Trials · Topics · Bleeding Disorders
Bleeding Disorders clinical trials
27 Bleeding Disorders trials (Classical Hematology) summarized for fellows and board review, newest first within each disease. Each summary covers design, results, toxicity and what changed in practice.
Jump to: Hemophilia · ITP · CLD-related thrombocytopenia · TTP · HIT
Hemophilia (10)
- FRONTIER4 trial Blood Adv 2026Every-2-weeks denecimig maintained near-zero bleeding (ABR 0.38; 84% with zero treated bleeds) over 26 weeks with no new safety signals, supporting flexible-interval...
- BASIS Blood 2025Weekly SC marstacimab substantially reduced ABR vs on-demand factor therapy and was superior to prior routine prophylaxis in severe hemophilia A or B without...
- ATLAS-A/B Lancet Haematol 2023Once-monthly fitusiran prophylaxis reduced ABR by ~90% compared to on-demand factor therapy in hemophilia A and B without inhibitors, with median ABR reaching 0.0...
- HOPE-B NEJM 2023A single infusion of etranacogene dezaparvovec reduced ABR by 64% and enabled 96% of patients to discontinue FIX prophylaxis at 18 months, the first approved gene...
- GENEr8-1 NEJM 2022A single infusion of valoctocogene roxaparvovec reduced ABR by 84.5% and allowed 85% of patients to discontinue FVIII prophylaxis at 2 years, establishing the first...
- HAVEN 2 trial, emicizumab, with inhibitor ≤12y/o Blood 2019In children with inhibitor-positive hemophilia A, emicizumab prophylaxis reduced bleeding by 99% vs prior bypassing-agent therapy, with no thrombotic events. Cost: 60...
- HAVEN-4 Lancet Haematol 2019Once-monthly emicizumab (6 mg/kg q4w) effectively reduced bleeding in hemophilia A with or without inhibitors, with 56% achieving zero treated bleeds, supporting q4w...
- HAVEN 3 trial, emicizumab, no inhibitor ≥12y/o NEJM 2018Emicizumab prophylaxis reduced bleeding by >95% vs no prophylaxis and was superior to prior FVIII prophylaxis in non-inhibitor hemophilia A. Cost: 60 mg/0.4 mL vial...
- HAVEN 1 trial, emicizumab, with inhibitor ≥12y/o NEJM 2017Emicizumab prophylaxis reduced bleeding by 87% vs no prophylaxis in inhibitor-positive hemophilia A. Concomitant aPCC drives thrombotic/TMA risk. Cost: 60 mg/0.4 mL...
- Joint Outcome Study NEJM 2007In boys with severe hemophilia A, prophylactic FVIII started before joint damage prevented joint destruction and reduced joint and other hemorrhages vs episodic...
ITP (10)
- ATRA + Eltrombopag ITP trial NEJM Evid 2026A 12-week course of ATRA added to eltrombopag nearly doubled 18-month sustained responses in steroid-resistant/relapsed ITP, with longer response duration and no...
- MEZAGITAMAB IN ITP NEJM 2026Mezagitamab 600 mg achieved platelet responses in 91% of patients vs 23% with placebo in persistent/chronic ITP, with an acceptable safety profile similar to placebo....
- PINES JAMA 2025Eltrombopag achieved significantly higher sustained platelet responses compared to standard first-line therapies in newly diagnosed pediatric ITP requiring treatment...
- FIT Am J Hematol 2018Fostamatinib achieved stable platelet response in 18% of heavily pretreated chronic ITP patients including those who failed TPO-RAs, establishing SYK inhibition as a...
- EXTEND Blood 2017Long-term eltrombopag maintains platelet counts ≥50×10⁷/L and reduces bleeding in most patients with persistent/chronic ITP, with a stable safety profile over years...
- PETIT2 Lancet 2015Romiplostim achieved durable platelet responses in 52% of pediatric patients with chronic ITP versus 10% on placebo, with significant reduction in rescue therapy...
- CHAMPION Lancet 2009Eltrombopag significantly increased platelet counts to ≥50×10⁹/L and reduced bleeding versus placebo in chronic ITP patients who had failed ≥1 prior therapy, with a...
- RAISE Lancet 2011Eltrombopag maintained platelet counts ≥50×10⁹/L in 59% of treatment weeks versus 16% on placebo, with significant reduction in bleeding symptoms, establishing...
- Romiplostim ITP Lancet 2008Romiplostim substantially increased durable platelet response rates compared to placebo in both splenectomized and non-splenectomized chronic ITP patients...
- Cheng 2003 (HD-Dex ITP) NEJM 2003A 4-day course of oral high-dose dexamethasone (40 mg/day) achieves initial platelet response in 85% of adults with newly diagnosed ITP and sustained response at 6 mo...
CLD-related thrombocytopenia (3)
- L-PLUS 2 Hepatology 2019Lusutrombopag 3 mg × ≤7 days significantly reduced platelet transfusion requirement before invasive procedures in CLD patients, with no portal vein thrombosis signal....
- ADAPT-1/2 Gastroenterology 2018Avatrombopag significantly increased platelet counts and reduced transfusion need pre-procedure in CLD patients across two replicate RCTs, with no signal of portal...
- ELEVATE NEJM 2012Eltrombopag significantly increased platelet counts and reduced transfusion need in CLD patients pre-procedure; however, it was stopped early due to a 4-fold excess...
TTP (3)
- HERCULES NEJM 2019Caplacizumab significantly reduced TTP recurrence and thromboembolic events and shortened time to platelet response in aTTP, establishing it as the first targeted...
- TITAN trial NEJM 2016Added to plasma exchange and immunosuppression, caplacizumab shortened time to platelet normalization and reduced relapse during treatment. Bleeding is the principal...
- Plasma Exchange for TTP NEJM 1991Plasma exchange was superior to plasma infusion for response and survival in TTP, establishing daily PE as the cornerstone of initial TTP therapy and the standard of...
HIT (1)
- Argatroban HIT (ARG-911) Circulation 2001Argatroban significantly reduced the composite of death, amputation, or new thrombosis in HIT patients vs historical controls, with faster platelet recovery...