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Haemophilia . 2020 Oct 21

Health-related quality of life and caregiver burden of emicizumab in children with haemophilia A and factor VIII inhibitors-Results from the HAVEN 2 study

Miguel J Lobo, Laia Reverte-Salisa, Ying-Chi Chao, Andreas Koschinski, Frank Gesellchen, Gunasekaran Subramaniam, He Jiang, Samuel Pace, Natasha Larcom, Ester Paolocci, Alexander Pfeifer, Sara Zanivan, Manuela Zaccolo, Helin Tercan, Niels P Riksen, Leo A B Joosten, Mihai G Netea, Siroon Bekkering, Maria Elisa Mancuso, Johnny Mahlangu, Robert Sidonio Jr, Peter Trask, Marianne Uguen, Tiffany Chang, Midori Shima, Guy Young, Johannes Oldenburg, Sylvia von Mackensen

Introduction: Persons with haemophilia A (PwHA) with factor (F)VIII inhibitors, including children, have impaired health-related quality of life (HRQoL). The HAVEN 2 study (NCT027955767) of paediatric PwHA with FVIII inhibitors demonstrated that subcutaneous emicizumab prophylaxis resulted in low annualizedbleed rates.

PMID: 33084175