ObjectiveAim of this paper is to illustrate the methodology, design, and development of the AutoInflammatory Disease Alliance (AIDA) International Registry dedicated to patients with the Periodic Fever, Aphthous stomatitis, Pharyngitis, and cervical Adenitis (PFAPA) syndrome.MethodsThis is a physician-driven, non-population- and electronic-based registry proposed to gather real-world demographics, clinical, laboratory, instrumental and socioeconomic data from PFAPA patients. Data recruitment is realized through the on-line Research Electronic Data Capture (REDCap) tool. This registry is thought to collect standardized information for clinical research leading to solid real-life evidence. The international scope and the flexibility of the registry will facilitate the realization of cutting-edge study projects through the constant updating of variables and the possible merging and transfer of data between current and future PFAPA registries.ResultsA total of 112 centers have already been involved from 23 countries and 4 continents starting from August 24th, 2021, to April 6th, 2022. In total 56/112 have already obtained the formal approval from their local Ethics Committees. The platform counts 321 users (113 principal investigators, 203 site investigators, two lead investigators, and three data managers). The registry collects retrospective and prospective data using 3,856 fields organized into 25 instruments, including PFAPA patient's demographics, medical histories, symptoms, triggers/risk factors, therapies, and impact on the healthcare systems.ConclusionsThe development of the AIDA International Registry for PFAPA patients will enable the on-line collection of standardized data prompting real-life studies through the connection of worldwide groups of physicians and researchers. This project can be found on https://clinicaltrials.gov NCT 05200715.
Patients with familial Mediterranean fever and spondylitis often fail to respond to conventional and biologic therapies. Achieving remission in these patients usually requires conventional and biologic treatment combinations. Combination of biologic agents may be a promising option for patients with familial Mediterranean fever and spondylitis who have refractory disease. Until recently, limited evidence existed regarding the efficacy and safety of this treatment strategy. To address this, our report presented a case series of 4 patients with familial Mediterranean fever and spondylitis who were resistant to standard treatments and in whom remission is achieved only with dual biologic therapy. The authors also conducted a literature search for studies that reported dual biological therapy in inflammatory diseases.
ObjectiveTo evaluate the potential role of Streptococcus salivarius K12 (SSK12) in controlling febrile flares in patients with Periodic Fever, Aphthous stomatitis, Pharyngitis, and cervical Adenitis (PFAPA) syndrome. Further aims were to assess the impact of SSK12 on (i) flare duration, (ii) variation in the degree of the highest body temperature during flares, (iii) steroid-sparing effect, and (iv) change of PFAPA accompanying symptoms before and after SSK12 introduction.Patients and methodsThe medical charts from 85 pediatric patients with PFAPA syndrome (49 males and 36 females) enrolled in the AIDA registry and treated with SSK12 for a median period of 6.00 ± 7.00 months in the period between September 2017 and May 2022 were examined. Children recruited had a median time of disease duration of 19.00 ± 28.00 months.ResultsThe number of febrile flares significantly decreased comparing the 12 months before [median (IQR), 13.00 (6.00)] and after SSK12 initiation [median (IQR), 5.50 (8.00), p < 0.001]. The duration of fever was significantly reduced from 4.00 (2.00) days to 2.00 (2.00) days [p < 0.001]. Similarly, the highest temperature in°C was found significantly lower in the last follow-up assessment [median (IQR), 39.00 (1.00)] compared to the period prior to SSK12 start [median (IQR), 40.00 (1.00), p < 0.001]. Steroid load (mg/year) of betamethasone (or any equivalent steroid) significantly decreased between 12 months before treatment with SSK12 [median (IQR), 5.00 (8.00) mg/year] and the last follow-up visit [median (IQR), 2.00 (4.00) mg/year, p < 0.001]. The number of patients experiencing symptoms including pharyngitis/tonsillitis (p < 0.001), oral aphthae (p < 0.001) and cervical lymphadenopathy (p < 0.001) significantly decreased following SSK12.ConclusionSSK12 prophylaxis given for at least 6.00 months was found to reduce febrile flares of PFAPA syndrome: in particular, it halved the total number per year of fever flares, shortened the duration of the single febrile episode, lowered body temperature by 1°C in the febrile flare, provided a steroid-sparing effect, and significantly reduced the accompanying symptoms related to the syndrome.
BackgroundFamilial Mediterranean fever (FMF) is an autoinflammatory disease characterized by recurrent attacks of fever, serositis, and musculoskeletal symptoms (1, 2). Arthritis is the most common musculoskeletal symptom of attacks and also included in diagnostic criteria of FMF (3). If it is specifically queried, myalgia may be detected in up to 20-25% of the patients with FMF.(4,5).ObjectivesIn our study, we aim to assess the localization of attack-related myalgia and associated parameters in patients with FMF.MethodsA total of 349 consecutive patients followed by FMF in our clinic were enrolled in the study and asked for attack-induced myalgia and if present, localization of muscle groups on the mannequin body parts diagram.Attack frequency, duration, and disease activity were evaluated with the AutoInflammatory Diseases Activity Index (AIDAI) scoring system (6). Patients were also asked for work/study day loss during attacks and patient acceptable symptom state (PASS) status (7).Results126 patients showed attack myalgia (36%); attack duration, frequency, severity were significantly higher in patients with attack-myalgia (p<0,005). Most common muscle groups were calves, lower back, and latissimus dorsi muscles in order. Myalgia was most commonly accompanied by arthritis (p<0,002). Patients with myalgia have a higher frequency of colchicine resistance and work/study day loss due to attacks.ConclusionOur results conclude that myalgia is an important domain of attacks and causes absenteeism and uncontrolled disease activity. Treatment of myalgia attacks may provide controlled disease activity, and prevent absenteeism from work/school.References[1]El-Shanti H, Majeed HA, El-Khateeb M. Familial mediterranean fever in Arabs.Lancet. 2006;367(9515):1016–24.[2]Majeed HA, Al-Qudah AK, Qubain H, Shahin HM. The clinical patterns of myalgia in children with familial Mediterranean fever. Semin Arthritis Rheum. 2000;30(2):138–43.[3]Gattorno M, Hofer M, Federici S, Vanoni F et al. Eurofever Registry and the Paediatric Rheumatology International Trials Organisation (PRINTO). Classification criteria for autoinflammatory recurrent fevers. Ann Rheum Dis. 2019 Aug;78(8):1025-1032. doi: 10.1136/annrheumdis-2019-215048.[4]Zemer D. Muscle pains in familial Mediterranean fever. Harefuah 1984; 106: 232-233.[5]Majeed HA. Differential diagnosis of fever of unknown origin in children. Curr Opin Rheumatol 2000; 12: 439-444.[6]Piram M, Frenkel J, Gattorno M et al. EUROFEVER and EUROTRAPS networks. A preliminary score for the assessment of disease activity in hereditary recurrent fevers: results from the AIDAI (Auto-Inflammatory Diseases Activity Index) Consensus Conference. Ann Rheum Dis. 2011 Feb;70(2):309-14. doi: 10.1136/ard.2010.132613.[7]Salaffi F, Carotti M, Gutierrez M, Di Carlo M, De Angelis R. (2015) Patient Acceptable Symptom State in Self-Report Questionnaires and Composite Clinical Disease Index for Assessing Rheumatoid Arthritis Activity: Identification of Cut-Off Points for Routine Care. Biomed Res Int. 2015:930756. doi: 10.1155/2015/930756.Table 1.Comparison of clinical and laboratory parameters between patients with /without myalgia attacksPatients with myalgia attackPatients without myalgia attackp valueAge (years)36,33 ±10, 6837,9±11, 48>0,05Sex (female/male)78/43138/87>0,05Follow-up time (years)16,117,6>0,05Dominant attack(number)Peritonitis4255>0,05Arthritis11390,012Pleuritis1310>0,05Only fever47>0,05AIDAI score (mean, 0-175)127,4080,68<0,05VAS score for pain (median score, during attack. 0-10)85<0,05Colchicine resistance (number ofpatients)69250,003Colchicine-resistant29840,002Colchicine-sensitivePASS status (number)Need additional treatment4160,016Satisfied from treatment34740,010Work/study day loss (number)4860,003AcknowledgementsAll study population signed informed consent for both participation and publication. Local Ethical Committee of the university approved the study.Disclosure of InterestsNone declared
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