College of Life Science and Technology, Huazhong University of Science and Technology, Wuhan, Hubei, PR China.
Medical Genetics Center, Maternal and Child Health Hospital of Hubei Province, Wuhan, Hubei, PR China.
JAMA Netw Open. 2024 Jun 3;7(6):e2412886. doi: 10.1001/jamanetworkopen.2024.12886.
Recent changes in China's social medical insurance reimbursement policy have impacted the financial burden of patients with phenylketonuria (PKU) for special foods. However, whether this policy change is associated with their blood phenylalanine (PHE) concentration is unclear.
To investigate the association between the reimbursement policy and blood PHE concentration in patients with PKU.
DESIGN, SETTING, AND PARTICIPANTS: This cohort study measured the blood PHE concentrations of 167 patients with PKU across 4 newborn screening centers in China from January 2018 to December 2021. The reimbursement policy for special foods for patients with PKU at 2 centers was canceled in 2019 and restored from 2020 onwards. In contrast, the other 2 centers consistently implemented the policy. Data were analyzed from September 10 to December 6, 2023.
The implementation and cancelation of the reimbursement policy for special foods of patients with PKU.
The blood PHE concentration was regularly measured from 2018 to 2021. A 1-sided Z test was used to compare the mean of the blood PHE concentration between different years.
Among 167 patients with PKU (mean [SD] age, 84.4 [48.3] months; 87 males [52.1%]), a total of 4285 measurements of their blood PHE concentration were collected from 2018 to 2021. For patients at the center that canceled the reimbursement policy in 2019, the mean (SD) of the blood PHE concentrations in 2019 was 5.95 (5.73) mg/dL, significantly higher than 4.84 (4.11) mg/dL in 2018 (P < .001), 5.06 (5.21) mg/dL in 2020 (P = .006), and 4.77 (4.04) mg/dL in 2021 (P < .001). Similarly, for patients at the other center that canceled the policy in 2019, the mean (SD) of the blood PHE concentrations in 2019 was 5.95 (3.43) mg/dL, significantly higher than 5.34 (3.45) mg/dL in 2018 (P = .03), 5.13 (3.15) mg/dL in 2020 (P = .003), and 5.39 (3.46) mg/dL in 2021 (P = .03). On the contrary, no significant difference was observed between any of the years for patients at the 2 centers that consistently implemented the policy.
In this cohort study of patients with PKU from multiple centers, the implementation of the reimbursement policy for special foods was associated with controlling the blood PHE concentration. Special foods expenditure for patients with PKU should be included in the scope of long-term social medical insurance reimbursement.
中国社会医疗保险报销政策的最近变化影响了苯丙酮尿症（PKU）患者特殊食品的经济负担。然而，尚不清楚该政策变化是否与患者的血苯丙氨酸（PHE）浓度有关。
本队列研究测量了 2018 年 1 月至 2021 年 12 月期间来自中国 4 个新生儿筛查中心的 167 例 PKU 患者的血 PHE 浓度。2019 年，2 个中心取消了 PKU 患者特殊食品的报销政策，并从 2020 年起恢复。相比之下，其他 2 个中心一直执行该政策。数据分析于 2023 年 9 月 10 日至 12 月 6 日进行。
该队列研究共纳入了 167 例 PKU 患者（平均[标准差]年龄，84.4[48.3]个月；男性 87 例[52.1%]），2018 年至 2021 年定期测量了他们的血 PHE 浓度。使用单边 Z 检验比较了不同年份血 PHE 浓度的平均值。
在取消 2019 年报销政策的中心的患者中，2019 年血 PHE 浓度的平均值（标准差）为 5.95（5.73）mg/dL，明显高于 2018 年的 4.84（4.11）mg/dL（P <.001）、2020 年的 5.06（5.21）mg/dL（P =.006）和 2021 年的 4.77（4.04）mg/dL（P <.001）。同样，在 2019 年取消政策的另一个中心的患者中，2019 年血 PHE 浓度的平均值（标准差）为 5.95（3.43）mg/dL，明显高于 2018 年的 5.34（3.45）mg/dL（P =.03）、2020 年的 5.13（3.15）mg/dL（P =.003）和 2021 年的 5.39（3.46）mg/dL（P =.03）。相比之下，在一直执行该政策的 2 个中心的患者中，任何一年之间均未观察到显著差异。
在这项来自多个中心的 PKU 患者的队列研究中，特殊食品报销政策的实施与控制血 PHE 浓度有关。PKU 患者的特殊食品支出应纳入长期社会医疗保险报销范围。
