Optimizing peripubertal growth in a child with short bowel syndrome on full oral feeding with glucagon-like peptide 2 analog.

Teduglutide is a glucagon-like peptide 2 (GLP-2) analog which acts by increasing intestinal absorption of the remnant bowel for children with short bowel syndrome (SBS) dependent on parenteral nutrition. We present a 13-year-old male patient with type 2 SBS (55 cm of jejunum) from necrotizing enterocolitis on full oral feeding from the age of 12 months. Because of faltering growth from the age of 11 despite oral hyperphagia, he started Teduglutide at the standard dose. Eighteen months after Teduglutide start the young boy gained 10 kg in weight and 13 cm in height with a significant reduction in bowel distension. No adverse events were reported during the treatment. Pubertal spurt might be impaired in children with SBS on full oral feeding if the caloric need is not met by the residual intestinal absorption rate. GLP-2 analog might represent an option to sustain pubertal spurt in SBS children on full oral feeding with hyperphagia.

Safety and efficacy of narsoplimab in pediatric and adult patients with transplant-associated thrombotic microangiopathy: a real-world experience.

Transplant-associated thrombotic microangiopathy (TA-TMA) is a severe complication following hematopoietic stem cell transplantation (HSCT). No approved treatments are currently available. This study presents real-world data obtained with narsoplimab, a human immunoglobulin G4 monoclonal antibody that inhibits MASP-2, the effector enzyme of the lectin pathway of the complement system. Between January 2018 and August 2023, 20 (13 adult and 7 pediatric) patients diagnosed with TA-TMA received narsoplimab under an ongoing compassionate use program. The diagnosis was based on internationally defined criteria for pediatric and adult patients. Fifteen patients fulfilled the criteria recently established by an international consensus on TA-TMA. Nineteen patients exhibited high-risk characteristics. Thirteen patients (65%) responded to narsoplimab, achieving transfusion independence and significant clinical improvement. The one-hundred-day Overall Survival (OS) post-TA-TMA diagnosis was 70%, and 100% for responders. Narsoplimab proved to be effective and safe in the treatment of high-risk TA-TMA, with no increased infectious complications or other safety signals of concern across all age groups. The high response rates and the encouraging survival outcomes underscore the potential of narsoplimab as a valuable therapeutic option, particularly for high-risk cases.

Evidence of vertical transmission of SARS-CoV-2 and interstitial pneumonia in second-trimester twin stillbirth in asymptomatic woman. Case report and review of the literature.

Data on the vertical transmission rate of COVID-19 in pregnancy are limited, although data reporting mother-fetal transmission in the second trimester of pregnancy are controversial. We described a case of second-trimester twin stillbirth in a woman with SARS-CoV-2 infection in which placental and fetal markers of infection were detected, despite the absence of respiratory syndrome. The patient developed clinical chorioamnionitis and spontaneously delivered 2 stillborn infants. Placental histology and immunohistochemistry demonstrated SARS-CoV-2 infection mostly within the syncytiotrophoblast, and fetal autopsy showed the development of interstitial pneumonia. Our findings demonstrated that in utero vertical transmission is possible in asymptomatic pregnant women with SARS-CoV-2 infection and that infection can lead to severe morbidity in the second trimester of pregnancy.