Searchable abstracts of presentations at key conferences on calcified tissues

ba0006p029 | (1) | ICCBH2017

Web-based surveys using Patient-Reported Outcome Measurement Information System (PROMIS) instruments allow documentation of important components of the disease experience among individuals with Osteogenesis Imperfecta

Tosi Laura , Floor Marianne , Holland Rosalie , Goerlich Cara , Hart Tracy , Cuthbertson David , Sutton V Reid , Krischer Jeffrey

Objectives: Two important goals of the Rare Diseases Clinical Research Network Brittle Bone Disorders Consortium (RDCRN BBD) are i) to perform collaborative clinical research in brittle bone disorders including a longitudinal observational study driven by genotypic association and ii) to explore use of the PROMIS tool to provide valid quality of life (QOL) measures in individuals with Osteogenesis Imperfecta (OI). This work has been motivated by the fact that all current outco...

ba0007oc7 | (1) | ICCBH2019

Comparison of zoledronate and pamidronate in children with skeletal disorders: Short term safety experience from a single institution

Tosi Laura , Estrada Andrea , Floor Marianne , Kim Mirini , Weigley Lindsay , Dollar Christina , Gillies Austin , Roberts Mary Scott , Gafni Rachel , Boyce Alison

Objectives: Bisphosphonates are frequently used in children with skeletal disorders, however optimal dosing and regimens are unknown. Early treatment focused on pamidronate (PAM), a second-generation formulation, however use of zoledronate (ZOL), a more potent third-generation bisphosphonate, has recently increased due to shorter and less frequent infusions. The objective of this study is to compare short-term safety of ZOL and PAM in a pediatric population.<p class="abste...

ba0002p180 | (1) | ICCBH2013

Beyond brittle bones: a preliminary report from the osteogenesis imperfecta adult natural history initiative

Tosi Laura , McKiernan Fergus , Oetgen Matthew , Rak Melanie , Tucker Carole , Mulroy Kyle , Simmonds Barbara , Mancuso Angela , Kennelly Ann , Greco Lauren , Blankenship Winslow , Floor Marianne , Huber Mary Beth , Hart Tracy

Background: Osteogenesis imperfecta (OI) is a heterogeneous, rare disorder most commonly affecting type I collagen. The OI Adult Natural History Initiative (OI ANHI) was established following a 2010 Ostegenesis Imperfecta Foundation (OIF) national meeting at which patient participants noted that i) there is little information about the natural history and progression of OI beyond childhood, and ii) most of the data available are focused on musculoskeletal issues. Adults with O...