が、2026/4/27FDAがdatalock後に9例でPrimary end pointの再調整があったと報告し、承認撤回を提案。再調整がなかった場合、52wでの有意差には影響が出たが、26w後の非劣性metには影響がなかった模様。これに対し、Amgenはまだ聴聞する機会があるようで、最終決定まではTavneosは米国市場に残るようです。
The two academic authors of the article by Jayne et al., Avacopan for the Treatment of ANCA Associated Vasculitis, N Engl J Med 2021;384:599-609,1 request retraction of the article because, according to an ongoing Food and Drug Administration investigation conducted after publication, and without the knowledge of these two authors, the primary end-point assessments in nine patients were readjudicated after database lock and trial unblinding. This was not disclosed in the article and is inconsistent with proper research conduct. The editors therefore retract the article. 2人の著者が論文撤回を要求した。出版後に進行中のFDAの審査に応じて。この2人の著者の知らない状況で9人のthe primary end-pointの評価がdatabaseロックの後、つまり試験の盲検化がなくなってから再調整された。この事は論文に記載されておらず、適切な調査行為に相当しない。それゆえ、editorsは論文を撤回する。
この撤回内容に相当するPrimary endpointとは？
A total of 331 patients underwent randomization; 166 were assigned to receive avacopan, and 165 were assigned to receive prednisone. The mean BVAS at baseline was 16 in both groups. Remission at week 26 (the first primary end point) was observed in 120 of 166 patients (72.3%) receiving avacopan and in 115 of 164 patients (70.1%) receiving prednisone (estimated common difference, 3.4 percent age points; 95% confidence interval [CI], −6.0 to 12.8; P<0.001 for noninferiority; P = 0.24 for superiority).
331例がランダムに割り付けを受け、166例がavacopan、165例がprednisone。平均BVASは両群とも16。26w後の寛解（最初のPrimary end point）はavacopan 120/166 (72.3%) 、 prednisone 115/164 (70.1%) で、estimated common difference, 3.4 percent age points; 95% confidence interval [CI], −6.0 to 12.8; P<0.001 for noninferiority; P = 0.24 for superiority).
Adjunctive therapy should be used in selected patients with GCA (refractory or relapsing disease, the presence or an increased risk of GC related adverse effects or complications) using tocilizumab. Methotrexate may be used as an alternative.
Recommendation: For patients with newly diagnosed GCA, we conditionally recommend the use of oral glucocorticoids with tocilizumab over oral glucocorticoids alone. A trial published in 2017 (8) demonstrated that tocilizumab has a significant glucocorticoid- sparing effect in GCA, and thus, tocilizumab should be considered for initial treatment. However, methotrexate with glucocorticoids, as well as glucocorticoids alone, can also be considered as initial treatment for newly diagnosed GCA. The decision to treat with tocilizumab and glucocorticoids, methotrexate and glucocorticoids, or glucocorti coid monotherapy for initial therapy should be made based on the physician’s experience and the patient’s clinical condition, values, and preferences. Lack of long- term follow-up data on tocilizumab and cost may limit its use (8,54). Abatacept with glucocorticoids can also be considered if these other agents are not effective (55).
