Evidence map›Paper›PMID 34999727›Full record

ArticleBone marrow transplantation2022

Allogeneic stem cell transplantation as a curative therapeutic approach for VEXAS syndrome: a case report.

Michael Loschi, Christian Roux, Isabelle Sudaka, Corinne Ferrero-Vacher, Alice Marceau-Renaut, Nicolas Duployez, Thierry Passeron, Thomas Cluzeau

Abstract readCase ReportsLetter
PubMed Publisher
In one paragraph

Article in Bone marrow transplantation, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
27citing papers in PubMed, 1 pooled it
10.2field-weighted citation impact, top 1% 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

27 citing papers in PubMed, 1 synthesis or guideline pooled it, 60 citations in OpenAlex.

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  13. [Macrocytic anemia and polychondritis: VEXAS syndrome].Zeitschrift fur Rheumatologie · 2024
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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

8 authors at 3 institutions in 1 country.

Michael LoschiHematology Department, Nice University Medical Center, Cote d'Azur University, Nice, France. loschi.m@chu-nice.fr.ORCID http://orcid.org/0000-0002-4460-5939
Christian RouxRheumatology Department, Nice University Medical Center, Nice, France.
Isabelle SudakaLaboratoire d'hématologie, CHU de Nice, Hospital Pasteur, Nice, France.
Corinne Ferrero-VacherLaboratoire d'hématologie, CHU de Nice, Hospital Pasteur, Nice, France.
Alice Marceau-RenautUniversity of Lille, CNRS, Inserm, CHU Lille, Institut de Recherche contre le Cancer de Lille, UMR9020-UMR-S 1277-Canther-Cancer Heterogeneity, Plasticity and Resistance to Therapies, 59000, Lille, France.
Nicolas DuployezUniversity of Lille, CNRS, Inserm, CHU Lille, Institut de Recherche contre le Cancer de Lille, UMR9020-UMR-S 1277-Canther-Cancer Heterogeneity, Plasticity and Resistance to Therapies, 59000, Lille, France.
Thierry PasseronCote d'Azur University, INSERM U1065, C3M, Nice, France.
Thomas CluzeauHematology Department, Nice University Medical Center, Cote d'Azur University, Nice, France.ORCID http://orcid.org/0000-0002-6745-1127
Inserm · FRCentre Hospitalier Universitaire de Nice · FRCentre National de la Recherche Scientifique · FR

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

to the editorVEXAS (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) syndrome is a newly described entity linked to somatic mutation of UBA1, encompassing inflammatory disorders and hematological malignancies. Patients experiments symptoms related to inflammatory manifestations on the skin, joints, lungs. Most patients are refractory to usual anti-inflammatory or immunosuppressive treatments. Half of them will develop hematological diseases, mostly myelodysplastic syndromes. VEXAS patients with hematological malignancies have a poor outcome and no curative option has been described so far. Because in the first reported cohort of VEXAS patients the UBA1 mutation was only found in hematopoietic stem cells but not in fibroblasts, we hypothesized that bone marrow transplantation would provide a cure for the disease. Here we report the case of a VEXAS patient who successfully received an allogeneic hematopoietic stem cell transplantation as a curative option.

Indexed as

Hematologic NeoplasmsHematopoietic Stem Cell TransplantationMyelodysplastic SyndromesHumansMutationUbiquitin-Activating EnzymesUbiquitin-Activating Enzymes

Identifiers

PMID34999727
OpenAlexW4205861608

What OpenQuestion holds

Textmetadata
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 OpenQuestion graph.