Increasing platelets without transfusion: is it time to introduce novel thrombopoietic agents in neonatal care?
The Food and Drug Administration recently approved two novel thrombopoiesis-stimulating agents, Romiplostim (AMG-531, Nplate) and Eltrombopag (Promacta), for the treatment of adults with immune thrombocytopenic purpura. For physicians taking care of critically ill neonates, this offers the opportunity of decreasing platelet transfusions and potentially improving the outcomes of neonates with severe and prolonged thrombocytopenia. However, several developmental factors need to be taken into consideration. First, the population of thrombocytopenic neonates likely to benefit from these agents needs to be carefully selected. Second, the mechanisms underlying neonatal and adult thrombocytopenia differ from each other and are incompletely understood, and pre-clinical evidence suggests that the response of neonates to thrombopoietic factors might be different from that of adults. Finally, the potential non-hematopoietic effects of thrombopoietin have not been well established. Here, we will discuss these issues in detail, and will highlight the critical developmental differences between neonates and adults that need to be considered as we think about introducing these agents into neonatal care.
Sallmon
H
H
Division of Newborn Medicine, Children's Hospital Boston and Harvard Medical School, 300 Longwood Avenue, Boston, MA 02115, USA.
Gutti
R K
RK
Ferrer-Marin
F
F
Liu
Z-J
ZJ
Sola-Visner
M C
MC
D000328:Adult; D000367:Age Factors; D017209:Apoptosis / Q000187:drug effects; D001565:Benzoates / Q000009:adverse effects / Q000627:therapeutic use*; D001921:Brain / Q000187:drug effects; D004305:Dose-Response Relationship, Drug; D017277:Drug Approval; D006801:Humans; D006834:Hydrazines / Q000009:adverse effects / Q000627:therapeutic use*; D007231:Infant, Newborn; D009474:Neurons / Q000187:drug effects; D018579:Patient Selection; D017713:Platelet Transfusion; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000097:blood / Q000188:drug therapy*; D011720:Pyrazoles / Q000009:adverse effects / Q000627:therapeutic use*; D011961:Receptors, Fc / Q000627:therapeutic use*; D053628:Receptors, Thrombopoietin / Q000819:agonists*; D011993:Recombinant Fusion Proteins / Q000009:adverse effects / Q000627:therapeutic use*; D054098:Thrombocytopenia, Neonatal Alloimmune / Q000097:blood / Q000188:drug therapy*; D034061:Thrombopoiesis / Q000187:drug effects*; D013926:Thrombopoietin / Q000009:adverse effects / Q000627:therapeutic use*; D014481:United States; D014486:United States Food and Drug Administration
D001565:Benzoates; D006834:Hydrazines; D011720:Pyrazoles; D011961:Receptors, Fc; D053628:Receptors, Thrombopoietin; D011993:Recombinant Fusion Proteins; D013926:Thrombopoietin; C488777:romiplostim; C520809:eltrombopag