In einer Phase I/II Studie wurde die tolerable Dosierung und antileukämische Wirksamkeit der Kombination von AMSA und Etoposid (VP 16-213) bei 20 Patienten mit refraktärer akuter myeloischer Leukämie untersucht. Dabeí erwies sich ein 5tägiger Therapiezyklus als tolerabel und wirksam: AMSA 210 mg/m2/die an den Tagen 2, 3, 4 und Etoposid an den Tagen 1 und 5 in fraktionierter Applikation von 100 mg/m2 über 1 h, gefolgt von einer 23stündigen Dauerinfusion von 230 mg/m2. 5 von 20 Patienten erreichten eine partielle Remission; 4 dieser Patienten waren primär resistent gegen zwei TAD Induktionskurse. 7 der 8 Patienten mit primärer TAD Refrakterität kamen in eine Knochenmarkaplasie ohne residuale Blastenpopulation. AMSA/Etoposid scheint daher eine antileukämische Effektivität ohne Kreuzresistenz gegen TAD zu besitzen.

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