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

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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

  • Goldenberg, NA
  • Abshire, T
  • Blatchford, PJ
  • Fenton, LZ
  • Halperin, JL
  • Hiatt, WR
  • Kessler, CM
  • Kittelson, JM
  • Manco‐Johnson, MJ
  • Spyropoulos, AC
  • Steg, PG
  • Stence, NV
  • Turpie, Alexander Graham Gri
  • Schulman, Sam
  • 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

publication date

  • September 2015

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