Mini-COMET study: Effects of 97 weeks of avalglucosidase alfa dosing on ptosis in participants with infantile-onset Pompe disease who were previously treated with alglucosidase alfa

Combinations of conventional androgen deprivation therapy (ADT) with docetaxel or abiraterone have shown encouraging results in several phase 3 randomized trials and were consequently implemented in current clinical guidelines. However, to date it is still not clear which patients benefit the most from combination therapy and whether there are still patients who are candidates for ADT monotherapy. Here we highlight evidence that indications for conventional ADT have been significantly reduced over the past years and should only be offered to highly selected patients. Since current knowledge is still based on subgroup analyses using mostly arbitrary post hoc cutoff values, general recommendations have to be given with caution.