Kasem AliSliman R, Regev S, Ghanim B, Hamad Saied M
Seminars in arthritis and rheumatism •
PFAPA (periodic fever, aphthous stomatitis, pharyngitis, adenitis) syndrome is the most common periodic fever disorder in children, causing recurrent debilitating episodes that impose a substantial burden on children and their families. Current therapeutic approaches primarily rely on corticosteroid administration, which provides rapid symptom resolution but fails to address the underlying inflammatory cascade, prevent future attacks, and carries the risk of side effects. To determine the therapeutic efficacy of colchicine prophylaxis in reducing attack frequency and extending disease-free intervals in PFAPA patients, compared to standard of care management alone. This retrospective cohort study included 55 pediatric PFAPA patients, 15 receiving colchicine prophylaxis, and 40 controls managed with standard care alone. The primary outcome was the change in inter-attack interval duration from baseline study completion. Secondary analyses examined treatment response by FMF genetic status and survival analysis for time to next attack. Both groups showed comparable baseline characteristics, except higher FMF mutation prevalence in the colchicine group (73.3% vs. 27.5%, P = 0.002). Patients receiving colchicine experienced a dramatic improvement in inter-attack intervals (median change: 60 days, IQR: 51) compared with controls (median change: 0 days, IQR: 0; P < 0.001). Colchicine's therapeutic benefit was consistent regardless of FMF genetic status. Colchicine prophylaxis significantly reduces PFAPA attack frequency, with therapeutic benefits that are independent of FMF genetic status. These findings support colchicine as an effective first-line prophylactic treatment for PFAPA patients with frequent episodes or families concerned about frequent steroid use, representing a paradigm shift from reactive to preventive management.
Otolaryngology--head and neck surgery : official journal of American Academy of Otolaryngology-Head and Neck Surgery •
To compare surgical versus medical treatment approaches for periodic fever, aphthous stomatitis, pharyngitis, and adenitis (PFAPA) syndrome. PubMed, Embase, Web of Science, and Cochrane. We followed the Preferred Reporting Items for Systematic Reviews and Meta-analyses-Network Meta-analyses checklist. Two authors independently reviewed the studies. Inclusion criteria comprised randomized controlled trials and cohort studies. Non-English studies, along with case series and review articles, were excluded. The primary outcome was the incidence of persistent symptoms following surgery, compared to medical treatment. Secondary outcomes included complications. The search identified 693 publications. After applying the inclusion and exclusion criteria, nine studies were included (three randomized controlled trials and six cohort studies), comprising a total of 691 patients (256 [37%] females, mean age: 2.97 years, and interquartile range: 2.3-3.3). Patients were treated with either tonsillectomy (n = 201), intracapsular tonsillectomy (IT, n = 24), or medical treatment (n = 466). Surgery was associated with a higher likelihood of symptom resolution compared to medical treatment (odds ratio [OR]: 11.7, 95% CI: 2.14-63.94). However, heterogeneity was observed across studies (I = 80.8%, P < .01). A sensitivity analysis was performed, including randomized controlled trials. Both tonsillectomy (OR: 34.15, 95% CI: 3.77-308.95) and IT (OR: 21, 95% CI: 1.5-293.25) were associated with a greater likelihood of symptom resolution. The pooled complication rate was 6.6%, with a higher incidence in patients who underwent tonsillectomy versus IT (46/201 vs 0/24, P = .008). The pooled rate of symptom recurrence was 13.6% in patients following tonsillectomy and 37.5% in IT. Surgical management of PFAPA with tonsillectomy was superior to medical treatment. Tonsillectomy resulted in a lower recurrence rate of symptoms compared to IT, with a comparable incidence of complications to existing literature. However, given the limited number of patients in the IT group, these findings should be interpreted with caution, and further randomized studies are warranted.
Amarilyo G, Harel L, Abu Ahmad S, Abu Rumi M, Brik R , et al.
The Journal of pediatrics •
To evaluate the ethnic distribution of Israeli patients with the syndrome of periodic fever, aphthous stomatitis, pharyngitis, and adenitis (PFAPA). The medical records of patients with PFAPA attending 2 pediatric tertiary medical centers in Israel from March 2014 to March 2019 were retrospectively reviewed. Patients with concomitant familial Mediterranean fever were excluded. Ethnicity was categorized as Mediterranean, non-Mediterranean, and multiethnic. Findings were compared with patients with asthma under treatment at the same medical centers during the same period. The cohort included 303 patients with PFAPA and 475 with asthma. Among the patients with PFAPA, 178 (58.7%) were of Mediterranean descent (Sephardic Jews or Israeli Arabs), 96 (33.0%) were multiethnic, and 17 (5.8%) were of non-Mediterranean descent (all Ashkenazi Jews). Patients with PFAPA had a significantly higher likelihood of being of Mediterranean descent than the patients with asthma (58.7% vs 35.8%; P < .0001). The Mediterranean PFAPA subgroup had a significantly earlier disease onset than the non-Mediterranean subgroup (2.75 ± 1.7 vs 3.78 ± 1.9 years, P < .04) and were younger at disease diagnosis (4.77 ± 2.3 vs 6.27 ± 2.9 years, P < .04). PFAPA was significantly more common in patients of Mediterranean than non-Mediterranean descent. Further studies are needed to determine the genetic background of these findings.