Objectives
We performed a retrospective analysis to investigate the clinical characteristics and therapeutic strategies of Chinese paroxysmal nocturnal hemoglobinuria (PNH) patients, and assessed the efficacy and safety of glucocorticoid in PNH patients.
Methods
The clinical data of 92 PNH cases in our hospital were analyzed, including clinical manifestation, laboratory examination, treatment efficacy, and survival.
Results
The main clinical manifestations of these patients included hemoglobinuria, anemia, fatigue, dyspnea, headache, abdominal pain, and erectile dysfunction. Glucocorticoid is still the first‐line treatment for PNH patients to control hemolytic attack, and the short‐term remission rate (12 months) is 79.01% (64/81). Meanwhile, the overall survival (OS) of 10 years after diagnosis was estimated as 70.77% (46/65). Moreover, Cox proportional risk model for multivariate analysis showed that the increase in LDH multiple, thrombosis complications, and complicated with bone marrow failure were the independent adverse prognostic factors affecting the survival of PNH patients.
Conclusion
Paroxysmal nocturnal hemoglobinuria patients in mainland China have various clinical features, while lower incidences of thrombosis and renal damage. Thrombosis and bone marrow failure are two complications with worse prognosis.
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.
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