Evidence map›Paper›PMID 39006867›Full record

ArticleJBMR plus2024

Imiglucerase, cholecalciferol, and bone-diet in skeletal health management of type I Gaucher disease patients: a pilot study and systematic review.

Antonio Barbato, Anita Vergatti, Alfonso Giaquinto, Ilaria Libera Pizzulo, Ludovica Perna, Giuseppe Perruolo, Veronica Abate, Michelina Sibilio, Ciro Mainolfi, Ernesto Soscia and 5 more

Abstract read
In one paragraph

Article in JBMR plus, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed
–field-weighted citation impact
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

4 citing papers in PubMed.

  1. Article
  2. Article
  3. Article
  4. Review
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.

Antonio BarbatoDepartment of Clinical Medicine and Surgery, Federico II University of Naples, 80131 Naples, Italy.
Anita VergattiDepartment of Clinical Medicine and Surgery, Federico II University of Naples, 80131 Naples, Italy.
Alfonso GiaquintoDepartment of Clinical Medicine and Surgery, Federico II University of Naples, 80131 Naples, Italy.
Ilaria Libera PizzuloDepartment of Clinical Medicine and Surgery, Federico II University of Naples, 80131 Naples, Italy.
Ludovica PernaDepartment of Clinical Medicine and Surgery, Federico II University of Naples, 80131 Naples, Italy.
Giuseppe PerruoloDepartment of Translational Medical Sciences, Federico II University of Naples, 80131 Naples, Italy.
Veronica AbateDepartment of Clinical Medicine and Surgery, Federico II University of Naples, 80131 Naples, Italy.
Michelina SibilioMetabolic Diseases Unit, Santobono-Pausilipon Children's Hospital, 80129 Naples, Italy.
Ciro MainolfiDepartment of Advanced Biomedical Sciences, Federico II University of Naples, 80131 Naples, Italy.
Ernesto SosciaInstitute of Biostructures and Bioimaging of the National Research Council - CNR, 80131 Naples, Italy.
Gianpaolo De FilippoAssistance Publique-Hôpitaux de Paris, Hôpital Robert-Debré, Service d'Endocrinologie-Diabétologie, 75019 Paris, France.
Pietro FormisanoDepartment of Translational Medical Sciences, Federico II University of Naples, 80131 Naples, Italy.
Ferruccio GallettiDepartment of Clinical Medicine and Surgery, Federico II University of Naples, 80131 Naples, Italy.
Pasquale StrazzulloFormer Professor of Internal Medicine, Federico II University, 80131 Naples, Italy.
Domenico RendinaDepartment of Clinical Medicine and Surgery, Federico II University of Naples, 80131 Naples, Italy.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Skeletal anomalies represent a characteristic feature of type 1 Gaucher disease (GD1). Here we evaluated the impact of an integrated therapy comprising enzyme-replacement therapy (ERT), cholecalciferol, and a normocalcemic-normocaloric-hyposodic diet (bone diet) on bone health in GD1 patients. We also performed a systematic review to compare our results with available data. From January 1, 2015 to February 28, 2019, all GD1 patients referred to Federico II University were enrolled and treated with the integrated therapy. Bone turnover markers and bone mineral density (BMD) were evaluated at baseline (T0) and after 24 months (T24). We enrolled 25 GD1 patients, all showing 25-hydroxy vitamin D (25OHD) levels < 50 nmol/l (hypovitaminosis D) at T0. Response to cholecalciferol treatment was effective, showing a direct relationship between 25OHD levels before and after treatment. At T0, 2 GD1 patients showed fragility fractures, 5 the Erlenmeyer flask deformity, 3 osteonecrosis, and 7 a BMD

Indexed as

bone markersenzyme-replacement therapyGaucher diseasevitamin D

Identifiers

PMID39006867
PMCPMC11245647

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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 OpenQuestion graph.