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Subject: search.filters.subject.hematopoietic stem cell transplantation

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    Hematopoietic Stem Cell Transplantation in Patients with Heterozygous STAT1 Gain-of-Function Mutation
    (SPRINGER/PLENUM PUBLISHERS, 2019-01-01) Kiykim, Ayca; Charbonnier, Louis Marie; Akcay, Arzu; Karakoc-Aydiner, Elif; Ozen, Ahmet; Ozturk, Gulyuz; Chatila, Talal A.; Baris, Safa
    PurposeHuman signal transducer and activator of transcription 1 (STAT1) gain-of-function (GOF) mutations present with a broad range of manifestations ranging from chronic mucocutaneous candidiasis and autoimmunity to combined immunodeficiency (CID). So far, there is very limited experience with hematopoietic stem cell transplantation (HSCT) as a therapeutic modality in this disorder. Here, we describe two patients with heterozygous STAT1 GOF mutations mimicking CID who were treated with HSCT.MethodsData on the HSC sources, conditioning regimen, graft-versus-host disease (GvHD) and antimicrobial prophylaxis, and the post-transplant course including engraftment, GvHD, transplant-related complications, infections, chimerism, and survival were evaluated. Pre- and post-transplant immunological studies included enumeration of circulating interferon gamma (IFN-)- and interleukin 17 (IL-17)-expressing CD4(+) T cells and analysis of IFN--induced STAT1 phosphorylation in patient 1 (P1)'s T cells.ResultsP1 was transplanted with cord blood from an HLA-identical sibling, and P2 with bone marrow from a fully matched unrelated donor using a reduced toxicity conditioning regimen. While P1 completely recovered from her disease, P2 suffered from systemic CMV disease and secondary graft failure and died due to severe pulmonary involvement and hemorrhage. The dysregulated IFN- production, suppressed IL-17 response, and enhanced STAT1 phosphorylation previously found in the CD4(+) T cells of P1 were normalized following transplantation.ConclusionHSCT could be an alternative and curative therapeutic option for selected STAT1 GOF mutant patients with progressive life-threatening disease unresponsive to conventional therapy. Morbidity and mortality-causing complications included secondary graft failure, infections, and bleeding.
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    GUT MICROBIOTA EFFECTS IN HEMATOPOIETIC STEM CELL TRANSPLANT PATIENTS
    (ISTANBUL UNIV, FAC MEDICINE, PUBL OFF, 2022-01-01) Gurer, Ekin Ece; Oguz, Fatma S. A. V. R. A. N.; Kalayoglu, Sevgi BEsIsIK.; Aktas, Zerrin; Gulbas, Zafer; Oncul, Mustafa Oral; Sezerman, Ugur
    Objective: In our study, we analyzed gut microbiota in allo-HSCT patients and aimed to evaluate the relationship of gut microbio-ta with transplant complications, mainly GVHD. Materials and Methods: A total of 25 adult recipients and do-nors who underwent allo-HSCT at Istanbul Anadolu Medical Center were included in the study. Stool samples were collected twice, before chemotherapy regimen and after allo-HSCT. Sam-ples were analyzed by High Melting (HRM) Analysis and Next Generation Sequencing (NGS) methods after nucleic acid iso-lation. Sequencing was done with Illumina MiSeq. Bacteria Silva database was used for taxonomic classification and QIIME 2 pro-grams were used for analysis. Statistical analyses were carried out with the R statistical programming language. Results: Twenty-five allo-HKHN recipients were included in the study. The mean age was 46.24 +/- 14.86 years in recipients and 43.40 +/- 13.20 years in donors. Gender distribution was M/F: 15/10 in patients and M/F: 17/8 in donors. Recipient and donor sib-ling HLA match was 10/10. The rate of GVHD associated with Allo-HSCT was 16\%, and the relapse rate was 16\%. It was ob-served that the Firmicutes and Proteobacteria phyla changed significantly before and after transplantation. The number of Entereccocus species was found to be higher in patients who developed GVHD and died. The loss of diversity was found to be statistically significant in the pre-transplant and post-engraft-ment samples of the patients. Conclusion: Gut microbiota diversity may guide the monitoring of GVHD and also may be manipulated for the treatment of GVHD. It is thought that increasing the diversity of commensal bacteria can also positively affect the prognosis of the disease.