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Clinical observation of low-dose combination chemotherapy in refractory/recurrent paroxysmal nocturnal hemoglobinuria patients: a single-centre retrospective analysis
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  • Liyan Li,
  • Hui Liu,
  • Honglei Wang,
  • Zhaoyun Liu,
  • Yingying Chen,
  • Chunyan Liu,
  • Xiaoyu Zhao,
  • Lijuan Li,
  • Huaquan Wang,
  • Zonghong Shao,
  • Rong Fu
Honglei Wang
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Zhaoyun Liu
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Yingying Chen
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Chunyan Liu
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Xiaoyu Zhao
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Huaquan Wang
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Zonghong Shao
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Rong Fu
Tianjin Medical University General Hospita
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Abstract

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.