Related Experiment Videos

The First Reported Case of VEXAS Syndrome in Lebanon: Efficacy of Azacitidine as a Therapeutic Option-Case Report

Roudy Issa1, Bassem Akiki2, Sophie Georgin-Lavialle3

  • 1Department of Internal Medicine and Clinical Immunology, Notre Dame des Secours University Hospital, Jbeil, Lebanon.

Case Reports in Hematology
|May 15, 2026
PubMed
Abstract

Insights

Azacitidine effectively treats VEXAS syndrome with myelodysplastic syndrome (MDS) in patients ineligible for stem cell transplant. This approach offers a viable path to remission and reduced steroid dependency.

Area of Science:

  • Hematology
  • Immunology
  • Genetics

Background:

  • Vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic (VEXAS) syndrome is a rare adult-onset autoinflammatory disease.
  • Management is challenging due to rarity and lack of treatment guidelines.

Purpose of the Study:

  • Report the first case of VEXAS syndrome in Lebanon.
  • Evaluate azacitidine's efficacy in a VEXAS syndrome patient with myelodysplastic syndrome (MDS).

Main Methods:

  • A 73-year-old male patient with VEXAS syndrome and intermediate-risk MDS was treated.
  • Therapeutic interventions included corticosteroids, methotrexate, erythropoietin, tocilizumab, and azacitidine.
  • Azacitidine regimen was adjusted to manage neutropenia and infections.

Main Results:

  • The patient achieved complete remission and became transfusion-independent.
  • Stable hemoglobin levels were maintained with reduced corticosteroid dependence.
  • Significant clinical and hematologic improvements were observed.

Conclusions:

  • Azacitidine demonstrates efficacy in managing VEXAS syndrome with MDS.
  • It offers a potential treatment for patients ineligible for hematopoietic stem cell transplantation.
  • This approach may lead to sustained remission and decreased reliance on corticosteroids.
Keywords:
VEXAS syndromeazacitidinecase reportmyelodysplastic syndrome

Related Concept Videos

Antiepileptic Drugs: Modulators of Neurotransmitter Release Mediated by SV2A Protein01:20

Antiepileptic Drugs: Modulators of Neurotransmitter Release Mediated by SV2A Protein

Antiepileptic drugs, such as levetiracetam (Keppra) and brivaracetam (Briviact), have emerged as crucial tools in managing epilepsy. These medications exert their therapeutic effects by targeting the synaptic vesicle protein SV2A, a transmembrane glycoprotein primarily found in the brain.
SV2A is a transmembrane glycoprotein located predominantly in the brain, modulating the release of neurotransmitters for neuronal communication. Both levetiracetam and brivaracetam exhibit a high affinity for...
Cancer Therapies02:49

Cancer Therapies

Cancer therapies are various modes of treatment, such as surgery, radiation therapy, and chemotherapy that are administered to cancer patients.
However, cancer treatments can pose several challenges, as therapies used to kill cancer cells are generally also toxic to normal cells. Moreover, cancer cells mutate rapidly and can develop resistance to chemical agents or radiation therapy. Besides, all types of cancer cells may not respond to the same therapy. Some cancer cells respond to one...
Antiepileptic Drugs: Potassium Channel Activators01:20

Antiepileptic Drugs: Potassium Channel Activators

Ezocgabine or retigabine, an antiepileptic drug of remarkable efficacy, has revolutionized the management of seizures. It is a potassium channel activator, explicitly targeting the family of Q subtype potassium channels. It enhances the transmembrane potassium currents, regulating neuronal excitability. This action stabilizes the resting membrane potential, a pivotal factor in mitigating the hyperexcitability that characterizes epilepsy.
Ezogabine has gained approval as an adjunctive treatment...
Targeted Cancer Therapies02:57

Targeted Cancer Therapies

The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against specific...
Nephrotic Syndrome I : Introduction01:24

Nephrotic Syndrome I : Introduction

Nephrotic Syndrome is a chronic kidney disorder defined by clinical findings such as severe proteinuria, hypoalbuminemia, hyperlipidemia, and edema. These symptoms result from damage to the glomeruli, the kidney’s filtering units, increasing their permeability to proteins.Definition and Meaning:Proteinuria, defined as the loss of more than 3.5 grams of protein per day in adults, is a crucial feature of nephrotic syndrome. This condition is often accompanied by edema, the accumulation of fluid...
Treatment Resistant Cancers02:56

Treatment Resistant Cancers

Cancer is the second leading cause of death in the United States. A cancer cell is genetically unstable and hence can mutate faster. They can also modify their microenvironment and escape immune surveillance. The difficulties in treating cancer are further compounded by the emergence of rapid resistance to anticancer drugs. The most common ways to attain resistance in cancer cells include alteration in drug transport and metabolism, modification of drug target, elevated DNA damage response, or...