Abstract Number: VPB0313
Meeting: ISTH 2022 Congress
Theme: Platelet Disorders, von Willebrand Disease and Thrombotic Microangiopathies » ADAMTS13 and TTP
Background: As a rare disorder, congenital thrombotic thrombocytopenic purpura (cTTP) is often severe in childhood and requires preventive treatment to avoid long-term adverse prognosis.
Aims: The purpose of this study was to understand the relationship between cTTP treatment and prognosis in China through single-center data analysis, and to provide a basis for standardized diagnosis and treatment.
Methods: Clinical data of children diagnosed with cTTP in our center were retrospectively collected, including onset age, treatment mode, acute episodes, renal and nervous system prognosis.
Results: There were 7 children, including 1 male and 6 female, with onset age ranging from several hours after birth to 28 months. The median follow-up time was 5.2years (0.6-6.9)years,and the median follow-up was 8 times (4-35 times).All patients were treated with fresh frozen plasma for replacement therapy, and 2 patients received preventive therapy. The average annual frequency of acute episodes was 1.05 (0.7-1.4) times, and no cerebral infarction or renal dysfunction occurred.Five patients were treated on demand, with an average annual acute episodes of 1.7 (1-6) times, and 3 patients had recent episodes of urinary protein (+).In case 3, the disease was poorly controlled, with the frequency of acute episodes as high as 9 times a year, and proteinuria and hematuria gradually appeared. The creatinine clearance rate decreased by 84ml/min and blood pressure increased, which could be controlled within the normal range by oral antihypertensive drug therapy.The annual frequency of episodes in children older than 10 years was lower than in all children younger than 10 years (1.05 vs 3.32).The average number of episodes per year was lower with prophylactic treatment than with on-demand treatment (1.05 vs 3.32). There was no significant difference in the severity of the episodes, which were all in grade 1.
Conclusion(s): Prophylactic treatment is expected to reduce the number of acute episodes.
To cite this abstract in AMA style:
fu l, Ma J, Wu R, wei y. Treatment and prognosis of children with inherited TTP: a single-center retrospective clinical study [abstract]. https://abstracts.isth.org/abstract/treatment-and-prognosis-of-children-with-inherited-ttp-a-single-center-retrospective-clinical-study/. Accessed September 29, 2023.« Back to ISTH 2022 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/treatment-and-prognosis-of-children-with-inherited-ttp-a-single-center-retrospective-clinical-study/