Clinical observation of low-dose combination chemotherapy in
refractory/recurrent paroxysmal nocturnal hemoglobinuria patients: a
single-centre retrospective analysis
Objective We performed a retrospective analysis to investigate the
clinical characteristics and therapeutic strategies of 20
refractory/recurrent PNH patients, including the clinical efficacy of
chemotherapy treatment and survival. Main Measures The clinical data of
20 classic PNH patients that were refractory/recurrent or had
glucocorticoid dependence in our hospital were analyzed, including
clinical manifestations, laboratory examinations, treatment efficacy and
survival. Key Results Seventeen patients had a marked improvement in
anemia after chemotherapy, 14 patients acquired blood transfusion
independence, and the Hb of 3 patients increased to normal levels.
Although 6 patients still needed blood transfusion, the transfusion
interval was significantly prolonged. The percentages of LDH, TBIL and
RET, which are indicators of hemolysis, were significantly lower than
those before chemotherapy. The dosage of adrenal glucocorticoids was
reduced by more than half compared with that before chemotherapy.
Conclusions Chemotherapy can reduce PNH clones, promote normal
hematopoiesis, and control hemolytic attack. It is a promising and
widely used therapeutic method.