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3. Targeted Treatments for Myasthenia Gravis in Children and Adolescents.

Author

Ramdas, Sithara, Painho, Teresa, Vanegas, Maria I., Famili, Dennis T., Lim, Ming J., and Jungbluth, Heinz

Subjects
*NEUROMUSCULAR diseases, *RANDOMIZED controlled trials, *MYONEURAL junction, *QUALITY of life, *PREVENTIVE medicine, *MYASTHENIA gravis
Abstract

Myasthenia gravis (MG) is an antibody-mediated disorder of the neuromuscular junction affecting children and adults. MG is a treatable condition with most patients requiring immunosuppression for disease control and/or remission. Juvenile myasthenia gravis (JMG) is rare in comparison with adult-onset MG but given the same underlying pathophysiology, treatment strategies are similar to those in adults. Until recently, there were only a few randomised controlled trials (RCTs) for MG treatments in adults and none in children, and management strategies were primarily based on expert consensus. In addition, treatment options for refractory MG cases have been severely limited, resulting in poor long-term quality of life in such patients due to the significant disease burden. Recently, there have been several RCTs focussing on novel therapeutic strategies with potentially promising outcomes, suggesting a change in MG management over the coming years and access to more effective and faster-acting drugs for MG patients. This paper will review current and new MG treatments including efgartigimod, eculizumab, rozanolixizumab, ravulizumab, and zilucoplan, with a focus on juvenile myasthenia gravis. [ABSTRACT FROM AUTHOR]

Published
2024
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4.  Pediatric Chronic Inflammatory Demyelinating Polyneuropathy: Challenges in Diagnosis and Therapeutic Strategies .

Author

Alawneh, Issa, Alenizi, Asmaa, Paiz, Freddy, Nigro, Elisa, Vajsar, Jiri, and Gonorazky, Hernan

Subjects
*CHRONIC inflammatory demyelinating polyradiculoneuropathy, *INTRAVENOUS immunoglobulins, *SYMPTOMS, *NEUROLOGICAL disorders, *AUTOIMMUNE diseases, *CEREBROSPINAL fluid examination
Abstract

Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare autoimmune neurological disorder seen in both pediatric and adult populations. CIDP typically presents with progressive and persistent weakness over at least 4 weeks in addition to sensory symptoms in the extremities. Although CIDP shares common clinical features between children and adults, it sometimes presents as a distinct clinical entity in children that requires close attention and recognition. A major caveat when diagnosing a child with CIDP is the clinical and diagnostic overlap with inherited neuropathies, most commonly Charcot–Marie–Tooth disease (CMT). Demyelinating CMT (dCMT) and CIDP might share similar clinical presentations, and sometimes it might be difficult to differentiate them on the basis of the electrodiagnostic findings or cerebrospinal fluid (CSF) albumino-cytological dissociation. This indeed merits early consideration for genetic testing in patients who do not respond to conventional CIDP therapies. Current treatment options for CIDP include intravenous immunoglobulins (IVIG), corticosteroids (CS), and plasmapheresis (PLEX). The need for novel therapies is essential in instances where patients continue to have symptoms despite the standard therapies or due to adverse effects of long-term use of standard therapies such as CS. This paper reviews the challenges in the diagnosis of CIDP in children and the current as well as novel therapies for CIDP. [ABSTRACT FROM AUTHOR]

Published
2024
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9. Effectiveness and Safety of Ustekinumab in Pediatric Ulcerative Colitis: A Multi-center Retrospective Study from the Pediatric IBD Porto Group of ESPGHAN

Author

Cohen, Shlomi, Rolandsdotter, Helena, Kolho, Kaija-Leena, Turner, Dan, Tzivinikos, Christos, Bramuzzo, Matteo, Pujol-Muncunill, Gemma, Scarallo, Luca, Urlep, Darja, Rinawi, Firas, Granot, Maya, Kang, Ben, Longueville, Ylva, Rodríguez-Belvís, Marta Velasco, Weintraub, Yael, Navas-López, Víctor Manuel, and Yerushalmy-Feler, Anat

Published
2024
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15. CROCuS, a Phase II Study Evaluating the Antiviral Activity, Clinical Outcomes, and Safety of Rilematovir in Children Aged ≥ 28 Days and ≤ 3 Years with Acute Respiratory Tract Infection Due to Respiratory Syncytial Virus

Author

Ferrero, Fernando, Lin, Chien-Yu, Liese, Johannes, Luz, Kleber, Stoeva, Tatyana, Nemeth, Agnes, Gijón, Manuel, Calvo, Cristina, Natalini, Silvina, Toh, Teck-Hock, Deleu, Sofie, Chen, Bohang, Rusch, Sarah, Sánchez, Beatriz López, Leipoldt, Illse, Vijgen, Leen, Huntjens, Dymphy, Baguet, Tristan, Bertzos, Kristi, Gamil, Mohamed, and Stevens, Marita

Published
2024
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