JAMA Network Open最新文献

Importance: The first peanut oral immunotherapy (OIT) for children was approved by the US Food and Drug Administration (FDA) in 2020. While clinical efficacy is established, evidence on cost-effectiveness-whether the benefits outweigh the costs and adverse effects-remains limited. A variant of OIT, known as probiotic and peanut OIT (PPOIT), has shown similar efficacy in trials.

Objective: To compare the cost-effectiveness of PPOIT, OIT, and no treatment.

Design, setting, and participants: This economic evaluation was conducted alongside a multicenter, randomized, placebo-controlled clinical trial in Australia between 2016 and 2019. Time horizon was 10 years, including 1.5 years of active treatment, 2 years of posttreatment follow-up, and 6.5 years of extrapolation. Data were analyzed from May 2024 to August 2025.

Interventions: PPOIT and OIT.

Main outcomes and measures: Effectiveness was measured using remission achieved and patient quality-adjusted life years (QALYs) gained. Costs were evaluated from a health care payer perspective, including active treatment and adverse event costs, and were calculated in Australian dollars. Incremental cost-effectiveness ratios were estimated. Sensitivity analyses were conducted to capture uncertainty.

Results: A total of 201 children aged 1 to 10 years were recruited, 79 in PPOIT, 83 in OIT, and 39 in no treatment (mean [SD] age, 5.9 [2.8] years; 129 [64.2%] male). Over a 10-year horizon, mean (SD) cost per patient was A$3956 (A$67) for PPOIT (treatment, A$3579 [A$0]; adverse events, A$377 [A$67]), A$3582 [A$57] for OIT (treatment, A$3179 [A$0]; adverse events, A$402 [A$57]), and A$249 (A$87) for no treatment (treatment, A$0 [A$0]; adverse events, A$249 [A$87]). Mean (SD) annual remission was 34.1% (12.7%) for PPOIT, 35.1% (15.4%) for OIT, and 7.3% (8.1%) for no treatment. The total QALYs gained for PPOIIT, OIT, and no treatment were 0.096, 0.055, and 0, respectively. PPOIT was slightly more costly than OIT, achieved similar remission, and achieved better quality-of-life. Compared with no treatment, both treatments were more costly with minor risks, achieved higher remission (PPOIT, A$1384; 95% CI, A$1269-A$1415; and OIT, A$1199; 95% CI, A$1091-A$1217 per year of remission achieved, respectively), and improved quality of life (PPOIT, A$38 435; 95% CI, A$31 058-A$48 668 and OIT, A$60 840; 95% CI, A$49 479-A$86 531 per QALY gained, respectively).

Conclusions and relevance: This economic evaluation found that for remission, both PPOIT and OIT were cost-effective and good value compared with no treatment, with OIT associated with a larger effect size but no clinically meaningful difference. When QALYs are prioritized, PPOIT offers the best value. Key factors associated with cost-effectiveness were treatment product pricing and patient quality of life.

Importance: Although primary hyperparathyroidism (PHPT) increases the risk of osteoporotic fractures, estimation of fracture probability among patients with PHPT remains challenging. Use of the Fracture Risk Assessment Tool (FRAX) among these patients remains poorly characterized, and its utility in identifying patients who may experience fracture risk reduction following parathyroidectomy (PTX) remains unknown.

Objective: To examine the calibration of FRAX in adults with PHPT and to describe the association between estimated probability and differential fracture risk with PTX compared with nonsurgical management.

Design, setting, and participants: This retrospective cohort study was performed from January 1, 2000, to January 1, 2024, using the TriNetX electronic health record US dataset. Participants included adults aged 40 to 90 years with diagnosis or biochemistry findings consistent with PHPT, excluding those with missing or out-of-range FRAX parameters.

Exposures: PTX vs nonsurgical management.

Main outcomes and measures: Observed major osteoporotic fracture (MOF) and hip fracture. Ten-year FRAX estimations of fracture probability were calculated without bone mineral density (BMD) findings.

Results: Among 59 194 patients with PHPT (mean [SD] age, 65.9 [10.8] years; 44 540 [75.2%] female), 14 783 (25.0%) were treated with PTX. Observed MOF and hip fractures were slightly greater than estimated by FRAX for all deciles of risk, with an MOF calibration y-intercept of 2.0% and slope of 1.17 and hip fracture calibration y-intercept of 1.4% and slope of 1.02. Overall, PTX was associated with a 12% decrease in MOF (hazard ratio [HR], 0.88; 95% CI, 0.77-1.02) and a 13% decrease in hip fracture (HR, 0.87; 95% CI, 0.72-1.07). The MOF score above which PTX was associated with consistently lower MOF hazard was 1.2%, and the corresponding hip score above which PTX was associated with consistently lower hip fracture hazard was 2.7%. Of the cohort not meeting traditional guideline-based surgical criteria, 6522 (25.0%) met this hip score for consistent fracture reduction associated with PTX.

Conclusions and relevance: In this cohort study of US patients with PHPT, FRAX estimation exhibited acceptable performance. FRAX may effectively stratify fracture risk and inform surgical decision-making even in absence of BMD. A larger subset of patients than previously identified by guidelines may benefit from parathyroidectomy from the standpoint of fracture prevention.

Importance: Venovenous extracorporeal membrane oxygenation (VV ECMO) is a resource-intensive, life-sustaining technology to support patients with severe refractory respiratory failure. Its precise indications and contraindications are not standardized, and expert opinions are frequently changing, leading to variation in why and to whom VV ECMO is offered.

Objective: To characterize the ways clinicians approach candidacy selection, the criteria considered, and the relative weight given to such variables.

Design, setting, and participants: This qualitative study conducted virtual semistructured interviews of clinicians between September and December 2024. Transcripts were qualitatively analyzed from January to June 2025 using both inductive and deductive approaches to thematic analysis and line-by-line consensus coding. Participants were physicians and ECMO coordinators from various urban, rural, public, and private medical centers in 9 countries.

Main outcomes and measures: Themes and subthemes that reflected clinicians' experiences and views.

Results: A purposeful sample of 45 clinicians directly involved in ECMO candidacy selection were contacted, of whom 24 (19 males [79%]) enrolled. Among these 24 participants interviewed, 21 (88%) were physicians and 3 (12%) were ECMO center coordinators; 8 (33%) practiced outside of the US. Five main themes were identified: (1) clinicians vary in their interpretation and incorporation of patient age, body mass index, and time on mechanical ventilation when selecting VV ECMO candidates; (2) perceived contraindications to VV ECMO are often flexible depending on various ethical and social criteria; (3) cognitive biases and heuristics affect the VV ECMO decision-making process; (4) institutional and cultural contexts shape individual VV ECMO candidacy decisions; and (5) participants provided suggestions to improve consistency in VV ECMO candidacy selection.

Conclusions and relevance: In this qualitative study, decisions to pursue VV ECMO for patients with severe respiratory failure were largely based on clinical judgments of suitability rather than objective guidelines. Variability in candidacy decision-making may lead to inconsistent or inequitable allocation.

Importance: There is limited large-scale evidence to guide the optimal mode of birth for patients with moyamoya disease (MMD).

Objective: To evaluate whether the mode of birth (cesarean vs vaginal) is associated with stroke risk after childbirth for women with MMD.

Design, setting, and participants: This cohort study evaluated stroke outcomes up to 3 years after childbirth. A nationwide, population-based analysis was performed using data from the Health Insurance Review and Assessment Service of South Korea. Individuals with MMD from January 1, 2002, to December 31, 2023 were identified. Among 31 750 patients, those with birth-related procedure codes were selected. The study population was restricted to women aged 19 to 49 years, and those with a diagnosis of malignant neoplasm within 3 years before the index date (date of childbirth) were excluded. Data were analyzed from June 11 to September 8, 2025.

Exposure: Mode of birth.

Main outcomes and measures: The primary outcome was any stroke, defined as a composite of ischemic or hemorrhagic stroke. Secondary outcomes included ischemic stroke, hemorrhagic stroke, and transient ischemic attack.

Results: Of 1683 women analyzed (mean [SD] age, 33.6 [7.8] years), 1077 (64.0%) had cesarean births, and 606 (36.0%) had vaginal births. Post partum (3 months), any stroke incidence was 63.49 and 33.33 per 1000 person-years for cesarean and vaginal births, respectively. Multivariable analyses showed no significant risk differences for any stroke by birth mode at 3 months (adjusted hazard ratio [aHR], 0.71 [95% CI, 0.26-1.97]; P = .52) or 3 years (aHR, 0.90 [95% CI, 0.55-1.47]; P = .67). A significant interaction was observed between the mode of birth and the clinical onset type of MMD for the risk of any stroke (interaction P = .04 after Bonferroni correction); the adjusted HR for vaginal vs cesarean birth was 0.10 (95% CI, 0.01-0.79) in the asymptomatic or nonvascular onset subgroup, 1.49 (95% CI, 0.73-3.03) in the ischemic onset subgroup, and 0.94 (95% CI, 0.50-1.77) in the hemorrhagic onset subgroup. Notably, stroke incidence peaked in the early postpartum period (≤6 months: 35.7 per 1000 person-years), decreased at 1 year, and thereafter remained at a similar level.

Conclusions and relevance: In this cohort study of women with MMD, MMD itself was not found to be an absolute indication for cesarean birth; birth planning should be individualized based on obstetric factors and clinical onset type rather than routine preference for cesarean birth. In addition, vigilant monitoring and preventive strategies during the early postpartum period are warranted.