Evidence map›Paper›PMID 39060513›Full record

SynthesisBone marrow transplantation2024

Assessing the efficacy of allogeneic hematopoietic cell transplantation in VEXAS syndrome: results of a systematic review and meta-analysis.

Razan Mohty, Tea Reljic, Nayef Abdel-Razeq, Omer Jamy, Talha Badar, Ambuj Kumar, Mahmoud Aljurf, Mohamed A Kharfan-Dabaja

Abstract readSystematic ReviewMeta-Analysis
PubMed Publisher
In one paragraph

Synthesis in Bone marrow transplantation, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed, 1 pooled it
–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

8 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Guideline
  2. Article
  3. Article
  4. Article
  5. Expert Perspective: Hematologic Malignancies and Vasculitis.Arthritis & rheumatology (Hoboken, N.J.) · 2025
    Review
  6. Article
  7. VEXAS Syndrome: An Overlooked Threat.Balkan medical journal · 2025
    Article
  8. [Treatment of VEXAS syndrome].Zeitschrift fur Rheumatologie · 2024
    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

8 authors.

Razan MohtyDivision of Hematology-Oncology and O'Neal Comprehensive Cancer Center, University of Alabama at Birmingham, Birmingham, AL, USA.
Tea ReljicResearch Methodology and Biostatistics Core, Office of Research Morsani College of Medicine, University of South Florida, Tampa, FL, USA.ORCID http://orcid.org/0000-0003-2029-6753
Nayef Abdel-RazeqDivision of Hematology-Oncology and Blood and Marrow Transplantation and Cellular Therapy Program and Mayo Clinic Comprehensive Cancer Center, Mayo Clinic, Jacksonville, FL, USA.
Omer JamyDivision of Hematology-Oncology and O'Neal Comprehensive Cancer Center, University of Alabama at Birmingham, Birmingham, AL, USA.
Talha BadarDivision of Hematology-Oncology and Blood and Marrow Transplantation and Cellular Therapy Program and Mayo Clinic Comprehensive Cancer Center, Mayo Clinic, Jacksonville, FL, USA.ORCID http://orcid.org/0000-0003-1548-918X
Ambuj KumarResearch Methodology and Biostatistics Core, Office of Research Morsani College of Medicine, University of South Florida, Tampa, FL, USA.
Mahmoud AljurfKing Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia.ORCID http://orcid.org/0000-0003-3942-7370
Mohamed A Kharfan-DabajaDivision of Hematology-Oncology and Blood and Marrow Transplantation and Cellular Therapy Program and Mayo Clinic Comprehensive Cancer Center, Mayo Clinic, Jacksonville, FL, USA. KharfanDabaja.Mohamed@mayo.edu.ORCID http://orcid.org/0000-0001-7394-5185

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

VEXAS syndrome is an X-linked monogenic disease with adult-onset inflammatory disease and myeloid dysplasia, with clinical presentation ensuing in the fifth decade of life or later. Inflammatory symptoms associated with VEXAS syndrome are treated with several lines of therapy, eventually requiring allogeneic hematopoietic cell transplantation (allo-HCT). No evidence from randomized controlled trials exists on allo-HCT versus other treatments in patients not responding to front-line therapy(ies). We show results of a systematic review/meta-analysis (SR/MA) following a search using EMBASE, PUBMED/MEDLINE and Web of Science on April 5, 2024. We extracted outcomes based on benefits (overall response rate (ORR), complete remission (CR), event-free survival (EFS) and overall survival (OS), and harms (non-relapse mortality (NRM) and acute and chronic graft-versus-host disease (GVHD)). The search identified 88 studies. Four studies (39 patients) met inclusion criteria. Median follow-up time after allo-HCT ranged from 8 to 18.5 months. Pooled EFS and OS rates were 56% and 86%, respectively. Pertaining to harms, pooled NRM rate was 14%. Pooled rates of acute and chronic GVHD were 42% and 13%, respectively. Allo-HCT is an effective treatment for VEXAS syndrome. We hope these results would increase awareness about this underdiagnosed and underreported disease.

Indexed as

Hematopoietic Stem Cell TransplantationMyelodysplastic SyndromesSkin Diseases, GeneticAllograftsGraft vs Host DiseaseHumansTransplantation, Homologous

Identifiers

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.