脑动静脉畸形患者管理实用研究中的患者选择
Patient Selection in a Pragmatic Study on the Management of Patients with Brain Arteriovenous Malformations.
作者信息
Raymond Jean, Benomar Anass, Gentric Jean-Christophe, Magro Elsa, Nico Lorena, Bacchus Emma, Klink Ruby, Iancu Daniela, Weill Alain, Roy Daniel, Bojanowski Michel W, Chaalala Chiraz, Eker Omer, Pelissou-Guyotat Isabelle, Piotin Michel, Aldea Sorin, Barbier Charlotte, Gaberel Thomas, Papagiannaki Chrysanthi, Derrey Stéphane, Nguyen Thanh N, Abdalkader Mohamad, Cognard Christophe, Januel Anne-Christine, Sabatier Jean-François, Jecko Vincent, Barreau Xavier, Costalat Vincent, Le Corre Marine, Gauvrit Jean-Yves, Morandi Xavier, Biondi Alessandra, Thines Laurent, Desal Hubert, Bourcier Romain, Beaujeux Rémy, Proust François, Viard Geraldine, Gevry Guylaine, Darsaut Tim E
机构信息
Department of Radiology, Service of Neuroradiology, Centre Hospitalier de l'Université de Montréal (CHUM), Montreal, Quebec, Canada.
Department of Radiology, Service of Neuroradiology, Centre Hospitalier de l'Université de Montréal (CHUM), Montreal, Quebec, Canada.
出版信息
World Neurosurg. 2023 Apr;172:e611-e624. doi: 10.1016/j.wneu.2023.01.098. Epub 2023 Feb 3.
BACKGROUND
The Treatment of Brain Arteriovenous Malformations Study (TOBAS) is an all-inclusive pragmatic study comprising 2 randomized clinical trials (RCTs). Patients excluded from the RCTs are followed in parallel treatment and observation registries, allowing a comparison between RCT and registry patients.
METHODS
The first randomized clinical trial (RCT-1) offers 1:1 randomized allocation of intervention versus conservative management for patients with arteriovenous malformation (AVM). The second randomized clinical trial (RCT-2) allocates 1:1 pre-embolization or no pre-embolization to surgery or radiosurgery patients judged treatable with or without embolization. Characteristics of RCT patients are reported and compared to registry patients.
RESULTS
From June 2014 to May 2021, 1010 patients with AVM were recruited; 498 patients were observed and 373 were included in the treatment registries. Randomized allocation in RCT-1 was applied to 139 (26%) of the 512 patients (including 127 of 222 [57%] with unruptured AVMs) considered for curative treatment. RCT-1 AVM patients differed (in rupture status, Spetzler-Martin grade and baseline modified Rankin Score) from those in the observation or treatment registries (P < 0.001). Most patients had small (<3 cm; 71%) low-grade (Spetzler-Martin I-II; 64%) unruptured (91%) AVMs. The allocated management was conservative (n = 71) or curative (n = 68), using surgery (n = 39), embolization (n = 16), or stereotactic radiosurgery (n = 13). Pre-embolization was considered for 179/309 (58%) patients allocated/assigned to surgery or stereotactic radiosurgery; 87/179 (49%) were included in RCT-2. RCT-2 patient AVMs differed in size, eloquence and grade from patients of the pre-embolization registry (P < 0.01). Most had small (<3 cm in 82%) low-grade (83%) AVMs in non-eloquent brain (64%).
CONCLUSIONS
Patients included in the RCTs differ significantly from registry patients. Meaningful results can be obtained if multiple centers actively participate in the TOBAS RCTs.
背景
脑动静脉畸形治疗研究（TOBAS）是一项综合性实用研究，包括两项随机临床试验（RCT）。被排除在随机临床试验之外的患者在平行的治疗和观察登记处进行随访，从而可以对随机临床试验患者和登记处患者进行比较。
方法
第一项随机临床试验（RCT - 1）为患有动静脉畸形（AVM）的患者提供1:1随机分配干预与保守治疗。第二项随机临床试验（RCT - 2）为判断可通过栓塞或不通过栓塞进行手术或立体定向放射外科治疗的患者按1:1分配预先栓塞或不进行预先栓塞。报告随机临床试验患者的特征并与登记处患者进行比较。
结果
2014年6月至2021年5月，招募了1010例动静脉畸形患者；498例患者进行观察，373例纳入治疗登记处。RCT - 1中的随机分配应用于512例考虑进行根治性治疗的患者中的139例（26%）（包括222例未破裂动静脉畸形患者中的127例[57%]）。RCT - 1中的动静脉畸形患者与观察或治疗登记处的患者在（破裂状态、斯佩茨勒 - 马丁分级和基线改良Rankin评分方面）存在差异（P < 0.001）。大多数患者患有小的（<3 cm；71%）低级别（斯佩茨勒 - 马丁I - II级；64%）未破裂（91%）的动静脉畸形。分配的治疗为保守治疗（n = 71）或根治性治疗（n = 68），采用手术（n = 39）、栓塞（n = 16）或立体定向放射外科治疗（n = 13）。179/309（58%）被分配/指定进行手术或立体定向放射外科治疗的患者考虑进行预先栓塞；87/179（49%）纳入RCT - 2。RCT - 2患者的动静脉畸形在大小、功能区位置和分级方面与预先栓塞登记处的患者不同（P < 0.01）。大多数患者患有小的（82%<3 cm）低级别（83%）非功能区脑动静脉畸形（64%）。
结论
随机临床试验纳入的患者与登记处患者有显著差异。如果多个中心积极参与TOBAS随机临床试验，可获得有意义的结果。
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