Tag: pubmed

Stat Med. 2026 Mar;45(6-7):e70455. doi: 10.1002/sim.70455.

ABSTRACT

This commentary offers perspectives on delivering “rigorous causal inference on meaningful estimands” that differ from the opinions recently shared by Fleming et al. We (1) depict a more robust pathway for achieving this aim that incorporates clinical, causal and statistical reasoning, (2) suggest a tangibility criterion to judge the practical usefulness of an intercurrent event strategy, (3) illustrate the utility of causal inference methods in providing robust estimates when the clinical objective aligns with a hypothetical strategy, and (4) advocate for careful consideration of the tradeoffs between an estimand’s relevance and the required assumptions.

PMID:41847726 | DOI:10.1002/sim.70455

Diagn Interv Radiol. 2026 Mar 18. doi: 10.4274/dir.2026.263802. Online ahead of print.

ABSTRACT

PURPOSE: To evaluate the safety and efficacy of combined ethanol lavage and bleomycin sclerotherapy administered via a percutaneous pigtail catheter for the treatment of benign cervical cystic lesions.

METHODS: This retrospective study included 29 patients (mean age, 30 years; range, 4-60 years; male-to-female ratio, 16:13) who underwent bleomycin sclerotherapy following ethanol lavage via a pigtail catheter for benign cervical cystic lesions, including branchial cleft cysts, ranulas, thyroglossal duct cysts, lymphatic malformations, and epidermoid cysts, between March 2009 and September 2022. To explore potential predictors of treatment response, clinical diagnosis, baseline cyst size, and the total volume of injected sclerosant were evaluated. Statistical analyses included the paired t-test, chi-square test, and Mann-Whitney U test.

RESULTS: All patients were followed up for a mean duration of 18.2 months (range, 3-72 months) after the final treatment session. Complete cyst resolution was achieved in 17 of the 29 patients (59%), 8 patients (27%) demonstrated a volume reduction greater than 75%, and 2 patients (7%) exhibited a reduction of less than 75%; recurrence occurred in 2 patients (7%) despite repeated sclerotherapy. There were no significant differences between responders and nonresponders with respect to clinical diagnosis, baseline cyst volume, or total sclerosant dose. Minor procedure-related complications occurred in three patients (10.34%); no major complications were observed.

CONCLUSION: Combined ethanol lavage and bleomycin sclerotherapy administered via a percutaneous pigtail catheter is a safe and feasible treatment option for benign cervical cystic lesions.

CLINICAL SIGNIFICANCE: Combined ethanol and bleomycin sclerotherapy represents a safe, minimally invasive treatment option for benign cervical cystic lesions in routine clinical practice, with favorable outcomes and potential to reduce the need for surgical intervention.

PMID:41847722 | DOI:10.4274/dir.2026.263802

Stat Med. 2026 Mar;45(6-7):e70452. doi: 10.1002/sim.70452.

ABSTRACT

While the use of estimands in randomized trials is increasing, there is little guidance on which intercurrent event strategies should be used. The article by Fleming et al. seeks to address this gap. They argue that strategies such as hypothetical, principal stratum, and while-alive generally cannot be used to reliably inform decision making, and that treatment policy (and composite for mortality) strategies should be used instead. In this Commentary we argue that there are a variety of settings where strategies such as hypothetical, principal stratum, and while-alive can reliably inform decision-making and are preferable to a treatment policy strategy. We provide an alternative approach for selecting intercurrent event strategies, which systematically considers the trade-off between relevance (whether it addresses a useful question) and reliability (the ability to be estimated such that stakeholders can have confidence in the results) of each strategy in order to identify those that can be used to robustly inform decision-making. Our overall conclusion is that there is no single intercurrent event strategy that is appropriate in all settings; all strategies can be beneficial when used in appropriate settings, but harmful when used in inappropriate settings.

PMID:41847719 | DOI:10.1002/sim.70452

Stat Med. 2026 Mar;45(6-7):e70453. doi: 10.1002/sim.70453.

NO ABSTRACT

PMID:41847717 | DOI:10.1002/sim.70453

J Gerontol A Biol Sci Med Sci. 2026 Mar 17:glag076. doi: 10.1093/gerona/glag076. Online ahead of print.

ABSTRACT

BACKGROUND: Predicting Alzheimer’s disease (AD)-related cognitive impairment (CI) among cognitively normal (CN) adults enables meaningful disease modification through early intervention and enrichment of clinical trials.

METHODS: A deep survival model is trained to predict CI conversion risk in 1,415 CN adults from the National Alzheimer’s Coordinating Center. Converters’ (N = 212) and non-converters’ (N = 1,203) baseline clinical measures and magnetic resonance images are used to estimate their conversion probability up to 22 years after baseline observation.

RESULTS: After 20-fold cross-validation, the model predicts conversion probability with a c-index of 0.88, and classification accuracy of 75% and AUC ROC of 0.89, outperforming previous machine learning models.

CONCLUSIONS: This is one of few studies on the important challenge of predicting future CI among unimpaired subjects. Deep survival modelling can improve the identification of preclinical AD and suggests that uncertainty in AD risk estimation is due to potentially modifiable lifestyle factors.

PMID:41844537 | DOI:10.1093/gerona/glag076

Hematology. 2026 Dec;31(1):2642515. doi: 10.1080/16078454.2026.2642515. Epub 2026 Mar 17.

ABSTRACT

BACKGROUND: Relapsed immune thrombocytopenia (ITP) poses challenges in treatment. Thrombopoietin receptor agonists (TPO-RAs) are recommended as second-line therapy, but the efficacy of monotherapy is limited to about 60%. Our preliminary studies suggest sirolimus is effective for relapsed ITP. This study aimed to evaluate the efficacy and safety of TPO-RA combined with sirolimus in treating relapsed ITP.

METHODS: A retrospective analysis was conducted on 50 patients with relapsed ITP, all of whom received TPO-RAs (hetrombopag and eltrombopag) combined with sirolimus therapy. The primary endpoints were the overall response rate (ORR) at 4, 8, and 12 weeks of treatment. Secondary endpoints included safety of this regimen and subgroup analysis.

RESULTS: ORR at 4, 8, and 12 weeks was 61%, 76%, and 82%, respectively, with a median time to response of 7 days. Mean platelet counts at 2-, 4-, 8-, and 12-weeks post-treatment were 71 ± 12.7 × 10⁹/L, 86 ± 10.9 × 10⁹/L, 128 ± 19.7 × 10⁹/L and 131 ± 17.2 × 10⁹/L, respectively. Whether it is hetrombopag or eltrombopag, when combined with sirolimus, there is no statistically significant difference in efficacy between the two subgroups. TPORAs combined with sirolimus as second-line treatment and as third-line or beyond treatment showed no difference in efficacy. Among ANA-positive patients, the ORR shows no significant difference compared to the ANA-negative group (p > 0.05). Patients with ITP for less than one year are more likely to benefit from this treatment regimen (p = 0.003).

CONCLUSIONS: The combination of TPO-RAs and sirolimus significantly improved platelet counts and sustained response rates. The regimen demonstrated a manageable safety profile, offering a novel therapeutic option for relapsed ITP.

PMID:41844533 | DOI:10.1080/16078454.2026.2642515

J Clin Ultrasound. 2026 Mar 17. doi: 10.1002/jcu.70229. Online ahead of print.

ABSTRACT

BACKGROUND AND OBJECTIVES: Ultrasonography is routinely used after pediatric kidney transplantation, but the prevalence and longitudinal pattern, and prognostic value of abnormal findings are not well defined. We described the frequency and temporal trends of post-transplant ultrasound abnormalities in children and evaluated their association with graft outcomes.

METHODS: We retrospectively included patients (< 18 years) who underwent kidney transplantation at Children’s Medical Center Hospital between 2018 and 2024. Sonographic features (parenchymal changes, peritransplant collections, urinary tract abnormalities, and Doppler indices) were recorded within six predefined follow-up windows. We used descriptive statistics and Kaplan-Meier survival curves stratified by the presence of any abnormal ultrasound finding at each window. Analyses were performed in R (version 4.3).

RESULTS: Sixty-three patients (mean age 12.0 ± 3.9 years) were included. Abnormal findings were present in 43%-56% of scans across follow-up windows. Peritransplant collections (33.9%) and hematomas (24.6%) were common early findings, whereas hydronephrosis and increased echogenicity became more frequent over time. Resistive index elevation (> 0.8) was uncommon (< 3.5%). Three-year graft survival was 55.7%. Any abnormality on baseline ultrasound was associated with lower 36 month survival (40.9% vs. 73.1%, log-rank p = 0.025), while abnormalities detected at later windows were not associated with survival differences.

CONCLUSION: Abnormal ultrasound findings were common after pediatric kidney transplantation, particularly in the early postoperative period. Baseline abnormalities were associated with poorer long-term graft survival, which may reflect perioperative complications or early allograft injury. This supports the clinical value of baseline ultrasound for early risk stratification and guiding the follow-up intensity.

PMID:41844519 | DOI:10.1002/jcu.70229

Clin Oncol (R Coll Radiol). 2026 Jan 27;53:104059. doi: 10.1016/j.clon.2026.104059. Online ahead of print.

ABSTRACT

AIMS: Healthcare systems data (HSD) can potentially improve the efficiency and costs of clinical trials (CTs) and reduce the burden of follow-up. To be used in CTs, HSD need to be accessible, accurate and complete. We compared data from trial-specific case-report forms (CRFs) with relevant HSD to assess whether HSD could replace hospital-based follow-up within an ongoing CT.

MATERIALS AND METHODS: Add-Aspirin (NCT02804815) is a multicentre, randomised, basket trial evaluating aspirin after potentially curative cancer therapy in four tumour types. The primary outcome measure is disease-free survival. HSD obtained from the English National Cancer Registration and Analysis Service (NCRAS) included initial cancer diagnosis and staging, recurrence, specific adverse events, and mortality for English participants during the first 3 years of the trial and was compared to CRF data collected at participating centres. Tabulations and descriptive statistics were used to assess agreement and explore differences between the datasets.

RESULTS: HSD was obtained for 3188/3538 (90%) of English participants. Tumour staging for all four tumour groups generally had good concordance, though this was variable (ranging from 85% (Cohen’s weighted kappa = 0.32) to 99% (kappa = 0.84)) and was lower with neoadjuvant therapy and/or radical radiotherapy. HSD identified all CRF-reported deaths and some further deaths not in the trial database due to loss to follow-up or lag time in data submission. Cancer registry recurrence data captured <50% of recurrences. However, most recurrences could be identified by utilising a combination of NCRAS datasets, including treatment and hospital events. Specific trial-defined adverse events were challenging to identify within HSD.

CONCLUSION: In this example, we found that HSD could not replace CRFs for later trial follow-up as anticipated, because at that time it could not capture all key outcomes accurately . Focussed, hospital-based follow-up was continued for a further 5 years. Selective use of HSD to improve trial efficiency retains potential.

TRIAL REGISTRATION NUMBER: NCT02804815.

PMID:41844499 | DOI:10.1016/j.clon.2026.104059

Gynecol Oncol. 2026 Mar 16;207:66-73. doi: 10.1016/j.ygyno.2026.02.035. Online ahead of print.

ABSTRACT

OBJECTIVE: To compare the long-term survival outcomes of robotic-assisted versus traditional laparoscopic surgery for treatment of stage I endometrial cancer.

METHODS: We performed a retrospective cohort study using the National Cancer Database, including patients with stage I endometrial cancer diagnosed from 2010 to 2017 (with follow-up through 2020) who underwent treatment with either robotic-assisted or traditional laparoscopic surgery. Demographics, clinical characteristics, and outcomes were summarized using descriptive statistics. Overall survival (OS) was compared using a Cox proportional hazard model, adjusting for demographic and clinical factors; hazard ratios and 95% confidence intervals reported.

RESULTS: A total of 127,342 patients with stage I endometrial cancer who underwent minimally invasive surgery were included. The median follow-up among those still known to be alive was 69.7 months. Almost three-quarters (74.5%) received robotic-assisted surgery, while 25.5% received traditional laparoscopic surgery. Demographic and clinical factors were similar between groups. No differences in OS were observed by surgery modality (5-year OS: 91.7% for robotic vs. 91.4% for traditional laparoscopic surgery) and this remained after adjustment for demographic and clinical factors (HR = 1.00, 95% CI 0.96-1.04).

CONCLUSIONS: There was no difference in OS for patients with stage I endometrial cancer treated with robotic-assisted versus traditional laparoscopic surgery. These data demonstrate excellent survival outcomes in this cohort and do not favor a robotic-assisted or traditional laparoscopic approach. However, the full extent of a route-of-surgery decision may not be detected given the potential for late recurrences and deaths, highlighting the need for large, prospective studies with long duration of follow-up.

PMID:41844495 | DOI:10.1016/j.ygyno.2026.02.035

JACC Adv. 2026 Mar 16;5(4):102653. doi: 10.1016/j.jacadv.2026.102653. Online ahead of print.

ABSTRACT

BACKGROUND: Peripartum cardiomyopathy (PPCM) is a leading cause of heart failure in pregnancy and contributes significantly to maternal morbidity and mortality. Black women are disproportionately affected and experience worse outcomes compared with other groups.

OBJECTIVES: This study aimed to quantify differences in risk factors and outcomes between Black and White women in the United States diagnosed with PPCM.

METHODS: We conducted a systematic review and meta-analysis of observational studies published after 2002 including U.S. women with PPCM and race-stratified risk factors and outcomes. Investigated outcomes included mortality, major adverse cardiac events, and recovery of left ventricular ejection fraction. Random-effects meta-analysis estimated the pooled prevalence of risk factors and outcomes. Logistic regression, forest plots, and I² statistics were utilized for analysis.

RESULTS: Compared with controls, cases had higher rates of obesity, preeclampsia, hypertension, diabetes, multiple gestations, and tobacco use. Compared to White cases, Black cases had higher prevalence of diabetes (14% vs 5%; P = 0.027) and utilization of public payer (72% vs 30%; P < 0.001). At presentation, mean left ventricular ejection fraction was 26% in Black women and 29% in White women. White women experienced higher rates of recovery in ejection fraction (63% vs 40%; P < 0.0001). Mortality rates were higher among Black women (8% vs 2%; P = 0.013).

CONCLUSIONS: Black women with PPCM experienced lower recovery and higher mortality rates compared with White women. With the exception of a significant difference in payer status, modest differences in previously identified risk factors were observed between racial groups to account for worse outcomes (Disparities in risk factors and outcomes between Black and White US women with peripartum cardiomyopathy: A systematic review and meta-analysis; CRD42023439228).

PMID:41844488 | DOI:10.1016/j.jacadv.2026.102653