Contributions
Abstract: PB1829
Type: Publication Only
Background
Paroxysmal nocturnal hemoglobinuria is not well recognized in pediatric age group.
Aims
To Study the clinical profile and laboratory data of children with PNH positive clone.
Methods
We analyzed the hospital records for patients of classical paroxysmal nocturnal hemoglobinuria and aplastic anemia/hypocellular bone marrow patients in the pediatric age group (<18years age) from 2013 to 2017. The clinical and laboratory data of patients with PNH clones were collected from the hospital records. PNH clone was identified by flowcytometry.
Patients with classical PNH were treated with a combination of steroids with or without androgens and those with PNH/AA were treated with cyclosporine+/-androgens.
Results
Table 1:
Symptoms at diagnosis | Classical PNH n=10(%) | PNH/AA n=29(%) |
Pallor/Fatigue | 10(100.00) | 29(100.00) |
Jaundice | 5(50.00) | 2(6.89) |
Hemoglobinuria | 5(50.00) | 3(10.34) |
Bleeding (Petechiae/Purpura/wet bleeds) | 0 | 11(37.93) |
Seizures | 1(10.00) | 0 |
Fever | 5(50.00) | 14(48.27) |
Thrombosis | 1(10.00) | 0 |
Dysphagia/Chest pain/Abdominal Pain | 2(20.00) | 1(3.44) |
Table 2:
| Classical PNH | PNH/AA | Total |
Median age to presentation, years (range) | 16.0 (13.7-16.7) | 16.0(14.0-18.0) | 16.0(14.0-18.0) |
Median age to diagnosis, years (range) | 18.0(16.0-19.0) | 16.0(14.0-18.0) | 16.0(15.0-18.0) |
Duration of symptoms, months (range) | 30(24-36) | 3(2-6) | 5(2-12) |
Laboratory results |
|
|
|
Hb, g/dL | 5.8(4.6-8.2) | 5.4(4.5-7.3) | 5.8(4.5-7.5) |
WBC x 109/L | 4.1(3.2-5.6) | 3.1(2.5-3.6) | 3.2(2.6-3.7) |
ANC x 109/L | 1.6(0.8-3.0) | 0.5(0.3-0.7) | 0.6(0.4-1.1) |
Platelet x 109/L | 74.0(52.0-150.0) | 20.0(10.0-40.0) | 30.0(15.0-59.5) |
Reticulocyte x 109/L | 57.0(50.0-67.0) | 1.0(0.06-2.0) | 20.0(0.9-40.0) |
Total Bilirubin, mg/dl | 1.4(0.8-2.0) | 0.7(0.5-0.8) | 0.8(0.5-1.1) |
PNH clone size |
|
|
|
Granulocyte clone size, %, median (range) | 79.0(40.6-86.0) | 3.0(1.0-8.0) | 5.2(2.0-28.2) |
Monocyte clone size, %, median (range) | 91.5(86.3-97.5) | 6.0(3.2-14.4) | 10.8(3.8-64.6)
|
14 patients were in CR, 11 patients were in PR and rest did not have any response. Only one patient was able to undergo allogenic stem cell transplantation.
Conclusion
There is significant delay in the diagnosis of the classic form of PNH in children and more awareness is to be created regarding this disorder in children. Thrombotic events were also less common in our patient population.
Session topic: 12. Bone marrow failure syndromes incl. PNH - Clinical
Keyword(s): Pediatric, PNH
Abstract: PB1829
Type: Publication Only
Background
Paroxysmal nocturnal hemoglobinuria is not well recognized in pediatric age group.
Aims
To Study the clinical profile and laboratory data of children with PNH positive clone.
Methods
We analyzed the hospital records for patients of classical paroxysmal nocturnal hemoglobinuria and aplastic anemia/hypocellular bone marrow patients in the pediatric age group (<18years age) from 2013 to 2017. The clinical and laboratory data of patients with PNH clones were collected from the hospital records. PNH clone was identified by flowcytometry.
Patients with classical PNH were treated with a combination of steroids with or without androgens and those with PNH/AA were treated with cyclosporine+/-androgens.
Results
Table 1:
Symptoms at diagnosis | Classical PNH n=10(%) | PNH/AA n=29(%) |
Pallor/Fatigue | 10(100.00) | 29(100.00) |
Jaundice | 5(50.00) | 2(6.89) |
Hemoglobinuria | 5(50.00) | 3(10.34) |
Bleeding (Petechiae/Purpura/wet bleeds) | 0 | 11(37.93) |
Seizures | 1(10.00) | 0 |
Fever | 5(50.00) | 14(48.27) |
Thrombosis | 1(10.00) | 0 |
Dysphagia/Chest pain/Abdominal Pain | 2(20.00) | 1(3.44) |
Table 2:
| Classical PNH | PNH/AA | Total |
Median age to presentation, years (range) | 16.0 (13.7-16.7) | 16.0(14.0-18.0) | 16.0(14.0-18.0) |
Median age to diagnosis, years (range) | 18.0(16.0-19.0) | 16.0(14.0-18.0) | 16.0(15.0-18.0) |
Duration of symptoms, months (range) | 30(24-36) | 3(2-6) | 5(2-12) |
Laboratory results |
|
|
|
Hb, g/dL | 5.8(4.6-8.2) | 5.4(4.5-7.3) | 5.8(4.5-7.5) |
WBC x 109/L | 4.1(3.2-5.6) | 3.1(2.5-3.6) | 3.2(2.6-3.7) |
ANC x 109/L | 1.6(0.8-3.0) | 0.5(0.3-0.7) | 0.6(0.4-1.1) |
Platelet x 109/L | 74.0(52.0-150.0) | 20.0(10.0-40.0) | 30.0(15.0-59.5) |
Reticulocyte x 109/L | 57.0(50.0-67.0) | 1.0(0.06-2.0) | 20.0(0.9-40.0) |
Total Bilirubin, mg/dl | 1.4(0.8-2.0) | 0.7(0.5-0.8) | 0.8(0.5-1.1) |
PNH clone size |
|
|
|
Granulocyte clone size, %, median (range) | 79.0(40.6-86.0) | 3.0(1.0-8.0) | 5.2(2.0-28.2) |
Monocyte clone size, %, median (range) | 91.5(86.3-97.5) | 6.0(3.2-14.4) | 10.8(3.8-64.6)
|
14 patients were in CR, 11 patients were in PR and rest did not have any response. Only one patient was able to undergo allogenic stem cell transplantation.
Conclusion
There is significant delay in the diagnosis of the classic form of PNH in children and more awareness is to be created regarding this disorder in children. Thrombotic events were also less common in our patient population.
Session topic: 12. Bone marrow failure syndromes incl. PNH - Clinical
Keyword(s): Pediatric, PNH