Gene Therapy in Hemophilia: A Transformational Patient Experience.

Hemophilia is a bleeding disorder caused by a single absent/defective gene and characterized by a lack of functional clotting factors. People with hemophilia may experience joint damage, pain, and psychological impairments, all of which could contribute to reduced health-related quality of life (HRQoL). The current standard of care is clotting factor replacement, which is associated with regular infusions; therefore, alternative treatments such as gene therapy (GT) are in development. GT involves the delivery of a functional copy of the clotting factor 8/9 gene by a single infusion into the patient's cells, enabling them to produce their own clotting factor VIII/IX. The impact of treatment on patients' HRQoL can be assessed using hemophilia-specific patient-reported outcome (PRO) measures. Since these measures were designed before the advent of GT, there is a need for updated individualized PRO measures. Patient groups and regulatory authorities emphasize the need for increased patient engagement when considering clinical trial design. Here, we provide patients' perspective on undergoing GT and discuss how to capture the patient voice when measuring the therapy's transformative impact.

Secondary Traumatic Stress and Burnout Among Refugee Resettlement Workers: The Role of Coping and Emotional Intelligence.

To promote a better understanding of the impact of refugee resettlement work on refugee resettlement workers, this study examined the prevalence rates of deleterious mental health and occupational outcomes, such as secondary traumatic stress and burnout, among a sample of 210 refugee resettlement workers at six refugee resettlement agencies in the United States. The study also explored coping mechanisms used by service providers to manage work-related stress and the influence of such strategies and emotional intelligence on secondary traumatic stress and burnout. Our findings show that certain coping strategies, including self-distraction, humor, venting, substance use, behavioral disengagement, and self-blame, were strongly related to deleterious outcomes, ßs = .18 to .38, ps = .023 to < .001. Emotional intelligence was a negative correlate for all outcomes, ßs = -.25 to -.30, ps < .001, above and beyond the effects of trauma, coping styles, job, and demographic characteristics. These findings have potential implications for clinical training and organizational policy regarding refugee mental health.