This study involves the one-time collection of whole blood or saliva samples for the extraction and storage of DNA for use in ongoing and future ChiLDReN studies.

开始日期2022-04-06

申办/合作机构

Arbor Research Collaborative For Health

NCT04181138

/ RecruitingN/AIIT

Prospective Observational Study of Primary Sclerosing Cholangitis (PSC) in Children

Primary sclerosing cholangitis (PSC) is a rare liver disease that damages the liver's bile ducts. Bile ducts are tiny tubes that carry bile from the liver to the small intestine. Bile is a liquid produced by the liver that helps us absorb and use the nutrients in the food we eat. In people with PSC, the bile backs up into the liver and will damage it, causing scarring of the liver.
The purposes of this study are to:
* Collect medical and other data to learn more about PSC, how it progresses, and identify factors that may cause the disease to progress more quickly.
* Ask questions about how PSC symptoms affect your child's life to learn more about its impact on your child's daily functioning
* Children with PSC who are seen at one of the participating clinical sites in the Childhood Liver Disease Research Network (ChiLDReN) will be asked to contribute information, DNA, and other specimens. The information and specimens will be available to investigators to carry out approved research aimed at learning more about the possible causes and long-term effects of PSC.

开始日期2021-12-30

申办/合作机构

Arbor Research Collaborative For Health

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493

项与 Arbor Research Collaborative For Health 相关的文献（医药）

2025-12-01·Clinical and Experimental Nephrology

Association between blood pressure and cardiovascular events and mortality in patients on peritoneal dialysis: a systematic review and meta-analysis of observational studies

Review

作者: Pecoits-Filho, Roberto ; Bezerra, Rodrigo ; Ponce, Daniela ; Coelho, Jorge A P M ; Feitosa, Audes D M ; Rodrigues, Cibele I S ; Teles, Flavio ; Nadruz, Wilson

BACKGROUND:

The association between blood pressure (BP) and adverse outcomes in peritoneal dialysis (PD) remains uncertain. This study aims to address this knowledge gap.

MATERIALS AND METHODS:

We systematically searched five databases (1964-2025) for observational studies assessing associations between BP and mortality or cardiovascular (CV) outcomes in adults on PD. Risk of bias was evaluated using the Newcastle-Ottawa Scale and ROBINS-I. Meta-analyses were performed using random- or fixed-effects models, and pooled hazard ratios (HRs) with 95% confidence intervals (CIs) were calculated for continuous and categorical BP exposures.

RESULTS:

Twenty-four studies comprising 28,016 patients (55% males; hypertension prevalence ranging from 39 to 95%) were included. Higher pulse pressure (PP) was consistently associated with increased all-cause (HR per 10 mmHg: 1.20; 95%CI 1.02-1.40) and CV (HR per 10 mmHg: 1.35; 95%CI 1.16-1.58) mortality. For systolic BP (SBP), no significant association was found when analyzed as a continuous variable. However, predefined thresholds revealed that all-cause mortality was significantly associated with SBP < 120 mmHg (HR: 1.55; 95%CI 1.15-2.11) and with SBP > 140 mmHg (HR: 1.18; 95%CI 1.07-1.31). Diastolic BP was not significantly associated with mortality. Additional studies linked higher BP to left ventricular hypertrophy and non-fatal CV events.

CONCLUSION:

In PD patients, SBP < 120 mmHg and > 140 mmHg are associated with increased all-cause mortality, while elevated PP robustly predicts all-cause and CV mortality. These findings identify SBP and PP as key prognostic markers and potential targets in PD management.

2025-12-01·BONE

Advancing Patient Evidence in XLH (APEX): Baseline analysis of a global data unification program

Article

作者: Li, Zhiyi ; McCullough, Keith P ; Imel, Erik A ; Lee, Cheuk ; Ishii, Haruka ; Brandi, Maria Luisa ; Ozono, Keiichi ; Carpenter, Thomas O ; Joos-Vandewalle, Paul ; Sandilands, Kerry ; Fukumoto, Seiji ; Haffner, Dieter ; Kanematsu, Masanori

PURPOSE:

X-linked hypophosphatemia (XLH) is a rare, genetic, progressive, lifelong disorder caused by pathogenic variants in the PHEX gene, leading to excess fibroblast growth factor 23 (FGF23) and renal phosphate wasting. Advancing Patient Evidence in XLH (APEX) is a 10-year global data unification project that combines 3 regional observational studies (XLH Disease Monitoring Program [DMP], International XLH Registry [IXLHR], and SUNFLOWER). APEX aims to describe the burden and lifelong progression of XLH, to collect real-world data on treatment effectiveness and safety, and to investigate regional differences in treatment outcomes.

METHODS:

This was a baseline analysis of the characteristics and disease burden of a global group of patients with XLH.

RESULTS:

This analysis included 1556 participants (XLH DMP n = 598; IXLHR n = 736; SUNFLOWER n = 222), with 590 participants aged 0-12 years, 193 aged 13-17 years, and 773 aged ≥18 years. Overall, 66 % of participants were female. Family history of XLH and of a PHEX variant were reported for 55 % and 47 % of participants, respectively; 71 % of participants had a confirmed PHEX variant. Participants had reduced height, normal weight, and elevated body mass index compared with a reference population. Clinical histories demonstrated increasing prevalence of dental complications, fractures, and osteoarthritis with age. Median Z-scores for phosphate, tubular maximum reabsorption of phosphate to glomerular filtration rate, and urine calcium/creatinine ratio were below the scores for the reference population.

CONCLUSION:

This baseline analysis provides substantial information on the global characteristics and natural history of patients with XLH in the APEX program.

REGISTRATION:

ClinicalTrials.govNCT03651505 (registered August 24, 2018), NCT03193476 (registered June 13, 2017), NCT03745521 (registered November 6, 2018).

2025-11-01·ADVANCES IN THERAPY

Disease Progression, Clinical Outcomes and Treatment Challenges in Patients with Chronic Kidney Disease and High-Risk Proteinuria

Article

作者: Svensson, Maria K ; Ambery, Phil ; Bodegård, Johan ; Pecoits-Filho, Roberto ; Nam, You-Seon ; Thuresson, Marcus

INTRODUCTION:

A urine albumin-creatinine ratio (UACR) > 700 mg/g signifies severe kidney damage, and nephrology referral is recommended for intensified management. The impact of UACR ≥ 700 mg/g on outcomes and the efficacy of kidney protective therapies in patients have not been thoroughly examined.

METHODS:

Using claims healthcare data from the USA, we identified prevalent patients with chronic kidney disease and a UACR measurement on 1 January 2022, and grouped them by UACR level. We also identified new users of sodium-glucose cotransporter 2 inhibitors (SGLT2i) between 2021 and 2023. Outcomes included estimated glomerular filtration rate (eGFR) slopes and risks of adverse outcomes and mortality.

RESULTS:

Of the 46,626 patients with UACR ≥ 300 mg/g, 23,998 had UACR ≥ 700 mg/g. In the UACR ≥ 700 and UACR 300 to < 700 mg/g groups, mean age was 72 and 74 years, median eGFR was 42 and 50 mL/min/1.73 m², median UACR was 1376 and 437 mg/g at baseline and eGFR slopes were - 5.5 and - 3.1 mL/min/1.73 m² per year, respectively. Compared to the UACR < 10 mg/g group, adjusted hazard ratio of risk of cardiorenal hospitalizations was 5.83 (95% CI 5.55-6.12) and 3.53 (95% CI 3.34-3.73), atherosclerotic cardiovascular disease hospitalizations was 2.97 (95% CI 2.76-3.19) and 2.33 (95% CI 2.15-2.52), and all-cause death was 2.89 (95% CI 2.78-3.02) and 2.10 (95% CI 2.01-2.20) in the UACR ≥ 700 and UACR 300 to < 700 mg/g groups, respectively. Of the 5908 new users of SGLT2i with UACR ≥ 700 mg/g, UACR and eGFR slope improved as expected (to 720 mg/g and - 2.44 mL/min/1.73 m² per year, respectively).

CONCLUSIONS:

A UACR ≥ 700 mg/g identifies patients at high risk of accelerated kidney function decline and adverse outcomes. While emerging therapies such as SGLT2i had the expected benefits, the persistence of residual proteinuria/albuminuria and associated risks highlight an unmet need for optimized and new treatment strategies.

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