重度镰状细胞病患儿治疗策略的决策分析
Decision analysis of treatment strategies in children with severe sickle cell disease.
作者信息
O'Brien Sarah H, Hankins Jane S
机构信息
Center for Innovation in Pediatric Practice, The Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA.
出版信息
J Pediatr Hematol Oncol. 2009 Nov;31(11):873-8. doi: 10.1097/MPH.0b013e3181b83cab.
Abstract
We designed a decision analysis model comparing 4 treatment strategies for severe sickle cell disease: no intervention, hydroxyurea (HU), chronic transfusion, or stem cell transplant (SCT). The treatment strategy associated with the highest average utility (quality of life) was SCT (0.85). Average utilities for no treatment, chronic transfusion, and HU were 0.68, 0.71, and 0.80, respectively. Our model was quite sensitive to quality-of-life estimates, indicating that a true comparison of HU and transplantation cannot occur until investigators directly measure the health-related quality of life in children with sickle cell disease during HU therapy and after SCT.
摘要
我们设计了一个决策分析模型，比较了重度镰状细胞病的4种治疗策略：不干预、羟基脲（HU）、长期输血或干细胞移植（SCT）。与最高平均效用（生活质量）相关的治疗策略是SCT（0.85）。未治疗、长期输血和HU的平均效用分别为0.68、0.71和0.80。我们的模型对生活质量估计相当敏感，这表明在研究人员直接测量镰状细胞病患儿在HU治疗期间和SCT后的健康相关生活质量之前，无法对HU和移植进行真正的比较。
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