat’s surgery. Responses were compiled and analyzed to determine owner perception of welfare postsurgery, and to identify patterns in medical history and welfare outcomes. Results In total, 105 cat owners were eligible for the survey; 74 responded. Altogether, 100% of responding individuals reported at least the same quality of life postsurgery compared with the cat’s quality of life before demonstrating urinary problems (48% reported better). On a quality-of-life scale of 1–10 (10 = excellent), 100% of responding cat owners reported at least 7; 75% reported 10. Conclusions and relevance Quality-of-life outcomes for cats 5–29 months after receiving perineal urethrostomy surgery were reported to be very good. Given this finding, and that perineal urethrostomy surgery should mitigate future blockages, we suggest considering perineal urethrostomy surgery as a standard tool to manage urinary obstructions in cats. The needs of the specific client and patient, including risks, costs and welfare of the cats, should drive the options for management of urinary obstructions in male cats.

Can implant-retained overdenture components cause damage to the opposing natural dentition?

Dental Update ◽  
2021 ◽  
Vol 48 (7) ◽  
pp. 571-578
Author(s):  
Nahal Razaghi ◽  
Mike Hughes ◽  
Mital Patel
Keyword(s):  
Quality Of Life ◽  
Clinical Relevance ◽  
Clinical Case ◽  
Case Series ◽  
Patient Assessment ◽  
Careful Patient ◽  
Potential Problems ◽  
Natural Dentition

This clinical case series highlights an under-reported and often unidentified complication of implant-retained overdentures that may leave patients with a risk of long-term discomfort and preventable damage to the opposing dentition. A variety of cases is discussed to demonstrate potential problems and symptoms that can flag up signs of parafunctional habits in implant patients, and how these can be managed successfully. CPD/Clinical Relevance: Implant-retained overdentures provide a significant improvement in quality of life; however, careful patient assessment and management is essential to ensure the components do not cause damage to the opposing dentition.

scholarly journals Perceived Burden of Treatment and Quality of Life in Long-Term Follow Up after Oral Immunotherapy in Food Allergic Patients

2020 ◽  
Vol 145 (2) ◽  
pp. AB147
Author(s):  
William Collins ◽  
Olivia Raeber ◽  
Dana Tupa ◽  
Shu Cao ◽  
Kari Nadeau ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Oral Immunotherapy ◽  
Burden Of Treatment ◽  
Perceived Burden ◽  
Long Term Follow Up

Art Activities and Quality of Life for People With Dementia in the Long Term Care Setting: A Case Series

2014 ◽  
Vol 15 (3) ◽  
pp. B3
Author(s):  
Rebecca S. King-Tucker ◽  
Rebecca S. King-Tucker ◽  
Maria Knupp ◽  
Terry Edwards ◽  
Elizabeth Walters
Keyword(s):  
Quality Of Life ◽  
Care Setting ◽  
Long Term Care ◽  
Case Series ◽  
Term Care ◽  
People With Dementia ◽  
Art Activities

Successful Long-Term Prophylactic Treatment With Subcutaneous C1 Esterase Inhibitor in a Patient With Hereditary Angioedema

2019 ◽  
Vol 33 (6) ◽  
pp. 907-911
Author(s):  
Janina Hahn ◽  
Melanie Nordmann-Kleiner ◽  
Susanne Trainotti ◽  
Thomas K. Hoffmann ◽  
Jens Greve
Keyword(s):  
Quality Of Life ◽  
Hereditary Angioedema ◽  
Prophylactic Treatment ◽  
Upper Airway ◽  
Venous Access ◽  
Current Standard ◽  
C1 Esterase Inhibitor ◽  
Esterase Inhibitor

Background: Hereditary angioedema (HAE) patients suffer from recurrent swellings. Current standard therapy consists of C1 esterase inhibitor (C1-INH) and bradykinin receptor B2 antagonists. Severe courses require prophylactic treatment. For such patients, it has been demonstrated that the intravenous (IV) administration of C1-INH [C1-INH(IV)] is safe and effective. A new prophylactic option is subcutaneous (SC) treatment with C1-INH. Methods and Case: We present the case of an HAE patient placed on prophylactic C1-INH(IV) therapy due to frequent attacks when managed with on-demand therapy. An implanted port allowed the periodical and safe application of medication until the device was explanted due to an infection. Due to the poor venous access, repeated IV application failed. Therefore, we began a SC treatment with 1500 IU C1-INH [C1-INH(SC)] as long-term prophylaxis and analyzed the clinical course over 16 months. Results: Under the SC prophylaxis, the number of attacks were reduced to 1/month in comparison to 4.33/month with no prophylactic treatment and 1.83/month with C1-INH(IV). No severe attacks and no attack within the upper airway occurred over the 16 months of C1-INH(SC) treatment. As a result, quality of life improved, as measured by the Angioedema quality of life questionaire (AE-QoL). Conclusion: Self-administered SC prophylactic use of C1-INH over a period of 16 months seems to be a well tolerated and efficient. The patient’s quality of life improved, and by learning self-application, the patient gained independence.

scholarly journals The impact of bilateral endoscopic inferior turbinoplasty with or without adenoidectomy on the quality of life of children: a retrospective case series study

2019 ◽  
Vol 48 (1) ◽  
Author(s):  
Ahmed Mourad ◽  
Hussein Jaffal ◽  
Ismaeel El-Hakim ◽  
Hamdy El-Hakim
Keyword(s):  
Quality Of Life ◽  
Case Series ◽  
Retrospective Case ◽  
Retrospective Case Series ◽  
Case Series Study ◽  
The Impact ◽  
Series Study

Abstract Background Inferior turbinoplasty (IT) and adenoidectomy (Ad) are frequently resorted to in children with chronic rhinitis (CR) refractory to medical therapy. The aim of this study is to document the long-term improvement in quality of life (QOL) in children with CR following endoscopic IT with or without Ad. Methods A retrospective case series study was conducted. We searched a prospectively kept surgical database for children ≤18 years old who had CR who underwent endoscopic IT with or without Ad between 2009 and 2016 at a tertiary care children’s center. Patients with sinonasal pathologies other than CR, had craniofacial syndromes or dysmorphism and had other sinonasal procedures or trauma were excluded. Collected data included demographics, secondary diagnoses, duration of follow-up, and complications of procedures. The Glasgow Children’s Benefit Inventory (GCBI) was administered by phone to assess QOL improvement. Results One hundred sixty-five eligible subjects were identified. Eighty-nine subjects met the inclusion criteria. Data was collected for the 60 subjects that were reached. Forty-two patients had IT only while 18 had IT and Ad. The mean age was 10.7 ± 2.7 years, with 31 males and 29 females. The median duration of follow-up (25th, 75th percentile) was 38.1 months (24.6, 55.8). The median GCBI score (25th, 75th percentile) was 22.9 (6.3, 39.6) revealing an overall positive benefit in all domains. There was only one complication. Conclusions This study validates prior findings regarding improvement of QOL and safety of IT with or without Ad for children with CR and indicates it is maintained in the long term.

Long-term efficacy of spironolactone on pain, mood, and quality of life in women with fibromyalgia: An observational case series

2014 ◽  
Vol 5 (2) ◽  
pp. 63-71 ◽  
Author(s):  
Heinrich Wernze ◽  
Thomas Herdegen
Keyword(s):  
Quality Of Life ◽  
Rating Scale ◽  
Dose Range ◽  
Fibromyalgia Impact Questionnaire ◽  
Case Series ◽  
Treatment Resistant ◽  
Positive Effects ◽  
Long Term Treatment

AbstractObjectiveNo single drug is broadly efficacious in the long-term treatment of fibromyalgia syndrome (FMS). Spironolactone is known to ameliorate mood and tension headache or migraine in women with premenstrual syndrome or clinical signs of hyperandrogenism. In a case series of women with treatment resistant FMS spironolactone was therefore added to their medication, and they were observed for at least 12 months.Methods31 women with treatment-resistant FMS received spironolactone as add-on medication to various pain modulating drugs. 15 women responded to spironolactone and baseline data were compared with assessments over 12–14 months on treatment with spironolactone (ALDACTONE®) in dose range 100–200 mg/day. The efficacy was evaluated by the fibromyalgia impact questionnaire (FIQ) total score and 8 FIQ subtests, a German mood inventory (BSKE-EWL), and further assessments of changes in relevant psychological and physical complaints. 16 women had no effect and stopped the treatment early.ResultsThe subsequent data refer to the 15 responders. The FIQ total score (maximal score = 80) decreased from 56.6 ± 10.0 at baseline to 17.1 ± 11.9 (mean ± SD) 12–14 months later, and pain intensity on an 11 point numeric rating scale (NRS) decreased from 8.8 ± 1.6 to 2.6 ± 1.9 (mean ± SD). Similar changes in FIQ subscores were found for fatigue, morning tiredness, stiffness, anxiety, and depression. Emotional functioning consistently improved: positive mood from 20.0 ± 5.4 to 37.7 ± 5.4 (maximal score = 48), and negative mood from 35.4 ± 5.3 to 10.0 ± 4.4 (maximal score = 60) (each mean ± SD) as well as other mental and physical dysfunctions including non-restorative sleep. All these changes at 4–6 weeks remained on this level for 11–13 months. The drug was well-tolerated and safe, no serious adverse effects were observed. Regular monitoring of serum potassium did not reveal hyperkalemia. All 15 women were able to reduce or discontinue concomitant drugs.ConclusionFifteen of 31 women with otherwise treatment-resistant FMS experienced a number of prolonged beneficial effects from spironolactone on their complex pain-condition.Implications and discussionWe hypothesise that spironolactone affects several central and peripheral neurotransmitter systems such as γ-aminobutyric acid (GABA) activity and dopaminergic transmission. The high rate of non-responsive patients underlines that FMS may represent several subgroups. Pain relief and improvement of associated FHS-symptoms and positive effects on additional diseases or dysfunctions give reasons for marked and sustained improvement in the quality of life.Well-controlled, double-blind, and randomised trials are necessary to confirm our potentially very important observations.

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