Wendy K M Liew1, Christina A Pacak2, Nicole Visyak3, Basil T Darras3, Athos Bousvaros4, Peter B Kang5. 1. Department of Neurology, Boston Children's Hospital and Harvard Medical School, Boston, MA; Neurology Service, Department of Pediatrics, KK Women's and Children's Hospital, Singapore. 2. Child Health Research Institute, Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL. 3. Department of Neurology, Boston Children's Hospital and Harvard Medical School, Boston, MA. 4. Division of Gastroenterology, Hepatology, and Nutrition, Boston Children's Hospital and Harvard Medical School, Boston, MA. 5. Department of Neurology, Boston Children's Hospital and Harvard Medical School, Boston, MA; Division of Pediatric Neurology, Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL. Electronic address: pbkang@ufl.edu.
Abstract
OBJECTIVE: To characterize the longitudinal clinical and electrophysiological patterns of thalidomide neuropathy in children and adolescents. STUDY DESIGN: Retrospective analysis of clinical records at a tertiary care children's hospital, including serial electrophysiological studies. RESULTS: Sixteen patients aged 6-24 years received thalidomide to treat Crohn's disease from 2002 to 2012. Nine subjects had electrophysiological evidence of sensorimotor axonal polyneuropathy, 8 of whom had sensory and/or motor symptoms. The patients with polyneuropathy received thalidomide for 5 weeks to 52 months, with cumulative doses ranging from 1.4 to 207.7 g. All subjects with cumulative doses greater than 60 g developed polyneuropathy, and 4 of the 5 subjects who received thalidomide for more than 20 months developed polyneuropathy. The 7 subjects who had normal neurophysiological studies received therapy for 1 week to 25 months, with cumulative doses ranging from 0.7 to 47 g. In contrast to some previous reports, several patients had sensorimotor polyneuropathies, rather than pure sensory neuropathies. In patients with neuropathy who received therapy for more than 24 months and had 3 or more electromyography studies, the severity of the neuropathy plateaued. CONCLUSIONS: Factors in addition to the total dose may contribute to the risk profile for thalidomide neuropathy, including pharmacogenetic susceptibilities. The severity of the neuropathy does not worsen relentlessly. Children, adolescents, and young adults receiving thalidomide should undergo regular neurophysiological studies to monitor for neuropathy.
OBJECTIVE: To characterize the longitudinal clinical and electrophysiological patterns of thalidomideneuropathy in children and adolescents. STUDY DESIGN: Retrospective analysis of clinical records at a tertiary care children's hospital, including serial electrophysiological studies. RESULTS: Sixteen patients aged 6-24 years received thalidomide to treat Crohn's disease from 2002 to 2012. Nine subjects had electrophysiological evidence of sensorimotor axonal polyneuropathy, 8 of whom had sensory and/or motor symptoms. The patients with polyneuropathy received thalidomide for 5 weeks to 52 months, with cumulative doses ranging from 1.4 to 207.7 g. All subjects with cumulative doses greater than 60 g developed polyneuropathy, and 4 of the 5 subjects who received thalidomide for more than 20 months developed polyneuropathy. The 7 subjects who had normal neurophysiological studies received therapy for 1 week to 25 months, with cumulative doses ranging from 0.7 to 47 g. In contrast to some previous reports, several patients had sensorimotor polyneuropathies, rather than pure sensory neuropathies. In patients with neuropathy who received therapy for more than 24 months and had 3 or more electromyography studies, the severity of the neuropathy plateaued. CONCLUSIONS: Factors in addition to the total dose may contribute to the risk profile for thalidomideneuropathy, including pharmacogenetic susceptibilities. The severity of the neuropathy does not worsen relentlessly. Children, adolescents, and young adults receiving thalidomide should undergo regular neurophysiological studies to monitor for neuropathy.
Authors: Ellen M Lavoie Smith; Clare Kuisell; Grace A Kanzawa-Lee; Celia M Bridges; Paola Alberti; Guido Cavaletti; Rima Saad; Susanna Park Journal: Lancet Haematol Date: 2020-05 Impact factor: 18.959
Authors: Carlotta Spagnoli; Francesco Pisani; Francesco Di Mario; Gioacchino Leandro; Federica Gaiani; Gian Luigi De' Angelis; Carlo Fusco Journal: Acta Biomed Date: 2018-12-17