Evidence map›Paper›PMID 32708003›Full record

ArticleJournal of clinical medicine2020

A Cross-Sectional Retrospective Study of Non-Splenectomized and Never-Treated Patients with Type 1 Gaucher Disease.

Christine Serratrice, Jérôme Stirnemann, Amina Berrahal, Nadia Belmatoug, Fabrice Camou, Catherine Caillaud, Thierry Billette de Villemeur, Florence Dalbies, Bérengère Cador, Roseline Froissart and 17 more

Open access · goldAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Journal of clinical medicine, 2020. 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
0.7field-weighted citation impact, top 30% 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

4 citing papers in PubMed, 12 citations in OpenAlex.

  1. Gaucher disease, state of the art and perspectives.Journal of internal medicine · 2025
    Review
  2. Article
  3. A rare homozygous p.Arg87Trp variant of theClinical case reports · 2022
    Article
  4. 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

27 authors at 17 institutions in 2 countries.

Christine SerratriceDepartment of Internal Medicine for the Aged, Geneva University Hospitals, 1226 Thonex- Geneva, Switzerland.
Jérôme StirnemannDepartment of Internal Medicine, Department of Medicine, Geneva University Hospitals, 1205 Geneva, Switzerland.
Amina BerrahalUniversity Hospital of Clermont Ferrand, Hematology Biology Department, 63000 Clermont-Ferrand, France.
Nadia BelmatougDepartment of Internal Medicine, Centre de Référence des Maladies Lysosomales, AP-HP.Nord, Site Beaujon, Paris University, 92110 Clichy, France.
Fabrice CamouIntensive Care Unit, Hôpital Saint-André, CHU Bordeaux, 33000 Bordeaux, France.
Catherine CaillaudBiochemistry, Metabolomics, and Proteomics Department, Necker Enfants Malades University Hospital, AP-HP, Center-Paris University, 75015 Paris, France.
Thierry Billette de VillemeurDepartment of Pediatric Neurology, Hôpital Trousseau, AP-HP, 75012 Paris, France.
Florence DalbiesInstitute of Cancerology and Hematology, CHRU Morvan, 29200 Brest, France.
Bérengère CadorDepartment of Internal Medicine, CHU Pontchaillou, 35000 Rennes, France.
Roseline FroissartBiochemical and Molecular Biology Department, Centre de Biologie et de Pathologie Est, Hospices Civils de Lyon, 69500 Bron, France.
Agathe MasseauDepartment of Internal Medicine, CHU Hôtel Dieu, 44093 Nantes, France.
Anaïs BrassierReference Centre for Hereditary Metabolic Diseases, Hôpital Necker-Enfants Malades, AP-HP, IHU Institut Imagine, 75015 Paris, France.
Bénédicte HivertDepartment of Clinical Hematology, Hôpital Saint Vincent de Paul, Groupement des Hôpitaux de l'Institut Catholique de Lille, 59800 Lille, France.
Laure SwiaderDepartment of Internal Medicine, CHU Timone, 13005 Marseille, France.
Ivan BertchanskyDepartment of Internal Medicine, Saint-Eloi Hospital, CHU Montpellier, 34000 Montpellier, France.
Claire de MoreuilDepartment of Internal Medicine, Hôpital de la Cavale Blanche, CHRU de Brest, 29200 Brest, France.
Brigitte ChabrolDepartment of Pediatric Neurology, CHU Timone, 13005 Marseille, France.
Isabelle DurieuDepartment of Internal Medicine, Hospices Civils de Lyon, University Claude Bernard Lyon 1, 69495 Pierre-Bénite, France.
Vanessa Leguy SeguinDepartment of Internal Medicine and Clinical Immunology, CHU F. Mitterrand, 21000 Dijon, France.
Leonardo AstudilloDepartment of Internal Medicine, CHU Toulouse, 31300 Toulouse, France.
Sébastien HumbertDepartment of Internal Medicine, Besancon University Hospital, 25000 Besancon, France.
Samia PichardService of Metabolic Diseases, Hôpital Robert Debré, 75019 Paris, France.
Catherine MarcelDepartment of Internal Medicine, Centre de Référence des Maladies Lysosomales, AP-HP.Nord, Site Beaujon, Paris University, 92110 Clichy, France.
Isabelle Hau RainsardService of General Pediatry, CHI Créteil, 94000 Créteil, France.
Monia BengherbiaDepartment of Internal Medicine, Centre de Référence des Maladies Lysosomales, AP-HP.Nord, Site Beaujon, Paris University, 92110 Clichy, France.
Karima YousfiDepartment of Internal Medicine, Centre de Référence des Maladies Lysosomales, AP-HP.Nord, Site Beaujon, Paris University, 92110 Clichy, France.
Marc G BergerUniversity Hospital of Clermont Ferrand, Hematology Biology Department, 63000 Clermont-Ferrand, France.
Hôpital Beaujon · FRCentre Hospitalier Régional Universitaire de Brest · FRHôpital de la Timone · FRUniversité Clermont Auvergne · FRAssistance Publique – Hôpitaux de Paris · FRCentre Hospitalier Universitaire de Besançon · FRCHU Dijon Bourgogne · FRGroupe Hospitalier de l'Institut Catholique de Lille · FRHôpital Necker-Enfants Malades · FRHôpital Pontchaillou · FRHôpital Robert-Debré · FRHôpital Saint-André · FRHôpital Saint Eloi · FRHospices Civils de Lyon · FRSorbonne Université · FRUniversité Claude Bernard Lyon 1 · FRUniversity Hospital of Geneva · CH

Funding

Shire International GmbH, now part of Takeda IIR-FRA-001381
6 · The paper itself

Abstract

Patients with type 1 Gaucher disease (GD1) present thrombocytopenia, anemia, organomegaly, and bone complications. Most experts consider that the less aggressive forms do not require specific treatment. However, little is known about the disease course of these forms. The objective of this cross-sectional retrospective study was to compare the clinical, radiological, and laboratory characteristics of patients with less severe GD1 at diagnosis and at the last evaluation to identify features that might lead to potential complications. Non-splenectomized and never-treated patients (19 women and 17 men) were identified in the French Gaucher Disease Registry (FGDR). Their median age was 36.6 years (2.4-75.1), and their median follow-up was 7.8 years (0.4-32.4). Moreover, 38.7% were heterozygous for the

Indexed as

disease courseenzyme replacement therapyGaucher diseaselysosomal storage disorderprognosisrisk factors

Identifiers

PMID32708003
PMCPMC7464688
OpenAlexW3044024096

What OpenQuestion holds

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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 OpenQuestion graph.