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Transient hematologic and clinical effect of E21R in a child with end-stage juvenile myelomonocytic leukemia

Identifieur interne : 000960 ( PascalFrancis/Curation ); précédent : 000959; suivant : 000961

Transient hematologic and clinical effect of E21R in a child with end-stage juvenile myelomonocytic leukemia

Auteurs : Frédéric Bernard [France, Australie] ; Caroline Thomas ; Jean Francois Emile ; Timothy Hercus ; Bruno Cassinat ; Christine Chomienne ; Jean Donadieu

Source :

RBID : Pascal:02-0277138

Descripteurs français

English descriptors

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.
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C01 01    ENG  @0 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.
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C07 03  X  FRE  @0 Myéloprolifératif syndrome @5 38
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C07 03  X  SPA  @0 Mieloproliferativo síndrome @5 38
C07 04  X  FRE  @0 Myélodysplasique syndrome @5 39
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Pascal:02-0277138

Le document en format XML

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<s5>09</s5>
</fC03>
<fC03 i1="09" i2="X" l="SPA">
<s0>Estudio caso</s0>
<s5>09</s5>
</fC03>
<fC03 i1="10" i2="X" l="FRE">
<s0>Récepteur E21R</s0>
<s4>INC</s4>
<s5>86</s5>
</fC03>
<fC07 i1="01" i2="X" l="FRE">
<s0>Homme</s0>
</fC07>
<fC07 i1="01" i2="X" l="ENG">
<s0>Human</s0>
</fC07>
<fC07 i1="01" i2="X" l="SPA">
<s0>Hombre</s0>
</fC07>
<fC07 i1="02" i2="X" l="FRE">
<s0>Hémopathie maligne</s0>
<s5>37</s5>
</fC07>
<fC07 i1="02" i2="X" l="ENG">
<s0>Malignant hemopathy</s0>
<s5>37</s5>
</fC07>
<fC07 i1="02" i2="X" l="SPA">
<s0>Hemopatía maligna</s0>
<s5>37</s5>
</fC07>
<fC07 i1="03" i2="X" l="FRE">
<s0>Myéloprolifératif syndrome</s0>
<s5>38</s5>
</fC07>
<fC07 i1="03" i2="X" l="ENG">
<s0>Myeloproliferative syndrome</s0>
<s5>38</s5>
</fC07>
<fC07 i1="03" i2="X" l="SPA">
<s0>Mieloproliferativo síndrome</s0>
<s5>38</s5>
</fC07>
<fC07 i1="04" i2="X" l="FRE">
<s0>Myélodysplasique syndrome</s0>
<s5>39</s5>
</fC07>
<fC07 i1="04" i2="X" l="ENG">
<s0>Myelodysplastic syndrome</s0>
<s5>39</s5>
</fC07>
<fC07 i1="04" i2="X" l="SPA">
<s0>Mielodisplastico síndrome</s0>
<s5>39</s5>
</fC07>
<fC07 i1="05" i2="X" l="FRE">
<s0>Cytokine</s0>
<s5>53</s5>
</fC07>
<fC07 i1="05" i2="X" l="ENG">
<s0>Cytokine</s0>
<s5>53</s5>
</fC07>
<fC07 i1="05" i2="X" l="SPA">
<s0>Citoquina</s0>
<s5>53</s5>
</fC07>
<fC07 i1="06" i2="X" l="FRE">
<s0>Polypeptide</s0>
<s5>54</s5>
</fC07>
<fC07 i1="06" i2="X" l="ENG">
<s0>Polypeptide</s0>
<s5>54</s5>
</fC07>
<fC07 i1="06" i2="X" l="SPA">
<s0>Polipéptido</s0>
<s5>54</s5>
</fC07>
<fN21>
<s1>161</s1>
</fN21>
<fN82>
<s1>PSI</s1>
</fN82>
</pA>
</standard>
</inist>
</record>

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