RESUMO
BACKGROUND: Hereditary angioedema (HAE) is often caused by low serum levels or functional deficiency in C1 inhibitor (C1-INH); however, in some cases, C1-INH serum level and function are measured as normal (HAE-nl-C1INH). Management of HAE-nl-C1INH is similar to management of HAE with C1-INH deficiency, including on-demand therapy for angioedema attacks and, potentially, prophylaxis. Recombinant human C1 esterase inhibitor (rhC1-INH) is indicated for treatment of acute HAE attacks. This study assessed the clinical profile and treatment outcomes in an HAE-nl-C1INH population with a history of rhC1-INH treatment. METHODS: Medical records containing patient-reported outcomes from ten US treatment centers were analyzed retrospectively for medical history, angioedema attack characteristics, attack treatments, and clinical outcomes. RESULTS: Twenty-three patients were included, with wide US geographic representation. Most patients (87.0%) were female; median age was 36.0 years (range, 19-67 years). Of 20 patients with available data, 4 had their first angioedema attack during childhood (aged <12 years), 3 during adolescence (aged 12-17 years), and 13 during adulthood (aged 18-29 years, n = 7; aged ≥30 years, n = 6). Median age at HAE-nl-C1INH diagnosis was 31.5 years (range, 9-59 years). Previous failed treatments included high-dose antihistamines (n = 20) and corticosteroids (n = 20). Use of US Food and Drug Administration (FDA)-approved HAE therapy positively impacted patient-reported assessments of angioedema attacks. Most patients were taking rhC1-INH or lanadelumab as prophylaxis and icatibant or rhC1-INH for acute management. Most patients reported improved disease control with these therapies, including reductions in angioedema attack frequency and severity. Although most patients were receiving prophylactic therapy, availability of treatment for breakthrough attacks was important. CONCLUSION: Findings from this retrospective study support use of FDA-approved HAE medications for prophylaxis and acute treatment of HAE attacks in patients with HAE-nl-C1INH. Individualized HAE treatment regimens were needed to optimize therapeutic outcomes.
RESUMO
We retrospectively reviewed 25 patients with foot or ankle osteomyelitis reported to a registry who were treated with daptomycin. The patients' clinical experience was analyzed and described at a median dose of 6 mg/kg (range, 4-6.2 mg/kg) and a median duration of 38 days (range, 6-59 days). Twenty-three patients received daptomycin as secondary or tertiary therapy, primarily for not responding to their prior antibiotic therapy (n = 15). Concomitant antibiotics were given to 11 patients, mostly for Gram-negative and/or anaerobic coverage. Methicillin-resistant Staphylococcus aureus was the most common pathogen overall (15 of 25 patients). The median followup interval was 9 weeks (range, 0.5-77 weeks). Outcomes at the end of therapy were 16 patients' symptoms resolved, eight patients improved, and one patient did not respond to therapy; at followup, 19 patients' symptoms resolved, three patients improved, and three patients did not respond to therapy. Ongoing antibiotics were given to 52% of patients for a minimum of 8 days (median, 30 days; range, 8-232 days). Four patients with an implant (all removed) were successfully treated. Daptomycin appears promising for foot and ankle osteomyelitis caused by Gram-positive bacteria. Prospective, controlled clinical trials of daptomycin for osteomyelitis are warranted.