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一种新型评分指数的开发,用于量化儿童干细胞移植和基因治疗期间营养支持干预的需求

Development of A Novel Scoring Index to Quantify the Need for Nutritional Support Interventions During Stem Cell Transplant and Gene Therapy in Children.

作者信息

Gurumurthy Gerard, Dixon Rebecca, Whiteley Victoria, Horgan Claire, Mustafa Omima, Williams Nicola, Wynn Robert

机构信息

The University of Manchester, Manchester, UK.

Royal Manchester Children's Hospital, Manchester, UK.

出版信息

J Hum Nutr Diet. 2025 Oct;38(5):e70124. doi: 10.1111/jhn.70124.

Abstract

BACKGROUND

Paediatric patients undergoing stem cell and gene therapy frequently experience nutritional challenges. Although nutritional status is known to correlate with outcomes, there is no scoring index to quantify the intensity and duration of dietetic interventions in children. We developed a nutritional intensity score that assigns weighted values to different nutritional support strategies that reflects both intervention type and duration over the first 100 days post-transplant.

METHODS

We retrospectively analysed 131 paediatric patients after transplant or gene therapy. Nutritional interventions were categorised into five levels: routine dietetic review (1 point), oral nutritional support (2 points), enteral nutritional support (4 points), parenteral nutritional (PN) support (6 points), and bespoke PN support (7 points). The cumulative nutritional intensity score was calculated by multiplying the intervention points by the duration (in days) and summing across all interventions. Subgroup analyses compared median area under the curve (AUC) values in different groups. A further multivariate logistic regression was employed to assess predictors of ≥ PN need at Day 100 within the allogeneic group.

RESULTS

Nutritional intensity varied with different treatment modalities. Chimeric Antigen Receptor T-cell (CAR-T) patients showed notably lower median nutritional intensity score (53.0, 95% CI: 30-117) relative to allogeneic (326.0, 95% CI: 282-404) or stem cell gene therapy (124.5, 95% CI: 56-182, p < 0.05) recipients. Within the allogeneic group (n = 109), patients with malignant diseases had a median nutritional intensity score of 444.0 (95% CI: 350.0-466.0) compared to 261.5 (95% CI: 224.0, 322.0, p < 0.01) in those with nonmalignant conditions. Those receiving total body irradiation (TBI) had higher median nutritional intensity score values (476.0, 95% CI: 317-543) than those without TBI (302.0, 95% CI: 244-386; p < 0.01). In the multivariate analysis, higher nutritional intensity score, malignant disease status, and TBI were significant predictors of ≥ PN requirement at Day 100.

CONCLUSION

Our nutritional intensity score reflects the cumulative burden of nutritional interventions in paediatric patients, and will inform patient and service planning. Future prospective studies are required to validate its predictive value.

摘要

背景

接受干细胞和基因治疗的儿科患者经常面临营养挑战。虽然已知营养状况与治疗结果相关,但尚无评分指数来量化儿童饮食干预的强度和持续时间。我们制定了一个营养强度评分,为不同的营养支持策略赋予加权值,该评分反映了移植后前100天的干预类型和持续时间。

方法

我们回顾性分析了131例移植或基因治疗后的儿科患者。营养干预分为五个级别:常规饮食评估(1分)、口服营养支持(2分)、肠内营养支持(4分)、肠外营养(PN)支持(6分)和定制PN支持(7分)。累积营养强度评分通过将干预分数乘以持续时间(天数)并对所有干预进行求和来计算。亚组分析比较了不同组中曲线下面积(AUC)值的中位数。进一步采用多因素逻辑回归评估异基因组中第100天时≥PN需求的预测因素。

结果

营养强度因不同治疗方式而异。嵌合抗原受体T细胞(CAR-T)患者的中位营养强度评分(53.0,95%置信区间:30-117)显著低于异基因移植患者(326.0,95%置信区间:282-404)或干细胞基因治疗患者(124.5,95%置信区间:56-182,p<0.05)。在异基因组(n=109)中,患有恶性疾病的患者中位营养强度评分为444.0(95%置信区间:350.0-466.0),而非恶性疾病患者为261.5(95%置信区间:224.0,322.0,p<0.01)。接受全身照射(TBI)的患者中位营养强度评分值(476.0,95%置信区间:317-543)高于未接受TBI的患者(302.0,95%置信区间:244-386;p<0.01)。在多因素分析中,较高的营养强度评分、恶性疾病状态和TBI是第100天时≥PN需求的显著预测因素。

结论

我们的营养强度评分反映了儿科患者营养干预的累积负担,并将为患者和服务规划提供参考。未来需要进行前瞻性研究以验证其预测价值。

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