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Romiplostim for Immune Thrombocytopenia in Neuroblastoma Patients Receiving Chemotherapy

Fassel, Hannah, MD*,†; Bussel, James B., MD*,†; Roberts, Stephen S., MD*; Modak, Shakeel, MD*

Journal of Pediatric Hematology/Oncology: May 2019 - Volume 41 - Issue 4 - p e257–e259
doi: 10.1097/MPH.0000000000001187
Online Articles: Clinical and Laboratory Observations

Thrombocytopenia, a serious complication of myelosuppressive chemotherapy in cancer patients, is managed with platelet transfusions until recovery of platelet counts. However, children receiving chemotherapy can rarely develop immune thrombocytopenia (ITP) that is refractory to transfused platelets. This limits the ability to achieve adequate platelet counts and administer further myelosuppressive chemotherapy safely, especially if first-line ITP therapy is ineffective. We report 2 cases of intravenous immunoglobulin refractory ITP in children receiving chemotherapy for high-risk neuroblastoma. ITP was successfully treated with the thrombopoietin-receptor-agonist romiplostim, allowing safe and timely continuation of antineuroblastoma therapies in these high-risk patients.

*Department of Pediatrics, Memorial Sloan-Kettering Cancer Center

Department of Pediatrics, Division of Hematology-Oncology, Weill Cornell Medicine, New York, NY

Supported by the MSK Cancer Center Support Grant P30 CA008748.

J.B.B. has received clinical research support from Amgen and Novartis and served on advisory boards for them. The remaining authors declare no conflict of interest.

Reprints: Shakeel Modak, MD, Department of Pediatrics, Memorial Sloan-Kettering Cancer Center, 1275 York Avenue, New York, NY 10065 (e-mail:

Received November 17, 2017

Accepted March 27, 2018

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