Shamim, Ejaz A.

Abstract Objectives The lack of uniform terminology and abbreviations for anti-synthetase syndrome has led to significant challenges in research and clinical practice. This study aimed to establish an international consensus on a standardized nomenclature and abbreviation among a diverse group of global myositis experts and patient representatives. Methods This qualitative project was approved by the International Myositis Assessment and Clinical Studies Group (IMACS). Based on the outcomes of a literature review and preliminary survey, list of terms for nomenclature and abbreviations was finalized. Subsequently, two rounds of the Delphi survey were conducted among myositis experts and patient participants to ascertain current practices and preferences. Using Nominal Group Technique (NGT), the project steering committee developed a consensus nomenclature and abbreviation. Results A total of 241 and 132 myositis experts from six continents, 35 countries and 9 specialities participated in round 1 and round 2 of the Delphi survey, respectively. The results indicated a preference for the terms anti-synthetase syndrome, antisynthetase syndrome and anti-tRNA synthetase syndrome. The abbreviations ASS, ASyS and ASSD were most used, though ASS received notable disapproval. The steering committee, including twenty-five myositis experts and three patient representatives from diverse backgrounds and geographical locations, reached a consensus on ‘anti-synthetase syndrome’ as the preferred nomenclature and ASyS as the preferred abbreviation, with 100% and 93.1% votes, respectively. Conclusion The terms anti-synthetase syndrome and ASyS should be adopted as the standard nomenclature and abbreviation in both research and clinical practice. This standardization is expected to increase consistency within the literature.

Objective To evaluate the efficacy and tolerability of single pulse transcranial magnetic stimulation (sTMS) for the preventive treatment of migraine. Background sTMS was originally developed for the acute treatment of migraine with aura. Open label experience has suggested a preventive benefit. The objective of this trial was to evaluate the efficacy and tolerability of sTMS for migraine prevention. Methods The eNeura SpringTMS Post-Market Observational U.S. Study of Migraine (ESPOUSE) Study was a multicenter, prospective, open label, observational study. From December 2014 to March 2016, patients with migraine (n = 263) were consented to complete a 1-month baseline headache diary followed by 3 months of treatment. The treatment protocol consisted of preventive (four pulses twice daily) and acute (three pulses repeated up to three times for each attack) treatment. Patients reported daily headache status, medication use, and device use with a monthly headache diary. The primary endpoint, mean reduction of headache days compared to baseline, was measured over the 28-day period during weeks 9 to 12. The primary endpoint was compared to a statistically-derived placebo estimate (performance goal). Secondary endpoints included: 50% responder rate, acute headache medication consumption, HIT-6, and mean reduction in total headache days from baseline of any intensity. Results Of a total of 263 consented subjects, 229 completed a baseline diary, and 220 were found to be eligible based on the number of headache days. The device was assigned to 217 subjects (Safety Data Set) and 132 were included in the intention to treat Full Analysis Set. For the primary endpoint, there was a −2.75 ± 0.40 mean reduction of headache days from baseline (9.06 days) compared to the performance goal (−0.63 days) ( p < 0.0001). The 50% responder rate of 46% (95% CI 37%, 56%) was also significantly higher ( p < 0.0001) than the performance goal (20%). There was a reduction of −2.93 (5.24) days of acute medication use, headache impact measured by HIT-6, −3.1 (6.4) ( p < 0.0001), and total headache days of any intensity −3.16 days (5.21) compared to the performance goal (−0.63 days) ( p < 0.0001). The most common adverse events were lightheadedness (3.7%), tingling (3.2%), and tinnitus (3.2%). There were no serious adverse events. Conclusions This open label study suggests that sTMS may be an effective, well-tolerated treatment option for migraine prevention. Trial registration number NCT02357381