PMID- 35413092 OWN - NLM STAT- MEDLINE DCOM- 20230201 LR - 20230304 IS - 2473-9537 (Electronic) IS - 2473-9529 (Print) IS - 2473-9529 (Linking) VI - 7 IP - 3 DP - 2023 Feb 14 TI - A single-arm, long-term efficacy and safety study of subcutaneous romiplostim in children with immune thrombocytopenia. PG - 396-405 LID - 10.1182/bloodadvances.2021006014 [doi] AB - Romiplostim is a thrombopoietin (TPO) receptor agonist approved for children and adults with immune thrombocytopenia (ITP) for >/=6 months, recommended as second-line treatment. This phase 3b, single-arm, multicenter study investigated long-term efficacy and safety of romiplostim in children >/=1 to <18 years old with >/=6 months' ITP duration and platelet counts /=50 x 109/L without rescue medication within the preceding 4 weeks). Overall, 203 patients (median age, 10.0 years) received >/=1 dose of romiplostim, median treatment duration was approximately 3 years, and median average weekly dose was 6.9 mug/kg. Ninety-five (46.8%) discontinued (lack of efficacy, n = 43 [21.2%]). Platelet responses were achieved a median (interquartile range) of 50.0% (16.7%-83.3%) of the time during the first 6 months, increasing to 78.2% (26.7%-90.4%) during the overall 36-month treatment period. Eleven patients (5.4%) achieved sustained responses (consecutive counts >/=50 x 109/L without ITP medications for >/=24 weeks). Treatment-related adverse events (AEs) occurred in 56 patients (27.6%), with 8 (3.9%) experiencing serious treatment-related AEs; all of these led to discontinuation, including 4 cases of neutralizing antibodies (romiplostim, n = 3; TPO, n = 1). Bleeding occurred in 141 patients (69.5%), decreasing over time; grade >/=3 bleeding events occurred in 20 (9.9%). At year 2, eight of 63 evaluable patients (12.7%) had grade 2 reticulin. Long-term romiplostim resulted in sustained on-treatment platelet responses with an overall safety profile consistent with previous studies. This trial was registered at www.clinicaltrials.gov as #NCT02279173. CI - (c) 2023 by The American Society of Hematology. Licensed under Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International (CC BY-NC-ND 4.0), permitting only noncommercial, nonderivative use with attribution. All other rights reserved. FAU - Grainger, John AU - Grainger J AUID- ORCID: 0000-0003-2626-0647 AD - Department of Haematology, Royal Manchester Children's Hospital, Faculty of Medical & Human Sciences, University of Manchester, Central Manchester University Hospitals NHS Foundation Trust, Manchester Academic Health Science Centre, Manchester, UK. FAU - Bussel, James AU - Bussel J AD - Department of Pediatrics, Division of Hematology, Weill Cornell Medicine, New York, NY. FAU - Tarantino, Michael AU - Tarantino M AD - The Bleeding and Clotting Disorders Institute, University of Illinois College of Medicine-Peoria, Peoria, IL. FAU - Cooper, Nichola AU - Cooper N AD - Department of Haematology, Hammersmith Hospital, Imperial College, London, UK. FAU - Beam, Donald AU - Beam D AD - Cook Children's Medical Center, Fort Worth, TX. FAU - Despotovic, Jenny AU - Despotovic J AD - Texas Children's Hematology Center, Houston, TX. FAU - Maschan, Alexey AU - Maschan A AUID- ORCID: 0000-0002-0016-6698 AD - Dmitry Rogachev National Research Center for Pediatric Hematology, Oncology and Immunology, Moscow, Russian Federation. FAU - Wang, Kejia AU - Wang K AD - Amgen Inc., Thousand Oaks, CA. FAU - Eisen, Melissa AU - Eisen M AD - Amgen Inc., Thousand Oaks, CA. FAU - Bowers, Charles AU - Bowers C AD - Amgen Inc., Thousand Oaks, CA. LA - eng SI - ClinicalTrials.gov/NCT02279173 PT - Clinical Trial, Phase III PT - Journal Article PT - Multicenter Study PT - Research Support, Non-U.S. Gov't PL - United States TA - Blood Adv JT - Blood advances JID - 101698425 RN - GN5XU2DXKV (romiplostim) RN - 9014-42-0 (Thrombopoietin) RN - 0 (Receptors, Fc) RN - 0 (Recombinant Fusion Proteins) SB - IM MH - Adult MH - Humans MH - Child MH - Adolescent MH - *Purpura, Thrombocytopenic, Idiopathic/drug therapy/chemically induced MH - Thrombopoietin/adverse effects MH - Treatment Outcome MH - Platelet Count MH - *Thrombocytopenia/chemically induced MH - Receptors, Fc/therapeutic use MH - Recombinant Fusion Proteins/adverse effects PMC - PMC9979726 COIS- Conflict-of-interest disclosure: J.G. has received consulting fees from Novartis, Amgen Inc., Ono, Alexion, Biotest, Dova, and Octapharma; has received speakers bureau fees from Novartis and Amgen; and is an ITP Support Association medical advisor. J.B. has participated in advisory boards and received consultancy fees from Amgen Inc., Novartis, Dova, Rigel, UCB, Argenx, Momenta, Regeneron, Kezar, Principia, and CSL-Behring; has participated in speakers bureaus with Novartis and 3S Bio; and has received honoraria from UpToDate. M.T. has received funding for clinical trials from Amgen Inc., Grifols, Novo Nordisk, and Principia; has participated in advisory boards supported by Amgen Inc., Dova, Genentech, Grifols, Novo Nordisk, and Shire/Takeda; and received speakers bureau fees from Novo Nordisk and Shire/Takeda. N.C. has received honoraria for speaking engagements and participated in advisory boards with Novartis, Amgen Inc., Rigel, and Principia; and has received support for clinical trials from Amgen Inc., Novartis, Rigel, Principia, and UCB. J.D. has received research grants from Novartis and consulting fees from Dova and Novartis. A.M. has received lecturer fees from Novartis and Amgen Inc. K.W., M.E., and C.B. are employees of and may hold stock options in Amgen Inc. The remaining author declares no competing financial interests. EDAT- 2022/04/13 06:00 MHDA- 2023/02/02 06:00 PMCR- 2022/04/13 CRDT- 2022/04/12 17:12 PHST- 2022/03/27 00:00 [accepted] PHST- 2021/08/25 00:00 [received] PHST- 2022/04/13 06:00 [pubmed] PHST- 2023/02/02 06:00 [medline] PHST- 2022/04/12 17:12 [entrez] PHST- 2022/04/13 00:00 [pmc-release] AID - 484880 [pii] AID - 10.1182/bloodadvances.2021006014 [doi] PST - ppublish SO - Blood Adv. 2023 Feb 14;7(3):396-405. doi: 10.1182/bloodadvances.2021006014.