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Background <p>Paroxysmal nocturnal hemoglobinuria (PNH) clones are detected in up to 60% of patients with aplastic anemia (AA); however, their prognostic impact remains incompletely defined, particularly in the context of frontline immunosuppressive therapy (IST) or allogeneic hematopoietic stem cell transplantation (HSCT).</p> Methods <p>We retrospectively analyzed 207 patients with AA treated between 2004 and 2024 at a single institution. PNH clones were identified at diagnosis in 64 patients (30.9%). Treatment modalities included IST (<i>n</i> = 104) and HSCT (<i>n</i> = 103). Clinical outcomes, including overall survival (OS), event-free survival, graft-versus-host disease (GVHD), relapse, and non-relapse mortality, were compared between the PNH-positive and PNH-negative cohorts.</p> Results <p>At 5 years, PNH-positive patients treated with IST had significantly improved OS compared with PNH-negative patients (100% vs. 72.4%, <i>p</i> = 0.004). In the HSCT group, OS was 100% in PNH-positive patients versus 90% in PNH-negative patients (<i>p</i> = 0.09). The incidence of chronic GVHD after HSCT was significantly lower in the PNH-positive group (4% vs. 27%, <i>p</i> = 0.01), whereas the rates of acute GVHD, graft failure, and relapse were comparable. Clone size (small vs. large) did not affect survival or GVHD outcomes.</p> Conclusions <p>The presence of a PNH clone in AA was associated with superior survival following IST and a lower incidence of chronic GVHD following HSCT. These findings suggest that PNH positivity may serve as a prognostic and immunomodulatory biomarker in AA and support its integration into therapeutic decision-making and risk stratification algorithms.</p>

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Prognostic significance of PNH clones in aplastic anemia treated with immunosuppression or allogeneic HSCT: a 20-year single-center experience

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年4月22日

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  • Alfadil Haroon,
  • Hazzaa Alzahrani,
  • Mostafa F. Mohammed Saleh,
  • Ali Aalahmari,
  • Shaykhah Alotaibi,
  • Tusneem Elhassan,
  • Feras Alfraih,
  • Fahad Alsharif,
  • Syed O. Ahmed,
  • Fahad Almohareb,
  • Riad El Fakih,
  • Mahmoud Aljurf

摘要

Background

Paroxysmal nocturnal hemoglobinuria (PNH) clones are detected in up to 60% of patients with aplastic anemia (AA); however, their prognostic impact remains incompletely defined, particularly in the context of frontline immunosuppressive therapy (IST) or allogeneic hematopoietic stem cell transplantation (HSCT).

Methods

We retrospectively analyzed 207 patients with AA treated between 2004 and 2024 at a single institution. PNH clones were identified at diagnosis in 64 patients (30.9%). Treatment modalities included IST (n = 104) and HSCT (n = 103). Clinical outcomes, including overall survival (OS), event-free survival, graft-versus-host disease (GVHD), relapse, and non-relapse mortality, were compared between the PNH-positive and PNH-negative cohorts.

Results

At 5 years, PNH-positive patients treated with IST had significantly improved OS compared with PNH-negative patients (100% vs. 72.4%, p = 0.004). In the HSCT group, OS was 100% in PNH-positive patients versus 90% in PNH-negative patients (p = 0.09). The incidence of chronic GVHD after HSCT was significantly lower in the PNH-positive group (4% vs. 27%, p = 0.01), whereas the rates of acute GVHD, graft failure, and relapse were comparable. Clone size (small vs. large) did not affect survival or GVHD outcomes.

Conclusions

The presence of a PNH clone in AA was associated with superior survival following IST and a lower incidence of chronic GVHD following HSCT. These findings suggest that PNH positivity may serve as a prognostic and immunomodulatory biomarker in AA and support its integration into therapeutic decision-making and risk stratification algorithms.