Article published Open Source in: press.endocrine.org/journal/jcem, january 2016, 101(1):344-342, doi: 10.1210/jc.2015-3462
Authors: Michael P. Whyte, Cheryl Rockman-Greenberg, Keiichi Ozono, Richard Riese, Scott Moseley, Agustin Melian, David D. Thompson, Nicholas Bishop, and Christine Hofmann
What is this article about:
Asfotase alfa is a treatment for hypophosphatasia (HPP), a severe genetic disorder that can lead to high mortality rates in infants. This study compared outcomes of 37 infants treated with asfotase alfa to 48 historical controls. Key findings showed that the treatment significantly improved survival rates: 95% of treated infants survived to age one, compared to only 42% of controls. Additionally, 84% of treated infants were alive at age five versus 27% of controls. The treatment also improved skeletal health and respiratory function, allowing many infants to be weaned off ventilatory support. Asfotase alfa therefore appears to enhance both the quality of life and survival for infants with severe HPP.
Key Take Aways:
1) Asfotase alfa significantly improves survival rates in infants with severe HPP.
2) Survival rates at age one were 95% for treated patients versus 42% for controls.
3) At age five, 84% of treated patients survived compared to 27% of historical controls.
4) The treatment also improved skeletal mineralization and respiratory function.
5) Many infants were able to wean off ventilatory support thanks to the treatment.