Evidence map›Paper›PMID 37772407›Full record

ReviewClinical trials (London, England)2024

Potential endpoints for assessment of bone health in persons with neurofibromatosis type 1.

Andrea M Gross, Scott R Plotkin, Nelson B Watts, Michael J Fisher, Laura J Klesse, Andrés J Lessing, Miranda L McManus, A Noelle Larson, Beverly Oberlander, Jonathan J Rios and 4 more

Open access · greenAbstract readReview
In one paragraph

Review in Clinical trials (London, England), 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
0.2field-weighted citation impact, top 42% 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

1 citing paper in PubMed, 1 citations in OpenAlex.

  1. Article
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

14 authors at 13 institutions in 1 country.

Andrea M GrossPediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, Bethesda, MD, USA.ORCID 0000-0003-1646-4531
Scott R PlotkinDepartment of Neurology and Cancer Center, Massachusetts General Hospital, Boston, MA, USA.
Nelson B WattsMercy Health Osteoporosis and Bone Health Services, Cincinnati, OH, USA.
Michael J FisherDivision of Oncology, The Children's Hospital of Philadelphia and the University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.ORCID 0000-0003-3408-3839
Laura J KlesseDivision of Hematology/Oncology, Department of Pediatrics, UT Southwestern Medical Center, Dallas, TX, USA.ORCID 0000-0003-1323-7720
Andrés J LessingNeurofibromatosis Northeast, Burlington, MA, USA.ORCID 0000-0001-8250-0145
Miranda L McManusDepartment of Biology, College of Charleston, Charleston, SC, USA.ORCID 0009-0001-5390-0518
A Noelle LarsonDepartment of Orthopedic Surgery, Mayo Clinic, Rochester, MN, USA.
Beverly OberlanderNeurofibromatosis Network, Henderson, NV, USA.
Jonathan J RiosCenter for Pediatric Bone Biology and Translational Research, Scottish Rite for Children, McDermott Center for Human Growth and Development, UT Southwestern Medical Center, Dallas, TX, USA.
Herb SarnoffResearch and Development, Infixion Bioscience, Inc., San Diego, CA, USA.ORCID 0000-0002-3219-3113
Brittany N SimpsonDivision of Human Genetics, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH, USA.ORCID 0000-0002-8643-0161
Nicole J UllrichDepartment of Neurology, Boston Children's Hospital, Boston, MA, USA.
David A StevensonDivision of Medical Genetics, Department of Pediatrics, Stanford University, Stanford, CA, USA.
Boston Children's Hospital · USChildren's Hospital of Philadelphia · USCincinnati Children's Hospital Medical Center · USCollege of Charleston · USMassachusetts General Hospital · USMayo Clinic in Arizona · USMercy Health · USNational Cancer Institute · USNeurofibromatosis NetworkSouthwestern Medical Center · USStanford University · USTexas Neurofibromatosis Foundation · USTexas Scottish Rite Hospital for Children · US

Funding

Genetic Analysis and Manipulation Core (GAEC)P50HD105351 · NICHD · BOSTON CHILDREN'S HOSPITAL · PI SCOTT Loren POMEROY, MUSTAFA SAHIN · 2021 to 2026
$9.4M
Intramural NIH HHS Z99 CA999999NICHD NIH HHS P50 HD105351
6 · The paper itself

Abstract

Neurofibromatosis type 1 is a genetic syndrome characterized by a wide variety of tumor and non-tumor manifestations. Bone-related issues, such as scoliosis, tibial dysplasia, and low bone mineral density, are a significant source of morbidity for this population with limited treatment options. Some of the challenges to developing such treatments include the lack of consensus regarding the optimal methods to assess bone health in neurofibromatosis type 1 and limited data regarding the natural history of these manifestations. In this review, the Functional Committee of the Response Evaluation in Neurofibromatosis and Schwannomatosis International Collaboration: (1) presents the available techniques for measuring overall bone health and metabolism in persons with neurofibromatosis type 1, (2) reviews data for use of each of these measures in the neurofibromatosis type 1 population, and (3) describes the strengths and limitations for each method as they might be used in clinical trials targeting neurofibromatosis type 1 bone manifestations. The Response Evaluation in Neurofibromatosis and Schwannomatosis International Collaboration supports the development of a prospective, longitudinal natural history study focusing on the bone-related manifestations and relevant biomarkers of neurofibromatosis type 1. In addition, we suggest that the neurofibromatosis type 1 research community consider adding the less burdensome measurements of bone health as exploratory endpoints in ongoing or planned clinical trials for other neurofibromatosis type 1 manifestations to expand knowledge in the field.

Indexed as

NeurilemmomaNeurofibromatosesNeurofibromatosis 1Skin NeoplasmsBone DensityHumansProspective Studiesbonebone mineral densityclinical trialsNeurofibromatosis type 1osteoporosis

Identifiers

PMID37772407
PMCPMC10920397
OpenAlexW4387157734

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.