paroxysmal nocturnal hemoglobinuria
Conditions
Interventions
Self control group:Glucocorticoid
Sponsors
Jiangsu Province Hospital
Eligibility
Sex/Gender
All
Age
18 Years to 75 Years
Inclusion criteria
Inclusion criteria: 1. All patients were diagnosed with paroxysmal nocturnal hemoglobinuria by flow cytometry FLAER. Bone marrow smear and bone marrow biopsy were performed to exclude aplastic anemia and myelodysplastic syndrome. The peripheral blood cell count was consistent with that of red blood cell and hemoglobin lower than normal, the absolute value of reticulocyte could be increased or decreased, the white blood cell and platelet could be normal or decreased, and the biochemical indirect bilirubin and lactate dehydrogenase of peripheral blood increased. 2. Aged 18 to 75 years; 3. ECoG physical condition score <= 2; 4. Before entering the trial and during the whole trial, female subjects received effective contraceptive methods (such as oral contraceptive, injection contraceptive, intrauterine device, contraceptive patch, male partner sterilization); 5. Willing to abide by the prohibition and restriction of this test protocol; 6. The subjects must sign the informed consent, indicating that they understand the purpose, steps and contents of the trial and voluntarily participate in the trial.
Exclusion criteria
Exclusion criteria: 1. Have blood diseases other than this disease, such as autoimmune hemolytic anemia or immune thrombocytopenia; 2. There are other clonal hematopoiesis, such as MDS, AML, etc.; 3. There are other serious diseases, such as serious damage to important organ function: respiratory failure, heart failure, liver dysfunction in the decompensated period, renal insufficiency, etc.; 4. Those who are known to be HIV seropositive; 5. There are mental diseases or other diseases, and cannot fully cooperate with the requirements of trial treatment or follow-up; 6. Have a history of severe allergy to glucocorticoids or biological products (including antibiotics); 7. Pregnant or lactating women; 8. Other serious acute or chronic physiological or mental diseases, or abnormal laboratory examination, may affect the giving of the study program and the judgment and interpretation of the results of the study by the researchers. Such patients are not suitable for participating in this clinical trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Hemoglobin level;Frequency of hemoglobinuria;Transfusion dependence; | — |
Secondary
| Measure | Time frame |
|---|---|
| lactate dehydrogenase;Indirect bilirubin;D-Dimer;PNH clone;hospitalization data ;the quality of life; | — |
Countries
China
Contacts
Public ContactHe Guangsheng
Jiangsu Province Hospital
Outcome results
None listed