Analysis of clinical characteristics of 92 patients with paroxysmal nocturnal hemoglobinuria: A single institution experience in China

Abstract

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.

Clinical observation of low‐dose combination chemotherapy in refractory/recurrent paroxysmal nocturnal hemoglobinuria patients: A single‐center retrospective analysis

“…Glucocorticoids are still effective drugs for reducing hemolysis to control PNH. After years of clinical progress, the current use of glucocorticoids and hematopoietic therapy has allowed the remission rate of the disease reach more than 60%, but nearly 40% of patients are not sensitive to this treatment 16,17 . Even in patients who achieve remission after treatment, there is still more than a 10% possibility of relapse 18 .…”

“…After years of clinical progress, the current use of glucocorticoids and hematopoietic therapy has allowed the remission rate of the disease reach more than 60%, but nearly 40% of patients are not sensitive to this treatment. 16 , 17 Even in patients who achieve remission after treatment, there is still more than a 10% possibility of relapse. 18 At the end of the 1970s, Soviet scholars reported that cyclophosphamide or 6‐mercaptopurine achieved certain effects in the treatment of PNH.…”

Posthepatectomy jaundice induced by paroxysmal nocturnal hemoglobinuria: A case report

“…In terms of treatment, complement inhibition therapy in the form of eculizumab has improved treatment prospects for PNH patients in developed countries[ 6 , 7 ]. However, high costs and access difficulties have limited the utilization of eculizumab in China, and steroid therapy is still the preferred treatment to control hemolytic attacks in PNH patients[ 8 ]. In this case, the patient's hyperbilirubinemia was relieved after the administration of methylprednisolone, and the treatment achieved relatively favorable outcomes.…”

Clinical observation of low-dose combination chemotherapy in refractory/recurrent paroxysmal nocturnal hemoglobinuria patients: a single-centre retrospective analysis

“…Glucocorticoids are still effective drugs for reducing hemolysis to control PNH. After years of clinical progress, the current use of glucocorticoids and hematopoietic therapy has allowed the remission rate of the disease reach more than 60%, but nearly 40% of patients are not sensitive to this treatment [16,17]. Even in patients who achieve remission after treatment, there is still more than a 10% possibility of relapse [18].…”