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@article{Grainger:2023:10.1182/bloodadvances.2021006014,
author = {Grainger, J and Bussel, J and Tarantino, M and Cooper, N and Beam, D and Despotovic, J and Maschan, A and Wang, K and Eisen, M and Bowers, C},
doi = {10.1182/bloodadvances.2021006014},
journal = {Blood Adv},
pages = {396--405},
title = {A single-arm, long-term efficacy and safety study of subcutaneous romiplostim in children with immune thrombocytopenia.},
url = {http://dx.doi.org/10.1182/bloodadvances.2021006014},
volume = {7},
year = {2023}
}

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TY  - JOUR
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 ≤30 × 109/L. Children received weekly subcutaneous romiplostim (1 μg/kg titrated to 10 μg/kg) to maintain platelets within 50 to 200 × 109/L. A subset underwent bone marrow examinations. The primary end point was percentage of time with platelet response during the first 6 months' treatment (counts ≥50 × 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 ∼3 years, and median average weekly dose was 6.9 μg/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 × 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 ww
AU  - Grainger,J
AU  - Bussel,J
AU  - Tarantino,M
AU  - Cooper,N
AU  - Beam,D
AU  - Despotovic,J
AU  - Maschan,A
AU  - Wang,K
AU  - Eisen,M
AU  - Bowers,C
DO  - 10.1182/bloodadvances.2021006014
EP  - 405
PY  - 2023///
SP  - 396
TI  - A single-arm, long-term efficacy and safety study of subcutaneous romiplostim in children with immune thrombocytopenia.
T2  - Blood Adv
UR  - http://dx.doi.org/10.1182/bloodadvances.2021006014
UR  - https://www.ncbi.nlm.nih.gov/pubmed/35413092
VL  - 7
ER  -

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