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Pulmonary hypertension: smaller kids, smaller steps

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Cited by (13)

  • Meaningful and feasible composite clinical worsening definitions in paediatric pulmonary arterial hypertension: An analysis of the TOPP registry

    2019, International Journal of Cardiology
    Citation Excerpt :

    These limitations of applying outcomes appropriate for adults to the paediatric population need to be overcome. Non-invasive clinical outcomes tailored for use in paediatric PAH are required in order to assess the efficacy of therapies in a clinical trial setting [6]. The use of composite CW outcomes is increasingly encouraged in PAH clinical research [7–9].

  • Using registries to understand clinical practice: A lesson for rare disease

    2016, Journal of the American College of Cardiology
  • FUTURE-2: Results from an open-label, long-term safety and tolerability extension study using the pediatric FormUlation of bosenTan in pUlmonary arterial hypeRtEnsion

    2016, International Journal of Cardiology
    Citation Excerpt :

    The FUTURE-1 and FUTURE-2 studies reflect on the challenges of performing pediatric clinical trials for diseases such as PAH in which patient numbers are low. An additional challenge of performing studies in children, following the identification of sufficient treatment-naive patients, is to ensure adequate retention levels in the context of changing treatment standards [12,20]. Although the TOPP [30] and REVEAL (pediatric cohort only) [7] registries have generated important data on the symptoms, diagnosis, and clinical outcome of PAH-specific therapies in a real-life setting, there is still a paucity of data for these treatments in pediatric patients with PAH.

  • Prognostic factors in pediatric pulmonary arterial hypertension: A systematic review and meta-analysis

    2015, International Journal of Cardiology
    Citation Excerpt :

    The ongoing Tracking Outcomes and Practice in Paediatric PH (TOPP) registry encompasses the largest cohort of children with PAH to date and is expected to yield important new insights in survival and prognostic factors in pediatric PAH [46]. Although a powerful tool with regard to sample size, the usefulness of any registry depends on the predefined aims and might be hampered by the fact that frequency and mode of follow-up are often not dictated [47]. To be able to further investigate reported discrepancies and to increase sample size and statistical power, it could also be considered to merge existing patient cohorts on an individual patient level.

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