Study Will Examine Brain Development in Young People with Hemophilia

A new observational study funded by Novo Nordisk, which is currently in the recruitment phase, will seek to better understand how hemophilia may impact brain development, thinking and behavior in affected children and young adults.

A precursor to this type of research can be found in studies conducted in the late 1980s and early 1990s. These studies also focused on brain development, mostly in the context of hemophilia and HIV, the latter of which had far reaching effects on the hemophilia community at the time. One prominent example of this was the Hemophilia Growth and Development Study (HGDS), a four-year observational study designed to learn more about the impact of hemophilia and HIV on brain development. The study included 333 male children and adolescents with moderate to severe hemophilia.

Results of HGDS suggested that hemophilia was associated with substantial brain dysfunction, reflected in difficulties related to coordination and motor function, lower intelligence, academic and adaptive skills, plus more behavioral/emotional problems compared to “published norms.” Study investigators also recognized an association between greater difficulties in performance skills, academic achievement, language, and nonverbal intelligence and memory in people with an impaired immune system linked to HIV infection.

The decades that have followed HGDS have seen a series of therapeutic breakthroughs and advancements in clinical practice, most notably the establishment of prophylaxis with factor replacement therapy as an optimal treatment for hemophilia patients. The study being initiated by Novo Nordisk is designed to help determine whether these therapeutic advancements have resulted in better outcomes related to brain function.

The Evolving Treatment of Hemophilia’s Impact on Neurodevelopment, Intelligence, and Other Cognitive Functions (eTHINK) study will aim to recruit approximately 510 patients from U.S. hemophilia treatment centers. Study participants will include  boys and young adults (1 to 21 years old) with hemophilia A and B, across varying severity levels and therapy regimens. Investigators will conduct a comprehensive examination of participants’ brain function, including an assessment of intelligence, emotional behavior, adaptive behavior, executive function, and attention and processing speed. The assessments will be conducted primarily through standardized tests and questionnaires completed by patients or their parents/caregivers.

“The cross‐sectional eTHINK study will provide an updated [brain function] assessment across the spectrum of hemophilia severity and potentially point to predictors of risk for children with hemophilia,” the researchers stated.

The new study’s design and HGSD are described in “Neuropsychological Function in Children with Hemophilia: A Review of the Hemophilia Growth and Development Study and Introduction of the Current eTHINK study,” which was published October 8, 2019 in Pediatric Blood & Cancer.

Source: Hemophilia News Today, October 18, 2019

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