A group of three case reports recently published in Pediatric Blood & Cancer highlights the potential of romiplostim in treating different forms of thrombocytopenia in infants, possibly including fetal and neonatal alloimmune thrombocytopenia (FNAIT).
Thrombocytopenia, defined as low levels of platelets, can arise due to a number of causes including FNAIT, sepsis, genetic disorders and more. Although platelet transfusions are often administered to infants experiencing thrombocytopenia, the procedure carries risks including bleeding and allergic reactions.
Romiplostim promotes platelet production and is approved for patients one year of age and older with chronic immune thrombocytopenic purpura (ITP). Recent studies have begun exploring the use of romiplostim in other types of thrombocytopenia, as well as in patients younger than one year.
In this study, the authors present three cases of newborns with thrombocytopenia who were successfully treated with romiplostim.
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The first case detailed a male infant who was born at 23 weeks’ gestation. His platelet levels began dropping at three days of age, requiring several platelet transfusions. Although FNAIT and other forms of immune thrombocytopenia seemed unlikely based on clinical presentations, intravenous immunoglobulin temporarily improved the patient’s platelet counts, indicating possible immune involvement.
The infant was prescribed seven doses of romiplostim, after which he did not require any more platelet transfusions.
The second infant was a female born at 32 weeks’ gestation due to low amniotic fluid and fetal distress. She required resuscitation after delivery, mechanical ventilation and a form of dialysis known as continuous renal replacement therapy (CRRT). Daily platelet transfusions were ineffective at raising her platelet levels.
The patient received romiplostim for one week with minimal improvements in platelet counts. After the dose was increased and CRRT was discontinued, her platelet levels increased, making it difficult to determine the role of romiplostim versus CRRT discontinuation in improving platelet counts. Her platelet levels dropped slightly after resuming CRRT, but they have returned to normal since switching to a different form of dialysis.
The third case described a female who was born at 41 weeks’ gestation with severe anemia and thrombocytopenia. Her condition resulted from Rh incompatibility, meaning the mother did not have the Rh protein on her red blood cells while the infant did. This caused the mother to produce antibodies against the infant’s red blood cells.
The patient received one dose of intravenous immunoglobulin therapy, as well as daily blood transfusions and platelet transfusions. Romiplostim was then prescribed, and the patient’s platelet counts returned to normal levels after two doses.
“These three cases demonstrate the notable success of utilizing romiplostim in the [neonatal intensive care unit] to spare platelet transfusions without adverse events and invite further investigation into this topic,” the study concluded.
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