Abstract Number: PB1455
Meeting: ISTH 2020 Congress
Theme: Platelet Disorders and von Willebrand Disease » Platelet Antagonists and Novel Therapeutics
Background: To address the question whether it will be possible to apply the same HDD application as the first-line treatment of children ITP.
Aims: To evaluate results of HDD replacing full-dose prednisone as first-line treatment for newly diagnosed children primary immune thrombocytopenia (ITP).
Methods: A prospective randomized study. The cohort were randomized allocated to “HDD group” and “PDN group”. The response rate of platelet count in two short-term points and two long-term points, the bleeding score with the improving rate and Glucocorticoid related adverse effects in 1 month point after treatment were collected.
Results: 211 children were enrolled, allocated to HDD group (n = 110) and PDN group (n = 101).
(1) Platelet count response rate:
① The one-week point: Total response (TR) (92.7% vs 93%, P=1.000). Complete response (CR) (67.3% vs 78.2%, P=0.001) and Partial response (PR) (25.5% vs 14.9%, P=0.028).
②The one-month point: TR (93.6% vs 96.0%, P=0.542), CR (76.3% vs 82.2%, P=0.398) and PR (16.4% vs 13.8%, P=0.702)
③The response rate in 6m point and the 12m point without differences.
(2) Although fewer bleeding events in the HDD group during 1 month treatment (10% vs 15%), without different (P=0.105). Patients who couldn’t reach to CR at 1 month that does not recover at 6 months was similar in two groups (39.3% vs 50%, P=0.532), also at 12months (36% vs 38.9%, P=0.462)
(3) Glucocorticoid related adverse effects: high blood pressure, high intraocular pressure, abdominal discomfort, mood changes occurred in two group without differences (16.4% vs 14.9%, P=0.850;4.5% vs 1% P=0.449; 13.6% vs 19.8%，P=0.267, 5.5% vs 12.9%，P=0.266).Cushing face and infection in the PDN group were higher than HDD group (80% vs 10%, P=0.001, 26% vs 11.8%, P=0.012), with statistically differences.
Conclusions: With the same efficacy and less Glucocorticoid related side-effects, HDD could be the better first-line treatment for Children ITP comparing to traditional PND.
To cite this abstract in AMA style:
Fu L, Ma J, Ma J, Gu H, Chen Z, Wu R. High-Dose Dexamethasone Effectively Replaces Traditional Full-Dose Prednisone as the First-Line Treatment of Children Immune Thrombocytopenia: A Prospective Randomized Single-Center Study [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/high-dose-dexamethasone-effectively-replaces-traditional-full-dose-prednisone-as-the-first-line-treatment-of-children-immune-thrombocytopenia-a-prospective-randomized-single-center-study/. Accessed September 29, 2023.« Back to ISTH 2020 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/high-dose-dexamethasone-effectively-replaces-traditional-full-dose-prednisone-as-the-first-line-treatment-of-children-immune-thrombocytopenia-a-prospective-randomized-single-center-study/