TOPLINE:

SGLT2 inhibitors significantly improved glycaemic control and reduced the albumin-to-creatinine ratio without affecting body weight in patients with Prader-Willi syndrome and type 2 diabetes (T2D); however, a notable proportion of treated patients experienced adverse events.

METHODOLOGY:

• T2D affects approximately 20% of patients with Prader-Willi syndrome and often progresses rapidly to insulin dependence. Despite potential benefits, the use of SGLT2 inhibitors in Prader-Willi syndrome with T2D remains largely unexplored.
• Researchers in France conducted a retrospective, multicentre cohort study using electronic medical record data to evaluate the efficacy and safety of SGLT2 inhibitors in 48 patients with genetically confirmed Prader-Willi syndrome and T2D.
• They compared 24 patients who received SGLT2 inhibitors (median age, 34 years; median duration of T2D, 9.5 years) with an equal number of matched control patients who did not receive the drug.
• Glycaemic and renal parameters were analysed at baseline and at least once between 6 and 12 months.
• Acute kidney injury was defined according to the Kidney Disease: Improving Global Outcomes 2012 criteria.

TAKEAWAY:

• A1c levels at baseline were significantly higher in the SGLT2 inhibitor group than in the control group (8.8% vs 6.9%; P = .0003). After 6 months of SGLT2 inhibitor treatment, A1c levels fell to 7.3% (P < .0001); however, A1c levels in the control group remained stable.
• The albumin-to-creatinine ratio decreased significantly in the SGLT2 inhibitor group, from a median of 25.7 to 5.0 mg/mmol (P = .0078), whereas it increased in the control group; the estimated glomerular filtration rate remained stable with SGLT2 inhibitor therapy.
• Systolic blood pressure also decreased modestly in the SGLT2 inhibitor group; all these benefits occurred without any change in the body weight.
• Adverse events occurred in 37.5% of patients in the SGLT2 inhibitor group, with 8.3% experiencing acute kidney injury requiring hospitalisation; however, the control group reported no adverse events.

IN PRACTICE:

"SGLT2is [SGLT2 inhibitors] may offer valuable glycemic and renal benefits in adults with PWS [Prader-Willi syndrome] and T2D. However, due to the elevated risk of adverse events, careful monitoring of renal function, volume status, and co-medications is essential," the authors wrote.

SOURCE:

This study was led by Juliette Jacquot-Thierry, Sorbonne Université, Inserm, Assistance Publique Hôpitaux de Paris, Service de Nutrition, CRMR PRADORT, Hôpital de la Pitié-Salpêtrière, Paris, France. It was published online on September 18, 2025, in Diabetes & Metabolism.

LIMITATIONS:

Because the study was retrospective, causal relationships cannot be confirmed. Additionally, missing data existed in the control group, most notably regarding urinary albumin-to-creatinine ratios.

DISCLOSURES:

No funding source was reported for the study. One author reported serving as an advisory panel member and receiving lecture fees from various pharmaceutical companies. Another author reported that their institutions have received funding for clinical trial research from Rhythm Pharmaceuticals, Inc.

This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication.