@article{Michael D. Tarantino_James B. Bussel_Victor S. Blanchette_Donald Beam_John Roy_Jenny Despotovic_Ashok Raj_Nancy Carpenter_Bhakti Mehta_Melissa Eisen_2019, place={Pavia, Italy}, title={Long-term treatment with romiplostim and treatment-free platelet responses in children with chronic immune thrombocytopenia}, volume={104}, url={https://haematologica.org/article/view/9131}, DOI={10.3324/haematol.2018.202283}, abstractNote={Children with immune thrombocytopenia for ≥6 months completing a romiplostim study received weekly subcutaneous romiplostim (1-10 μg/kg targeting platelet counts of 50-200×10<sup>9</sup>/L) in this extension to examine romiplostim’s long-term safety and efficacy. Sixty-five children received romiplostim for a median of 2.6 years (range: 0.1-7.0 years). Median baseline age was 11 years (range: 3-18 years) and platelet count was 28×10<sup>9</sup>/L (range: 2-458×10<sup>9</sup>/L). No patient discontinued treatment for an adverse event. Median average weekly dose was 4.8 mg/kg (range: 0.1-10 mg/kg); median platelet counts remained >50×10<sup>9</sup>/L, starting at week 2. Nearly all patients (94%) had ≥1 platelet response (≥50×10<sup>9</sup>/L, no rescue medication in the previous 4 weeks), 72% had responded at ≥75% of visits, and 58% had responded at ≥90% of visits. Treatment-free response (platelets ≥50×10<sup>9</sup>/L ≥24 weeks without immune thrombocytopenia treatment) was seen in 15 of 65 patients while withholding romiplostim doses. At onset of treatment-free response, the nine girls and six boys had a median immune thrombocytopenia duration of four years (range: 1-12 years) and had received romiplostim for two years (range: 1-6 years). At last observation, treatment-free responses lasted for a median of one year (range: 0.4-2.1 years), with 14 of 15 patients still in treatment-free response. Younger age at first dose and platelet count >200×10<sup>9</sup>/L in the first four weeks were associated with treatment-free responses. In this 7-year open-label extension, three-quarters of the patients responded ≥75% of the time, and romiplostim was well tolerated, with no substantial treatment-related adverse events. Importantly, 23% of children maintained treatment-free platelet responses while withholding romiplostim and all other immune thrombocytopenia medications for ≥6 months. (Registered at <em><a href="http://clinicaltrials.gov">clinicaltrials.gov</a> identifier: 01071954</em&gt;)}, number={11}, journal={Haematologica}, author={Michael D. Tarantino and James B. Bussel and Victor S. Blanchette and Donald Beam and John Roy and Jenny Despotovic and Ashok Raj and Nancy Carpenter and Bhakti Mehta and Melissa Eisen}, year={2019}, month={Oct.}, pages={2283-2291} }