Preliminary data presented at Kidney Week 2020 Reimagined suggest pegloticase is safe and effective for treating uncontrolled gout in kidney transplant recipients.1

In the phase 4 PROTECT trial, a team of investigators tested pegloticase (8 mg infused every 2 weeks) in 15 kidney transplant recipients who had serum uric acid levels of 7 mg/dL or greater or hyperuricemia refractory to other urate-lowering therapies. Patients also had prior or current tophi, 2 or more gout flares within the past year, or gouty arthritis.

Before starting pegloticase, patients received gout flare prophylaxis, including colchicine and low-dose prednisone. They also had stable doses of 2 or more immunosuppressants, such as mycophenolate mofetil or tacrolimus. Formation of anti-pegloticase antibodies can decrease treatment efficacy, whereas immunosuppression along with pegloticase has shown better treatment response in phase 3 trials, according to investigators.

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Over 24 weeks of pegloticase treatment, all 15 patients had a rapid decrease in serum uric acid levels, most to a level of less than 1 mg/dL, Abdul Abdellatif, MD, of Baylor College of Medicine in Houston, Texas, and colleagues reported. Patients also had clinically important reductions in pain (Health Assessment Questionnaire [HAQ] pain score decreased by 33.6 points from baseline to week 24) and disability (HAQ-Disability Index decreased by 0.3 points from baseline to week 24). Estimated glomerular filtration rate remained stable at a mean 40.7 mL/min/1.73 m2 at 24 weeks.

Twelve of the 15 transplant recipients experienced an adverse event. Nearly half had gout flares. No patient had anaphylaxis or an immune-related event. Two patients experienced pyrexia, arthralgia, and nasal congestion. One patient had tachycardia, ocular hyperemia, photophobia, melena, asthenia, eye injury, reduced blood calcium, hyperglycemia, muscle spasms, osteonecrosis, headache, and oropharyngeal pain. 

Gout is common and often more severe in kidney transplant recipients due to reduced eGFR and use of calcineurin inhibitors and diuretics, the investigators noted. Early data from this ongoing clinical trial are “promising” for treating uncontrolled gout in kidney transplant recipients with pegloticase, they concluded.

In a separate study presented at the conference, Anthony J. Bleyer, MD, of Wake Forest University in Winston-Salem, North Carolina, and colleagues characterized how pegloticase is being used in patients on dialysis.2 The study is the first to report on pegloticase use in this patient population, according to the investigators.

The study included 42 patients (76.2% male; 54.8% White) who received a median 3 pegloticase infusions; 9 of the patients received at least 12 infusions. The mean dose was 9.7 mg and the median time between doses was 14 days, in keeping with the pegloticase label specifying biweekly infusion. Pegloticase was mostly prescribed by outpatient dialysis facilities (58.1%) and rheumatologists (37.7%). In addition, the vast majority of patients were receiving erythropoiesis-stimulating agents (ESAs). Monthly mean ESA dosing declined by a significant 14,574 units after pegloticase treatment.

Their findings “suggest that pegloticase can and has been used in dialysis patients with uncontrolled gout, though further research is warranted,” Dr Bleyer’s team noted.

Most patients in the study (76.2%) underwent hemodialysis, whereas 19% received continuous cycling and 4.8% continuous ambulatory peritoneal dialysis. Eighty-one percent of patients had hypertension, 45.2% had diabetes, and 23.8% had congestive heart failure. All adult age groups were represented in the cohort: 28.6% were aged 18-44 years; 54.8% were aged 45-64 years; and 16.7% were aged 65 years or older.

Disclosure: These studies were supported by Horizon Therapeutics. Please see the original references for a full list of authors’ disclosures.


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, 2020. Poster PO2481.  

Bleyer AJ, Zhang Y, Kshirsagar OS, Marder BA, LaMoreaux B. USRDS database study on the use of pegloticase in patients undergoing dialysis. Presented at: Kidney Week 2020 Reimagined, October 19-25, 2020. Poster PO1166.