Recombinant human C1 esterase inhibitor for hereditary angioedema attacks: A European registry.

BACKGROUND: Hereditary angioedema (HAE) due to C1 esterase inhibitor deficiency (C1-INH-HAE) is characterized by recurrent swelling attacks. A European treatment registry was established to review the adverse event profile and efficacy of recombinant human C1 esterase inhibitor (rhC1-INH) for HAE attacks. METHODS: Individuals with C1-INH-HAE were enrolled following a decision to treat with rhC1-INH and provision of written informed consent. Medical history and baseline HAE information were collected at screening. Healthcare providers entered data on HAE attacks, response to treatment, and adverse events using a web-based questionnaire. RESULTS: From July 1, 2011, through December 1, 2019, 71 patients with C1-INH-HAE (30 male/41 female; mean age, 47.3 years; age range, 19-78 years) in 9 countries reported 2356 attacks and were treated with rhC1-INH. Before registry entry, patients, including 20 (28.2%) who were on maintenance therapy/prophylaxis at registry enrollment, experienced a mean of 25 HAE attacks per year (median, 16 [range, 0-185]). Most treated HAE attacks were abdominal (46.1%), followed by peripheral (38.3%), oro-facial-pharyngeal (14.8%), urogenital (3.2%), and laryngeal (2.6%). The mean rhC1-INH dose was 3307 U (43.3 U/kg). Patients reported symptom improvement within 4 h for 97.8% of attacks (2305/2356) with rhC1-INH; most attacks (99.8%; 2351/2356) required only 1 dose. Five attacks were treated with a second dose (total rhC1-INH dose administered for attack, 4200 U). No hypersensitivity, thrombotic/thromboembolic events, or drug-related serious adverse events were reported. CONCLUSION: The rhC1-INH treatment registry provided real-world data on the treatment of 2356 HAE attacks that were consistent with clinical trial data of rhC1-INH in patients with C1-INH-HAE.

A Three-Year Course of House Dust Mite Sublingual Immunotherapy Appears Effective in Controlling the Symptoms of Allergic Rhinitis.

Background Allergic rhinitis is the most common allergic disorder. Although the management of the disease is successful in many patients, based on guidelines, some of them remain with symptoms uncontrolled with pharmacotherapy. Presently, there is no substantiated information on the control of allergic rhinitis in patients who underwent sublingual immunotherapy. Objective The purpose of this prospective follow-up study was to assess the control of allergic rhinitis in adults after a three-year course of house dust mite sublingual immunotherapy. Methods This prospective real-life study was designed to include adults with moderate to severe allergic rhinitis sensitized to house dust mite who underwent a three-year course of sublingual immunotherapy. Control of symptoms was assessed by Rhinitis Control Assessment Test (RCAT) after three years of house dust mite sublingual immunotherapy. Additionally, patients assessed their symptoms by utilizing a visual analog scale. Results A total number of 86 consecutively enrolled patients (46 (53.49%) men; mean age 26.10 years (SD = 5.85)) with moderate to severe allergic rhinitis and clinically relevant sensitization to house dust mite were evaluated. When assessed by RCAT on the third year, 74 (86.05%) had well-controlled symptoms and 20 (27.03%) of them were completely controlled. A significant reduction in visual analog scale scores-from 7.52 cm at baseline to 2.31 cm-was established ( P < 0.0001). There was a strong negative correlation between RCAT scores and visual analog scale (r = -0.65; P < 0.01). Conclusion This study provided evidence that a three-year course of house dust mite sublingual immunotherapy appears effective in controlling the symptoms of allergic rhinitis.

Clinical characteristics of patients seeking medical advice for nasal symptoms in Bulgaria with special focus on children.

BACKGROUND: In an attempt to circumvent low response rates and high cost of classical epidemiological trials, we carried out a real-life survey among practicing physicians consulting patients for nasal symptoms. In this fragment of our work we analyze similarities and differences between children and adults and within the different strata of pediatric age. METHODS: A survey was carried out by 69 physicians across Bulgaria (general practitioners, allergists and otorhinolaryngologists) and made possible calculation of the proportion of subjects with nasal symptoms from all other patients seen. Its structure allowed classification of rhinitis according the ARIA guidelines. RESULTS: Out of the 1685 completed survey forms, 506 pertained to the age group below 18 years. The gender predominance differed in children and adults: 57.3 % vs. 42.8 % of males respectively, P < 0.001. The prevalence of persistent rhinitis in children was 55.7 %, lower than in adults, 63.3 %, P = 0.004. In both pediatric and adult patients moderately severe and severe forms of rhinitis prevailed, 93.7 % vs. 94.6 %, with nasal obstruction as leading symptom: 59.9 % vs. 58.8 %. Cough was significantly more prevalent among children, 72.5 %, gradually decreasing until reaching adulthood, 58.7 %, P < 0.001. Prevalence of doctor diagnosed asthma was also higher among children, 25.1 %, than in adults, 19.5 %, P = 0.011. A gradient for characteristics, which were different in children, emerged across the pediatric age strata. DISCUSSION: Our study uses an unorthodox design targeting the patient population visiting physicians' offices because of nasal symptoms, achieving a much higher level of credibility of the results at minimal expense. As we base our survey on international guidelines, we believe this approach demonstrates the applicability of such consensus documents for practical purposes when in the hands of qualified physicians. CONCLUSIONS: Moderate and severe rhinitis symptoms motivate patients and their guardians to seek medical advice. While nasal congestion is a leading bothersome symptom in both adults and children, specific other features characterize the pediatric age and differ across its strata.

Characteristics of a patient population seeking medical advice for nasal symptoms in Bulgaria.

BACKGROUND: The proportion of patients visiting general practitioners (GPs), otorhinolaryngologists (ORLs), and allergologists (ALRGs) for nasal complaints is unknown but important in estimating the number of subjects with nasal symptoms bothersome enough to warrant physician consultations and assessing nasal pathological conditions' burden on a national health care system. OBJECTIVE: The Symptoms of Nasal Inconvenience Fact Finding (SNIFF) survey was developed to (1) assess incidence of physician visits attributable to nasal complaints; (2) characterize patients' nasal conditions; and (3) outline differences across physician categories. METHODS: The SNIFF survey was completed over 20 days by Bulgarian GPs, ORLs, and ALRGs whom patients consulted for nasal symptoms. Survey forms differentiated type and severity of patients' conditions according to Allergic Rhinitis and its Impact on Asthma (ARIA) guidelines and ranked bothersome symptoms. Smell impairment, comorbidities, and prescription practices were documented. RESULTS: Sixty-nine physicians (30 GPs, 8 ORLs, 31 ALRGs) completed 1,685 surveys. The proportion of patients with nasal symptoms over the total patients seen was 15.7%: ALRGs, 18.0%; GPs, 14.6%; ORLs, 13.1%. Patients were classified as having intermittent (38.8%) or persistent (61.2%) rhinitis, with most having moderate/severe symptoms (94.4%). Congestion was the leading symptom in 59.1%. Smell was impaired in 69.8% of patients, asthma was present in 21.4%, and cough in 62.9%. ALRGs were more likely to diagnose and manage patients per ARIA guidelines than were ORLs or GPs. CONCLUSION: The SNIFF survey results demonstrate congestion's role as a leading symptom motivating patients to seek medical advice. SNIFF also uncovered differences in practices among different categories of health care providers.

Rhinitis and sleep apnea.

The nose and pharynx begin the upper airway system and represent a continuum. This is the biologic basis for the mutual influences of rhinitis and obstructive sleep apnea (OSA). Sleep-disordered breathing has a large differential diagnosis that includes snoring, upper airway resistance syndrome, and severe OSA. Nasal obstruction is an independent risk factor for OSA, but there is no correlation of daytime nasal resistance with the severity of OSA. However, nasal resistance was an independent predictor of apnea-hypopnea index in a recent study of nonobese OSA patients. Rhinitis alone is associated with mild OSA, but commonly causes microarousals and sleep fragmentation. Reduction of nasal inflammation with topical treatment improves sleep quality and subsequent daytime sleepiness and fatigue. Patient compliance with the nasal continuous positive airway pressure (nCPAP) device is relatively low, in part due to adverse nasal effects.