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Cancer drug benefits patients with inherited kidney disease
Bosutinib may inhibit the growth of cysts in patients with autosomal dominant polycystic kidney disease, according to findings recently published in the Journal of the American Society of Nephrology.
As many as one in 1,000 people are affected by the hereditary condition, also known as ADPKD, the researchers said.
“Clinical approaches to managing ADPKD are primarily supportive, such as focusing on hypertension and other secondary complications,” Vladimir Tesar, MD, PhD, of the department of nephrology at General University Hospital in the Czech Republic, and colleagues wrote.
For their phase 2 trial, researchers randomly assigned 169 patients with ADPKD to 200 mg of bostunib daily, 400 mg of bosutinib daily or a placebo daily for 24 months or less. These patients had median estimated glomerular filtration rates (eGFR) of 85.9 mL/min/1.73 mL2 and a median total kidney volume of 1376.30 mL.
Those who received a 400-mg dose daily had it reduced to 200 mg during the trial per an amendment to the trial’s protocol. The primary endpoint was the annualized rate of kidney enlargement in patients who had received treatment for 2 or more weeks.
Tesar and colleagues found that the rate of kidney enlargement was lowered by 66% in patients assigned 200 mg of bosutinib vs. those assigned placebo (P = .005), and by 82% for pooled bosutinib vs. placebo (P < .001).
In addition, the annualized rate of eGFR decline for placebo vs. bosutinib was –2.54 vs. –3.09 for those taking 200 mg daily, –7.43 for those taking 400 mg daily and –4.76 for those who reduced from 400 mg to 200 mg daily. Researchers reported that eGFR decreased from baseline over time across all of the trial’s participants; there was a general trend toward dose-dependent worsening of eGFR with increasing bosutinib dose that was partially reversible during the 30-day washout following the first treatment period.
According to researchers, diarrhea, nausea, nasopharyngitis, vomiting and headache were among some of the adverse events reported by the study’s participants.
“The overall gastrointestinal and liver toxicity profile was consistent with the profile in prior studies of bosutinib; no new toxicities were identified,” Tesar and colleagues wrote. – by Janel Miller
Disclosure: Tesar was a paid consultant for Pfizer at the time of the study. Please see the study for a list of the other authors’ relevant financial disclosures.