Safety and Efficacy of Romiplostim in Maintaining Chemotherapy Interval and Dose Intensity in Pediatric Patients With Solid Tumors and Chemotherapy‐Induced Thrombocytopenia

BACKGROUND: Chemotherapy-induced thrombocytopenia (CIT) is a complication of myelosuppressive chemotherapy. Management consists of platelet transfusions, dose reductions, and delays in subsequent chemotherapy cycles, which can lead to a decrease in progression-free survival as well as overall survival in this patient population. Romiplostim is a thrombopoietin receptor agonist (TPO-RA) that binds to and activates the TPO receptor on megakaryocyte precursors, increasing platelet production. Literature to support the routine use of romiplostim in the setting of CIT in pediatric patients is limited. OBJECTIVE: To evaluate the safety profile and efficacy of romiplostim in achieving and maintaining platelets ≥75,000/mL to allow resumption of chemotherapy without recurrence of CIT in pediatric patients with solid tumors. METHODS: This single-center retrospective chart review was conducted at a tertiary pediatric hospital. Patients ≤22 years of age were included if they received romiplostim for CIT from July 1, 2020, to December 31, 2023. The primary outcome of this study was to determine the number of patients who were responsive to romiplostim. RESULTS: A total of 18 patients were included in the analysis. Twelve patients (70%) were responsive to romiplostim. There was an average of two dose reductions prior to romiplostim initiation versus one dose reduction during treatment with romiplostim (p = 0.46) and three delays prior to versus one delay during treatment with romiplostim (p = 0.0008). Average time to platelet count of ≥75,000/µL was 30 days prior to romiplostim versus 15 days during romiplostim. CONCLUSION: Romiplostim may decrease the number of chemotherapy dose reductions and delays in pediatric patients with CIT, but further prospective studies are necessary to determine its place in therapy.

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Journal
Pediatric Blood & Cancer
Published
2026-04-11
DOI
https://doi.org/10.1002/1545-5017.70337
Primary Topic
Platelet Disorders and Treatments
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article
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article

Safety and Efficacy of Romiplostim in Maintaining Chemotherapy Interval and Dose Intensity in Pediatric Patients With Solid Tumors and Chemotherapy‐Induced Thrombocytopenia

Suzanne Smolik, Erica Tonti, Zanette Kanani Bradley, Ashley Sabus
Pediatric Blood & Cancer
Platelet Disorders and Treatments
article

Safety and Efficacy of Romiplostim in Maintaining Chemotherapy Interval and Dose Intensity in Pediatric Patients With Solid Tumors and Chemotherapy‐Induced Thrombocytopenia

Suzanne Smolik, Erica Tonti, Zanette Kanani Bradley, Ashley Sabus
article en

Abstract

BACKGROUND: Chemotherapy-induced thrombocytopenia (CIT) is a complication of myelosuppressive chemotherapy. Management consists of platelet transfusions, dose reductions, and delays in subsequent chemotherapy cycles, which can lead to a decrease in progression-free survival as well as overall survival in this patient population. Romiplostim is a thrombopoietin receptor agonist (TPO-RA) that binds to and activates the TPO receptor on megakaryocyte precursors, increasing platelet production. Literature to support the routine use of romiplostim in the setting of CIT in pediatric patients is limited. OBJECTIVE: To evaluate the safety profile and efficacy of romiplostim in achieving and maintaining platelets ≥75,000/mL to allow resumption of chemotherapy without recurrence of CIT in pediatric patients with solid tumors. METHODS: This single-center retrospective chart review was conducted at a tertiary pediatric hospital. Patients ≤22 years of age were included if they received romiplostim for CIT from July 1, 2020, to December 31, 2023. The primary outcome of this study was to determine the number of patients who were responsive to romiplostim. RESULTS: A total of 18 patients were included in the analysis. Twelve patients (70%) were responsive to romiplostim. There was an average of two dose reductions prior to romiplostim initiation versus one dose reduction during treatment with romiplostim (p = 0.46) and three delays prior to versus one delay during treatment with romiplostim (p = 0.0008). Average time to platelet count of ≥75,000/µL was 30 days prior to romiplostim versus 15 days during romiplostim. CONCLUSION: Romiplostim may decrease the number of chemotherapy dose reductions and delays in pediatric patients with CIT, but further prospective studies are necessary to determine its place in therapy.

Pediatric Blood & Cancer
Children's Hospital Colorado (US), University of Colorado Denver (US)
Good health and well-being
Openalex Percentile: Top 17%
Platelet Disorders and Treatments
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