Safety and Efficacy of Asfotase Alfa in Patients With Hypophosphatasia (HPP)

Inclusion Criteria

• •Patients must meet one selection criteria of following "1", "2", and "3", and must meet the selection criteria of "4."
• •1. Patient who has been already treated with Asfotae Alfa (ALXN1215) out of this clinical trial
• •2. Patient who has been diagnosed as HPP
• •3. Documented diagnosis of HPP as indicated by:
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• •1. Total serum alkaline phosphatase below the lower limit of normal for age
• •2. Ultrasonographic features of prenatal, characterized by:
• •1\) severe short extremities (femur length \<-4SD in second and third trimesters) 2) extending into the metaphysis (femur metaphysis length or femur length \>0.33) 3) craniotabes 4) Hypoplastic thorax (Thoracic or abdominal circumference \<0.6) (3) Computed tomographic findings of prenatal, characterized by:
• •1. Generalized decreased ossification
• •2. Extreme shortening of tubular bones
• •3. Hypoplastic thorax (4) Radiographic evidence of HPP, characterized by:
• •1\) Flared and frayed metaphyses 2) Severe, generalized osteopenia 3) Widened growth plates 4) Areas of radiolucency or sclerosis (5) Two or more of the following HPP-related findings:
• •1. History or presence of:
• •\- Nontraumatic post-natal fracture
• •\- Delayed fracture healing
• •2. Nephrocalcinosis or history of elevated serum calcium
• •3. Functional craniosynostosis
• •4. Respiratory compromise or rachitic chest deformity
• •5. Vitamin B6 dependent seizures
• •6. Failure to thrive
• •7. Premature tooth loss (6) Patient who have the mutation of tissue non-specific ALP gene 4. Parent or legal guardian(s) must provide written informed consent prior to any study procedures being performed and must be willing to comply with all study-required procedures