The Health Institute, Institute for Clinical Research and Health Policy Studies, Tufts Medical Center, Boston, Massachusetts 02111, USA.
Pediatr Blood Cancer. 2012 Oct;59(4):725-31. doi: 10.1002/pbc.24036. Epub 2011 Dec 19.
Little is known regarding the health-related quality of life (HRQL) trajectory of children with sickle cell disease or thalassemia ("hemoglobinopathies") following hematopoietic stem cell transplantation (HSCT).
We serially evaluated the HRQL of 13 children with hemoglobinopathies who received HSCT during two prospective multi-center studies using the Child Health Ratings Inventories (CHRIs). The HRQL scores among children with hemoglobinopathies, as reported separately by the children and their parents were compared using repeated measures models to scores of a comparison group of children receiving HSCT for malignancies or severe aplastic anemia.
The sample included 13 children with hemoglobinopathies (median age: 8 years, range 5-18) and 268 children in the comparison group (median age: 11 years, range 5-18). There were similar rates of early infection, chronic graft versus host disease, and all-cause mortality between the two groups. There was no significant difference in recovery to baseline scores for physical, emotional, and role functioning by 3 months for either group. Children with hemoglobinopathies had higher HRQL scores for physical (beta = 12, se = 5.5, P = 0.01) and baseline emotional functioning (beta = 11.6, se = 5.5, P = 0.03) than the comparison group. For all domains for both groups, parent reports demonstrated a nadir at 45 days with recovery to baseline by 3 months following transplant. Children's ratings were higher than those of their parents in both diagnostic groups.
Children with hemoglobinopathies had higher physical and emotional functioning scores prior to HSCT and experienced a similar pattern of recovery to their baseline functioning by 3 months post-HSCT when compared to children receiving HSCT for acquired conditions.
对于接受造血干细胞移植（HSCT）后的镰状细胞病或地中海贫血症（“血红蛋白病”）患儿的健康相关生活质量（HRQL）轨迹知之甚少。
我们在两项前瞻性多中心研究中，使用儿童健康评分量表（CHRIs）连续评估了 13 名接受 HSCT 的血红蛋白病患儿的 HRQL。使用重复测量模型，将血红蛋白病患儿的 HRQL 评分与接受 HSCT 治疗恶性肿瘤或严重再生障碍性贫血的患儿进行比较，单独由患儿及其父母报告的评分。
样本包括 13 名血红蛋白病患儿（中位数年龄：8 岁，范围 5-18 岁）和 268 名比较组患儿（中位数年龄：11 岁，范围 5-18 岁）。两组之间早期感染、慢性移植物抗宿主病和全因死亡率的发生率相似。两组在 3 个月时身体、情绪和角色功能均未恢复到基线评分。血红蛋白病患儿的身体功能（β=12，se=5.5，P=0.01）和基线情绪功能（β=11.6，se=5.5，P=0.03）评分均高于对照组。对于两组的所有领域，父母报告的评分在 45 天达到最低点，在移植后 3 个月恢复到基线。在两个诊断组中，患儿的评分均高于其父母。
与接受 HSCT 治疗后天获得性疾病的患儿相比，接受 HSCT 前血红蛋白病患儿的身体和情绪功能评分较高，且在移植后 3 个月时，他们的功能恢复到基线水平的模式也相似。
