Medical Professional Publications

Another Option for Low Platelet Counts in Children After Intensive Chemotherapy?

Columbus, OH — May 2017

Platelet transfusion is the only widely accepted treatment for children who develop thrombocytopenia as a result of cancer therapy, but it is not always effective. The potential for adverse reactions, and the difficulty of maintaining platelet levels even after multiple transfusions, mean that the development of other options is crucial.

A recently published case series from physician-researchers at Nationwide Children’s Hospital shows that romiplostim, a drug initially approved for adult chronic immune thrombocytopenia, may be one of those options.

The Pediatric Blood & Cancer publication details the use of romiplostim in five children who were undergoing intensive chemotherapy and/or radiation therapy for solid tumors. Those therapies had to be reduced or delayed as a result of thrombocytopenia. Three patients received multiple platelet transfusions but continued to have difficulty stabilizing platelet counts; one patient had moderate thrombocytopenia with platelet counts low enough to delay therapy but not low enough for transfusion; and one patient’s parents had a religious objection to transfusion.

In each case, platelet counts rose enough to resume regular therapy and the romiplostim was well-tolerated.

“We extrapolated data from the adult literature in making our decision to use it,” says Bhuvana Setty, MD, a member of the Division of Hematology, Oncology and Blood and Marrow Transplant at Nationwide Children’s and senior author of the publication. “We knew the side effect profile was better than some other possibilities, and that there are no known adverse reactions with chemotherapy agents these patients were receiving. Romiplostim can be monitored closely in terms of platelet counts, so we decided to administer it in these patients who developed persistent thrombocytopenia midway through therapy.”

One patient developed thrombocytosis while on romiplostim, but this resolved when the medication was held for a week. Patients who received pelvic radiation therapy had more difficulty with platelet counts initially but also responded well to the drug.

Dr. Setty says romiplostim is easily available, and while clinicians needed to justify its use for two patients, all insurance plans ultimately approved the treatments. Nationwide Children’s is continuing to use the drug on patients in a specific, case-by-case manner, and continuing to see positive results.

“We don’t have enough data to say that romiplostim should be considered standard of care to guard against thrombocytopenia before chemotherapy begins, or know how long a patient should be on the medication.” says Dr. Setty. “We have had success with it, though, and prospective studies are warranted.”

Jacobson AE, Shah N, Setty BA. Romiplostim for therapy-related thrombocytopenia in pediatric malignancies. Pediatric Blood & Cancer. 2017 Feb 2. [Epub ahead of print]

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