Pre-Transplant Immune Dysregulation Predicts for Poor Outcome Following Allogeneic Haematopoietic Stem Cell Transplantation in Adolescents and Adults with Inborn Errors of Immunity (IEI)

Journal of Clinical Immunology, Journal Year: 2025, Volume and Issue: 45(1)

Published: Jan. 6, 2025

Allogeneic haematopoietic stem cell transplantation (alloHSCT) is safe and effective for adolescents adults with inborn errors of immunity (IEI) severe disease manifestations their disease. The comorbidity index (HCT-CI) score predicts transplant survival in non-malignant diseases, including IEIs. We hypothesised that immune dysregulation pre-transplant may also influence outcomes. calculated the activity (IDDA v2.1) 82 adolescent adult IEI patients (aged ≥ 13 years). Three-year overall (OS) whole cohort was 90% (n = 82) a median follow up 44.7 months (range 8.4 to 225.8). Events were defined as acute graft-versus-host (GvHD) grades II or above, chronic GvHD any grade, graft failure, death from cause. event free (EFS) 72%. In multivariable analysis IDDA v2.1 HCT-CI significantly impacted OS (hazard ratio 1.08, p 0.028) EFS 1.04, 0.0005). Importantly, 35% this had high (≥ 15) low (< 3) suggesting risks alloHSCT be underestimated proportion if used alone. These findings support potential improved outcomes following successful modulation pre-transplant. has utility an objective measurement providing additional information reagrding complications individual patient.

Language: Английский

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The causal effect of autoimmune diseases on myelodysplastic syndrome:a Mendelian randomization study

Research Square (Research Square), Journal Year: 2024, Volume and Issue: unknown

Published: June 19, 2024

Background The relationship between different types of autoimmune diseases and myelodysplastic syndrome (MDS) are inconclusive. Therefore,we employed Mendelian randomization (MR) to explore the causal associations MDS. Methods Single nucleotide polymorphisms (SNPs) significantly associated with 10 were extracted from summary statistics European genome-wide association studies (GWAS). A two-sample MR analysis was performed using summary-level sourced GWAS datasets. Inverse-variance weighting (IVW),MR‒Egger,and weighted median (WM) further supported by several sensitivity analyses. Results Four showed genetical predisposition MDS: rheumatoid arthritis(OR = 1.186,95%CI 1.028–1.367, P 0.019), multiple sclerosis(OR 1.247,95%CI 1.013–1.534, 0.037), myasthenia gravis(OR 1.326,95%CI 1.010–1.742, 0.042), hashimoto thyroiditis(OR 1.519,95%CI 1.008–2.290, 0.046).Nevertheless,no similar found remaining seven MDS.The accuracy robustness these findings confirmed tests. Conclusions We first use relationships mechanism this link needs be explored.

Language: Английский

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Hematopoietic Stem Cell Transplantation in the Management of Myelodysplastic Syndrome: A Retrospective, Current, and Future Perspective

Cell Transplantation, Journal Year: 2024, Volume and Issue: 33

Published: Jan. 1, 2024

Myelodysplastic syndrome (MDS) is a clonal disorder that affects hematopoietic stem cells (HSCs), primarily occurring in the elderly population. Lower-risk MDS characterized by decrease blood cells, whereas higher-risk associated with an increased risk of transformation to acute myeloid leukemia (AML). Currently, treatment still unsatisfactory, although demethylating agents, azacitidine (AZA), and decitabine (Dec) have been successfully used treat improve survival rates. However, cell transplantation (HSCT) remains only curative for patients, effectively increasing patient quality life. Nevertheless, treatment-related toxicity, graft-versus-host disease, infectious complications, relapse are major post-transplant issues. In this review, through retrospective analysis past present HSCT MDS, we provide insights future.

Language: Английский

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Association between autoimmune diseases and myelodysplastic syndrome:a Mendelian randomization study

Hematology, Journal Year: 2024, Volume and Issue: 29(1)

Published: Nov. 27, 2024

Background: The relationship between different types of autoimmune diseases and myelodysplastic syndrome (MDS) is inconclusive. Therefore, we employed Mendelian randomization (MR) to examine whether genetically predicted susceptibility ten associated with the risk MDS.

Language: Английский

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