Fig 1
Fig 2
Evolution of PNH population before and after treatment with eculizumab
| PNH population | 27/10/20 | 16/3/21 | 23/11/21 |
|---|---|---|---|
| Granulocyte | 82.8% | 85.3% | 85.5% |
| Monocyte | 78.6% | 83.6% | 82.7% |
| Erythrocyte | 55.2% | 57.4% | 55.4% |
The oscillation of biochemical parameter after eculizumab treatment
| Date | TBIL (μmol/l) | AST (μkat/l) | LD (μkat/l) | ERY (1012/L) | RET (1012/L) |
|---|---|---|---|---|---|
| 12/01/21 | 84.6 | 0.44 | 4.76 | 4.45 | 369 |
| 04/03/21 | 67.9 | 2.36 | 24.98 | 4.09 | 185.7 |
| 16/03/21 | 61.2 | 0.52 | 4.64 | 4.45 | 239.2 |
| 06/07/21 | 54.2 | 0.81 | 7.66 | 3.8 | 363.5 |
| 16/09/21 | 90.9 | 0.48 | 3.79 | 4.55 | 232.9 |
| 09/12/21 | 79.1 | 1.96 | 13.93 | 4.87 | 194.3 |
Laboratory result during attack of haemolysis
| Laboratory result | |
|---|---|
| Haemoglobin | 130 (g/L) |
| MCV | 98.9 (fL) |
| Reticulocyte absolute count | 225.9 (1012/L) |
| Reticulocyte relative count | 5.7 (%) |
| Ferritin | 275.30 (μg/l) |
| Lactate dehydrogenase | 68 (μkat/L) |
| Haptoglobin | 0.08 (g/l) |
| Direct bilirubin | 81.1 (μmol/l) |
| Indirect bilirubin | 9.5 (μmol/l) |
Indication for screening for paroxysmal nocturnal hemoglobinuria
|
1. Chronic Coombs negative intravascular haemolysis with LD elevated above 1.5 UNL, decreased haptoglobin or kidney injury |
|
2. Thrombosis in atypical location, or thrombosis in a young patient without other explanation and sings of haemolysis |
|
3. Dysphagia, abdominal pain, erective dysfunction and sing of haemolysis |
|
4. Signs of haemolysis and iron deficiency |
|
5. Patient with aplastic anaemia or myelodysplastic syndrome |
|
6. Idiopathic cytopenia without other explanation |
Differential diagnosis of paroxysmal nocturnal haemoglobinuria
| 1. Haemolytic anaemia |
| 1.1. Autoimmune haemolytic anaemia |
| 1.2. Drugs induce haemolytic anaemia |
| 1.3. Intoxication |
| 1.4. Enzymopathies |
| 1.4.1. G6PD deficiency |
| 1.4.2. Pyruvate kinase deficiency |
| 1.5. Hemoglobinopathies |
| 1.5.1. Sickle cell anaemia |
| 1.5.2. Thalassemia |
| 1.6. Membranopaties |
| 1.6.1. Spherocytosis |
| 1.6.2. Elliptocytoses |
| 1.7. Infections (malaria, HIV) |
| 1.8. Microangiopathies |
| 1.8.1. TTP |
| 1.8.2. HUS |
| 1.8.3. HELLP |
| 1.8.4. Uremic syndrome |
| 1.8.5. DIC |
| 1.8.6. Drug induced macroangiopathic haemolytic anaemia |
| 1.9. Systemic diseases |
| 1.9.1. Systemic Lupus erythematosus |
| 1.9.2. Vasculitis |
| 2. Thromboembolic events with atypical localized thrombus |
| 3. Bone marrow diseases |
| 3.1. Myelodysplastic syndrome |
| 3.2. Aplastic anaemia |
Frequency of symptoms in patients with proximal nocturnal hemoglobinuria
| Symptoms | Frequency |
|---|---|
| Fatigue | 80% |
| Dyspnoea | 64% |
| Headache | 63% |
| Hemoglobinuria | 62% |
| Icterus | 50% |
| Abdominal pain | 50% |
| Erective dysfunction | 38% |
| Chest pain | 33% |
| Dysphagia | 25% |