Slavc I
Universitäts-Kinderklinik, Graz, Osterreich.
Padiatr Padol. 1992;27(6):167-75.
Bone marrow transplantation (BMT) is an established therapy in pediatric oncology and is increasingly used as curative approach in the treatment of congenital, non-oncologic diseases of the lymphohematopoietic system. There is increasing evidence, however, that BMT can be followed by severe long term effects including neuroendocrine, ophthalmologic, dental and central nervous system abnormalities, particularly in children. Therefore the indication to BMT depends on the results obtained by conventional therapy. Due to the high cure rates of leukemia with conventional therapy BMT is only warranted following relapse except for certain forms with poor prognosis factors. For patients with chronic myelogeneous leukemia, however, BMT is the only chance of cure. In solid tumors the role of BMT is more difficult, because there is no clear evidence that BMT is superior to conventional therapy with regard to long term survival. In severe aplastic anemia, however, the long term results of BMT are clearly better than those obtained by conventional therapy. Other undisputed indications for BMT are severe combined immuno deficiencies and other congenital diseases for which BMT is currently the only curative therapy. Progress with matched unrelated donor transplantations by better histocompatibility testing and more specific immunosuppressive therapy to reduce graft-versus-host disease, still a major problem of allogeneic BMT, as well as the perspectives of gen therapy in the future will offer a chance of cure to many patients without a matched sibling donor.
骨髓移植(BMT)是儿科肿瘤学中一种既定的治疗方法,并且越来越多地被用作治疗先天性淋巴造血系统非肿瘤性疾病的治愈性方法。然而,越来越多的证据表明,骨髓移植之后可能会出现严重的长期影响,包括神经内分泌、眼科、牙科和中枢神经系统异常,尤其是在儿童中。因此,骨髓移植的适应症取决于传统疗法所取得的结果。由于传统疗法对白血病的治愈率很高,除了某些预后因素较差的特定类型外,只有在复发后才需要进行骨髓移植。然而,对于慢性粒细胞白血病患者,骨髓移植是唯一的治愈机会。在实体瘤中,骨髓移植所起的作用更为复杂,因为没有明确证据表明骨髓移植在长期生存方面优于传统疗法。然而,在严重再生障碍性贫血中,骨髓移植的长期效果明显优于传统疗法。骨髓移植的其他无可争议的适应症包括严重联合免疫缺陷以及其他先天性疾病,目前骨髓移植是这些疾病唯一的治愈性疗法。通过更好的组织相容性检测和更具特异性的免疫抑制疗法来减少移植物抗宿主病(这仍然是异基因骨髓移植的一个主要问题),在匹配无关供体移植方面所取得的进展,以及未来基因治疗的前景,将为许多没有匹配同胞供体的患者提供治愈的机会。