According to early data from an ongoing clinical trial, pegloticase treatment is safe and effective for kidney transplant recipients with uncontrolled gout, investigators reported at the American Society of Nephrology’s Kidney Week 2020 Reimagined virtual conference.

Of 15 patients enrolled in the PROTECT (PROspective sTudy of pEglotiCase in Transplant patients) trial as of September 14, 2020, 5 have completed treatment and all experienced substantial and sustained reductions in serum uric acid throughout treatment. All patients had rapid declines in serum uric acid levels after starting pegloticase treatment, and most had a serum uric acid level less than 1 mg/dL at their last available assessment. Eight patients continue to receive the medication. After completing 24 weeks of treatment, patients demonstrated clinically important decreases in pain and disability, the investigators reported.

Study patients are receiving pegloticase 8 mg once every 2 weeks for 24 weeks to determine response rate during month 6. The investigators defined response as a serum uric acid level less than 6 mg/L for least 80% of the time.

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With regard to safety, 12 patients (80%) experienced adverse events, the majority of which were mild, according to the investigators. For all patients, the estimated glomerular filtration rate (eGFR) remained stable throughout treatment. In the 5 patients who completed treatment, the mean eGFR was 40.9 mL/min/1.73 m2 at baseline and 40.7 mL/min/1.73 m2 at week 24.

“Strategies to effectively manage uncontrolled gout within the vulnerable post-transplant population are important given medications to prevent organ rejection can contribute to increased uric acid levels and lead to higher rates of uncontrolled gout,” primary investigator Abdul Abdellatif, MD, an assistant professor at Baylor College of Medicine in Houston, Texas, said in a press release issued by Horizon Therapeutics, which is funding the study. “Early data of this ongoing clinical trial are encouraging and suggest [pegloticase] is safe and effective for treating uncontrolled gout in this very sensitive transplant population without compromising kidney function.”

Marketed as Krystexxa, pegloticase received US Food and Drug Administration approval in 2010 for treating severe treatment-refractory chronic gout.

Disclosure: Abdul Abdellatif, MD, is a consultant and paid speaker for Horizon Therapeutics, and other investigators are employees of and hold stock in the company or serve as a consultant for company.


Abdellatif AA, Zhao L, Peloso PM, et al. Pegloticase for uncontrolled gout in kidney transplant recipients: Early data report of a multicenter, open-label efficacy and safety study. Presented at: Kidney Week 2020 Reimagined, October 19-25. Poster PO2481.