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本文引用的文献

1
Preferences for potential benefits and risks for gene therapy in the treatment of sickle cell disease.对基因疗法治疗镰状细胞病的潜在益处和风险的偏好。
Blood Adv. 2023 Dec 12;7(23):7371-7381. doi: 10.1182/bloodadvances.2023009680.
2
Increased incidence of hematologic malignancies in SCD after HCT in adults with graft failure and mixed chimerism.造血系统恶性肿瘤在成人移植物失败和混合嵌合体后 HCT 后 SCD 中的发生率增加。
Blood. 2022 Dec 8;140(23):2514-2518. doi: 10.1182/blood.2022017960.
3
Debating the Future of Sickle Cell Disease Curative Therapy: Haploidentical Hematopoietic Stem Cell Transplantation vs. Gene Therapy.镰状细胞病治愈性疗法的未来辩论：单倍体相合造血干细胞移植与基因疗法
J Clin Med. 2022 Aug 16;11(16):4775. doi: 10.3390/jcm11164775.
4
Long-Term Health Effects of Curative Therapies on Heart, Lungs, and Kidneys for Individuals with Sickle Cell Disease Compared to Those with Hematologic Malignancies.与血液系统恶性肿瘤患者相比，镰状细胞病患者的心脏、肺和肾脏的治愈性疗法的长期健康影响。
J Clin Med. 2022 May 31;11(11):3118. doi: 10.3390/jcm11113118.
5
Discrete Choice Experiments to Elicit Patient Preferences for Decision Making in Transplantation.运用离散选择实验来获取患者在器官移植决策中的偏好。
Transplantation. 2021 May 1;105(5):960-967. doi: 10.1097/TP.0000000000003500.
6
Choice of Donor Source and Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Sickle Cell Disease.镰状细胞病造血干细胞移植的供体来源选择和预处理方案
J Clin Med. 2019 Nov 15;8(11):1997. doi: 10.3390/jcm8111997.
7
Patient Perspectives on Gene Transfer Therapy for Sickle Cell Disease.镰状细胞病患者对基因转移治疗的看法。
Adv Ther. 2017 Aug;34(8):2007-2021. doi: 10.1007/s12325-017-0587-7. Epub 2017 Jul 17.
8
Constructing experimental designs for discrete-choice experiments: report of the ISPOR Conjoint Analysis Experimental Design Good Research Practices Task Force.构建离散选择实验的实验设计：ISPOR 联合分析实验设计良好实践工作组报告。
Value Health. 2013 Jan-Feb;16(1):3-13. doi: 10.1016/j.jval.2012.08.2223.
9
Comparing abstract numerical and visual depictions of risk in survey of parental assessment of risk in sickle cell hydroxyurea treatment.镰状细胞羟基脲治疗中父母风险评估调查里抽象数字与视觉风险描述的比较
J Pediatr Hematol Oncol. 2011 Jan;33(1):4-9. doi: 10.1097/MPH.0b013e3181f46884.
10
Pulmonary, gonadal, and central nervous system status after bone marrow transplantation for sickle cell disease.骨髓移植治疗镰状细胞病后的肺部、性腺和中枢神经系统状况。
Biol Blood Marrow Transplant. 2010 Feb;16(2):263-72. doi: 10.1016/j.bbmt.2009.10.005. Epub 2009 Oct 12.

The challenge of eliciting opinions of gene therapy for SCD.

作者信息

Limerick Emily M, Fitzhugh Courtney D

机构信息

Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD.

出版信息

Blood Adv. 2023 Dec 12;7(23):7369-7370. doi: 10.1182/bloodadvances.2023011606.

DOI:10.1182/bloodadvances.2023011606
PMID:38085553
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10726235/
Abstract
摘要