{
  "abstract": "This retrospective, single‐center study aimed to characterize clonal dynamics of GPI‐deficient cells in patients with paroxysmal nocturnal hemoglobinuria (PNH) or PNH/aplastic anemia (AA) syndrome using multiparameter flow cytometry including FLAER. Among 108 patients diagnosed and treated at our center, 83 had longitudinal monitoring of GPI‐deficient neutrophils; 53 of these also had data on monocytes and lymphocytes. Median observation time was 29.4 months (range 1.1–162.7 months). Clone size in neutrophils showed a strong correlation with monocytes, but not with lymphocytes. Clonal persistence represented the most frequent individual pattern of clone evolution observed during follow‐up; clonal persistence was observed in 32/83 patients, while 24 patients exhibited clonal expansion (≥ 10%) and 13 showed regression. Expansion frequently involved all three lineages, suggesting ongoing biological mechanisms contributing to clonal selection. Regression was more likely in patients with small initial clones. In six patients undergoing allogeneic stem cell transplantation, complete eradication of PNH clones was achieved. Clonal dynamics were independent of eculizumab treatment; however, regression was observed in some patients treated with ATG and cyclosporine A. These findings support the need for longitudinal clone size monitoring, particularly in neutrophils and monocytes. Further studies are warranted to elucidate factors influencing clone expansion and remission.\n\nTrial Registration: 25‐12 634‐BO",
  "abstract_shown": true,
  "affiliations": [
    "Department of Hematology and Stem Cell Transplantation, West German Cancer Center University Hospital Essen, University of Duisburg‐Essen Essen Germany",
    "Department of Hematology and Central Hematology Laboratory, Inselspital University Hospital Bern, University of Bern Bern Switzerland"
  ],
  "article_type": "research article",
  "arxiv": null,
  "author_total": 7,
  "authors": [
    {
      "affs": [
        0,
        1
      ],
      "name": "Sandra M. Frey",
      "orcid": "0000-0002-8725-6484"
    },
    {
      "affs": [
        0
      ],
      "name": "Friederike Poppenborg"
    },
    {
      "affs": [
        0
      ],
      "name": "Ute Schmücker"
    },
    {
      "affs": [
        0
      ],
      "name": "Nicole Preising"
    },
    {
      "affs": [
        0
      ],
      "name": "H. Christian Reinhardt",
      "orcid": "0000-0001-5706-9349"
    },
    {
      "affs": [
        0
      ],
      "name": "Alexander Röth",
      "orcid": "0000-0003-4414-7699"
    },
    {
      "affs": [
        0
      ],
      "name": "Ferras Alashkar",
      "orcid": "0000-0001-7530-8892"
    }
  ],
  "bibtex": "https://research.draconitic.com/paper/0arj3vjd38k2t.bib",
  "cited_by": 0,
  "doi": "10.1111/ejh.70293",
  "fulltext_url": "https://pmc.ncbi.nlm.nih.gov/articles/PMC13729962/",
  "id": "0arj3vjd38k2t",
  "issns": [
    "0902-4441",
    "1600-0609"
  ],
  "landing_url": "https://doi.org/10.1111/ejh.70293",
  "language": "en",
  "licence": "CC-BY-4.0",
  "licence_label": "CC BY 4.0",
  "licence_source": "article",
  "licence_url": "https://creativecommons.org/licenses/by/4.0/",
  "metadata_licence": null,
  "native_subjects": [
    [
      "heading",
      "Original Article"
    ],
    [
      "openalex",
      "2403|Complement system in diseases"
    ]
  ],
  "openalex": "W7203941801",
  "pdf_url": "https://onlinelibrary.wiley.com/doi/pdfdirect/10.1111/ejh.70293",
  "pmcid": "PMC13729962",
  "pmid": "42625551",
  "published": "2026-08-21",
  "publisher": null,
  "ris": "https://research.draconitic.com/paper/0arj3vjd38k2t.ris",
  "sources": [
    {
      "first_seen": "2026-11-07T12:22:40Z",
      "native_id": "PMC13729962",
      "source": "pmc",
      "url": "https://doi.org/10.1111/ejh.70293"
    }
  ],
  "subjects": [
    {
      "field": "biology",
      "origin": "openalex:2403|Complement system in diseases",
      "subfield": "immunology"
    }
  ],
  "title": "Clonal Dynamics of GPI‐Deficient Cells in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH): A Retrospective Follow‐Up Analysis",
  "title_text": "Clonal Dynamics of GPI‐Deficient Cells in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH): A Retrospective Follow‐Up Analysis",
  "type": "peer-reviewed",
  "updated_at": "2026-11-07T12:22:40Z",
  "url": "https://research.draconitic.com/paper/0arj3vjd38k2t",
  "venue": "European Journal of Haematology",
  "versions": []
}