Bergeron A, Chagnon K, Feuillet S, Chevret S, Tazi A
Service de Pneumologie, Hôpital Saint-Louis, Assistance Publique- Hôpitaux de Paris, Université Denis Diderot, Paris 7, Paris, France.
Rev Mal Respir. 2009 Sep;26(7):794-800. doi: 10.1016/s0761-8425(09)72433-7.
Although it has not been evaluated prospectively, the usual treatment for obstructive airway disease after allogeneic hematopoietic stem cell transplantation, which is related to graft versus host disease, consists of intensification of systemic immunosuppressive therapy. However, this treatment has a limited efficacy and is associated with a significant number of serious adverse effects, particularly infectious. Alternative treatments are therefore required. Recently, clinical and functional improvement in patients with obstructive airway disease following allogenic hematopoietic stem cell transplantation treated with inhaled combined Budesonide/Formoterol has been retrospectively reported.
The present prospective multi-centered, randomised double-blind trial is designed to evaluate the efficacy of the combination of budesonide/formoterol (400/12 microg 2 inhalations bid) versus placebo in patients with moderate to severe obstructive airway disease, not requiring initiation or intensification of systemic immunosuppressive therapy for extra thoracic graft versus host disease. The primary outcome will be the improvement of FEV1 at 1 month of treatment. The secondary outcomes will be the clinical and functional pulmonary improvements at 6 months.
The leading hypothesis is that patients treated with inhaled combined Budesonide/Formoterol will show significant improvement of their clinical symptoms and pulmonary functional testing.
尽管尚未进行前瞻性评估，但异基因造血干细胞移植后与移植物抗宿主病相关的阻塞性气道疾病的常规治疗包括强化全身免疫抑制治疗。然而，这种治疗效果有限，且伴有大量严重不良反应，尤其是感染。因此需要替代治疗方法。最近，有回顾性报道称，接受吸入性布地奈德/福莫特罗联合治疗的异基因造血干细胞移植后阻塞性气道疾病患者的临床和功能有所改善。
本前瞻性多中心随机双盲试验旨在评估布地奈德/福莫特罗联合用药（400/12微克，每日两次，每次2吸）与安慰剂相比，对中度至重度阻塞性气道疾病患者的疗效，这些患者无需因胸外移植物抗宿主病而开始或强化全身免疫抑制治疗。主要结局将是治疗1个月时第一秒用力呼气容积（FEV1）的改善情况。次要结局将是6个月时临床和肺功能的改善情况。
主要假设是接受吸入性布地奈德/福莫特罗联合治疗的患者临床症状和肺功能测试将有显著改善。
