美国接受酶替代疗法治疗的庞贝病患者经济负担的回顾性队列研究

数据集获取（Figshare）

• 源站标题：A retrospective cohort study of the economic burden of Pompe disease in patients treated with enzyme replacement therapy in the United States
• DOI：10.6084/m9.figshare.30050401.v1
• 发布日期：2025-09-04
• 文件数：1 个
• 文件总体积：171.25 KB
• 源站页面：https://tandf.figshare.com/articles/dataset/A_retrospective_cohort_study_of_the_economic_burden_of_Pompe_disease_in_patients_treated_with_enzyme_replacement_therapy_in_the_United_States/30050401

命令行下载

curl -L -o "ijme_a_2556629_sm6265.xlsx" "https://ndownloader.figshare.com/files/57655993"

文件清单

• ijme_a_2556629_sm6265.xlsx（171.25 KB）

文件总体积 171.25 KB（源站 API 实测）。

数据加载示例（图像类）

from PIL import Image
import glob, os

files = (glob.glob(os.path.join(path, "**", "*.png"), recursive=True)
       + glob.glob(os.path.join(path, "**", "*.jpg"), recursive=True)
       + glob.glob(os.path.join(path, "**", "*.tif"), recursive=True))
print("图像文件数:", len(files))
img = Image.open(files[0]); print("尺寸/模式:", img.size, img.mode)

# torchvision Dataset 方式:
# from torchvision import datasets
# ds = datasets.ImageFolder(path)  # 要求 子目录=类别

目录组织与标注格式以源站说明和下载后实际文件为准。

数据说明

To describe the economic burden of Pompe disease in patients treated with enzyme replacement therapy (ERT) in the US.
A retrospective cohort study (1 January 2012–30 September 2022) was conducted using the Merative™ MarketScan Research Databases. Patients with ≥2 outpatient or ≥1 inpatient claims of Pompe disease, ≥1 claim of ERT, and continuous enrollment in medical/prescription coverage for ≥90 days prior to the diagnosis date for patients ≥2 years and ≥1-month post-index were included. Patients were stratified into infantile-onset Pompe disease (IOPD) or late-onset Pompe disease (LOPD) cohorts using age at diagnosis and clinical presentation. Healthcare resource utilization and costs were evaluated over the follow-up period.
A total of 105 patients were included (IOPD: n = 50; LOPD: n = 55 [mean age at diagnosis: IOPD: 0.3 years, LOPD: 32.2 years; male: IOPD: 66%, LOPD: 60%]). Mean follow-up length was 914 and 987 days in the IOPD and LOPD cohorts, respectively. Forty-seven (94.0%) patients with IOPD and 49 (89.1%) with LOPD utilized ≥1 all-cause outpatient service, while 23 (46.0%) and 16 (29.0%), respectively, had ≥1 all-cause hospitalization. Mean total all-cause costs were $950,380 in patients with IOPD and $185,7823 in those with LOPD. Mean all-cause outpatient costs were $559,888 and $122,2078, while mean all-cause inpatient costs were $169,781 and $60,584 in the IOPD and LOPD cohorts, respectively. The all-cause outpatient ERT cost was $308,421 in patients with IOPD and $778,190 in those with LOPD.
Pompe disease incurs a substantial economic burden in patients despite treatment with ERT. For both types of Pompe disease, the primary drivers of costs were outpatient- and ERT-related; however, patients with IOPD had higher inpatient and supportive care utilization and costs, whereas patients with LOPD had higher costs for outpatient services.

许可

CC BY 4.0

引用

学术使用请引用 DOI 10.6084/m9.figshare.30050401.v1（source: https://tandf.figshare.com/articles/dataset/A_retrospective_cohort_study_of_the_economic_burden_of_Pompe_disease_in_patients_treated_with_enzyme_replacement_therapy_in_the_United_States/30050401）。