Leukemic transformation of polycythemia vera

BACKGROUND: Acute leukemia (AL) may occur as rare and late event of polycythemia vera (PV). METHODS: The current study included 23 patients who developed acute leukemia in a cohort of 414 consecutive PV patients with long-term observation (3208 person years of follow-up). Kaplan-Meier Product-Limit method was used to estimate the cumulative probability of survival; Gehan-Wilcoxon test was applied to compare survival in different groups of patients. RESULTS: Median age was 68 years, and 18 patients (78%) were > 60 years of age. At diagnosis of AL, most patients had a white blood count > 10 x 10(9)/L (n = 17; 74%), Hgb 50 x 10(9)/L (n = 17; 74%). Of 14 patients in whom cytogenetic analysis was available at leukemic transformation, 12 showed high-risk abnormalities including complex karyotype (n = 10), del (7)(q22) sole (n = 1) and del (X)(q26) sole (n = 1), whereas 2 had a normal karyotype. In patients whose karyotype was available at diagnosis of PV, cytogenetic evolution was documented at progression to AL. Treatment consisted of supportive care and/or low-dose chemotherapy (n = 15), or induction chemotherapy (n = 8). This included idarubicin plus cytarabine (n = 3), high-dose cytarabine (n = 4), and fludarabine-based regimen (n = 1). Allogenic stem cell transplantation was offered to a single patient, who is alive at Day + 70. The outcome of patients was poor, with a median survival of 2.9 months (range, 0.6-20.1 mos), with no significant differences between palliation and intensive treatments. CONCLUSIONS: AL following PV has distinct clinical and biologic features. Outcome of patients is poor irrespective of the treatment employed.