Determining meaningful health-related quality-of-life improvement in persons with haemophilia A using the Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL).
Sylvia von MackensenOlivier CatalaniElina AsikaniusIdo Paz-PrielMichaela LehlePeter TraskPublished in: Haemophilia : the official journal of the World Federation of Hemophilia (2020)
Our responder thresholds are mostly consistent with those proposed by Wyrwich et al (cut-offs of -10 and -7 for PH and TS, respectively). The majority of responders to emicizumab prophylaxis had improvements greater than the previously reported 10-point reduction in PH and 7-point reduction in TS.