Yousef S, Benden C, Boyer D, Elidemir O, Frischer T, Goldfarb S, Lopez-Mitnik G, Mallory G, Visner G, Westall G, Schecter M G
Division of Pediatric Pulmonology, Miller School of Medicine, University of Miami, Miami, FL 33136, USA.
Pediatr Transplant. 2013 May;17(3):231-6. doi: 10.1111/petr.12023. Epub 2012 Dec 6.
Allogenic BMT has been successfully performed as a treatment for hematologic diseases with an expected long-term survival. This survival is truncated by respiratory complications including airway obstruction especially BO. Chronic GVHD has been reported to precede almost all cases reported. LTx has become a therapeutic life-saving option for patients with end-stage lung disease that maybe offered for the treatment of GVHD. We report a multi-center experience of pediatric LTx following BMT in 11 patients age- and gender-matched with 11 controls who received LTx for end-stage lung disease secondary to CF. Overall death was 36.4% over a follow-up period of 19 months (range 3-36 months) for the cases and 27.3% for the control group followed for 17 months (range 8-32 months). Median FEV1 one yr post-transplant for the cases was 78% predicted compared with 67.3% predicted for the controls. The median for episodes of infection was comparable at a median of one episode per patient through the entire follow-up period among both groups. Acute rejection episodes were significantly higher in the control group with a median of one episode per patient in the control group compared to none within the cases. Our data suggest that LTx may be a valuable therapeutic option for children with end-stage lung disease post-BMT with comparable survival outcome to that after LTx in children for other indications such as CF. Hospital stay was significantly longer in our case group. Infection rate was comparable between groups albeit type of infection varied. Significantly and of interest is that acute rejection episodes were non-existent in these cases.
异基因骨髓移植已成功用于治疗血液系统疾病，并有望实现长期生存。但这种生存会因包括气道阻塞尤其是细支气管炎（BO）在内的呼吸系统并发症而缩短。据报道，几乎所有已报告的病例都先出现慢性移植物抗宿主病（GVHD）。肺移植（LTx）已成为终末期肺病患者的一种挽救生命的治疗选择，可用于治疗GVHD。我们报告了11例接受骨髓移植后进行小儿肺移植的多中心经验，这些患儿在年龄和性别上与11例因囊性纤维化（CF）继发终末期肺病而接受肺移植的对照组相匹配。在19个月（范围3 - 36个月）的随访期内，病例组的总死亡率为36.4%，对照组随访17个月（范围8 - 32个月），总死亡率为27.3%。病例组移植后1年的第一秒用力呼气容积（FEV1）中位数为预测值的78%，而对照组为预测值的67.3%。两组在整个随访期间每位患者感染发作的中位数相当，均为1次。对照组的急性排斥反应发作明显更高，对照组每位患者的中位数为1次发作，而病例组无急性排斥反应发作。我们的数据表明，对于骨髓移植后患有终末期肺病的儿童，肺移植可能是一种有价值的治疗选择，其生存结果与因其他适应症（如CF）接受肺移植的儿童相当。我们的病例组住院时间明显更长。两组之间的感染率相当，尽管感染类型有所不同。值得注意的是，这些病例中不存在急性排斥反应发作。
