Multicenter randomized controlled trial on Duration of Therapy for Thrombosis in Children and Young Adults (the Kids-DOTT trial): pilot/feasibility phase findings. Academic Article uri icon

Overview

abstract

  • BACKGROUND: 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. METHODS: 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. RESULTS: 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 (k-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%). CONCLUSIONS: 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.

authors

  • Kucine, Nicole Elena
  • 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

publication date

  • August 11, 2015

Research

keywords

  • Anticoagulants
  • Venous Thrombosis

Identity

PubMed Central ID

  • PMC4561031

Scopus Document Identifier

  • 84940900498

Digital Object Identifier (DOI)

  • 10.1111/jth.13038

PubMed ID

  • 26118944

Additional Document Info

volume

  • 13

issue

  • 9