The potential impact of gene therapy on health-related quality of life (HRQoL) domains in haemophilia
Bullinger M, Gardner D, Lewis H, Miesbach W, Nolte S, O’Hara J, O’Mahony B, Pollard D, Skinner M, Quinn J. The Journal of Haemophilia Practice. 2021;8(1): 56-68. https://doi.org/10.17225/jhp00176
Patient-relevant health outcomes for hemophilia care: development of an international standard outcomes set
van Balen EC, O'Mahony B, Cnossen MH, Dolan G, Blanchette VS, Fischer K, Gue D, O'Hara J, Iorio A, Jackson S, Konkle BA, Nugent DJ, Coffin D, Skinner MW, Smit C, Srivastava A, van Eenennaam F, van der Bom JG, Gouw SC. Res Pract Thromb Haemost. 2021;5:e12488. https://doi.org/10.1002/rth2.12488
Getting older with a bleeding disorder: a needs assessment
O'Callaghan S, Jayaram P, Caris S. Melbourne: HFA, 2020. Detailed PROBE findings are in Appendix 2
Multi-stakeholder Approaches to Improve Evidence-Based Decisions in Rare Diseases: Engagement of Patients and Patient Organizations
Granados A, Mullin T, Moseley J, Meyer F, Avetisyan R, Wong-Rieger D, Kaatee M, Skinner M, Leyden S.Report of HTAi 2016 Panel Session. Health Technology Assessment International – Canada. (2016). Published at: http://www.htai.org 19 Sep 2016
Guidelines for the management of hemophilia
Srivastava A, Brewer AK, Mauser-Bunschoten EP, Key NS, Kitchen S, Llinas A, Ludlam CA, Mahlangu JN, Mulder K, Poon MC, Street A and (2013). Haemophilia. 19: e1-e47. https://doi.org/10.1111/j.1365-2516.2012.02909.x
