Clinical characteristics predict response to antithymocyte globulin in paroxysmal nocturnal haemoglobinuria

Ronald L. Paquette, Ryan Yoshimura, Charles Veiseh, Lori Kunkel, James Gajewski, Peter J. Rosen

    Research output: Contribution to journalArticle

    37 Scopus citations

    Abstract

    Seven patients with paroxysmal nocturnal haemoglobinuria (PNH) were treated with antithymocyte globulin (ATG). Each patient received ATG (20 mg/kg/d) for 8 d and prednisone to prevent or control serum sickness. Three patients experienced a sustained improvement in at least one peripheral blood cytopenia, including one patient who had a complete trilineage response. Several pretreatment clinical features appeared to be associated with response to ATG. All responding patients had hypoproliferative features including depressed platelet counts (<30 x 109/l), and a minor degree of chronic haemolysis as indicated by relatively low reticulocyte counts (<100 x 109/l), lactate dehydrogenase (<1000 U/l) and total bilirubin (<17 μmol/l) levels. Responding patients continued to have chronic low-grade haemolysis after their response to immunosuppression that was similar to that observed prior to treatment. The non-responding patients had a classic haemolytic form of PNH characterized by elevated reticulocyte counts (>100 x 109/l), lactate dehydrogenase (>2000 U/l) and total bilirubin (>17 μmol/l) levels. The impaired haemopoiesis that occurs in hypoproliferative PNH may respond to ATG treatment, but the haemolytic component of the disease, and hence the PNH clone, is not altered by immunosuppressive therapy.

    Original languageEnglish (US)
    Pages (from-to)92-97
    Number of pages6
    JournalBritish Journal of Haematology
    Volume96
    Issue number1
    DOIs
    StatePublished - Feb 5 1997

    Keywords

    • antithymocyte globulin
    • aplastic anaemia
    • immunosuppressive therapy
    • paroxysmal nocturnal haemoglobinuria

    ASJC Scopus subject areas

    • Hematology

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