AUTOINFLAMMATORY DISEASES IN HEMATOLOGY FROM DIAGNOSIS TO TREATMENT

AUTOINFLAMMATORY_DISEASES_IN_HEMATOLOGY_FROM_DIAGNOSIS_TO_TREATMENTkapak

ALLOGENEIC STEM CELL TRANSPLANTATION

Tuğrul Elverdi

İstanbul University, Cerrahpaşa Faculty of Medicine Department of Hematology, İstanbul, Türkiye

Elverdi T. Allogeneic Stem Cell Transplantation. In: Kurt Yüksel M, editor. Autoinflammatory Diseases in Hematology from Diagnosis to Treatment. 1st ed. Ankara: Türkiye Klinikleri; 2025. p.241-246.

ABSTRACT

VEXAS is a hemato-inflammatory syndrome caused by a somatic mutation in “UBA1”. Allogeneic transplantation is the only potential curative option for this syndrome; however, the available evidence is scarce, non-randomized, and retrospective. Here, we aim to collect and discuss the existing data. Further prospective trials with larger patient cohorts are needed to determine the optimal timing and indication for allogeneic transplantation in this rare syndrome.

Keywords: Hemato-inflammatory syndrome; Allogeneic stem cell transplantation; Auto-inflammation; Graft failure; Graft versus host disease

Referanslar

  1. Nakajima H, Kunimoto H. VEXAS syndrome. Int J Hematol. 2024. [Crossref]  [PubMed]
  2. Dias AL, Groarke EM, Hickstein D, Patel B. Role of allogeneic hematopoietic cell transplantation in VEXAS syndrome. Ann Hematol. 2024;103(11):4427-36. [Crossref]  [PubMed]  [PMC]
  3. Georgin-Lavialle S, Terrier B, Guedon AF, Heiblig M, Comont T, Lazaro E, et al. Further characterization of clinical and laboratory features in VEXAS syndrome: large-scale analysis of a multicentre case series of 116 French patients. Br J Dermatol. 2022;186(3):564-74. [Crossref]  [PubMed]
  4. Comont T, Heiblig M, Riviere E, Terriou L, Rossignol J, Bouscary D, et al. Azacitidine for patients with Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic syndrome (VEXAS) and myelodysplastic syndrome: data from the French VEXAS registry. Br J Haematol. 2022;196(4):969-74. [Crossref]  [PubMed]
  5. Ali SB, Gurnari C. Allogenic haematopoietic stem cell transplantation in VEXAS: A review of 33 patients. Clin Rheumatol. 2024;43(11):3565-75. [Crossref]  [PubMed]  [PMC]
  6. Mohty R, Reljic T, Abdel-Razeq N, Jamy O, Badar T, Kumar A, et al. Assessing the efficacy of allogeneic hematopoietic cell transplantation in VEXAS syndrome: results of a systematic review and meta-analysis. Bone Marrow Transplant. 2024;59(10):1423-7. [Crossref]  [PubMed]
  7. Gurnari C, Koster L, Baaij L, Heiblig M, Yakoub-Agha I, Collin M, et al. Allogeneic hematopoietic cell transplantation for VEXAS syndrome: results of a multicenter study of the EBMT. Blood Adv. 2024;8(6):1444-8. [Crossref]  [PubMed]  [PMC]
  8. Gutierrez-Rodrigues F, Kusne Y, Fernandez J, Lasho T, Shalhoub R, Ma X, et al. Spectrum of clonal hematopoiesis in VEXAS syndrome. Blood. 2023;142(3):244-59. [Crossref]  [PubMed]  [PMC]
  9. Mangaonkar AA, Langer KJ, Lasho TL, Finke C, Litzow MR, Hogan WJ, et al. Reduced intensity conditioning allogeneic hematopoietic stem cell transplantation in VEXAS syndrome: Data from a prospective series of patients. Am J Hematol. 2023;98(2):E28-E31. [Crossref]
  10. Diarra A, Duployez N, Fournier E, Preudhomme C, Coiteux V, Magro L, et al. Successful allogeneic hematopoietic stem cell transplantation in patients with VEXAS syndrome: a 2-center experience. Blood Adv. 2022;6(3):998-1003. [Crossref]  [PubMed]  [PMC]
  11. Al-Hakim A, Poulter JA, Mahmoud D, Rose AMS, Elcombe S, Lachmann H, et al. Allogeneic haematopoietic stem cell transplantation for VEXAS syndrome: UK experience. Br J Haematol. 2022;199(5):777-81. [Crossref]  [PubMed]
  12. Stiburkova B, Pavelcova K, Belickova M, Magaziner SJ, Collins JC, Werner A, et al. Novel Somatic UBA1 Variant in a Patient With VEXAS Syndrome. Arthritis Rheumatol. 2023;75(7):1285-90. [Crossref]  [PubMed]  [PMC]
  13. Mascaro JM, Rodriguez-Pinto I, Poza G, Mensa-Vilaro A, Fernandez-Martin J, Caminal-Montero L, et al. Spanish cohort of VEXAS syndrome: clinical manifestations, outcome of treatments and novel evidences about UBA1 mosaicism. Ann Rheum Dis. 2023;82(12):1594-605. [Crossref]  [PubMed]  [PMC]
  14. van Leeuwen-Kerkhoff N, de Witte MA, Heijstek MW, Leavis HL. Case report: Up-front allogeneic stem cell transplantation in a patient with the VEXAS syndrome. Br J Haematol. 2022;199(3):e12-e5. [Crossref]  [PubMed]
  15. Loschi M, Roux C, Sudaka I, Ferrero-Vacher C, Marceau-Renaut A, Duployez N, et al. Allogeneic stem cell transplantation as a curative therapeutic approach for VEXAS syndrome: a case report. Bone Marrow Transplant. 2022;57(2):315-8. [Crossref]  [PubMed]