A comparative analysis of clinical characteristics of patients with paroxysmal nocturnal hemoglobinuria between Asia and Europe/America

Abstract

To accurately analyze the clinical characteristics of paroxysmal nocturnal hemoglobinuria (PNH) in different ethnic backgrounds, we retrieved all retrospective studies on clinical characteristics of PNH with a median follow-up period >60 months published after 2000, analyzed the clinical characteristics of PNH patients in Asia and European/America, and statistically compared enumeration data in these studies. We included 1665 patients in this analysis. The proportion of female patients in Asia was significantly lower than that in Europe/America (P = 0.000). Incidence rates of hemoglobinuria and thromboembolism in Asia were significantly lower than in Europe/America (both P values were 0.000). Within the subgroups of patients with thromboembolism, Asian patients had a higher proportion of arterial thrombosis while Western patients had a higher proportion of abdominal venous thrombosis. Bone marrow failure was not clearly defined in most studies. The proportion of patients with pancytopenia was higher in China than in France (P = 0.048). The total death rates were similar in both ethnic groups (P > 0.05). In Europe/America the major cause of death was thromboembolism and in Asian countries was serious infections. Differences in population characteristics of PNH patients among different ethnic groups indicate the possibility of differential pathogenesis and may be informative for treatment decisions.

Eculizumab in paroxysmal nocturnal haemoglobinuria and atypical haemolytic uraemic syndrome: 10‐year pharmacovigilance analysis

“…In the only double‐blind, randomised, placebo‐controlled trial of eculizumab in 87 patients with PNH (Hillmen et al , ), serious nonmeningococcal infections were reported in similar proportions of eculizumab‐treated and untreated patients with PNH (2% vs. 6%, respectively). Outside of the clinical trial programme, severe infections have been reported in up to 18% (Nishimura et al , ) and were identified as the cause of death in 25–40% of mortalities reported in untreated patients with PNH (Jang et al , ; Yu et al , ). Infections have been identified as precipitating events in patients with aHUS (Noris et al , ; Schifferli et al , ; Geerdink et al , ; Fremeaux‐Bacchi et al , ; Campistol et al , ); it is important to note that immunosuppression may play a role in these infections because many aHUS patients receive dialysis or renal transplantation (Noris et al , ; Schifferli et al , ; Geerdink et al , ; Fremeaux‐Bacchi et al , ; Lok & Foley, ; Campistol et al , ).…”

“…However, some analyses comparing cohorts of treated and untreated patients showed no difference in cumulative incidence of clonal evolution to MDS or acute myeloid leukaemia (AML) (Loschi et al , ). In addition, in a systematic review of published studies in patients with PNH ( N = 1665), 5·1% progressed to MDS or AML, and malignancy was the cause of death in 11·9% (Yu et al , ). Similar to patients with PNH, patients with aHUS who undergo transplantation or who otherwise receive long‐term immunosuppressive treatment are at increased risk of malignancy, which is unlikely to be related to eculizumab (Villeneuve et al , ).…”

Clinical Manifestation and Errors in the Diagnosis of Classical Paroxysmal Nocturnal Hemoglobinuria:

“…По литературным данным, с учетом различий в критериях диагностики классическая форма составляет 26-71 и 18-62 % в европейских и азиатских когортах соответственно [5,[7][8][9][10]17]. В целом формы с выраженной цитопенией чаще выявляются в исследованиях из Азии [18].…”

Pregnancy with paroxysmal nocturnal hemoglobinuria: A case series with review of the literature

“…It can also mimic other hematological disorders such as aplastic anemia (AA) and myelodysplastic diseases (MDS), although PNH can have a combination of both AA and MDS. [ 2 3 ] According to Brando et al ,[ 4 ] classical signs and symptoms of PNH include hemoglobinuria or hemosiderinuria, unexplained direct antiglobulin tests, negative hemolysis, aplastic anemia, thrombosis at unusual sites and dystonic symptoms (abdominal pain or dysphagia). For confirmatory diagnosis, flow cytometry is used, as it can demonstrate the absence or deficiency in the expression of GPI-anchored protein in a sizable portion of peripheral blood, mainly in red blood cells (RBC), neutrophils and monocytes, called as 'PNH clones,' which are fundamentals in diagnosing PNH.…”