Multicenter randomized controlled trial on Duration of Therapy for Thrombosis in Children and Young Adults (the Kids‐DOTT trial): pilot/feasibility phase findings. (11th August 2015)

• Record Type: Journal Article
• Authors: Goldenberg, N. A.; Abshire, T.; Blatchford, P. J.; Fenton, L. Z.; Halperin, J. L.; Hiatt, W. R.; Kessler, C. M.; Kittelson, J. M.; Manco‐Johnson, M. J.; Spyropoulos, A. C.; Steg, P. G.; Stence, N. V.; Turpie, A. G. G.; Schulman, S.; the Kids‐DOTT Trial Investigators; Punzalan, R.; Wang, M.; Jaffray, J.; Young, G.; Rajpurkar, M.; Carpenter, S.; Diab, Y.; Verdun, N.; Tarango, C.; Acharya, S.; Torres, M.; Kucine, N.; Mitchell, B.; Shah, N.; Thornburg, C.; Takemoto, C.; Kulkarni, R.; O'Brien, S.; Haley, K.; Recht, M.; Knoll, C.; Thornburg, C.; Geddis, A.; Ahuja, S.; Simpson, M.; Srivaths, L.; Journeycake, J.; Zia, A.;
• Is Part Of: Journal of thrombosis and haemostasis. Volume 13:Number 9(2015:Sep.)
• Page Start: 1597
• Page End: 1605
• Publication Date: 2015-08-11
• Subjects: Thrombosis -- Periodicals; Hemostasis -- Periodicals; Blood coagulation disorders -- Periodicals; 616.1
• DOI: 10.1111/jth.13038
• Languages: English
• ISSNs: 1538-7933

Abstract

<abstract abstract-type="main" id="jth13038-abs-0001"> <title>Summary</title> <sec id="jth13038-sec-0001" sec-type="section"> <title>Background</title> <p>Randomized controlled trials (RCTs) on pediatric venous thromboembolism (VTE) treatment have been challenged by unsubstantiated design assumptions and/or poor accrual. Pilot/feasibility (P/F) studies are critical to future RCT success.</p> </sec> <sec id="jth13038-sec-0002" sec-type="section"> <title>Methods</title> <p>The Kids‐DOTT trial is a multicenter RCT investigating non‐inferiority of a 6‐week (shortened) versus 3‐month (conventional) duration of anticoagulation in patients aged < 21 years with provoked venous thrombosis. Primary efficacy and safety endpoints are symptomatic recurrent VTE at 1 year and anticoagulant‐related, clinically relevant bleeding. In the P/F phase, 100 participants were enrolled in an open, blinded‐endpoint, parallel‐cohort RCT design.</p> </sec> <sec id="jth13038-sec-0003" sec-type="section"> <title>Results</title> <p>No eligibility violations or randomization errors occurred. Of the enrolled patients, 69% were randomized, 3% missed the randomization window, and 28% were followed in prespecified observational cohorts for completely occlusive thrombosis or persistent antiphospholipid antibodies. Retention at 1 year was 82%. Interobserver agreement between local and blinded central determination of venous occlusion by imaging at 6 weeks after diagnosis was strong (<italic>k</italic>‐statistic = 0.75; 95% confidence interval [CI] 0.48–1.0). The primary efficacy and safety event rates were 3.3% (95% CI 0.3–11.5%) and 1.4% (95% CI 0.03–7.4%).</p> </sec> <sec id="jth13038-sec-0004" sec-type="section"> <title>Conclusions</title> <p>The P/F phase of the Kids‐DOTT trial has demonstrated the validity of vascular imaging findings of occlusion as a randomization criterion, and defined randomization, retention and endpoint rates to inform the fully powered RCT.</p> </sec> </abstract>