Kulkarni S, Powles R, Sirohi B, Treleaven J, Saso R, Horton C, Atra A, Ortin M, Rudin C, Goyal S, Sankpal S, Meller S, Pinkerton C R, Mehta J, Singhal S
Leukaemia and Myeloma Units, The Royal Marsden Hospital, Downs Road, Sutton, Surrey SM2 5PT, UK.
Bone Marrow Transplant. 2003 Jul;32(2):165-70. doi: 10.1038/sj.bmt.1704033.
Thalidomide was used to treat acute (n=21) or chronic (n=59) graft-vs-host disease (GVHD) in 80 haematopoietic stem cell allograft recipients after failure to respond to the combination of cyclosporine and corticosteroids with or without other agents. The median time to onset of acute GVHD was 11 days, and thalidomide was started at a median of 48 days post transplant. In addition to corticosteroids and cyclosporine, 13 patients had also received other agents before thalidomide. None of the patients responded and all died of acute GVHD. For chronic GVHD (limited in 13, extensive in 46), thalidomide was started at a median of 385 days post transplant. In addition to corticosteroids and cyclosporine, 34 patients received azathioprine concomitantly. In all patients, thalidomide was added to the ongoing immunosuppressive regimen. The median duration of therapy with thalidomide was 60 days (range, 11-1210; <2 weeks in 11). In total, 13 patients (22%) had complete response, eight (14%) partial response and 38 (64%) no response. Response rates were comparable for limited (39%) and extensive (33%) chronic GVHD. At a median of 53 months, 19 patients are alive, 13 without evidence of chronic GVHD. Survival was significantly better in patients who responded to thalidomide. The principal causes of death were progressive chronic GVHD (n=29) and relapsed leukaemia (n=7). In conclusion, thalidomide has no activity in acute GVHD, but has some activity in chronic GVHD in combination with other agents.
沙利度胺用于治疗80例造血干细胞同种异体移植受者的急性（n = 21）或慢性（n = 59）移植物抗宿主病（GVHD），这些患者在使用或未使用其他药物的情况下，对环孢素和皮质类固醇联合治疗无反应。急性GVHD的中位发病时间为11天，沙利度胺在移植后中位48天开始使用。除皮质类固醇和环孢素外，13例患者在使用沙利度胺之前还接受了其他药物治疗。所有患者均无反应，均死于急性GVHD。对于慢性GVHD（局限性13例，广泛性46例），沙利度胺在移植后中位385天开始使用。除皮质类固醇和环孢素外，34例患者同时接受了硫唑嘌呤治疗。在所有患者中，沙利度胺被添加到正在进行的免疫抑制方案中。沙利度胺的中位治疗持续时间为60天（范围11 - 1210天；11例患者治疗时间<2周）。总共有13例患者（22%）完全缓解，8例（14%）部分缓解，38例（64%）无反应。局限性（39%）和广泛性（33%）慢性GVHD的缓解率相当。在中位53个月时，19例患者存活，13例无慢性GVHD证据。对沙利度胺有反应的患者生存率明显更好。主要死亡原因是进行性慢性GVHD（n = 29）和白血病复发（n = 7）。总之，沙利度胺对急性GVHD无活性，但与其他药物联合使用时对慢性GVHD有一定活性。
