Guy Young, MD: Prophylactic or Gene Therapy, and Shared Decision Making in Hemophilia

Deciphering the best treatment options for hemophilia patients involves a process of shared decision-making, considering eligibility, patient preferences, and risk tolerance, Guy Young, MD, director of the Hemostasis and Thrombosis Center at Children's Hospital Los Angeles, said in an interview.

​​”Gene therapy is such a new platform that any patient who takes it on, or who agrees to receive gene therapy, has to understand that there's long term unknowns,” he said.

Informed discussions about the pros and cons of different treatments help guide the decision-making process. When it comes to treatment adherence and overall quality of life, patient lifestyle plays a significant role.

Psychological factors also come into play, as patients may have varying appetites for risk and preferences for treatment regimens, Young explained. Some individuals are comfortable with the long-term uncertainties associated with gene therapy, while others may be more hesitant.

“We inform the patient about the different options, we discuss the pros and cons of each of the options,” Young shared. “We discuss what they're looking for in a treatment–what is it that's most important to them? We discussed, how risk averse they are? Are they risk averse, or are they willing to take on some risk?”

Patient age, marital status, presence of children, and the impact of treatment on family and work are additional factors considered when determining the best treatment approach.

The decision-making process takes into account both clinical factors and non-clinical elements related to patients' lifestyles, values, and treatment goals. It aims to find a treatment option that not only effectively manages bleeding but also enhances patients' quality of life and aligns with their individual circumstances.