Bone turnover and mineral metabolism in adult patients with hypophosphatasia treated with asfotase alfa

• DOI: 10.1007/s00198-021-06025-y
• Publication Date: 2021-07-02T18:03:08Z
• Authors: L. Seefried; D. Rak; A. Petryk; F. Genest
• Keywords: Bone remodeling; N-terminal telopeptide; Hypophosphatasia

There is limited understanding of how asfotase alfa affects mineral metabolism and bone turnover in adults with pediatric-onset hypophosphatasia. This study showed that hypophosphatasia treated experienced significant changes biochemical markers metabolism, possibly reflecting enhanced remodeling previously osteomalacic bone. Hypophosphatasia (HPP), due to a tissue nonspecific alkaline phosphatase (TNSALP) deficiency, can cause impaired mineralization turnover. Although HPP may be alfa, an enzyme replacement therapy, data are available on treatment HPP. ALP substrates, markers, density (BMD) from EmPATHY, single-center, observational (≥ 18 years) (NCT03418389), were collected during routine clinical care analyzed baseline through 24 months treatment. Data 21 patients significantly increased activity reduced urine phosphoethanolamine (PEA)/creatinine (Cr) ratios after transient increases parathyroid hormone 1-84 (PTH), osteocalcin, procollagen type 1 N-propeptide (P1NP) levels at 3 6 tartrate-resistant acid 5b (TRAP5b) months, decrease N-terminal telopeptide collagen (NTX) months. Lumbar spine BMD T scores continuously Significant suggest treatment-mediated enable unmineralized surfaces. Urine PEA/Cr useful parameter monitoring care.