SGLT2抑制剂作为慢性肾脏疾病儿童患者的肾保护药物
By Fortrea
When a child is diagnosed with chronic kidney disease (CKD), their physician faces limited treatment options and often prescribes "off-label" medications, which means an FDA-approved drug is used for an unapproved use to treat a disease or medical condition.
To help address this knowledge gap, NephCure and the Kidney Health Initiative hosted a workshop, assembling a steering committee with advisors from the FDA and academic pediatric nephrologists. Dr. Barbara Gillespie, Vice President, Therapeutic Head of Nephrology at Fortrea and Adjunct Professor at the University of North Carolina, also served on this steering committee and shared her clinical trial insight with the workshop participants. Global attendees included children living with CKD and their parents, FDA, NIH, academic nephrologists (for both pediatrics and adults), and industry representatives.
Collaborating to understand the challenge of supporting pediatric patients
The goal of the workshop was to gather stakeholder perspectives and define the best path forward to assess the safety and efficacy of sodium-glucose cotransporter-2 (SGLT2) inhibitors for renoprotection in children and adolescents living with CKD. This class of drugs was initially approved by the FDA for adults with Type 2 diabetes. Subsequent trial data supported label extensions, leading the FDA to also approve SGLT2 inhibitors to reduce the risk of heart failure and preserve renal function in adults.
"Recent trials focused on CKD patients have demonstrated safety and efficacy in SGLT2 inhibitors for adults living with CKD," explained Dr. Gillespie. "The goals of the meeting were to gather key stakeholders to understand that these drugs are already being used off-label in pediatric CKD populations, and to agree that cohesive efforts in generating data in these patients are needed to more thoroughly assess their safety and efficacy for our children."
Sharing workshop highlights and listening to the voice of the patient
As a result of the workshop, Dr. Gillespie and other co-authors are currently developing a manuscript to publish for a summary of the discussion, which touched on current data, gaps in the data, and paths forward toward generating evidence in pediatric CKD patients.
The co-authors will also summarize one of the highlights during the workshop, a Voice of the Patient panel, which featured adolescents and their parents sharing their CKD journey.
When asked what they hated most about kidney disease, one teenager on the panel answered, "Kids are mean. I'm already shorter than my classmates because of my kidney disease and the only available medication is steroids, which makes me puffy and ugly."
Dr. Gillespie noted, “These kids just want to have a normal childhood, and they certainly deserve proven medications without the deleterious side effects of steroids, which are used in many pediatric kidney diseases that fall under the category of glomerular disease.”
Looking ahead to guide targeted pediatric research
Dr. Gillespie hopes her contributions to this collaborative team—alongside her work at Fortrea—will help improve outcomes for children living with kidney disease.
"A common saying in pediatrics is, 'Kids are not just small adults,'" said Dr. Gillespie. "They need to be studied separately. Whenever I'm talking to a sponsor about their clinical development plans, I ask them early on about their pediatric investigational plan (PIP). Sponsors need to start thinking sooner, rather than later, about how they will include a pediatric population during their development so they can plan appropriately. We can help them prepare for these important trials as part of our commitment to our sponsors and the patients they can potentially impact with these life-changing treatments."
Further reading on sodium-glucose cotransporter-2 (SGLT2) inhibitors:
· General information on Forxiga (dapagliflozin) from the EMA
· Studying dapagliflozin in children with chronic kidney disease
· The EMA-submitted paediatric investigation plan for Invokana (canagliflozin)
