Consensus statement helps assess patient readiness for gene therapy in sickle cell disease
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Patient readiness will greatly affect the success of gene therapy for sickle cell disease at the individual and collective levels, according to a consensus statement published in JAMA Network Open.
The safety, efficacy and acceptance of gene therapy will determine whether the modality truly can transform sickle cell disease treatment, according to the authors. However, historic medical exploitation of racial and ethnic minority individuals necessitate “extreme care” to assure readiness for gene therapies, they added.
To address these concerns, the Cure Sickle Cell Initiative established the Patient Readiness and Resilience Working Group in 2020.
The group generated consensus recommendations over the past 2 years and identified the following goals to help clinicians evaluate patient readiness: promoting open dialogue; providing information about psychosocial factors that might affect uptake of gene therapy; understanding patient strengths that may help ensure well-being before, during and after gene therapy; and identifying and managing psychosocial risks.
“My hope is that the working group's recommendations will lead institutions and providers to consider adding evaluations of psychosocial readiness to their pre-gene therapy workflows,” statement lead author Steven J. Hardy, PhD, director of behavioral health services and a pediatric psychologist in the divisions of hematology, oncology and blood and marrow transplantation at Children’s National Hospital, told Healio. “We understand that psychosocial issues are going to play a major role in gene therapy outcomes — both in terms of physical and mental health — whether you assess them or not.”
Healio spoke with Hardy about the origins of the working group, the importance of the consensus statement, and how he hopes these recommendations will help guide use of gene therapy for sickle cell disease.
Healio: What led to establishing this working group?
Hardy: The working group was really borne out of advocacy from the sickle cell community about the importance of mental health. Leaders of the NHLBI's Cure Sickle Cell Initiative heard from patients and caregivers who wanted to know how to support their own or, in some cases, their children’s mental health and well-being during the COVID-19 pandemic. In recognition of how much the sickle cell community really wanted guidance on this topic, a group was formed to consider how psychosocial issues may factor into one's readiness for gene therapy and what forms of care would be needed for those who decide to undergo gene therapy.
Healio: What prompted the development of this consensus statement?
Hardy: We felt a consensus statement was needed because there really was not much guidance in the literature about how best to evaluate and support psychosocial readiness for gene therapy in sickle cell disease. We aimed to grow awareness about psychosocial readiness as a concept worth attending to, planning for and prioritizing, while also providing concrete recommendations about how they could be carried out effectively.
The recommendations were developed from 2021 to 2023, and a white paper had been produced prior to the FDA’s first approval of a gene therapy for sickle cell disease. However, with these treatments becoming commercially available, we felt there was a need for further peer review of our recommendations and dissemination to a broad audience.
Healio: What are the five key recommendations the group makes?
Hardy: First, we recommended evaluating the patient's understanding of gene therapy and their own perception of their readiness for the treatment. Second, the evaluation should be conducted in a way that creates space for and evokes a candid dialogue about any concerns the patient may have about gene therapy. Providers should aim to conceptualize psychosocial factors that could influence decision-making about pursuing gene therapy or ultimately affect the success of the treatment. We recommended assessing psychosocial risks that the treating teams should be aware of so that concerns can be addressed before, during and after gene therapy. Finally, the working group advised identifying patients' strengths or resilience factors, which could be leveraged to promote optimal treatment experiences and long-term health.
Healio: Are there any recommendations you would like to highlight as particularly important?
Hardy: Evaluating knowledge and understanding of gene therapy is especially critical. Patients should have a strong understanding of what causes sickle cell disease and sickle cell-related complications because that information helps connect the dots to how gene therapy works and why it could be helpful. We want to know that patients understand the details of what is going to happen before, during and after treatment; how they are going to feel; and what the potential short- and long-term outcomes of gene therapy might be. This treatment has great potential to positively affect individuals living with sickle cell disease, but it is complex and not without risks. We need to ensure that patients have realistic expectations for what they may experience if they choose to pursue this type of treatment.
With the consensus statement and associated recommendations, we have provided a roadmap for evaluating key factors that will help determine a patient's readiness for gene therapy and identify specific areas of concern that can be addressed to enhance readiness.
Reference:
For more information:
Steven J. Hardy, PhD, can be reached at sjhardy@childrensnational.org.
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