Achondroplasia, the most common skeletal dysplasia, is characterized by a variety of medical, functional and psychosocial challenges across the lifespan. The condition is caused by a common, recurring, gain-of-function mutation in FGFR3, the gene that encodes fibroblast growth factor receptor 3. This mutation leads to impaired endochondral ossification of the human skeleton. The clinical and radiographic hallmarks of achondroplasia make accurate diagnosis possible in most patients. However, marked variability exists in the clinical care pathways and protocols practised by clinicians who manage children and adults with this condition. A group of 55 international experts from 16 countries and 5 continents have developed consensus statements and recommendations that aim to capture the key challenges and optimal management of achondroplasia across each major life stage and sub-specialty area, using a modified Delphi process. The primary purpose of this first International Consensus Statement is to facilitate the improvement and standardization of care for children and adults with achondroplasia worldwide in order to optimize their clinical outcomes and quality of life.
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http://dx.doi.org/10.1038/s41574-021-00595-x | DOI Listing |
JAMA Netw Open
January 2025
Division of Neonatology, Department of Pediatrics, Willem-Alexander Children's Hospital, Leiden University Medical Center, Leiden, the Netherlands.
Importance: Preventive efforts in pregnancy-related alloimmunization have considerably decreased the prevalence of hemolytic disease of the fetus and newborn (HDFN). International studies are therefore essential to obtain a deeper understanding of the postnatal management and outcomes of HDFN. Taken together with numerous treatment options, large practice variations among centers may exist.
View Article and Find Full Text PDFMed Care
February 2025
RTI International, Research Triangle Park, NC.
Background: There is a lack of consensus on the effectiveness of audio-based care to manage chronic conditions. This knowledge gap has implications for health policy decisions and for health equity, as underserved populations are more likely to access care by telephone.
Objectives: We compared the effectiveness of audio-based care to usual care for managing chronic conditions (except diabetes).
Cochrane Database Syst Rev
January 2025
Neonatal Intensive Care Unit, Copenhagen University Hospital - Rigshospitalet, Copenhagen, Denmark.
Background: Esophageal atresia is one of the most common life-threatening congenital malformations and is defined as an interruption in the continuity of the esophagus with or without fistula to the trachea or bronchi. Definitive treatment is surgical ligation of the fistula if present and esophageal end-to-end anastomosis of the two pouches, thereby reconstructing the continuity of the esophagus. During this procedure, the surgeon may choose to either ligate or preserve the azygos vein, a major draining vein for the esophagus and surrounding structures, but no definitive consensus on the matter exists.
View Article and Find Full Text PDFCardiovasc Diagn Ther
December 2024
Department of Cardiology, St. Luke's International Hospital, Tokyo, Japan.
Tetralogy of Fallot (TOF) is a condition that often leads to long-term enlargement of the aortic root in after surgery. The aortic dilation is believed to be caused by histological abnormalities of the aortic media and the hemodynamic characteristics of increased aortic flow, compared to pulmonary flow. Severe cyanosis, severe right ventricular outflow tract (RVOT) obstruction, older age at repair, a larger aortic size at the time of repair, and a history of an aortopulmonary shunt parameters related to long-standing volume overload of the aortic root were the reported risk factors.
View Article and Find Full Text PDFNPJ Prim Care Respir Med
January 2025
US Primary Care Respiratory Group, Winnsboro, SC, USA.
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