Department of Medicine, Pulmonary, Critical Care and Sleep Division, Tufts Medical Center, Boston, MA.
Department of Medicine, Pulmonary, Critical Care and Sleep Division, Tufts Medical Center, Boston, MA.
Chest. 2014 Sep;146(3):e81-e83. doi: 10.1378/chest.13-2795.
Pulmonary hypertension (PH) is a known complication of Gaucher disease (GD) and splenectomy. Although it resembles World Health Organization (WHO) group 1 pulmonary arterial hypertension (PAH), PH due to GD or splenectomy is part of WHO group 5. There are no clinical trials testing therapies in PH due to GD or splenectomy. Several reports suggest that PAH-specific therapies are beneficial in patients with PH due to GD, although data are insufficient to formulate a treatment algorithm for these patients. The tyrosine kinase inhibitor imatinib has been investigated in the treatment of severe PAH, but not in PH WHO group 5. We report a patient with GD and splenectomy who developed PH that progressed while on conventional PAH-specific therapy and improved once imatinib was added to her treatment regimen. This is the first report, to our knowledge, describing significant subjective and objective improvements in response to imatinib in a patient with WHO group 5 PH.
肺高血压（PH）是戈谢病（GD）和脾切除术的已知并发症。尽管它类似于世界卫生组织（WHO）第 1 组肺动脉高压（PAH），但由于 GD 或脾切除术引起的 PH 属于 WHO 第 5 组。目前尚无临床试验测试针对 GD 或脾切除术后 PH 的治疗方法。有几项报告表明，PAH 特异性治疗对 GD 引起的 PH 患者有益，尽管数据不足以为这些患者制定治疗方案。酪氨酸激酶抑制剂伊马替尼已被研究用于治疗严重的 PAH，但不适用于 WHO 第 5 组 PH。我们报告了一名患有 GD 和脾切除术的患者，其在接受常规 PAH 特异性治疗期间 PH 进展，而一旦添加伊马替尼到她的治疗方案中，病情就得到改善。据我们所知，这是第一份描述患者对伊马替尼治疗有明显主观和客观改善的报告，该患者患有 WHO 第 5 组 PH。
