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Evaluation of treatment responses and colony-forming progenitor cells in 50 patients with aplastic anemia after immunosuppressive therapy or hematopoietic stem cell transplantation: a single-center experience

Therapieerfolg und Vorläuferzellzahl bei 50 Patienten mit Aplastischer Anämie nach immunsuppressiver Therapie oder Stammzelltransplantation: Erfahrungen aus einem Schwerpunkt-Zentrum

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Summary

We analyzed the clinical course and outcome in 50 patients (27 males, 23 females) suffering from aplastic anemia (AA), treated in our department between 1987 and 2007. The median age was 37 years (range: 14–70 years). A total of 42 patients received antithymocyte globulin and cyclosporine A (CSA). Seven patients were transplanted using a matched sibling donor upfront, and one patient was treated with CSA and growth factors only. A total of 34 patients (68 %) achieved a complete remission, and 7 (14 %), a partial remission. Eight patients (16 %) did not respond to treatment, and one died shortly after transplantation. Relapses of AA occurred in eight patients (20 %). No obvious correlations between clinical parameters, including age, karyotype, existence of paroxysmal nocturnal hemoglobinuria clones, pretreatment blood counts, progenitor cell counts, and the response to immunosuppressive therapy (IST), were found. We also examined the numbers of colony-forming progenitor cells (CFUs) before and after therapy. In most responding patients, CFU numbers increased substantially after successful therapy. However, even in patients without a substantial increase in CFU, stable remissions were observed. Together, both IST and stem cell transplantation are reasonable treatment options for patients with AA.

Zusammenfassung

In der vorliegenden Studie wurden die Daten von 50 Patienten (27 Männer/23 Frauen) mit Aplastischer Anämie (AA), die von 1987–2007 an unserer Klink behandelt wurden, analysiert. Das mediane Alter betrug 37 Jahre (14–70 Jahre). Zweiundvierzig Patienten erhielten Antithymozytenglobulin/ CyclosporinA (CSA) und 7 wurden primär mit einem Geschwisterspender transplantiert. Ein Patient erhielt nur CSA und Wachstumsfaktoren. Vierunddreißig Patienten (68 %) erreichten eine komplette Remission und 7 (14 %) eine partielle Remission. Acht Patienten sprachen nicht auf die Therapie an und einer verstarb kurz nach der Transplantation. Rezidive traten bei 8 Patienten (20 %) auf. Es konnten keine offensichtlichen Korrelationen zwischen klinischen Parametern wie Alter, Karyotyp, Vorhandensein eines paroxysmal-nächtliche Hämoglobinurie (PNH) – Klons, Blutbilder vor Therapie, Vorläuferzellzahl und dem Ansprechen auf die immunsuppressive Therapie gefunden werden. Wir untersuchten auch die Vorläuferzellzahl vor und nach Therapie. In den meisten Patienten stieg die Vorläuferzellzahl nach erfolgreicher Therapie deutlich an. Allerdings erreichten sogar manche Patienten ohne einen substantiellen Anstieg der Vorläuferzellzahl stabile Remissionen. Insgesamt sind sowohl die immunsuppressive Therapie als auch die Stammzelltransplantation wirksame Therapieoptionen bei Patienten mit Aplastischer Anämie.

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Authors and Affiliations

  1. Division of Hematology & Hemostaseology, Department of Internal Medicine I, AKH, Medical University of Vienna, Währinger Gürtel 18–20, 1090, Vienna, Austria

    Wolfgang Füreder, Andrea Paulitsch-Buckingham, Wolfgang R. Sperr & Peter Valent

  2. Bone Marrow Transplantation Unit, Department of Internal Medicine I, AKH, Medical University of Vienna, Währinger Gürtel 18–20, 1090, Vienna, Austria

    Werner Rabitsch

  3. Clinical Institute of Medical & Chemical Laboratory Diagnostics, Department of Internal Medicine I, AKH, Medical University of Vienna, Währinger Gürtel 18–20, 1090, Vienna, Austria

    Eva Jäger & Ilse Schwarzinger

Authors
  1. Wolfgang Füreder
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  2. Andrea Paulitsch-Buckingham
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  3. Werner Rabitsch
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  4. Eva Jäger
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  5. Ilse Schwarzinger
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  6. Wolfgang R. Sperr
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  7. Peter Valent
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Correspondence to Wolfgang Füreder.

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Füreder, W., Paulitsch-Buckingham, A., Rabitsch, W. et al. Evaluation of treatment responses and colony-forming progenitor cells in 50 patients with aplastic anemia after immunosuppressive therapy or hematopoietic stem cell transplantation: a single-center experience. Wien Klin Wochenschr 126, 119–125 (2014). https://doi.org/10.1007/s00508-013-0484-2

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