Therapeutic Outcomes in <scp>VEXAS</scp> Syndrome: A Multicenter Comparative Cohort of Allogeneic Hematopoietic Stem Cell Transplantation and Hypomethylating Agents

Hypomethylating agents (HMA) and allogeneic hematopoietic stem cell transplantation (alloHSCT) have both demonstrated remissions in VEXAS; however, comparative data is lacking. We conducted a multicenter, retrospective analysis of 66 patients diagnosed with VEXAS syndrome treated with HMA (n = 35) or alloHSCT (n = 31). Baseline characteristics such as genetics, co-morbidities, and performance status were balanced between the groups, except older age in the HMA group. Median follow-up from therapy initiation was 18 months (95% CI: 11-26), and 14 (21%) deaths were reported (alloHSCT n = 3; HMA n = 11). Among all evaluable patients within the alloHSCT cohort, all patients achieved molecular remission, and a substantial proportion of patients discontinued glucocorticoids (58%). In contrast, HMA therapy was associated with lower but meaningful rates of molecular remission (22%) and glucocorticoid discontinuation (6%). In a real-world setting, HMA therapy was associated with a high discontinuation rate related to toxicity or lack of response. On multivariable analysis adjusted for age and Charlson Comorbidity Index, alloHSCT was associated with improved overall survival (HR = 0.20, 95% CI: 0.05-0.81; p = 0.024). This association remained consistent across multiple ancillary sensitivity analyses, including restriction to transplant-eligible patients, patients aged ≤ 75 years, 1:1 matching, and propensity score-based weighted analyses. Although limited by retrospective design, these findings suggest that alloHSCT remains an attractive and potentially curative strategy in selected patients with VEXAS. Prospective validation of these findings is warranted.

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Journal
American Journal of Hematology
Published
2026-06-08
DOI
https://doi.org/10.1002/ajh.70401
Primary Topic
Otitis Media and Relapsing Polychondritis
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article
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article

Therapeutic Outcomes in VEXAS Syndrome: A Multicenter Comparative Cohort of Allogeneic Hematopoietic Stem Cell Transplantation and Hypomethylating Agents

Naseema Gangat, Terra L. Lasho, David Dingli, A. Saliba et al.
American Journal of Hematology
Otitis Media and Relapsing Polychondritis
article

Therapeutic Outcomes in VEXAS Syndrome: A Multicenter Comparative Cohort of Allogeneic Hematopoietic Stem Cell Transplantation and Hypomethylating Agents

Naseema Gangat, Terra L. Lasho, David Dingli, A. Saliba, Matthew J. Koster, M. Hefazi, Hemant S. Murthy, M. R. Litzow, Horatiu Olteanu, Urshila Durani, Saurabh Chhabra, Ronald S. Go, James M. Foran, Aasiya Matin, Joseph P. McGuirk, Amelia Langston, David B. Beck, Hassan B. Alkhateeb, Yael N. Kusne, Talha Badar, Kebede H. Begna, Saubia Fathima, William J. Hogan, Gary Ho, Kimberly J. Langer, Maria Sirenko, Nathan Punwani, Jesus Gonzalez-Lugo, Aref Al-Kali, Abhishek A. Mangaonkar, Megan Sullivan, Kaaren K. Reichard, Mary Riwes, Ali Alsugair, Christy Finke, Tariq Kewan, Susan M. Geyer, Jeanne Palmer, Margaret Mei‐Kay Wong, Maher Abdul‐Hay, Elizabeth F. Krakow, Mithun V. Shah, Jun Choi, Taxiarchis Kourelis, Jade Kutzke, Gabriel Bartoo, Mrinal M. Patnaik, Kristen McCullough, Kenneth J. Warrington
article en

Abstract

Hypomethylating agents (HMA) and allogeneic hematopoietic stem cell transplantation (alloHSCT) have both demonstrated remissions in VEXAS; however, comparative data is lacking. We conducted a multicenter, retrospective analysis of 66 patients diagnosed with VEXAS syndrome treated with HMA (n = 35) or alloHSCT (n = 31). Baseline characteristics such as genetics, co-morbidities, and performance status were balanced between the groups, except older age in the HMA group. Median follow-up from therapy initiation was 18 months (95% CI: 11-26), and 14 (21%) deaths were reported (alloHSCT n = 3; HMA n = 11). Among all evaluable patients within the alloHSCT cohort, all patients achieved molecular remission, and a substantial proportion of patients discontinued glucocorticoids (58%). In contrast, HMA therapy was associated with lower but meaningful rates of molecular remission (22%) and glucocorticoid discontinuation (6%). In a real-world setting, HMA therapy was associated with a high discontinuation rate related to toxicity or lack of response. On multivariable analysis adjusted for age and Charlson Comorbidity Index, alloHSCT was associated with improved overall survival (HR = 0.20, 95% CI: 0.05-0.81; p = 0.024). This association remained consistent across multiple ancillary sensitivity analyses, including restriction to transplant-eligible patients, patients aged ≤ 75 years, 1:1 matching, and propensity score-based weighted analyses. Although limited by retrospective design, these findings suggest that alloHSCT remains an attractive and potentially curative strategy in selected patients with VEXAS. Prospective validation of these findings is warranted.

American Journal of Hematology
Mayo Clinic (US), University of Michigan (US), Jacksonville College (US), WinnMed (US), NYU Langone Health (US), Fred Hutch Cancer Center (US), The University of Kansas Cancer Center (US), Piedmont Cancer Institute (US), Center for Human Genetics (US), Mayo Clinic in Arizona (US), Mayo Clinic Hospital (US), Mayo Clinic in Florida (US), New York University (US)
Good health and well-being
Openalex Percentile: Top 5%
Otitis Media and Relapsing Polychondritis
DOISourceOpenAlex
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