Evidence map›Paper›PMID 38568107›Full record

Trial reportVascular medicine (London, England)2024

Pilot study to evaluate the safety and effectiveness of etidronate treatment for arterial calcification due to deficiency of CD73 (ACDC).

Elisa A Ferrante, Cornelia D Cudrici, Mahmood Rashidi, Yi-Ping Fu, Rebecca Huffstutler, Katherine Carney, Marcus Y Chen, Cynthia St Hilaire, Kevin Smith, Hadi Bagheri and 5 more

Open access · greenAbstract readClinical Trial
In one paragraph

Trial report in Vascular medicine (London, England), 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
10citing papers in PubMed, 1 pooled it
6.6field-weighted citation impact, top 3% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

10 citing papers in PubMed, 1 synthesis or guideline pooled it, 12 citations in OpenAlex.

  1. Effect of osteoporosis medications on vascular and valvular calcification: a systematic review and meta-analysis.Osteoporosis international : a journal established as result of cooperation between the European Foundation for Osteoporosis and the National Osteoporosis Foundation of the USA · 2025
    Pooled it
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  5. Considerations on the Development of Therapeutics in Vascular Calcification.Journal of cardiovascular development and disease · 2025
    Review
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

15 authors at 3 institutions in 1 country.

Elisa A FerranteNational Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA.ORCID 0000-0002-0352-709X
Cornelia D CudriciNational Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA.
Mahmood RashidiNational Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA.
Yi-Ping FuNational Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA.
Rebecca HuffstutlerNational Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA.
Katherine CarneyNational Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA.
Marcus Y ChenNational Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA.
Cynthia St HilaireDepartments of Medicine and Bioengineering, Vascular Medicine Institute, University of Pittsburg, PA, USA.ORCID 0000-0003-1871-6915
Kevin SmithClinical Center Nursing Department, Hatfield Clinical Center at the National Institutes of Health, Bethesda, MD, USA.
Hadi BagheriRadiology and Imaging Sciences, National Institutes of Health Clinical Center, Bethesda, MD, USA.
James D KatzNational Institute of Arthritis and Musculoskeletal and Skin Diseases, National Institutes of Health, Bethesda, MD, USA.ORCID 0000-0002-2098-8883
Carlos R FerreiraNational Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.
William A GahlNational Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.
Manfred BoehmNational Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA.
Alessandra BrofferioNational Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA.
National Institutes of Health · USNational Institutes of Health Clinical Center · USPittsburg State University · US

Funding

Human Biochemical GeneticsZIAHG000215 · NHGRI · NATIONAL HUMAN GENOME RESEARCH INSTITUTE · PI GAHL, WILLIAM · 2009 to 2025
$72.6M
Vascular remodeling in patients with rare genetic disordersZIAHL006077 · NHLBI · NATIONAL HEART, LUNG, AND BLOOD INSTITUTE · PI BOEHM, MANFRED · 2010 to 2025
$22.9M
Cardiovascular CT Clinical ResearchZIAHL006220 · NHLBI · NATIONAL HEART, LUNG, AND BLOOD INSTITUTE · PI CHEN, MARCUS · 2017 to 2025
$22.5M
Cardiovascular Clinical Section StudiesZICHL006199 · NHLBI · NATIONAL HEART, LUNG, AND BLOOD INSTITUTE · PI SACK, MICHAEL · 2017 to 2025
$8.8M
Skeletal GenomicsZIAHG200407 · NHGRI · NATIONAL HUMAN GENOME RESEARCH INSTITUTE · PI FERREIRA, CARLOS · 2020 to 2024
$3.7M
Undiagnosed Diseases ProgramZIAHG200421 · NHGRI · NATIONAL HUMAN GENOME RESEARCH INSTITUTE · PI GAHL, WILLIAM · 2023 to 2025
$825k
Regulation of Vascular Calcification by Adenosine SignalingK22HL117917 · NHLBI · UNIVERSITY OF PITTSBURGH AT PITTSBURGH · PI ST. HILAIRE, CYNTHIA · 2015 to 2017
$731k
NHLBI NIH HHS K22 HL117917
6 · The paper itself

Abstract

backgroundArterial calcification due to deficiency of CD73 (ACDC; OMIM 211800) is a rare genetic disease resulting in calcium deposits in arteries and small joints causing claudication, resting pain, severe joint pain, and deformities. Currently, there are no standard treatments for ACDC. Our previous work identified etidronate as a potential targeted ACDC treatment, using in vitro and in vivo disease models with patient-derived cells. In this study, we test the safety and effectiveness of etidronate in attenuating the progression of lower-extremity arterial calcification and vascular blood flow based on the computed tomography (CT) calcium score and ankle-brachial index (ABI).

methodsSeven adult patients with a confirmed genetic diagnosis of ACDC were enrolled in an open-label, nonrandomized, single-arm pilot study for etidronate treatment. They took etidronate daily for 14 days every 3 months and were examined at the NIH Clinical Center bi-annually for 3 years. They received a baseline evaluation as well as yearly follow up after treatment. Study visits included imaging studies, exercise tolerance tests with ABIs, clinical blood and urine testing, and full dental exams.

resultsEtidronate treatment appeared to have slowed the progression of further vascular calcification in lower extremities as measured by CT but did not have an effect in reversing vascular and/or periarticular joint calcifications in our small ACDC cohort.

conclusionsEtidronate was found to be safe and well tolerated by our patients and, despite the small sample size, appeared to show an effect in slowing the progression of calcification in our ACDC patient cohort.

Indexed as

5'-NucleotidaseEtidronic AcidGPI-Linked ProteinsVascular CalcificationAdultAgedAnkle Brachial IndexBone Density Conservation AgentsComputed Tomography AngiographyDisease ProgressionFemaleGenetic Predisposition to DiseaseHumansLower ExtremityMaleMiddle Aged5'-NucleotidaseBone Density Conservation AgentsEtidronic AcidGPI-Linked ProteinsNT5E protein, humanACDCadenosine signalingbisphosphonatesCD73 deficiencyetidronatejoint calcificationNT5Erare diseasevascular calcification

Identifiers

PMID38568107
PMCPMC11608424
OpenAlexW4393871289

What Socratic holds

Textmetadata
LicenceTDM
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.