Why this review matters for Bardet-Biedl syndrome
Obesity is a major health concern in childhood, and it has particular relevance for children with Bardet-Biedl syndrome (BBS). The new JAMA review, “Obesity in Children Aged 2 to 12 Years: A Review,” summarizes how obesity is identified and treated in young children—and highlights a treatment specifically indicated for obesity associated with BBS.
For families affected by BBS, conversations about weight should be connected to overall health, daily wellbeing, and access to specialized care. The review underscores that childhood obesity is not simply a future concern: it can be associated with health complications during childhood and can persist into adulthood. Importantly, it also recognizes that some children have rare genetic conditions, including BBS, for which targeted pharmacologic treatment may be part of care.
Childhood obesity: common, consequential, and measurable
In the United States, obesity affects approximately 15% of children ages 2 to 5 years and 22% of children ages 6 to 11 years. Across children generally, obesity is associated with dyslipidemia (unhealthy blood lipid levels), high blood pressure, metabolic dysfunction-associated steatotic liver disease, and obstructive sleep apnea. Children with obesity also report lower quality of life than their normal-weight peers and are five times more likely to have obesity as adults.
Clinicians commonly screen for obesity beginning at age 2 years using body mass index (BMI), a calculation based on weight and height. Because children are growing, their BMI is interpreted using charts standardized by age and sex rather than a single adult cutoff. Obesity is defined as a BMI at or above the 95th percentile. Severe obesity is defined as BMI at or above 120% of the 95th percentile.
These measurements are tools for recognizing elevated risk and guiding care. For children with BBS, regular assessment can help families and clinicians identify needs early, track changes over time, and address obesity alongside other aspects of the child’s health.
Lifestyle treatment remains a foundation
The review identifies intensive health behavior and lifestyle treatment as an evidence-based approach for childhood obesity. This is more substantial than a brief conversation during a routine appointment. It involves at least 26 hours of face-to-face, group- or family-based activities over three to 12 months.
Programs include education and support around nutrition, physical activity, and behavior change, with both the child and caregivers involved. In the studies reviewed, this intensive treatment reduced BMI by 0.7 compared with children who did not take part.
The family-based nature of these programs is especially meaningful. Young children rely on caregivers for meals, routines, access to activities, and healthcare visits. A care plan that includes the whole family can focus on practical, sustainable changes rather than placing responsibility on the child alone.
A targeted medicine for obesity associated with BBS
A central point for the BBS community is the review’s discussion of setmelanotide. Setmelanotide is a melanocortin-4 receptor agonist and is currently the only obesity medication approved by the US Food and Drug Administration for children younger than 12 years.
The medication is indicated for several rare forms of obesity, including obesity associated with Bardet-Biedl syndrome. It is also indicated for certain forms of monogenic obesity involving variants in the LEPR, POMC, or PCSK1 genes, as well as acquired hypothalamic obesity.
This distinction is important: obesity in BBS is recognized within the review as part of a rare-condition context in which an FDA-approved targeted treatment exists. It also illustrates the growing role of precision medicine in obesity care. Rather than treating all childhood obesity as biologically identical, clinicians can consider whether a child’s underlying diagnosis may affect available treatment options.
Other medicines and surgery: an evolving evidence base
The review also discusses liraglutide, which is FDA-approved for youth ages 12 years and older. In a randomized clinical trial involving children ages 6 to younger than 12 years, liraglutide reduced BMI by 7.4% compared with placebo. However, it is not currently FDA-approved for obesity in children under age 12.
Metabolic and bariatric surgery is rarely used in children younger than 12 years. The review notes that evidence supporting surgery in this age group remains limited.
Together, these findings reinforce that treatment decisions in young children should be individualized. Intensive lifestyle care is a core evidence-based approach, while medications may provide additional benefit in appropriate circumstances. The review emphasizes that short- and long-term safety and efficacy data for pharmacotherapy are still limited.
Looking ahead: earlier recognition, more tailored care
The JAMA review makes a strong case for identifying and addressing obesity early. Earlier treatment may reduce the risk of type 2 diabetes, dyslipidemia, hypertension, and mortality later in life.
For the BBS community, the inclusion of setmelanotide in a broad review of childhood obesity is a meaningful sign of progress. It recognizes BBS-associated obesity within mainstream clinical discussions while pointing to the value of diagnosis-informed treatment. Future research will need to clarify long-term outcomes of medications in young children, refine how behavioral and pharmacologic approaches work together, and continue expanding care that reflects the distinct biology and needs of rare genetic conditions such as BBS.