Although gene therapy is anticipated to bring positive changes for people with hemophilia, choosing to receive gene therapy will require empowerment, participation, and shared decision-making, investigators write.
Gene therapy brings a new era for hemophilia treatment, challenging health care providers (HCPs) and patients to adapt to a supportive paradigm.
The successful implementation of gene therapy for hemophilia hinges on effective communication and collaboration, investigators emphasized in a new review article published in Haemophilia.
According to the article, hemophilia treatment centers, HCPs, patients, and various stakeholders must synchronize their efforts with transparent discussions, robust education, and a deep understanding of individual patient needs.
In the evolving landscape of medical advancements, the emergence of adeno-associated virus (AAV)–based gene therapy brings a transformation to the treatment of hemophilia among adults. Treatment centers and HCPs are at the frontier of this cutting-edge therapeutic approach, which requires adaptation and preparedness for a new treatment journey for patients and institutions.
“While principles in this publication reflect experiences and expertise leveraged from US hemophilia treatment centers, concepts discussed within may have applicability for facilities and teams involved in the management of hemophilia patients across the globe,” the review stated.
Understanding the Patient Journey
Before infusion, the patient journey begins with thorough information gathering, where patients learn about the risks and benefits of gene therapy, including lifestyle changes.
Decision-making is a critical phase and demands comprehensive education and discussion. Patient eligibility and choice play pivotal roles, accentuating the necessity for informed decision-making processes as a part of standard practice.
Following this, a detailed patient assessment will be conducted. Preparation for the gene therapy infusion involves not only physical readiness but also mental and emotional preparedness. On the day of infusion, patients will reconfirm their agreement for treatment and final eligibility will be verified.
“Although gene therapy is anticipated to bring positive changes for people with hemophilia and their long-term outlook, choosing to receive gene therapy will require people with hemophilia and their families to feel empowered to participate in informed and shared decision-making,” investigators wrote. “Not all people will hemophilia will be eligible for, or decide to receive, gene therapy.”
Peri- and postinfusion periods will entail short- and long-term monitoring, lifestyle modifications, and potential immunosuppressive treatments. Directly after therapy, the patient will continue to be observed. Long-term monitoring requires collaboration with a referral center or physician.
Institutional Adaptations for Dosing Center
Simultaneously, treatment centers must also work on integrating gene therapy into their existing frameworks. The article addressed biosafety procedures, which demand meticulous consideration to ensure the safety of both patients and health care providers. Planning for product procurement, storage, handling, and administration necessitates precision and adherence to protocols.
Detailed protocols and guidance documents are imperative to the institutional journey. Contingency planning for managing immunosuppressive treatments and hemostatic requirements also needs to be incorporated.
When it comes to staffing, the evaluation of clinical capabilities and the training of staff are necessary to meet the demands of the treatment. The article also stated the coordination of efforts across multidisciplinary teams and seamless collaboration between various centers is pivotal for the success of gene therapy implementation.
“As current electronic medical record systems don't allow full interaction/sharing between sites, hemophilia treatment centers will also need to identify a streamlined process for appropriately exchanging the necessary health information (including eligibility assessment and laboratory results) between the referral/follow-up center and the dosing center,” investigators wrote.
Reference
Pipe, S, Douglas, K, Hwang, N, Young, G, Patel, P, Fogarty, P. Delivery of gene therapy in haemophilia treatment centres in the United States: Practical aspects of preparedness and implementation. Haemophilia. Published online September 25, 2023. doi:10.1111/hae.14867
Perioperative Nivolumab Boosts NSCLC Survival: CheckMate 77T Trial
May 16th 2024This interim analysis of the CheckMate 77T trial, outcomes were compared between adult patients receiving neoadjuvant nivolumab plus chemotherapy or neoadjuvant chemotherapy plus placebo for resectable non–small cell lung cancer (NSCLC).
Read More
Frameworks for Advancing Health Equity: Urban Health Outreach
May 9th 2024In the series debut episode of "Frameworks for Advancing Health Equity," Mary Sligh, CRNP, and Chelsea Chappars, of Allegheny Health Network, explain how the Urban Health Outreach program aims to improve health equity for individuals experiencing homelessness.
Listen
Decitabine, Venetoclax Combo Boosts Survival in Patients With AML and MDS
May 16th 2024Study findings demonstrate that combining decitabine with venetoclax enhances molecular response rates in patients with acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS), correlating strongly with improved clinical outcomes and overall survival.
Read More
Congress Urged to Repeal Comstock Act Threatening Reproductive Rights and Public Health
May 16th 2024In a joint letter addressed to Congress, Healthcare Across Borders, Take Back the Court Action Fund, and UltraViolet Action called out the resurgence of the Comstock Act, urging immediate action to repeal this century-old law that threatens reproductive rights and public health in the US.
Read More