Open-label pilot study of romiplostim for thrombocytopenia after autologous hematopoietic cell transplantation

•There are no prior prospective studies of thrombopoietin receptor agonists to enhance platelet recovery after auto-HCT.•As studied, romiplostim did not shorten the duration and depth of the platelet nadir, but it enhanced platelet recovery after day +15. [Display omitted] There are no standard trea...

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Published in:	Blood advances 2023-04, Vol.7 (8), p.1536-1544
Main Authors:	Scordo, Michael, Gilbert, Leah J., Hanley, Danielle M., Flynn, Jessica R., Devlin, Sean M., Nguyen, Linh K., Ruiz, Josel D., Shah, Gunjan L., Sauter, Craig S., Chung, David J., Landau, Heather J., Lahoud, Oscar B., Lin, Richard J., Dahi, Parastoo B., Perales, Miguel-Angel, Giralt, Sergio A., Soff, Gerald A.
Format:	Article
Language:	eng
Subjects:	Clinical Trials and Observations Hematopoietic Stem Cell Transplantation - adverse effects Humans Multiple Myeloma - drug therapy Pilot Projects Retrospective Studies Thrombocytopenia - drug therapy Thrombocytopenia - etiology
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Summary:	•There are no prior prospective studies of thrombopoietin receptor agonists to enhance platelet recovery after auto-HCT.•As studied, romiplostim did not shorten the duration and depth of the platelet nadir, but it enhanced platelet recovery after day +15. [Display omitted] There are no standard treatments to prevent or hasten the recovery from severe conditioning-regimen–induced thrombocytopenia occurring after autologous hematopoietic cell transplantation (auto-HCT). We conducted an open-label, single-arm pilot study of romiplostim, a thrombopoietin receptor agonist, to enhance platelet recovery in patients with multiple myeloma or lymphoma undergoing auto-HCT. All patients were treated weekly with romiplostim starting day +1 after auto-HCT until the platelet count was >50 × 109/L without transfusion. Compared with contemporary retrospective data from romiplostim-naïve patients (N = 853), romiplostim-treated patients (N = 59) had a similar median number of days of grade 4 thrombocytopenia or days requiring transfusions, time to platelet engraftment, and number of platelets transfusions during the auto-HCT. However, romiplostim-treated patients had enhanced platelet recovery to normal values beginning at approximately day +15. In matched cohort multivariable analyses, romiplostim treatment was associated with higher platelet counts by an average of 40 × 109/L (95% confidence interval (CI) (14, 67), P = .003) and 118 × 109/L (95% CI [84, 152], P
ISSN:	2473-9529 2473-9537