Gonzalez-Ryan L, Haut P R, Coyne K, Syckle K V, Duerst R, Haro D, Kletzel M
Children's Memorial Hospital, Stem Cell Transplant Program at Children's Memorial Hospital, 2300 Children's Plaza, Chicago IL 60614, USA.
Front Biosci. 2001 Aug 1;6:G1-5. doi: 10.2741/ryan.
We describe the development of a pediatric outpatient transplant program and our initial experience with autologous and allogeneic transplants performed partially or completely in the outpatient setting. Forty-eight autologous and seven allogeneic transplants have been performed in our institution in the outpatient setting between June 1994 and July 2000. The ablation used for the autologous outpatient transplants was VP-16 1000 mg/m2/ day as a continuous infusion for 2 days and Carboplatinum 667mg/m2/day for 2 days. The autologous inpatient transplants received Thio-tepa 300-mg/ m2per day x 3 days and cyclophosphamide 60 mg/kg/day for 4 days. For those patients who received an immune-ablative allogeneic outpatient transplant, the regimen consisted of Fludarabine 30 mg/m2/day for 6 days, followed by busulfan for children less than five years of age 1 mg/kg/dose x 8 doses and for those five years and older 0.8 mg/kg/dose x 8 doses, followed by ATG 40mg/kg/day x 4 days. Engraftment was complete in all transplants achieving an ANC >500 for the outpatient transplant in 15 days (10-35) vs. the inpatient in 15 days (14-58). This was not statistically significant. They achieved un-sustained platelets >20.0 by day 19 (14-58) for the outpatients and day 32 10-64) for the inpatient. The allogeneic immune ablative transplants were considered engrafted when their VNTRs were greater that 30% which was achieved at a median of 13 days (10-27). The economic data showed a statistically significant decrease in charges and direct costs between the outpatient (median charges $30 775, direct costs $8 389) and the inpatient (median charges $99 838, direct costs $42 757) transplants (p0.001). There was no difference in morbidity and mortality between the two groups but the use of empiric amphotericin B was markedly decreased in the outpatient transplants. In conclusion it is feasible and less costly to perform autologous hematopoietic stem cell transplants in the outpatient setting with no increase in morbidity and mortality. For the allogeneic transplants there is not yet enough data to establish a similar conclusion.
我们描述了一个儿科门诊移植项目的发展情况以及我们在门诊环境中部分或完全进行自体和异体移植的初步经验。1994年6月至2000年7月期间，我们机构在门诊环境中进行了48例自体移植和7例异体移植。用于自体门诊移植的预处理方案为：VP - 16 1000mg/m²/天，持续输注2天；卡铂667mg/m²/天，共2天。自体住院移植接受硫鸟嘌呤300mg/m²/天×3天以及环磷酰胺60mg/kg/天×4天。对于接受免疫清除性异体门诊移植的患者，方案包括氟达拉滨30mg/m²/天，共6天，随后对于5岁以下儿童给予白消安1mg/kg/剂量×8剂，5岁及以上儿童给予0.8mg/kg/剂量×8剂，接着给予抗胸腺细胞球蛋白40mg/kg/天×4天。所有移植均实现植入，门诊移植在15天（10 - 35天）时中性粒细胞绝对值（ANC）>500，住院移植在15天（14 - 58天）时达到相同水平。这在统计学上无显著差异。门诊患者在第19天（14 - 58天）血小板计数>20.0×10⁹/L，住院患者在第32天（10 - 64天）达到该水平。异体免疫清除性移植当其可变数目串联重复序列（VNTRs）大于30%时被视为植入，这在中位时间13天（10 - 27天）时实现。经济数据显示，门诊移植（中位费用30775美元，直接成本8389美元）和住院移植（中位费用99838美元，直接成本42757美元）之间的费用和直接成本在统计学上有显著降低（p<0.001）。两组在发病率和死亡率方面无差异，但门诊移植中经验性使用两性霉素B明显减少。总之，在门诊环境中进行自体造血干细胞移植是可行的且成本更低，发病率和死亡率没有增加。对于异体移植，尚无足够数据得出类似结论。
