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 journalArticle

4 Citations (Scopus)

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

Fingerprint

National Heart, Lung, and Blood Institute (U.S.)
Specimen Handling
Factor VIII
Observational Studies
Hemophilia A
Cohort Studies
Education
Antibodies
Patient Participation
Ethics
Public Health
Research
Population

Keywords

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

ASJC Scopus subject areas

  • Hematology
  • Genetics(clinical)

Cite this

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 (2019). The national blueprint for 21st century data and specimen collection and observational cohort studies: NHLBI State of the Science Workshop on factor VIII inhibitors. Haemophilia, 25(4), 590-594. https://doi.org/10.1111/hae.13772

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.

In: Haemophilia, Vol. 25, No. 4, 01.07.2019, p. 590-594.

Research output: Contribution to journalArticle

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 2019, 'The national blueprint for 21st century data and specimen collection and observational cohort studies: NHLBI State of the Science Workshop on factor VIII inhibitors', Haemophilia, vol. 25, no. 4, pp. 590-594. https://doi.org/10.1111/hae.13772
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. The national blueprint for 21st century data and specimen collection and observational cohort studies: NHLBI State of the Science Workshop on factor VIII inhibitors. Haemophilia. 2019 Jul 1;25(4):590-594. https://doi.org/10.1111/hae.13772
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. / The national blueprint for 21st century data and specimen collection and observational cohort studies : NHLBI State of the Science Workshop on factor VIII inhibitors. In: Haemophilia. 2019 ; Vol. 25, No. 4. pp. 590-594.
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