Hereditary leiomyomatosis and renal cell carcinoma (HLRCC)-associated papillary RCC is an aggressive form of kidney cancer that is passed down in families (inherited). Most patients die from progressive disease, with an average survival of less than 2 years. There is currently no effective treatment for patients with advanced HLRCC-associated papillary RCC.

A recent phase 2 study testing a combination of bevacizumab (an immunotherapy) with erlotinib (a TKI) has shown promising anti-cancer results in both HLRCC-associated papillary RCC and papillary RCC. Nearly three quarters of patients responded to treatment with bevacizumab plus erlotinib. The average time to when the treatment stopped working and the cancer started growing again (progression-free survival, PFS) was nearly 2 years (21.1 months) and the average overall survival time was (OS) was more than 3 and a half years (44.6 months).

Every patient in the study had at least one treatment-related side effect, and about half had a severe or life-threatening side effect. The most common side effects were rash, diarrhoea, protein in the urine (proteinuria), and dry skin. High blood pressure was the most common severe or life-threatening side effect.

The combination of bevacizumab and erlotinib showed anti-cancer activity in patients with HLRCC-associated or sporadic papillary RCC. Side effects were those known to be associated with this combination.

Read more in MedpageToday here and The New England Journal of Medicine here