[Efficacy of Low Dose Combined Chemotherapy for Patients with Relapsed and Refractory Aplastic Anemia-Paroxysmal Nocturnal Hemoglobinuria Syndrome].

Objective: To evaluate the clinical efficacy of low dose combined chemotherapy（LDCC） for patients with relapsed and refractory aplastic anemia-paroxysmal nocturnal hemoglobinuria（AA-PNH） syndrome, and to analyze the advantages of LDCC in the treatment of AA-PNH syndrome.
Methods: The clinical characteristics and the curative effect of LDCC in 9 patients with relapsed and refractory AA-PNH syndrome were retrospectively analyzed. Five patients were treated with MP therapy［melphalan 2 mg/(m ² ·d); prednisone 0.5 mg/(kg·d)］, and the other 4 patients were treated with HA therapy(HHT 2 mg/d iv drip, for 5 days; Ara-C 100 mg/d iv drip, for 5 days). The changes of PNH clone, dosage of corticosteroid, hemolysis and the relapse of disease, hematological parameters and adverse reactions were compared before and after therapy. All patients were treated for 1-2 courses.
Results: Seven out of 9 patients responded well, the dosage of corticosteroid and the bilirubin concentration decreased significantly and anemia was relieved in 7 patients (P<0.05). One patient relapsed in one year. PNH clone of 3 patients turned negative. Five patients did not rely on blood transfusion in 1 year. There was no bone marrow failure to be found in all patients.
Conclusion: The LDCC has better efficacy and safety in the treatment of patients with AA-PNH syndrome, moreover, the patients is more tolerant to LDCC, thus the LDCC may be a selection for treatment of patients with relapsed and refractory AA-PNH syndrome.