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Romiplostim for temozolomide-induced thrombocytopenia in glioblastoma: The PLATUM trial
Emilie Le Rhun1, Patrick Devos1, Caroline Houillier1
1From the General and Stereotaxic Neurosurgery Service, Neuro-oncology (E.L.R.), EA 2694-Santé Publique: Épidémiologie et Qualité des Soins (P.D.) and Onco thAI, INSERM U1189 (N.R.), Université de Lille, Research Federation (C.L.), and Department of Neurosurgery (N.R., F.D.), CHU Lille; Service de Neurologie 2-Mazarin, APHP, Sorbonne Université, IHU, ICM (C.H.), and Service de Neurologie 2-Mazarin (A.L.D.S.), Groupe Hospitalier Pitié-Salpêtrière, Paris; Service de Neuro-oncologie (S.C.), Hospices Civils de Lyon, Hôpital Neurologique; Service de Neuro-Oncologie (O.C.), Aix-Marseille University, AP-HM, CHU Timone, Marseille, France; and Department of Neurology & Brain Tumor Center (M.W.), University Hospital and University of Zurich, Switzerland.
Objective:
To determine the efficacy of the thrombopoietin receptor agonist romiplostim for the prevention of temozolomide-induced thrombocytopenia in newly diagnosed glioblastoma.
Methods:
In the PLATUM phase II open-label, multicenter, single-arm trial, patients diagnosed with Common Terminology Criteria for Adverse Events grade 3 or 4 thrombocytopenia during chemoradiotherapy received weekly subcutaneous romiplostim injections. PLATUM aimed at demonstrating that the percentage of thrombocytopenic patients treated with romiplostim able to complete 6 cycles of maintenance temozolomide chemotherapy exceeded 10% (p0 = 0.10; pA = 0.35). Using type I error equal to 0.05% and 95% power, 31 patients had to be recruited. According to a Fleming 2-step design with a preplanned interim analysis after recruitment of 20 patients (step 1), the trial was terminated early for success.
Results:
Twenty patients were enrolled in step 1. Median age was 61 years (range 33-73). Twelve patients received 6 temozolomide cycles, corresponding to a success rate of 60% (95% confidence interval 36%-81%). Four patients discontinued temozolomide because they did not respond to romiplostim, 2 for progression, and 2 for adverse events unrelated to romiplostim.
Conclusion:
The thrombopoietin receptor agonist romiplostim improves exposure to chemotherapy in patients with glioblastoma experiencing temozolomide-induced thrombocytopenia.
Clinicaltrialsgov Identifier:
NCT02227576.
Classification Of Evidence:
This study provides Class IV evidence that for patients with glioblastoma and thrombocytopenia, romiplostim is effective for the secondary prophylaxis of temozolomide-induced thrombocytopenia.
