Deletion of the Pyrophosphate Generating Enzyme ENPP1 Rescues Craniofacial Abnormalities in the TNAP Mouse Model of Hypophosphatasia and Reveals FGF23 as a Marker of Phenotype Severity.

Hypophosphatasia is a rare heritable metabolic disorder caused by deficient Tissue Non-specific Alkaline Phosphatase (TNAP) enzyme activity. A principal function of TNAP is to hydrolyze the tissue mineralization inhibitor pyrophosphate. ENPP1 (Ectonucleotide Pyrophosphatase/Phosphodiesterase 1) is a primary enzymatic generator of pyrophosphate and prior results showed that elimination of ENPP1 rescued bone hypomineralization of skull, vertebral and long bones to different extents in TNAP null mice.

Pharmacokinetics, Safety, and Efficacy of Chemoembolization with Doxorubicin-Loaded Tightly Calibrated Small Microspheres in Patients with Hepatocellular Carcinoma.

Purpose: This study examines safety, efficacy, and pharmacokinetics of chemoembolization with loadable microspheres ≤100 μm for hepatocellular carcinoma.

Materials And Methods: A pilot safety study was performed in 19 patients with size and dose escalation and then 52 patients were enrolled prospectively and randomly assigned to chemoembolization with TANDEM™ loaded with 150 or 100 mg of doxorubicin.

Results: The mean diameter of the tumors was 7.

Chemoembolization with DC Bead™ for the treatment of hepatocellular carcinoma: an update.

In this review, DC Bead™ for the treatment of hepatocellular carcinoma is discussed. The embolic device and its mechanism of action is described, focusing on the clinical application and the preclinical background. Guidelines for patient selection and management, along with technical considerations for the appropriate use are provided.

Prospective randomized comparison of chemoembolization with doxorubicin-eluting beads and bland embolization with BeadBlock for hepatocellular carcinoma.

The purpose of this study was to evaluate the added role of a chemotherapeutic in transarterial chemoembolization (TACE) of intermediate-stage hepatocellular carcinoma (HCC). The issue is of major importance since, as suggested by recent evidence, hypoxia or incomplete devascularization of the tumor is a potent stimulator of angiogenesis, and there are not many papers supplying level one evidence confirming the value of a chemotherapeutic. The hypothesis was that since drug-eluting bead (DEB)-TACE is standardized and reproducible, a comparison with bland TACE can readily reveal the potential value of the chemotherapeutic.