The national blueprint for 21st century data and specimen collection and observational cohort studies: NHLBI State of the Science Workshop on factor VIII inhibitors

on behalf of the members of Working Group 2, the NHLBI State of the Science Workshop on factor VIII inhibitors: Generating a national blueprint for future research

Research output: Contribution to journalArticlepeer-review

4 Scopus citations

Abstract

Introduction: A devastating complication of hemophilia A, the congenital deficiency of coagulation factor VIII (FVIII), therapy is the development of inhibitory antibodies (inhibitors) to infused FVIII concentrate affecting up to 30% of people with the most severe form of the disease. Although the number of patients affected by hemophilia A with inhibitors is small, the physical, emotional, financial, and public health impact is overwhelming to the patient, family, and medical system. To best serve this patient population, as well as enhance knowledge around this complication, a robust data and specimen collection strategy must be designed. Aim: Working Group 2 (WG2) of the National Heart, Lung, and Blood Institute (NHLBI)- sponsored State of the Science (SOS) workshop on factor VIII inhibitors in 2018 was tasked with developing recommendations around the conduct of research, modeling efficient data and specimen collection, developing infrastructure support, establishing partnerships with all stakeholders, including international collaborators, addressing ethical issues, maximizing patient/family engagement and promoting training opportunities. Methods: A group with diverse expertise was assembled who reviewed the current state of data and specimen collection in patients with hemophilia and developed recommendations for the future. Results: Our results were presented at the SOS Workshop where additional feedback was gained. Conclusion: Our charge and recommendations are summarized in this manuscript.

Original languageEnglish (US)
Pages (from-to)590-594
Number of pages5
JournalHaemophilia
Volume25
Issue number4
DOIs
StatePublished - Jul 1 2019

Keywords

  • antibodies
  • data
  • haemophilia A
  • inhibitors
  • repository
  • specimen

ASJC Scopus subject areas

  • Hematology
  • Genetics(clinical)

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