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


Authors: Scordo, M.; Gilbert, L. J.; Hanley, D. M.; Flynn, J. R.; Devlin, S. M.; Nguyen, L. K.; Ruiz, J. D.; Shah, G. L.; Sauter, C. S.; Chung, D. J.; Landau, H. J.; Lahoud, O. B.; Lin, R. J.; Dahi, P. B.; Perales, M. A.; Giralt, S. A.; Soff, G. A.
Article Title: Open-label pilot study of romiplostim for thrombocytopenia after autologous hematopoietic cell transplantation
Abstract: 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<.001) at days +21 and +30, respectively, compared with those of no romiplostim. Only 1 adverse event was deemed possibly attributable to romiplostim: a low-risk pulmonary embolism in a patient with multiple myeloma. In conclusion, romiplostim showed promising activity and safety after auto-HCT, but the improvement in platelet counts occurred later than the goal of shortening the duration and depth of the platelet nadir. © 2023 by The American Society of Hematology.
Journal Title: Blood Advances
Volume: 7
Issue: 8
ISSN: 2473-9529
Publisher: American Society of Hematology  
Date Published: 2023-04-25
Start Page: 1536
End Page: 1544
Language: English
DOI: 10.1182/bloodadvances.2022007838
PUBMED: 36409612
PROVIDER: scopus
PMCID: PMC10130608
DOI/URL:
Notes: The MSK Cancer Center Support Grant (P30 CA008748) is acknowledged in the PDF -- Corresponding author is MSK author: Michael Scordo -- Source: Scopus
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MSK Authors
  1. Sergio Andres Giralt
    1053 Giralt
  2. Miguel-Angel Perales
    915 Perales
  3. Heather Jolie Landau
    419 Landau
  4. David Chung
    240 Chung
  5. Parastoo Bahrami Dahi
    295 Dahi
  6. Michael Scordo
    367 Scordo
  7. Gunjan Lalitchandra Shah
    419 Shah
  8. Oscar Boutros Lahoud
    133 Lahoud
  9. Richard Jirui Lin
    124 Lin
  10. Josel Dumo Ruiz
    54 Ruiz
  11. Linh Nguyen
    3 Nguyen
  12. Danielle Marie Hanley
    6 Hanley