Cambridge, Mass. — The Collaborative Trajectory Analysis Project said a newly published prognostic score may help predict loss of ambulation in patients with Duchenne muscular dystrophy.
The score, published May 26 in Neurology, the medical journal of the American Academy of Neurology, is based on two common functional assessments: rise from floor and the 10-meter walk/run. cTAP said the tool provides more refined risk stratification and may better inform clinical decision-making and research than previously reported models.
The publication is the latest addition to cTAP’s suite of open-access prognostic models and insights developed to support improved trial design and understanding of Duchenne muscular dystrophy.
“This new prognostic score represents a significant advancement in managing Duchenne muscular dystrophy,” said Craig McDonald, MD, Professor, University of California at Davis Medical Center. “By providing clinicians with a simple yet powerful tool to predict time to loss of ambulation, we can better counsel patients and families, optimize trial design, and ultimately improve the quality of care for individuals living with Duchenne.”
The prognostic score was developed using data from more than 600 boys with Duchenne muscular dystrophy from natural history databases and clinical trial placebo arms. Researchers used classification and regression tree models, prioritizing functional measures that are easy to obtain in clinical settings. The two predictors were used to classify patients into five distinct risk groups.
“The loss of ambulation remains one of the most significant challenges faced by individuals living with Duchenne and their families,” said Debra Miller, founder and CEO of CureDuchenne, a founding patient advocacy partner and provider of initial seed funding to cTAP. “This novel prognostic score will help improve research for treatments that aim to stop or delay Duchenne’s relentless progression and support clinicians to better assist families in planning ahead to navigate this transition. We are eager to see the score put into use, and we are grateful to have played a part by contributing critical patient data from a prospectively defined natural history study to help develop the model. This cTAP effort is an example of the importance of partnerships that bring patient organizations, academics, and industry together to solve problems.”
According to cTAP, the study showed that the prognostic score effectively stratified patients into well-separated risk groups, with median time to loss of ambulation of more than four years in the lowest-risk group and 0.9 years in the highest-risk group. The score was validated using an independent dataset, confirming its robustness and generalizability.
cTAP said the score outperformed existing classifications based on individual functional tests, offering a more accurate prediction of loss-of-ambulation timelines.
The organization said the tool may support clinical practice by helping healthcare providers offer more personalized guidance on mobility devices, home modifications and psychosocial support. In clinical trials, it may help improve patient stratification and research efficiency by identifying subgroups with similar prognostic trajectories.
