AustralieFrV1, Main, Exploration, bibRecord, 006856

Outcomes after related and unrelated umbilical cord blood transplantation for hereditary bone marrow failure syndromes other than Fanconi anemia

Identifieur interne : 006856 ( Main/Exploration ); précédent : 006855; suivant : 006857

Outcomes after related and unrelated umbilical cord blood transplantation for hereditary bone marrow failure syndromes other than Fanconi anemia

Auteurs : Renata Bizzetto [France, Brésil] ; Carmen Bonfim [Brésil] ; Vanderson Rocha [France] ; Gérard Socie [France] ; Franco Locatelli [Italie] ; Kawah Chan [États-Unis] ; Oscar Ramirez [Colombie] ; Joel Stein [Israël] ; Samir Nabhan [Brésil] ; Eliana Miranda [Brésil] ; Jakob Passweg [Suisse] ; Carmino Antonio De Souza Ca [Brésil] ; Eliane Gluckman [France]

Source :

Haematologica : (Roma) [ 0390-6078 ] ; 2011.

RBID : Pascal:11-0165931

Descripteurs français

Pascal (Inist)
- Homogreffe, Pronostic, Donneur non apparenté, Insuffisance médullaire, Cordon ombilical, Cellule souche, Syndrome de De Toni-Debré-Fanconi, Cellule hématopoïétique, Cellule sanguine, Moelle osseuse, Héréditaire, Prise greffe, Système HLA, Système histocompatibilité majeur, Compatibilité, Hématologie, Aminoacidurie, Syndrome de Fanconi.
Wicri :
- topic : Cellule souche.

English descriptors

KwdEn :
- Aminoaciduria, Blood cell, Bone marrow, Bone marrow failure, Compatibility, De Toni-Debré-Fanconi syndrome, Engraftment, HLA-System, Hematology, Hematopoietic cell, Hereditary, Homograft, Major histocompatibility system, Prognosis, Stem cell, Umbilical cord, Unrelated donor.

Abstract

Background Allogeneic stem cell transplantation is the only curative option for patients with hereditary bone marrow failure syndromes. Umbilical cord blood is an alternative source of stem cells for allogeneic transplantation. Design and Methods This multicenter, retrospective study is based on data reported to the Eurocord Registry about patients with hereditary bone marrow failure syndrome who underwent umbilical cord blood transplantation. Results Sixty-four patients with hereditary bone marrow failure syndromes were transplanted from related (n=20) or unrelated donors (n=44). Diagnoses were Diamond-Blackfan anemia (21 patients), congenital amegakaryocytic thrombocytopenia (16 patients), dyskeratosis congenita (8 patients), Shwachman-Diamond syndrome (2 patients), severe congenital neutropenia (16 patients) and unclassified (1 patient). In the group of patients who received grafts from related donors, all patients but one received an HLA-matched sibling transplant. The median number of total nucleated cells infused was 5×10⁷/kg. The cumulative incidence of neutrophil recovery at 60 days was 95%. Two patients had grade II-IV acute graft-versus-host disease, while the 2-year cumulative incidence of chronic graft-versus-host disease was 11 %. The 3-year overall survival rate was 95%. In the group of patients who received grafts from unrelated donors, 86% had HLA-mismatched grafts and three received two umbilical cord blood units. The median number of total nucleated cells infused was 6.1×10⁷/kg. The cumulative incidence of neutrophil recovery at day 60 in this group was 55%. The 100-day cumulative incidence of grade II-IV acute graft-versus-host disease was 24%, while the 2-year cumulative incidence of chronic graft-versus-host disease was 53%. The 3-year overall survival rate was 61 %; better overall survival was associated with age less than 5 years (P=0.01) and 6.1×10⁷/kg or more total nucleated cells infused (P=0.05). Conclusions In patients with hereditary bone marrow failure syndromes, related umbilical cord blood transplantation is associated with excellent outcomes while increasing cell dose and better HLA matching might provide better results in unrelated umbilical cord blood transplantation.

Affiliations:

Brésil, Colombie, France, Israël, Italie, Suisse, États-Unis
Texas, Île-de-France
Paris

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Le document en format XML

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<author><name sortKey="Socie, Gerard" sort="Socie, Gerard" uniqKey="Socie G" first="Gérard" last="Socie">Gérard Socie</name>
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<author><name sortKey="Passweg, Jakob" sort="Passweg, Jakob" uniqKey="Passweg J" first="Jakob" last="Passweg">Jakob Passweg</name>
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<author><name sortKey="Antonio De Souza Ca, Carmino" sort="Antonio De Souza Ca, Carmino" uniqKey="Antonio De Souza Ca C" first="Carmino" last="Antonio De Souza Ca">Carmino Antonio De Souza Ca</name>
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<author><name sortKey="Gluckman, Eliane" sort="Gluckman, Eliane" uniqKey="Gluckman E" first="Eliane" last="Gluckman">Eliane Gluckman</name>
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<profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Aminoaciduria</term>
<term>Blood cell</term>
<term>Bone marrow</term>
<term>Bone marrow failure</term>
<term>Compatibility</term>
<term>De Toni-Debré-Fanconi syndrome</term>
<term>Engraftment</term>
<term>HLA-System</term>
<term>Hematology</term>
<term>Hematopoietic cell</term>
<term>Hereditary</term>
<term>Homograft</term>
<term>Major histocompatibility system</term>
<term>Prognosis</term>
<term>Stem cell</term>
<term>Umbilical cord</term>
<term>Unrelated donor</term>
</keywords>
<keywords scheme="Pascal" xml:lang="fr"><term>Homogreffe</term>
<term>Pronostic</term>
<term>Donneur non apparenté</term>
<term>Insuffisance médullaire</term>
<term>Cordon ombilical</term>
<term>Cellule souche</term>
<term>Syndrome de De Toni-Debré-Fanconi</term>
<term>Cellule hématopoïétique</term>
<term>Cellule sanguine</term>
<term>Moelle osseuse</term>
<term>Héréditaire</term>
<term>Prise greffe</term>
<term>Système HLA</term>
<term>Système histocompatibilité majeur</term>
<term>Compatibilité</term>
<term>Hématologie</term>
<term>Aminoacidurie</term>
<term>Syndrome de Fanconi</term>
</keywords>
<keywords scheme="Wicri" type="topic" xml:lang="fr"><term>Cellule souche</term>
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<front><div type="abstract" xml:lang="en">Background Allogeneic stem cell transplantation is the only curative option for patients with hereditary bone marrow failure syndromes. Umbilical cord blood is an alternative source of stem cells for allogeneic transplantation. Design and Methods This multicenter, retrospective study is based on data reported to the Eurocord Registry about patients with hereditary bone marrow failure syndrome who underwent umbilical cord blood transplantation. Results Sixty-four patients with hereditary bone marrow failure syndromes were transplanted from related (n=20) or unrelated donors (n=44). Diagnoses were Diamond-Blackfan anemia (21 patients), congenital amegakaryocytic thrombocytopenia (16 patients), dyskeratosis congenita (8 patients), Shwachman-Diamond syndrome (2 patients), severe congenital neutropenia (16 patients) and unclassified (1 patient). In the group of patients who received grafts from related donors, all patients but one received an HLA-matched sibling transplant. The median number of total nucleated cells infused was 5×10<sup>7</sup>
/kg. The cumulative incidence of neutrophil recovery at 60 days was 95%. Two patients had grade II-IV acute graft-versus-host disease, while the 2-year cumulative incidence of chronic graft-versus-host disease was 11 %. The 3-year overall survival rate was 95%. In the group of patients who received grafts from unrelated donors, 86% had HLA-mismatched grafts and three received two umbilical cord blood units. The median number of total nucleated cells infused was 6.1×10<sup>7</sup>
/kg. The cumulative incidence of neutrophil recovery at day 60 in this group was 55%. The 100-day cumulative incidence of grade II-IV acute graft-versus-host disease was 24%, while the 2-year cumulative incidence of chronic graft-versus-host disease was 53%. The 3-year overall survival rate was 61 %; better overall survival was associated with age less than 5 years (P=0.01) and 6.1×10<sup>7</sup>
/kg or more total nucleated cells infused (P=0.05). Conclusions In patients with hereditary bone marrow failure syndromes, related umbilical cord blood transplantation is associated with excellent outcomes while increasing cell dose and better HLA matching might provide better results in unrelated umbilical cord blood transplantation.</div>
</front>
</TEI>
<affiliations><list><country><li>Brésil</li>
<li>Colombie</li>
<li>France</li>
<li>Israël</li>
<li>Italie</li>
<li>Suisse</li>
<li>États-Unis</li>
</country>
<region><li>Texas</li>
<li>Île-de-France</li>
</region>
<settlement><li>Paris</li>
</settlement>
</list>
<tree><country name="France"><region name="Île-de-France"><name sortKey="Bizzetto, Renata" sort="Bizzetto, Renata" uniqKey="Bizzetto R" first="Renata" last="Bizzetto">Renata Bizzetto</name>
</region>
<name sortKey="Gluckman, Eliane" sort="Gluckman, Eliane" uniqKey="Gluckman E" first="Eliane" last="Gluckman">Eliane Gluckman</name>
<name sortKey="Rocha, Vanderson" sort="Rocha, Vanderson" uniqKey="Rocha V" first="Vanderson" last="Rocha">Vanderson Rocha</name>
<name sortKey="Rocha, Vanderson" sort="Rocha, Vanderson" uniqKey="Rocha V" first="Vanderson" last="Rocha">Vanderson Rocha</name>
<name sortKey="Socie, Gerard" sort="Socie, Gerard" uniqKey="Socie G" first="Gérard" last="Socie">Gérard Socie</name>
</country>
<country name="Brésil"><noRegion><name sortKey="Bizzetto, Renata" sort="Bizzetto, Renata" uniqKey="Bizzetto R" first="Renata" last="Bizzetto">Renata Bizzetto</name>
</noRegion>
<name sortKey="Antonio De Souza Ca, Carmino" sort="Antonio De Souza Ca, Carmino" uniqKey="Antonio De Souza Ca C" first="Carmino" last="Antonio De Souza Ca">Carmino Antonio De Souza Ca</name>
<name sortKey="Bonfim, Carmen" sort="Bonfim, Carmen" uniqKey="Bonfim C" first="Carmen" last="Bonfim">Carmen Bonfim</name>
<name sortKey="Miranda, Eliana" sort="Miranda, Eliana" uniqKey="Miranda E" first="Eliana" last="Miranda">Eliana Miranda</name>
<name sortKey="Nabhan, Samir" sort="Nabhan, Samir" uniqKey="Nabhan S" first="Samir" last="Nabhan">Samir Nabhan</name>
</country>
<country name="Italie"><noRegion><name sortKey="Locatelli, Franco" sort="Locatelli, Franco" uniqKey="Locatelli F" first="Franco" last="Locatelli">Franco Locatelli</name>
</noRegion>
</country>
<country name="États-Unis"><region name="Texas"><name sortKey="Chan, Kawah" sort="Chan, Kawah" uniqKey="Chan K" first="Kawah" last="Chan">Kawah Chan</name>
</region>
</country>
<country name="Colombie"><noRegion><name sortKey="Ramirez, Oscar" sort="Ramirez, Oscar" uniqKey="Ramirez O" first="Oscar" last="Ramirez">Oscar Ramirez</name>
</noRegion>
</country>
<country name="Israël"><noRegion><name sortKey="Stein, Joel" sort="Stein, Joel" uniqKey="Stein J" first="Joel" last="Stein">Joel Stein</name>
</noRegion>
</country>
<country name="Suisse"><noRegion><name sortKey="Passweg, Jakob" sort="Passweg, Jakob" uniqKey="Passweg J" first="Jakob" last="Passweg">Jakob Passweg</name>
</noRegion>
</country>
</tree>
</affiliations>
</record>

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Outcomes after related and unrelated umbilical cord blood transplantation for hereditary bone marrow failure syndromes other than Fanconi anemia

Outcomes after related and unrelated umbilical cord blood transplantation for hereditary bone marrow failure syndromes other than Fanconi anemia

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