Objectives: The use of rapid rituximab infusion in certain pediatric populations has generally been regarded as safe. The safety of our institution's rapid rituximab protocol was evaluated.
Methods: The primary end point was the number of and severity of adverse drug reactions. Secondary end points included a description of the patient population defined by the indication, dose, and number of rituximab infusions administered. Additionally, the difference in infusion times in hours of those receiving rapid rituximab infusions versus the theoretical infusion time of subsequent administration rate schedules was defined.
Results: A total of 88 infusions for 22 patients were reviewed. No dose-limiting adverse reactions were observed. Three patients experienced grade 1 isolated infusion-related adverse events during a single infusion encounter. Two of the three patients received additional doses of rapid rituximab infusions without incident, whereas the other patient no longer required rituximab therapy.
Conclusions: The use of a 90-minute rituximab infusion protocol in pediatric patients with non-rheumatic diseases was well tolerated.
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http://dx.doi.org/10.5863/1551-6776-25.3.215 | DOI Listing |
Medicine (Baltimore)
January 2025
Department of Neurology (Nerve-Muscle Unit), Reference Center for Neuromuscular Diseases "AOC," ALS Reference Center, University Hospitals of Bordeaux (Pellegrin Hospital), University of Bordeaux, Bordeaux, France.
Rationale: Locked-in syndrome (and its variant, completely locked-in state) generally has a high mortality rate in the acute setting; however, when induced by conditions such as acute inflammatory polyradiculoneuropathy, it may well be curable such that an attempt at cure should be systematically sought by clinicians.
Patient Concerns: A 52-year-old man presented with acute tetraparesia and areflexia, initially diagnosed as Guillain-Barré syndrome. Despite appropriate treatment, his condition deteriorated, evolving into a completely locked-in state.
BMJ Case Rep
January 2025
Faculty of Health and Medical Sciences, The University of Adelaide, Adelaide, South Australia, Australia.
Immune-mediated necrotising myopathy (IMNM) can be associated with autoantibodies to 3-hydroxy-3-methylglutaryl coenzyme A reductase (anti-HMGCR). We present a case of a man in his 60s with a 13-year history of relapsing anti-HMGCR-positive IMNM, intermittently partially responsive to various treatments including corticosteroids, methotrexate, mycophenolate, intravenous immunoglobulin, abatacept and rituximab. After a repeat presentation with severe weakness, plasmapheresis was commenced, resulting in rapid and significant improvement in muscle strength and biochemical markers, which was sustained for several months.
View Article and Find Full Text PDFNeurol Neuroimmunol Neuroinflamm
March 2025
Department of Neurology, Mayo Clinic, Rochester, MN.
Background And Objectives: While it is well characterized in adults, little is known about the clinical features of neurofascin 155-IgG4 autoimmune nodopathy (NF155-IgG4 AN) in the pediatric population. In this study, we aimed to describe the clinical features and treatment outcomes in children diagnosed with neurofascin 155-IgG4 autoimmune nodopathy (NF155-IgG4 AN).
Methods: Pediatric and adult patients with NF155-IgG4 AN were identified retrospectively through the Mayo Clinic Neuroimmunology Laboratory database.
Mol Genet Metab Rep
March 2025
Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA.
Background: The current standard of care for infantile-onset Pompe disease (IOPD), a severe form of acid α-glucosidase enzyme activity deficiency is: (1) detection by newborn screening, (2) early initiation of intravenous enzyme replacement therapy (ERT) using recombinant human acid alpha-glucosidase (rhGAA), with higher doses of rhGAA increasingly used to improve clinical outcomes, and (3) immune tolerization induction (ITI) using to prevent anti-rhGAA antibody formation, with methotrexate (MTX), rituximab, and IVIG used for patients who are cross-reactive immunologic material negative (CRIM-) and monotherapy with MTX used in patients who are cross-reactive immunologic material positive (CRIM+).
Objectives/methods: A pilot study evaluates a dose-intensive therapy (DIT) using high-dose ERT (40 mg/kg/week) and more frequent exposure to ERT (i.e.
J Clin Med
January 2025
Department of Paediatrics, Nephrology and Hypertension, Medical University of Gdansk, 80-210 Gdansk, Poland.
: Idiopathic nephrotic syndrome (INS) is the most common cause of nephrotic syndrome in children. A hallmark of the disease is the rapid remission of proteinuria following a high dose of steroids. Recurrent disease or steroid dependence are common, leading to a high steroid burden and the introduction of steroid sparing therapy.
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