Clinical characteristics and real-life diagnostic approaches in all Danish children with hereditary angioedema

Hereditary angioedema (HAE) due to C1-inhibitor (C1-INH) deficiency is a rare disease characterized by recurrent swellings. This study aims to determine (i) the clinical characteristics of the HAE patient population from Poland, and (ii) real-life patients’ treatment practices. A cross-sectional study involved 138 adult HAE patients (88 females, 50 males) treated in six regional HAE centers in Poland. Consecutive patients during routine follow-up visits underwent a structured medical interview on the clinical characteristics of the course and treatment of HAE attacks within the last six months. A total of 118 of 138 patients was symptomatic. They reported in total 2835 HAE attacks predominantly peripheral and abdominal, treated with plasma-derived C1-INH (61.4%), icatibant (36.7%) and recombinant C1-INH (1.9%). An amount of 116 patients carried the rescue medication with them while traveling, and 74 patients self-administrated on demand treatment. There were twice as many symptomatic women (n = 78) as there were men (n = 40). Women treated their HAE attacks significantly more often than men. Older patients (≥65 years) reported a longer delay in diagnosis, and practiced the self-administration of rescue medication less frequently in comparison to other patients. Clinical features of the surveyed population are similar to other European, but not Asian, HAE patient groups. Self-administration still remains an unmet medical need. Some distinct HAE patients may require special attention due to the severe course of the disease (females) or a delay in diagnosis (the elderly).

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Individually Tailored Prophylaxis with C1-INH Concentrate in four Pediatric Patients with Hereditary Angioedema (HAE) – Real Life Data

10.26226/morressier.5acc8ad5d462b8028d89aa9b ◽

2018 ◽

Author(s):

Inmaculada Martinez Saguer

Keyword(s):

Hereditary Angioedema ◽

Pediatric Patients ◽

Real Life ◽

Life Data ◽

Real Life Data

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Mortality trends in an ambulatory multidisciplinary heart failure unit from 2001 to 2018

Scientific Reports ◽

10.1038/s41598-020-79926-3 ◽

2021 ◽

Vol 11 (1) ◽

Author(s):

Giosafat Spitaleri ◽

Josep Lupón ◽

Mar Domingo ◽

Evelyn Santiago-Vacas ◽

Pau Codina ◽

...

Keyword(s):

Heart Failure ◽

Linear Regression ◽

Cardiovascular Mortality ◽

Clinical Characteristics ◽

Real Life ◽

Cardiovascular Death ◽

Progressive Decrease ◽

Mortality Trends ◽

Heart Failure Unit ◽

Ambulatory Patients

AbstractTo assess mortality trends at 1 and 3 years from 2001 to 2018 in a real-life cohort of HF outpatients from different etiologies with depressed and preserved LVEF. A total of 2368 consecutive patients with HF (mean age 66.4 ± 12.9 years, 71% men, 15.4% with preserved LVEF) admitted to a HF clinic from August 2001 to September 2018 were included in the study. Patients were divided into five quintiles (Q) according to the period of admission. Trends for all-cause and cardiovascular mortality from Q1 to Q5 were assessed by linear regression. Patients with LVEF < 50% had a progressive decrease in the rates of all-cause and cardiovascular death at 1 year (12.1% in Q1 to 6.5% in Q5, p = 0.003; and 8.4% in Q1 to 3.8% in Q5, p = 0.007, respectively) and 3 years (30.5% in Q1 to 17.0% in Q5, p = 0.003; and 23.9% in Q1 to 9.8% in Q5, p = 0.003, respectively). These trends remained significant after adjusting for clinical characteristics and risk. No significant trend in mortality was observed in patients with LVEF ≥ 50%. In a cohort of real-life ambulatory patients with HF, mortality progressively declined in patients with LVEF < 50%, but the same trend was not observed in patients with preserved LVEF.

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P-116: The impact of SLiM criteria in myeloma in a real-life population: clinical characteristics, treatment and outcome from the Australian and New Zealand Myeloma and Related Diseases Registry (MRDR)

Clinical Lymphoma Myeloma & Leukemia ◽

10.1016/s2152-2650(21)02243-6 ◽

2021 ◽

Vol 21 ◽

pp. S98

Author(s):

P. Joy Ho ◽

Elizabeth Moore ◽

Cameron Wellard ◽

Hang Quach ◽

Hilary Blacklock ◽

...

Keyword(s):

New Zealand ◽

Clinical Characteristics ◽

Real Life ◽

The Impact

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Practicalities of a reduced volume formulation of a C1-INH concentrate for the treatment of hereditary angioedema: real-life experience

Allergy Asthma & Clinical Immunology ◽

10.1186/s13223-018-0267-4 ◽

2018 ◽

Vol 14 (1) ◽

Cited By ~ 2

Author(s):

John Dempster

Keyword(s):

Hereditary Angioedema ◽

Life Experience ◽

Real Life ◽

Reduced Volume

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PsART-ID inception cohort: clinical characteristics, treatment choices and outcomes of patients with psoriatic arthritis

Rheumatology ◽

10.1093/rheumatology/keaa663 ◽

2020 ◽

Author(s):

Gizem Ayan ◽

Sibel Zehra Aydin ◽

Gezmis Kimyon ◽

Cem Ozisler ◽

Ilaria Tinazzi ◽

...

Keyword(s):

Psoriatic Arthritis ◽

Clinical Characteristics ◽

Life Experience ◽

Real Life ◽

Treatment Strategies ◽

Inception Cohort ◽

Disease Pattern ◽

Involvement Rate ◽

Axial Involvement ◽

Nail Involvement

Abstract Objectives Our aim is to understand clinical characteristics, real-life treatment strategies, outcomes of early PsA patients and determine the differences between the inception and established PsA cohorts. Methods PsArt-ID (Psoriatic Arthritis- International Database) is a multicentre registry. From that registry, patients with a diagnosis of PsA up to 6 months were classified as the inception cohort (n==388). Two periods were identified for the established cohort: Patients with PsA diagnosis within 5–10 years (n = 328), ≥10 years (n = 326). Demographic, clinical characteristics, treatment strategies, outcomes were determined for the inception cohort and compared with the established cohorts. Results The mean (s.d.) age of the inception cohort was 44.7 (13.3) and 167/388 (43.0%) of the patients were male. Polyarticular and mono-oligoarticular presentations were comparable in the inception and established cohorts. Axial involvement rate was higher in the cohort of patients with PsA ≥10 years compared with the inception cohort (34.8% vs 27.7%). As well as dactylitis and nail involvement (P = 0.004, P = 0.001 respectively). Both enthesitis, deformity rates were lower in the inception cohort. Overall, 13% of patients in the inception group had a deformity. MTX was the most commonly prescribed treatment for all cohorts with 10.7% of the early PsA patients were given anti-TNF agents after 16 months. Conclusion The real-life experience in PsA patients showed no significant differences in the disease pattern rates except for the axial involvement. The dactylitis, nail involvement rates had increased significantly after 10 years from the diagnosis and the enthesitis, deformity had an increasing trend over time.

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