Objective: To observe the clinical effects of low-dose thalidomide (THAL) and prednisone (PRED) with or without danazol (DANA) in patients with primary myelofibrosis (PMF) associated anemia.
Methods: A cohort of 58 PMF patients with anemia (Hb<100 g/L) were retrospectively studied. Of them, 28 patients were treated with THAL and PRED (THAL-PRED group), and the rest with THAL, PRED and DANA (THAL-PRED-DANA group). The hematological response was assessed according to the modified criteria of the International Working Group in 2006, and the myelofibrosis degree was evaluated at 3 and 12 month after treatment.
Results: The total response rate was 56.9%(33/58) including 1.7% (1/58) partial remission (PR) and 55.2% (32/58) clinical improvement (CI). There was no statistical difference in the response rate between THAL-PRED and THAL-PREDDANA groups (50.0% vs 63.3%, P=0.306). However, the median response duration of clinical improvement, erythroid response (CI-E) and total response prolonged in THAL-PRED-DANA than THALPRED group (61.5w vs 22w, P=0.015; 75w vs 30w, P=0.007, respectively). Myelofibrosis degree at 3 and 12 months after treatment decreased significantly than before treatment (P=0.000 and 0.005, respectively). Side-effects in both groups were only grade 1-2.
Conclusion: Low-dose THAL together with PRED appeared to be effective in the treatment of PMF-associated anemia, and the response duration would prolong significantly if combined with DANA.