Hobbs J R, Jones K H, Shaw P J, Lindsay I, Hancock M
Lancet. 1987 May 16;1(8542):1111-5. doi: 10.1016/s0140-6736(87)91673-4.
2 patients with fast and 4 with medium varieties of Gaucher's syndrome were treated by displacement bone marrow transplantation to install a donor enzyme factory for life. Immunoprophylaxis was given to prevent host impairment of normal enzymes. 5 patients showed rapid and remarkable improvement and were leading fully active lives at 310-1207 days postgraft. Hypersplenism caused the death of 1 patient and a stormy time for another. Pre-transplant elective splenectomy hastened recovery.
2例患有快速型和4例患有中型戈谢病的患者接受了置换骨髓移植,以建立一个终身的供体酶工厂。给予免疫预防以防止宿主对正常酶的损害。5例患者显示出快速且显著的改善,在移植后310 - 1207天过着完全活跃的生活。脾功能亢进导致1例患者死亡,另1例患者经历了一段艰难时期。移植前的择期脾切除术加速了恢复。