Contributions
Abstract: EP598
Type: E-Poster Presentation
Session title: Bone marrow failure syndromes incl. PNH - Clinical
Background
The characters update of patients with paroxysmal nocturnal hemoglobinuria (PNH) in China has not been well addressed. We retrospectively reviewed patients with complete data who visited Peking Union Medical College Hospital from 2009 to 2018 and had been followed up for more than 2 years.
Aims
To update the characters of patients with PNH in China.
Methods
Retrospectively analyzed 512 patients diagnosed with PNH in the past ten years from a single center in China.
Results
There were 512 patients, including 56.3% males and 43.8% females. The median age at disease onset was 33 (9~80) years. Classic PNH, bone marrow failure (BMF)/PNH and subclinical PNH accounted for 52.3%, 46.3% and 1. 6% of the patients, respectively. Most of the patients were at 21~40 years (50.6%) and 41~60 years (25.4%). The most common symptoms and positive lab findings were hemolytic and BMF related. Most of the patients had >50% of Flare- granulocytes. PNH clone size correlated with hematoglubinuria (R2=0.038, P=0.001), erectile dysfunction (R2=0.03, P=0.019), reticulocyte (R2=0.164, P<0.01) and LDH level (R2=0.268, P<0.001). Thrombosis events (TEs) occurred in 18.0% of patients. 88.8% were venous and 11.2% were arterial events. 77.2% of the patients had single-site thrombosis with the abdominal vein (44.8%) the most common site. 22.8% of patients experienced repeated or multiple-site thrombosis. Renal deficiency happened in 16.9% of patients. Patients with classic PNH had longer disease duration, bigger PNH clone size, higher LDH, TBil, DBil, AST, Scr, and blood cell count, lower ferritin compared with those with BMF/PNH. Although the rate of thrombosis was similar for patients with classic PNH (17.3%) and BMF/PNH (19.1%, P=0.66), patients with larger PNH clone and BMF/PNH had higher chance of renal impairment (P<0.05). Immunosuppressive agents, glucocorticoids were the most common treatments and only 39.1% of patients with thrombosis received anticoagulants. The chance of pregnancy was rare and the abortions were high for women in need of fertility. More evolution from BMF/PNH into classic PNH (26.2%) and less from classic PNH into AA/PNH (2.2%) and even less into malignancies were observed at a median of 93.5 (24~144) months follow-up. The major causes of deaths were thrombosis (29.6%), hemorrhage (18.5%) and infections (18.5%). Thrombosis (HR, 3.462; P =0.013) and renal deficiency (HR, 2.105; P =0.032) were the independent risk factors for overall survival (OS).
Conclusion
Although most PNH patients survived without complement inhibitors, more hemolytic features of PNH in China might be greatly improved with complement inhibitors in the future.
Keyword(s):
Abstract: EP598
Type: E-Poster Presentation
Session title: Bone marrow failure syndromes incl. PNH - Clinical
Background
The characters update of patients with paroxysmal nocturnal hemoglobinuria (PNH) in China has not been well addressed. We retrospectively reviewed patients with complete data who visited Peking Union Medical College Hospital from 2009 to 2018 and had been followed up for more than 2 years.
Aims
To update the characters of patients with PNH in China.
Methods
Retrospectively analyzed 512 patients diagnosed with PNH in the past ten years from a single center in China.
Results
There were 512 patients, including 56.3% males and 43.8% females. The median age at disease onset was 33 (9~80) years. Classic PNH, bone marrow failure (BMF)/PNH and subclinical PNH accounted for 52.3%, 46.3% and 1. 6% of the patients, respectively. Most of the patients were at 21~40 years (50.6%) and 41~60 years (25.4%). The most common symptoms and positive lab findings were hemolytic and BMF related. Most of the patients had >50% of Flare- granulocytes. PNH clone size correlated with hematoglubinuria (R2=0.038, P=0.001), erectile dysfunction (R2=0.03, P=0.019), reticulocyte (R2=0.164, P<0.01) and LDH level (R2=0.268, P<0.001). Thrombosis events (TEs) occurred in 18.0% of patients. 88.8% were venous and 11.2% were arterial events. 77.2% of the patients had single-site thrombosis with the abdominal vein (44.8%) the most common site. 22.8% of patients experienced repeated or multiple-site thrombosis. Renal deficiency happened in 16.9% of patients. Patients with classic PNH had longer disease duration, bigger PNH clone size, higher LDH, TBil, DBil, AST, Scr, and blood cell count, lower ferritin compared with those with BMF/PNH. Although the rate of thrombosis was similar for patients with classic PNH (17.3%) and BMF/PNH (19.1%, P=0.66), patients with larger PNH clone and BMF/PNH had higher chance of renal impairment (P<0.05). Immunosuppressive agents, glucocorticoids were the most common treatments and only 39.1% of patients with thrombosis received anticoagulants. The chance of pregnancy was rare and the abortions were high for women in need of fertility. More evolution from BMF/PNH into classic PNH (26.2%) and less from classic PNH into AA/PNH (2.2%) and even less into malignancies were observed at a median of 93.5 (24~144) months follow-up. The major causes of deaths were thrombosis (29.6%), hemorrhage (18.5%) and infections (18.5%). Thrombosis (HR, 3.462; P =0.013) and renal deficiency (HR, 2.105; P =0.032) were the independent risk factors for overall survival (OS).
Conclusion
Although most PNH patients survived without complement inhibitors, more hemolytic features of PNH in China might be greatly improved with complement inhibitors in the future.
Keyword(s):