What are the challenges involved in enrolling pediatric patients in cGvHD clinical trials?

The GvHD Hub was pleased to speak with Andrew Harris, Memorial Sloan Kettering Cancer Center, New York, US. We asked, What are the challenges involved in enrolling pediatric patients in cGvHD clinical trials?  

In this interview, Harris discusses key barriers to enrolling pediatric patients with chronic graft-versus-host disease (cGvHD) in clinical trials, including the limited number of specialist centers with expertise in managing pediatric cGvHD and the resulting need for some children and their families to travel considerable distances to participating sites. He also highlights how the rarity of cGvHD in children may make it difficult to identify eligible patients for clinical trials. 

Key points 

• Given the limited treatment options available for pediatric patients with cGvHD, addressing barriers to clinical trial participation in this population is particularly important.¹ 
• Several practical factors may pose challenges to enrolling pediatric patients in cGvHD clinical trials, particularly when considering patients with a low body weight, for whom certain trial procedures, such as intravenous access and blood sampling, may be technically challenging.²  
• The need for specialized centers with the expertise and resources required to treat pediatric cGvHD may restrict the number of centers able to participate in clinical trials in this setting. 
• In turn, the limited number of participating centers in pediatric cGvHD clinical trials can mean that children and their families may need to travel considerable distances to access a trial.^3–5 
• This can be particularly challenging when frequent visits are required – for example, when investigational therapies require intravenous or subcutaneous administration at the trial center. Where appropriate, oral therapies that can be administered at home may help reduce this logistical burden. 
• In addition, the relatively low incidence of cGvHD in children can make it challenging for centers to recruit sufficient numbers of eligible participants.^1,6 
  • This challenge may be further compounded by the heterogeneous presentation and clinical course of GvHD in children, which can make it difficult to identify patients who meet the specific eligibility criteria of individual trials.⁷ 
• The need for parental consent may present an additional challenge to enrollment, with parental approval influenced by several factors, including socioeconomic status, the age of their child, and perceived risks and benefits.¹ 
• In summary, several practical and logistical challenges can impact the enrollment of pediatric patients in cGvHD clinical trials, with the limited number of centers with appropriate expertise representing a key limiting factor. 

This educational resource is independently supported by Sanofi. All content is developed by SES in collaboration with an expert steering committee. Funders are allowed no influence.