CLINICAL CHARACTERISTICS OF CHINESE PAROXYSMAL NOCTURNAL HEMOGLOBINURIA DIAGNOSED BY FLAER
(Abstract release date: 05/19/16)
EHA Library. He G. 06/09/16; 134665; PB1765
Dr. Guangsheng He
Contributions
Contributions
Abstract
Abstract: PB1765
Type: Publication Only
Background
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare bone marrow failure disorder caused by the absence of glycosylphosphatidylinositol (GPI) on the membranes of blood cells. It was claimed that FLAER gave a more accurate assessment of the GPI anchor deficit in PNH .It can detect as few as 1% PNH monocytes and neutrophils in aplastic anemia, that were otherwise undetectable using CD55 and CD59 on RBC’s. Therefore, the clinical characteristics of PNH patients diagnosised by FLAER method might be different from previous CD55/CD59 and Ham test used before in China.
Aims
To find the clinical characteristics of Chinese paroxysmal nocturnal hemoglobinuria diagnosed by FLAER.
Methods
The clinical data of 98 cases diagnosed by FLAER methods from September 2011 to March 2014 were analyzed retrospectively, including demography, clinical features, laboratory examination results and complications.
Results
48 were male and 50 were female, median age 30.5 years (1~74 years), the median follow-up time were 24 months (12 ~30 months). 11 patients with PNH less than 18 years of age belong to adolescents, 6 patients of them were male, median age was 13 years (1~16 years). There were 43 cases of classic PNH (43.9%), 45 of PNH combined with other specific bone marrow disorders (45.9%), 10 cases of subclinical PNH (10.2%). The clinical features (Table 1) showed many patients with bone marrow failures. Fatigue (71.4%), hemoglobinuria (41.8%), headache/dizziness (38.8%), bleeding (22.4%), and dyspnea (19.4%) were commonly encountered symptoms. Thrombosis in 9 cases (9.18%), 3 cases of deep vein thrombosis, 1 case of pulmonary thromboembolism, 2 cases of mesenteric venous thrombosis, and 2 cases of hepatic portal thrombosis happened in group of classic PNH. Only one case of hepatic portal thrombosis was found in PNH with aplastic anemia. 17 cases cooccurrented with renal impairment respectively, there were 14 patients (14.5%), 16 patients (16.3%) and 1 patient (1%) at stage of CKD1, CKD2, and CKD3 respectively. Only 2 cases of PNH were suffered with pulmonary hypertension. The estimated OS at 3 years after diagnosis as 82.0%, 11 patients who died during follow-up period. Comparison of OS among the three subcategories was no statistical significance(P=0.978). Univariate analysis revealed that thrombocytopenia (P=0.048),abdominal pain(P<0.001), thrombotic events (P<0.001),and recurrent infections(P<.001) were of risk factors affecting survival. Multivariate analysis further confirmed that thrombotic events and recurrent infections remained to be statistically significant risk factors affecting survival (Table 2).Table 1 Clinical features of patients with PNH
PNH with another specified bone marrow disorder PNH SBMD
Conclusion
Bone marrow failure is frequently found in the Chinese Patients with PNH. Renal, liver impairment and thrombosis in rare sites was relatively common. But pulmonary arterial hypertension happened seldom. Thrombotic events and recurrent infections influenced survival of patients with PNH.
Session topic: E-poster
Keyword(s): Infection, Paroxysmal nocturnal hemoglobinuria (PNH), Survival, Thrombosis
Type: Publication Only
Background
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare bone marrow failure disorder caused by the absence of glycosylphosphatidylinositol (GPI) on the membranes of blood cells. It was claimed that FLAER gave a more accurate assessment of the GPI anchor deficit in PNH .It can detect as few as 1% PNH monocytes and neutrophils in aplastic anemia, that were otherwise undetectable using CD55 and CD59 on RBC’s. Therefore, the clinical characteristics of PNH patients diagnosised by FLAER method might be different from previous CD55/CD59 and Ham test used before in China.
Aims
To find the clinical characteristics of Chinese paroxysmal nocturnal hemoglobinuria diagnosed by FLAER.
Methods
The clinical data of 98 cases diagnosed by FLAER methods from September 2011 to March 2014 were analyzed retrospectively, including demography, clinical features, laboratory examination results and complications.
Results
48 were male and 50 were female, median age 30.5 years (1~74 years), the median follow-up time were 24 months (12 ~30 months). 11 patients with PNH less than 18 years of age belong to adolescents, 6 patients of them were male, median age was 13 years (1~16 years). There were 43 cases of classic PNH (43.9%), 45 of PNH combined with other specific bone marrow disorders (45.9%), 10 cases of subclinical PNH (10.2%). The clinical features (Table 1) showed many patients with bone marrow failures. Fatigue (71.4%), hemoglobinuria (41.8%), headache/dizziness (38.8%), bleeding (22.4%), and dyspnea (19.4%) were commonly encountered symptoms. Thrombosis in 9 cases (9.18%), 3 cases of deep vein thrombosis, 1 case of pulmonary thromboembolism, 2 cases of mesenteric venous thrombosis, and 2 cases of hepatic portal thrombosis happened in group of classic PNH. Only one case of hepatic portal thrombosis was found in PNH with aplastic anemia. 17 cases cooccurrented with renal impairment respectively, there were 14 patients (14.5%), 16 patients (16.3%) and 1 patient (1%) at stage of CKD1, CKD2, and CKD3 respectively. Only 2 cases of PNH were suffered with pulmonary hypertension. The estimated OS at 3 years after diagnosis as 82.0%, 11 patients who died during follow-up period. Comparison of OS among the three subcategories was no statistical significance(P=0.978). Univariate analysis revealed that thrombocytopenia (P=0.048),abdominal pain(P<0.001), thrombotic events (P<0.001),and recurrent infections(P<.001) were of risk factors affecting survival. Multivariate analysis further confirmed that thrombotic events and recurrent infections remained to be statistically significant risk factors affecting survival (Table 2).Table 1 Clinical features of patients with PNH
| Characteristic | Classic PNH | PNH SBMD | Subclinical PNH | Total | ||
| Hemoglobinuria | 30(69.8%) | 11(24.4%) | 0 (0%) | 41 (41.8%) | ||
| Abdominal pain | 4 (9.3%) | 2 (4.4%) | 0 (0%) | 6 (6.1%) | ||
| Dysphagia | 4 (9.3%) | 0 (0%) | 0 (0%) | 4 (4.1%) | ||
| Headache/ dizziness | 13(30.2%) | 25(55.6%) | 0 (0%) | 38 (38.8%) | ||
| Pectoralgia | 0(0%) | 1(2.2%) | 0(0%) | 1 (1.0%) | ||
| Back pain | 3(7.0%) | 0(0%) | 0 (0%) | 3 (3.1%) | ||
| Dyspnoea | 9(20.9%) | 10(22.2%) | 0(0%) | 19 (19.4%) | ||
| Bleeding | 2 (4.7%) | 19(42.2%) | 1 (10.0%) | 22(22.4%) | ||
| Fatigue | 30 (69.8%) | 34 (75.6%) | 6 (60.0%) | 70 (71.4%) | ||
| Infections | 0 (0%) | 9(20%) | 1(10%) | 10 (10.2%) | ||
Conclusion
Bone marrow failure is frequently found in the Chinese Patients with PNH. Renal, liver impairment and thrombosis in rare sites was relatively common. But pulmonary arterial hypertension happened seldom. Thrombotic events and recurrent infections influenced survival of patients with PNH.
Session topic: E-poster
Keyword(s): Infection, Paroxysmal nocturnal hemoglobinuria (PNH), Survival, Thrombosis
Abstract: PB1765
Type: Publication Only
Background
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare bone marrow failure disorder caused by the absence of glycosylphosphatidylinositol (GPI) on the membranes of blood cells. It was claimed that FLAER gave a more accurate assessment of the GPI anchor deficit in PNH .It can detect as few as 1% PNH monocytes and neutrophils in aplastic anemia, that were otherwise undetectable using CD55 and CD59 on RBC’s. Therefore, the clinical characteristics of PNH patients diagnosised by FLAER method might be different from previous CD55/CD59 and Ham test used before in China.
Aims
To find the clinical characteristics of Chinese paroxysmal nocturnal hemoglobinuria diagnosed by FLAER.
Methods
The clinical data of 98 cases diagnosed by FLAER methods from September 2011 to March 2014 were analyzed retrospectively, including demography, clinical features, laboratory examination results and complications.
Results
48 were male and 50 were female, median age 30.5 years (1~74 years), the median follow-up time were 24 months (12 ~30 months). 11 patients with PNH less than 18 years of age belong to adolescents, 6 patients of them were male, median age was 13 years (1~16 years). There were 43 cases of classic PNH (43.9%), 45 of PNH combined with other specific bone marrow disorders (45.9%), 10 cases of subclinical PNH (10.2%). The clinical features (Table 1) showed many patients with bone marrow failures. Fatigue (71.4%), hemoglobinuria (41.8%), headache/dizziness (38.8%), bleeding (22.4%), and dyspnea (19.4%) were commonly encountered symptoms. Thrombosis in 9 cases (9.18%), 3 cases of deep vein thrombosis, 1 case of pulmonary thromboembolism, 2 cases of mesenteric venous thrombosis, and 2 cases of hepatic portal thrombosis happened in group of classic PNH. Only one case of hepatic portal thrombosis was found in PNH with aplastic anemia. 17 cases cooccurrented with renal impairment respectively, there were 14 patients (14.5%), 16 patients (16.3%) and 1 patient (1%) at stage of CKD1, CKD2, and CKD3 respectively. Only 2 cases of PNH were suffered with pulmonary hypertension. The estimated OS at 3 years after diagnosis as 82.0%, 11 patients who died during follow-up period. Comparison of OS among the three subcategories was no statistical significance(P=0.978). Univariate analysis revealed that thrombocytopenia (P=0.048),abdominal pain(P<0.001), thrombotic events (P<0.001),and recurrent infections(P<.001) were of risk factors affecting survival. Multivariate analysis further confirmed that thrombotic events and recurrent infections remained to be statistically significant risk factors affecting survival (Table 2).Table 1 Clinical features of patients with PNH
PNH with another specified bone marrow disorder PNH SBMD
Conclusion
Bone marrow failure is frequently found in the Chinese Patients with PNH. Renal, liver impairment and thrombosis in rare sites was relatively common. But pulmonary arterial hypertension happened seldom. Thrombotic events and recurrent infections influenced survival of patients with PNH.
Session topic: E-poster
Keyword(s): Infection, Paroxysmal nocturnal hemoglobinuria (PNH), Survival, Thrombosis
Type: Publication Only
Background
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare bone marrow failure disorder caused by the absence of glycosylphosphatidylinositol (GPI) on the membranes of blood cells. It was claimed that FLAER gave a more accurate assessment of the GPI anchor deficit in PNH .It can detect as few as 1% PNH monocytes and neutrophils in aplastic anemia, that were otherwise undetectable using CD55 and CD59 on RBC’s. Therefore, the clinical characteristics of PNH patients diagnosised by FLAER method might be different from previous CD55/CD59 and Ham test used before in China.
Aims
To find the clinical characteristics of Chinese paroxysmal nocturnal hemoglobinuria diagnosed by FLAER.
Methods
The clinical data of 98 cases diagnosed by FLAER methods from September 2011 to March 2014 were analyzed retrospectively, including demography, clinical features, laboratory examination results and complications.
Results
48 were male and 50 were female, median age 30.5 years (1~74 years), the median follow-up time were 24 months (12 ~30 months). 11 patients with PNH less than 18 years of age belong to adolescents, 6 patients of them were male, median age was 13 years (1~16 years). There were 43 cases of classic PNH (43.9%), 45 of PNH combined with other specific bone marrow disorders (45.9%), 10 cases of subclinical PNH (10.2%). The clinical features (Table 1) showed many patients with bone marrow failures. Fatigue (71.4%), hemoglobinuria (41.8%), headache/dizziness (38.8%), bleeding (22.4%), and dyspnea (19.4%) were commonly encountered symptoms. Thrombosis in 9 cases (9.18%), 3 cases of deep vein thrombosis, 1 case of pulmonary thromboembolism, 2 cases of mesenteric venous thrombosis, and 2 cases of hepatic portal thrombosis happened in group of classic PNH. Only one case of hepatic portal thrombosis was found in PNH with aplastic anemia. 17 cases cooccurrented with renal impairment respectively, there were 14 patients (14.5%), 16 patients (16.3%) and 1 patient (1%) at stage of CKD1, CKD2, and CKD3 respectively. Only 2 cases of PNH were suffered with pulmonary hypertension. The estimated OS at 3 years after diagnosis as 82.0%, 11 patients who died during follow-up period. Comparison of OS among the three subcategories was no statistical significance(P=0.978). Univariate analysis revealed that thrombocytopenia (P=0.048),abdominal pain(P<0.001), thrombotic events (P<0.001),and recurrent infections(P<.001) were of risk factors affecting survival. Multivariate analysis further confirmed that thrombotic events and recurrent infections remained to be statistically significant risk factors affecting survival (Table 2).Table 1 Clinical features of patients with PNH
| Characteristic | Classic PNH | PNH SBMD | Subclinical PNH | Total | ||
| Hemoglobinuria | 30(69.8%) | 11(24.4%) | 0 (0%) | 41 (41.8%) | ||
| Abdominal pain | 4 (9.3%) | 2 (4.4%) | 0 (0%) | 6 (6.1%) | ||
| Dysphagia | 4 (9.3%) | 0 (0%) | 0 (0%) | 4 (4.1%) | ||
| Headache/ dizziness | 13(30.2%) | 25(55.6%) | 0 (0%) | 38 (38.8%) | ||
| Pectoralgia | 0(0%) | 1(2.2%) | 0(0%) | 1 (1.0%) | ||
| Back pain | 3(7.0%) | 0(0%) | 0 (0%) | 3 (3.1%) | ||
| Dyspnoea | 9(20.9%) | 10(22.2%) | 0(0%) | 19 (19.4%) | ||
| Bleeding | 2 (4.7%) | 19(42.2%) | 1 (10.0%) | 22(22.4%) | ||
| Fatigue | 30 (69.8%) | 34 (75.6%) | 6 (60.0%) | 70 (71.4%) | ||
| Infections | 0 (0%) | 9(20%) | 1(10%) | 10 (10.2%) | ||
Conclusion
Bone marrow failure is frequently found in the Chinese Patients with PNH. Renal, liver impairment and thrombosis in rare sites was relatively common. But pulmonary arterial hypertension happened seldom. Thrombotic events and recurrent infections influenced survival of patients with PNH.
Session topic: E-poster
Keyword(s): Infection, Paroxysmal nocturnal hemoglobinuria (PNH), Survival, Thrombosis
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