Long-term follow-up of 64 children with classical infantile-onset Pompe disease since 2004: A French real-life observational study.

Background: Classical infantile-onset Pompe disease (IOPD) is the most severe form of Pompe disease. Enzyme replacement therapy (ERT) has significantly increased survival but only a few studies have reported long-term outcomes.

Methods: We retrospectively analyzed the outcomes of classical IOPD patients diagnosed in France between 2004 and 2020.

Severe hemolysis after plasma transfusion in a neonate with necrotizing enterocolitis, Clostridium perfringens infection, and red blood cell T-polyagglutination.

Background: Red blood cell (RBC) Thomsen-Friedenreich antigen exposure (T activation) in infants with necrotizing enterocolitis (NEC) has occasionally been associated with posttransfusional intravascular hemolysis thought to be due to anti-T antibodies in the donor plasma.

Study Design And Methods: We describe an infant with NEC and Clostridium perfringens infection complicated by severe hemolysis after plasma transfusion. After this case, infants with confirmed NEC were prospectively evaluated for T activation.

Variability of Care in Infants with Severe Bronchiolitis: Less-Invasive Respiratory Management Leads to Similar Outcomes.

Objective: To compare the management of children with severe bronchiolitis requiring intensive care (based on duration of ventilatory support and duration of pediatric intensive care unit [PICU] stay) in 2 countries with differing pediatric transport and PICU organizations.

Study Design: This was a prospective observational care study in 2 PICUs of tertiary care university hospitals, 1 in France and 1 in Canada. All children with bronchiolitis who required admission to the PICU between November 1, 2013, and March 31, 2014, were included.

Red blood cell Thomsen-Friedenreich antigen expression and galectin-3 plasma concentrations in Streptococcus pneumoniae-associated hemolytic uremic syndrome and hemolytic anemia.

Background: Pneumococcal hemolytic uremic syndrome (P-HUS) is a rare but severe complication of invasive pneumococcal disease (IPD) in young children. Consensual biologic diagnosis criteria are currently lacking.

Study Design And Methods: A prospective study was conducted on 10 children with culture-confirmed IPD.

Cerebral venous thrombosis after embolization of pediatric AVM with jugular bulb stenosis or occlusion: management and prevention.

Purpose: Thrombosis of cerebral arteriovenous malformation after embolization is rare, but can involve the normal venous network with extensive venous thrombosis. We report angioarchitecture findings, our management and prevention strategy for this complication in pediatric AVMs.

Methods: In this 5.