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Primary endpoints: pharmacokinetics, pharmacodynamics (CD19\n                      <jats:sup>+<\/jats:sup>\n                      B-cell count); secondary endpoint: safety; exploratory endpoints: MRI activity, protocol-defined relapses, Expanded Disability Status Scale (EDSS) score change.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>A total of 23 patients (cohort 1: n\u2009=\u20096, age 10\u201312\u00a0years, BW 27.0\u201339.0\u00a0kg; cohort 2: n\u2009=\u200917, age 11\u201317\u00a0years, BW 42.1\u2013108.4\u00a0kg) were enrolled. Median treatment duration was 120 (range, 24\u2013193) weeks at the primary analysis cutoff (October 5, 2023). Overall, the pharmacokinetic data were within the range observed at 600\u00a0mg in adult patients with MS; however, the exposure at 300\u00a0mg in patients\u2009&lt;\u200940\u00a0kg was lower than with 600\u00a0mg in patients\u2009\u2265\u200940\u00a0kg. Shifting the cutoff to 35\u00a0kg would provide better exposure to patients with 35\u201340\u00a0kg body weight. Sustained, near-complete B-cell depletion was observed. The safety profile was consistent with that in adults. EDSS scores remained stable; no clinical relapses were observed.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>A dosing regimen of 300\u00a0mg ocrelizumab for patients\u2009&lt;\u200935\u00a0kg, and 600\u00a0mg for patients\u2009\u2265\u200935\u00a0kg (every 24\u00a0weeks), was selected for the phase 3 OPERETTA II trial (NCT05123703).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Trial registration<\/jats:title>\n                    <jats:p>\n                      ClinicalTrials.gov:\n                      <jats:underline>NCT04075266<\/jats:underline>\n                      .\n                    <\/jats:p>\n                  <\/jats:sec>","DOI":"10.1007\/s00415-024-12879-z","type":"journal-article","created":{"date-parts":[[2025,1,15]],"date-time":"2025-01-15T08:36:10Z","timestamp":1736930170000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":11,"title":["Ocrelizumab dose selection for treatment of pediatric relapsing\u2013remitting multiple sclerosis: results of the OPERETTA I study"],"prefix":"10.1007","volume":"272","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-8449-2003","authenticated-orcid":false,"given":"Soe","family":"Mar","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Massimiliano","family":"Valeriani","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Barbara","family":"Steinborn","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Teri","family":"Schreiner","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emmanuelle","family":"Waubant","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Massimo","family":"Filippi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katarzyna","family":"Kotulska","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maria","family":"Mazurkiewicz-Beldzinska","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bouchra","family":"El Azzouzi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chien-Ju","family":"Lin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yun-An","family":"Shen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Heidemarie","family":"Kletzl","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joanna","family":"Evershed","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alexandra","family":"Hogea","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Corinne","family":"Manlius","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ulrike","family":"Bonati","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brenda","family":"Banwell","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2025,1,15]]},"reference":[{"key":"12879_CR1","doi-asserted-by":"publisher","first-page":"1285","DOI":"10.1007\/s40263-022-00965-7","volume":"36","author":"L Freeman","year":"2022","unstructured":"Freeman L, Longbrake EE, Coyle PK, Hendin B, Vollmer T (2022) High-efficacy therapies for treatment-na\u00efve individuals with relapsing-remitting multiple sclerosis. 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Massimiliano Valeriani is Chief Editor of\n                      Pain Research and Management\n                      and Associate Editor of\n                      Frontiers in Neurology\n                      and\n                      BMC Neurology\n                      ; received compensation for speaking activities from Novartis and Roche; and participates in clinical trials funded by F. Hoffmann-La Roche Ltd, Amgen, Biogen, Eli Lilly, and Alexion. Barbara Steinborn participates in clinical trials funded by F. Hoffmann-La Roche Ltd, Biogen, and Novartis. Teri Schreiner has received consultancy fees from Roche and Cycle Pharmaceuticals. She has received support from the National MS Society and the Center for Disease Control and Prevention, and she participates in clinical trials funded by Roche. Emmanuelle Waubant is funded by the National Institutes of Health, National MS Society, Department of Defense, Consortium of Multiple Sclerosis Centers, Race to Erase MS, and Patient-Centered Outcomes Research Institute. She is a site investigator for trials with Biogen, Alexion, and Roche. She has received honoraria for talks from Yoga Moves MS, Neurology Live, and Advanced Curriculum. Massimo Filippi is Editor-in-Chief of the\n                      Journal of Neurology\n                      and Associate Editor of\n                      Human Brain Mapping\n                      ,\n                      Neurological Sciences\n                      , and\n                      Radiology\n                      ; received compensation for consulting services from Alexion, Almirall, Biogen, Merck, Novartis, Roche, and Sanofi; received fees for speaking activities from Bayer, Biogen, Celgene, Chiesi Italia SpA, Eli Lilly, Genzyme, Janssen, Merck-Serono, Neopharmed Gentili, Novartis, Novo Nordisk, Roche, Sanofi, Takeda, and Teva; participated in advisory boards for Alexion, Biogen, Bristol Myers Squibb, Merck, Novartis, Roche, Sanofi, Sanofi-Aventis, Sanofi-Genzyme, and Takeda; provided scientific direction of educational events for Biogen, Merck, Roche, Celgene, Bristol Myers Squibb, Eli Lilly, Novartis, and Sanofi-Genzyme; and receives research support from Biogen Idec., Merck-Serono, Novartis, Roche, the Italian Ministry of Health, the Italian Ministry of University and Research, and Fondazione Italiana Sclerosi Multipla. Katarzyna Kotulska is a principal investigator in Roche, Novartis, and Biogen clinical studies; and has participated in advisory boards for Roche, Biogen, and Novartis. Maria Mazurkiewicz-Beldzinska is a principal investigator in Roche clinical studies and has participated in advisory boards for Roche. Alexandra Hogea, Joanna Evershed, and Chien-Ju Lin are employees of and shareholders in Roche Products Ltd. Yun-An Shen is an employee of Genentech, Inc., South San Francisco, CA, USA, and a shareholder in F. Hoffmann-La Roche Ltd. Heidemarie Kletzl, Corinne Manlius, Ulrike Bonati, and Bouchra El Azzouzi are employees of and shareholders in F. Hoffmann-La Roche Ltd. Brenda Banwell has served as a consultant to Novartis, Biogen, F. Hoffmann-La Roche Ltd, UCB, Teva Neuroscience, Janssen, and the University of Texas Southwestern Medical Center.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of interest"}}],"article-number":"137"}}