Clonal Dynamics of GPI‐Deficient Cells in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH): A Retrospective Follow‐Up Analysis
- 1Department of Hematology and Stem Cell Transplantation, West German Cancer Center University Hospital Essen, University of Duisburg‐Essen Essen Germany
- 2Department of Hematology and Central Hematology Laboratory, Inselspital University Hospital Bern, University of Bern Bern Switzerland
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.
Trial Registration: 25‐12 634‐BO
Related
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- [Eculizumab resistance caused by C5 gene polymorphism in a patient with paroxysmal nocturnal hemoglobinuria: a case report].
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- Iptacopan combined with immunosuppressive therapy for aplastic anemia-paroxysmal nocturnal hemoglobinuria syndrome: A case report.
- FACIT-fatigue score and treatment quality assessment for paroxysmal nocturnal hemoglobinuria patients treated with Eculizumab in Türkiye
BibTeX
@article{frey2026clonal,
title = {{Clonal Dynamics of GPI‐Deficient Cells in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH): A Retrospective Follow‐Up Analysis}},
author = {Sandra M. Frey and Friederike Poppenborg and Ute Schmücker and Nicole Preising and H. Christian Reinhardt and Alexander Röth and Ferras Alashkar},
journal = {European Journal of Haematology},
year = {2026},
month = aug,
doi = {10.1111/ejh.70293},
url = {https://doi.org/10.1111/ejh.70293},
note = {CC BY 4.0},
}
RIS
TY - JOUR
TI - Clonal Dynamics of GPI‐Deficient Cells in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH): A Retrospective Follow‐Up Analysis
AU - Sandra M. Frey
AU - Friederike Poppenborg
AU - Ute Schmücker
AU - Nicole Preising
AU - H. Christian Reinhardt
AU - Alexander Röth
AU - Ferras Alashkar
PY - 2026
DA - 2026/08/21
JO - European Journal of Haematology
DO - 10.1111/ejh.70293
UR - https://doi.org/10.1111/ejh.70293
AB - 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. Trial Registration: 25‐12 634‐BO
SN - 0902-4441
SN - 1600-0609
ER -