A Survey to Assess the Knowledge and Perceptions about Gene Therapy in Indian Haemophilia Patients

Novel molecules including gene therapy are used to treat haemophilia. Little is known on patient perceptions about gene therapy. Present survey is to assess the knowledge and identify key educational gaps and concerns about gene therapy in Indian persons with haemophilia (PWH). An explorative, qualitative, closed questionnaire based survey was conducted recruiting adult PWH from 3 Centers in India. Data were collected through face-to-face surveys or the patients themselves filling the survey forms. Of the 415 complete questionnaires received from adult severe PWH, approximately half of the PWH felt that gene therapy is the best option for PWH, but only 31.2% showed their willingness to be a part of any gene therapy clinical trial, if available. About 57% of the PWH were unable to decide about participation in clinical trials and 11.7% of the participants would never consider receiving gene therapy. Major concerns were cost, side effects, duration of expression and rejection, infection and risk of liver cancer. The PWH expressed their desire for information on subjects like eligibility criteria, earlier trial data, and details on safety. The survey shows a low level of knowledge about gene therapy in PWH and identifies gaps in knowledge and a strong willingness to understand more on the potential risk: benefit profile of gene therapy.

The online version contains supplementary material available at 10.1007/s12288-024-01945-1.