ReviewBone marrow transplantation2022
Update on VEXAS and role of allogeneic bone marrow transplant: Considerations on behalf of the Chronic Malignancies Working Party of the EBMT.
Review 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 19 papers, 2 of them syntheses that pooled 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.
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.
Who cites it
19 citing papers in PubMed, 2 syntheses or guidelines pooled it, 37 citations in OpenAlex.
- Hypomethylating agents in vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic syndrome (VEXAS): A systematic review.British journal of haematology · 2026Pooled it
- Emerging treatment approaches for VEXAS syndrome: a systematic review and meta-analysis.Annals of hematology · 2025Pooled it
- VEXAS Syndrome: Genetics, Gender Differences, Clinical Insights, Diagnostic Pitfalls, and Emerging Therapies.International journal of molecular sciences · 2025Review
- Exploring patient-reported outcomes and morbidity burden of patients with VEXAS syndrome: a scoping review.EClinicalMedicine · 2025Review
- Real-world outcomes of vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic syndrome at a tertiary referral centre.British journal of haematology · 2025Article
- Clinical features and treatments of VEXAS syndrome in critical care: a scoping review.Critical care (London, England) · 2025Article
- Allogenic haematopoietic stem cell transplantation in VEXAS: A review of 33 patients.Clinical rheumatology · 2024Review
- Role of allogeneic hematopoietic cell transplantation in VEXAS syndrome.Annals of hematology · 2024Review
- De-escalation of corticosteroids and clonal remission inHaematologica · 2024Article
- A vexing case of a 73-year-old man with fevers, orbital cellulitis, and asymptomatic interstitial lung disease.Respirology case reports · 2024Article
- Long-term genetic and clinical remissions after cessation of azacitidine treatment in patients with VEXAS syndrome.HemaSphere · 2024Article
- [The most frequent febrile syndromes and autoinflammatory diseases in adulthood].Zeitschrift fur Rheumatologie · 2024Review
- Allogeneic hematopoietic cell transplantation for VEXAS syndrome: results of a multicenter study of the EBMT.Blood advances · 2024Article
- VEXAS syndrome: complete molecular remission after hypomethylating therapy.Annals of hematology · 2024Article
- Review
- Intrapatient competition of VEXAS syndrome and CML clones.Blood advances · 2023Article
- VEXAS Syndrome-Review.Global medical genetics · 2023Review
- How I Manage Transplant Ineligible Patients with Myelodysplastic Neoplasms.Clinical hematology international · 2023Review
- Hematopoietic Stem Cell Transplantation for Myelodysplastic Syndromes: The Current Landscape and Future Directions.Cancer journal (Sudbury, Mass.)Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 2 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
VEXAS (acronym for Vacuoles, E1 enzyme, X-linked, Autoinflammatory, Somatic) is a fascinating new entity encompassing a variety of clinical manifestations, spanning from auto-inflammatory symptoms to hematologic disorders, including myelodysplastic syndromes and plasma cell dyscrasias. Genetically defined by somatic mutations of the X-linked gene UBA1 in hematopoietic stem and progenitor cells, VEXAS typically manifests in males during the fifth/sixth decade of life. Since its discovery, several groups have documented pleomorphic clinical phenotypes, in addition to a plethora of therapeutic options (e.g., JAK inhibitors, hypomethylating agents, and allogeneic stem cell transplant, allo-HCT) in retrospective case series. However, no treatment guidelines have been validated to date, VEXAS patients are typically steroid-dependent and may manifest life-threatening inflammatory symptoms refractory to multiple lines of therapy. To date, the only curative option appears to be allo-HCT in suitable individuals. Nonetheless, this procedure carries an inherent risk of morbidity and mortality that must be judiciously evaluated against a phenotypically diverse disorder where the optimal therapeutic algorithm remains ill-defined. Herein, we provide an overview of the current VEXAS data/ therapeutic evidence and discuss the curative potential of allo-HCT whilst highlighting the efforts required for generation of robust data able to inform therapeutic decisions.
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Registered trials
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.