OC 52.4 - The impact of steroids following gene therapy for haemophilia: results from the Exigency study

Background: With gene therapy (GT) for haemophilia A (HA) and B (HB) now licensed in Europe and the US, understanding the impacts of these treatments on people with haemophilia (PwH) and their families will ensure effective provision of care and support during treatment and follow-up. Post-gene therapy, clinical trials have used corticosteroids as immunosuppression to support factor expression; the lived experience of this on PwH has not been widely studied..

Aims: To describe the impact of corticosteroid use in PwH who have participated in GT clinical trials.

Methods: In-depth qualitative interviews were conducted with PwH who had received GT and, where available, a family member. Interviews were analysed using inductive thematic methodology. The Exigency study is registered on ClinicalTrials.gov NCT04723680.

Results: 16 PwH (11 HA; 5 HB) and 10 partners/spouses were interviewed. 13 PwH (81%) were prescribed steroids (HA mean 17.9 weeks, HB 21 weeks; range 6-36 weeks). Side effects reported included mood changes (6), insomnia (6), energy-mania (6) and weight gain (4). All those reporting mood changes said this had a negative impact on their family; 2 PwH and 3 partners/spouses identified a lack of psychological support to help cope with mood changes caused by steroids. Often reported together, some described insomnia and energy-mania in negative terms (4/6 and 3/6 respectively); others found them positive (2/6 and 3/6 respectively). 7 described steroids as the most difficult part of having GT; 2 reported no adverse effects. Positively, 4 reported improved joint pain while taking steroids.

Conclusion(s): Over half of PwH described steroids as the worst part of GT, impacting themselves and their families due to adverse side effects. Ensuring these impacts are understood, identified quickly, and dealt with appropriately and effectively is essential if PwH are to obtain the maximum benefit from GT. There remains a need for studies of less toxic approaches to immunosuppression.