We have partnered with international experts to provide rare bone diseases highlights from ASBMR 2021.
This is the third episode in a series of four podcasts. In this episode Charlene Waldman (RBD Alliance) and Inês Alves (European Rare Bone Forum) discuss their highlights.
In this second episode of a three-episode podcast series, COR2ED and ASBMR have partnered with Professor Eric Rush (Children’s Mercy Hospital) to provide rare bone disease highlights from ASBMR 2022 Annual Meeting. Prof. Rush opens with detailed information on fibrodysplasia ossificans progressiva (FOP): symptoms of this severe congenital condition include permanent heterotopic ossifications preceded by painful soft tissue swelling. Nearly all patients have the same ACVR1 receptor variant, and Prof. Rush shares therapy options for the disease. He then offers insights on hypochondroplasia (HCH), a developmental disorder caused by a defect in the FGFR3 gene. An animal model for HCH has been developed, in which infigratinib, a selective FGFR tyrosine kinase inhibitor, shows positive effects on growth as well as proportion. He then describes a clinical study in osteogenesis imperfects (OI), in which allogenic transplantation of fetal liver-derived mesenchymal stem cells was tested in children with type III and type IV OI. While increased bone density and height were observed, Prof. Rush cautions that both the number of study participants and the study duration were low. He closes the podcast with personal insights from the in-person Annual Meeting, including opportunities for engagement and exchange at “Meet the Professor” sessions. ...
COR2ED Medical Education: Pablo Peinado, Research Nurse Coordinator, and Dr. Elena Castro, medical oncologist, who both work at Hospital Universitario Virgen de la Victoria in Malaga in Spain, discuss ‘The potential of PARP inhibitors in prostate cancer: What nurses need to know’. In this podcast, the experts discuss PARP inhibitors (PARPi’s), how they work and key data from the registration trials for rucaparib and olaparib, TRITON2 and PROfound. They also discuss how to select PARPi’s for their prostate cancer patients and where they fit in the overall treatment sequence. Pablo provides some insight into communicating with patients, and the types of questions and concerns they often have in relation to genetic testing and treatment with PARPi’s. In addition, they also discuss the PARPi’s currently in late-stage development such as niraparib and talazoparib as well as the potential future use of PARPi’s in combination with immunotherapies or androgen receptor pathway inhibitors. ...
In this second episode of the podcast series Women and Girls Bleed Too, Haematologist Dr. Roseline d’Oiron (Reference Centre for Haemophilia and RBD, Bicêtre Hospital AP-HP and University of Paris-Saclay in France) and Obstetrician/Gynaecologist Prof. Rezan Abdul-Kadir (The Royal Free Foundation Hospital in London, UK) discuss how to identify women and girls who are likely to have a bleeding disorder. Prof. Abdul-Kadir addresses the steps she takes when she suspects a potential bleeding disorder in one of her patients. Dr. d’Oiron confirms it is key to get a clear understanding of the personal bleeding history and family history. There are several screening tools available to help assess the patient’s signs and symptoms. The experts discuss when a patient should be referred to a Haematologist, it is important to know that Haematologists are easily accessible for referral when in doubt. Finally, Dr. d’Oiron and Prof. Abdul-Kadir address the types of testing that should and should not be done in first line. It turns out there are many misconceptions about laboratory testing for bleeding disorders. For example, contrary to common belief a normal aPTT and PT does not exclude a bleeding disorder. Furthermore, referring to a Haematologist for testing does not mean the patient should remain untreated. Starting basic treatment is crucial to limit the bleeding and address any iron deficiency as quickly as possible. ...
