title: Transient hematologic and clinical effect of E21R in a child with end-stage juvenile myelomonocytic leukemia.
abstract: E21R is a modified granulocyte macrophage-colony-stimulating factor (GM-CSF) protein which results in antagonism of GM-CSF function via selective binding to the GM-CSF receptor complex. Juvenile chronic myelomonocytic leukemia (JMML) is a rare leukemia where spontaneous proliferation of myeloid and monocytic precursors in patients' bone marrow cultures is dependent on GM-CSF. For patients who progress after systemic chemotherapy, there are no effective therapies. In vitro and in vivo studies in an animal model demonstrating that E21R exerts an antileukemic action prompted us to consider its potential utility in a child with end-stage JMML. E21R was well-tolerated during the 3 courses of subcutaneous treatment. A clear in vivo efficacy was observed after 2 courses of E21R but the disease appeared completely refractory during the third course. This novel therapeutic approach clearly deserves further evaluation in JMML.
authors.affiliation: Pediatric Department III, Hemato-Oncology Unit, Hôpital Arnaud de Villeneuve, Avenue du Doyen Giraud, F-34000 CHU Montpellier, France. f-bernard@chu-montpellier.fr
authors.forename: Frédéric
authors.initials: F
authors.lastname: Bernard
authors.forename: Caroline
authors.initials: C
authors.lastname: Thomas
authors.forename: Jean François
authors.initials: JF
authors.lastname: Emile
authors.forename: Timothy
authors.initials: T
authors.lastname: Hercus
authors.forename: Bruno
authors.initials: B
authors.lastname: Cassinat
authors.forename: Christine
authors.initials: C
authors.lastname: Chomienne
authors.forename: Jean
authors.initials: J
authors.lastname: Donadieu
mesh_terms: D019943:Amino Acid Substitution; D001120:Arginine; D002648:Child; D016903:Drug Monitoring; D018698:Glutamic Acid; D016178:Granulocyte-Macrophage Colony-Stimulating Factor / Q000097:blood / Q000235:genetics* / Q000627:therapeutic use; D006801:Humans; D015479:Leukemia, Myelomonocytic, Acute / Q000097:blood / Q000188:drug therapy / Q000235:genetics*; D008297:Male; D020125:Mutation, Missense; D016896:Treatment Outcome
chemical_list: D018698:Glutamic Acid; D016178:Granulocyte-Macrophage Colony-Stimulating Factor; D001120:Arginine