Autologous hematopoietic stem cell transplantation for autoimmune diseases: an observational study on 12 years’ experience from the European Group for Blood and Marrow Transplantation Working Party on Autoimmune Diseases
Identifieur interne : 007C51 ( Main/Exploration ); précédent : 007C50; suivant : 007C52Autologous hematopoietic stem cell transplantation for autoimmune diseases: an observational study on 12 years’ experience from the European Group for Blood and Marrow Transplantation Working Party on Autoimmune Diseases
Auteurs : Dominique Farge [France] ; Myriam Labopin [France] ; Alan Tyndall [Suisse] ; Athanasios Fassas [Grèce] ; Gian Luigi Mancardi [Italie] ; Jaap Van Laar [Royaume-Uni] ; Jian Ouyang [République populaire de Chine] ; Tomas Kozak [République tchèque] ; John Moore [Australie] ; Ina Kötter [Allemagne] ; Virginie Chesnel [France] ; Alberto Marmont [Italie] ; Alois Gratwohl [Suisse] ; Riccardo Saccardi [Italie]Source :
- Haematologica [ 0390-6078 ] ; 2009.
Abstract
Autologous hematopoietic stem cell transplantation has been used since 1996 for the treatment of severe autoimmune diseases refractory to approved therapies. We evaluated the long-term outcomes of these transplants and aimed to identify potential prognostic factors.
In this observational study we analyzed all first autologous hematopoietic stem cell transplants for autoimmune diseases reported to the European Group for Blood and Marrow Transplantation (EBMT) registry between 1996–2007. The primary end-points for analysis were overall survival, progression-free survival and transplant-related mortality at 100 days.
Nine hundred patients with autoimmune diseases (64% female; median age, 35 years) who underwent a first autologous hematopoietic stem cell transplant were included. The main diseases were multiple sclerosis (n=345), systemic sclerosis (n=175), systemic lupus erythematosus (n=85), rheumatoid arthritis (n=89), juvenile arthritis (n=65), and hematologic immune cytopenia (n=37). Among all patients, the 5-year survival was 85% and the progression-free survival 43%, although the rates varied widely according to the type of autoimmune disease. By multivariate analysis, the 100-day transplant-related mortality was associated with the transplant centers’ experience (
This largest cohort studied worldwide shows that autologous hematopoietic stem cell transplantation can induce sustained remissions for more than 5 years in patients with severe autoimmune diseases refractory to conventional therapy. The type of autoimmune disease, rather than transplant technique, was the most relevant determinant of outcome. Results improved with time and were associated with the transplant centers’ experience. These data support ongoing and planned phase III trials to evaluate the place of autologous hematopoietic stem cell transplantation in the treatment strategy for severe autoimmune diseases.
Url:
DOI: 10.3324/haematol.2009.013458
PubMed: 19773265
PubMed Central: 2817032
Affiliations:
- Allemagne, Australie, France, Grèce, Italie, Royaume-Uni, République populaire de Chine, République tchèque, Suisse
- Bade-Wurtemberg, Bohême centrale, District de Tübingen, Nouvelle-Galles du Sud, Île-de-France
- Paris, Prague, Sydney, Tübingen
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Le document en format XML
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France</nlm:aff><country xml:lang="fr">France</country><wicri:regionArea> Internal Medicine, Saint Louis Hospital, Paris</wicri:regionArea><placeName><region type="region">Île-de-France</region><region type="old region">Île-de-France</region><settlement type="city">Paris</settlement></placeName></affiliation></author><author><name sortKey="Labopin, Myriam" sort="Labopin, Myriam" uniqKey="Labopin M" first="Myriam" last="Labopin">Myriam Labopin</name><affiliation wicri:level="3"><nlm:aff id="af2-0950284"> Hôpital Saint Antoine, Service d’hématologie et Thérapie Cellulaire, AP-HP, UPMC Univ Paris 06, UMR-S 938, CEREST-TC EBMT, Paris, France</nlm:aff><country xml:lang="fr">France</country><wicri:regionArea> Hôpital Saint Antoine, Service d’hématologie et Thérapie Cellulaire, AP-HP, UPMC Univ Paris 06, UMR-S 938, CEREST-TC EBMT, Paris</wicri:regionArea><placeName><region type="region">Île-de-France</region><region type="old region">Île-de-France</region><settlement type="city">Paris</settlement></placeName></affiliation></author><author><name sortKey="Tyndall, Alan" sort="Tyndall, Alan" uniqKey="Tyndall A" first="Alan" last="Tyndall">Alan Tyndall</name><affiliation wicri:level="1"><nlm:aff id="af3-0950284"> Rheumatology, Rheumatologische Universtätsklinik, Basel, Switzerland</nlm:aff><country xml:lang="fr">Suisse</country><wicri:regionArea> Rheumatology, Rheumatologische Universtätsklinik, Basel</wicri:regionArea><wicri:noRegion>Basel</wicri:noRegion></affiliation></author><author><name sortKey="Fassas, Athanasios" sort="Fassas, Athanasios" uniqKey="Fassas A" first="Athanasios" last="Fassas">Athanasios Fassas</name><affiliation wicri:level="1"><nlm:aff id="af4-0950284"> Neurology and Haematology, The George Papanicolau Hospital, Thessaloniki, Greece</nlm:aff><country xml:lang="fr">Grèce</country><wicri:regionArea> Neurology and Haematology, The George Papanicolau Hospital, Thessaloniki</wicri:regionArea><wicri:noRegion>Thessaloniki</wicri:noRegion></affiliation></author><author><name sortKey="Mancardi, Gian Luigi" sort="Mancardi, Gian Luigi" uniqKey="Mancardi G" first="Gian Luigi" last="Mancardi">Gian Luigi Mancardi</name><affiliation wicri:level="1"><nlm:aff id="af5-0950284"> Neurosciences, Ophthalmology and Genetics, Univ. of Genova, Genova, Italy</nlm:aff><country xml:lang="fr">Italie</country><wicri:regionArea> Neurosciences, Ophthalmology and Genetics, Univ. of Genova, Genova</wicri:regionArea><wicri:noRegion>Genova</wicri:noRegion></affiliation></author><author><name sortKey="Van Laar, Jaap" sort="Van Laar, Jaap" uniqKey="Van Laar J" first="Jaap" last="Van Laar">Jaap Van Laar</name><affiliation wicri:level="1"><nlm:aff id="af6-0950284"> Institute of Cellular Medicine, Newcastle Univ., Newcastle Upon Tyne, United Kingdom</nlm:aff><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea> Institute of Cellular Medicine, Newcastle Univ., Newcastle Upon Tyne</wicri:regionArea><wicri:noRegion>Newcastle Upon Tyne</wicri:noRegion></affiliation></author><author><name sortKey="Ouyang, Jian" sort="Ouyang, Jian" uniqKey="Ouyang J" first="Jian" last="Ouyang">Jian Ouyang</name><affiliation wicri:level="1"><nlm:aff id="af7-0950284">Hematology, Hospital of Nanjing, Nanjing, China;</nlm:aff><country xml:lang="fr">République populaire de Chine</country><wicri:regionArea>Hematology, Hospital of Nanjing, Nanjing</wicri:regionArea><wicri:noRegion>Nanjing</wicri:noRegion></affiliation></author><author><name sortKey="Kozak, Tomas" sort="Kozak, Tomas" uniqKey="Kozak T" first="Tomas" last="Kozak">Tomas Kozak</name><affiliation wicri:level="3"><nlm:aff id="af8-0950284">Clinical Haematology, Charles Univ. Prague, Prague, Czech Republic;</nlm:aff><country xml:lang="fr">République tchèque</country><wicri:regionArea>Clinical Haematology, Charles Univ. Prague, Prague</wicri:regionArea><placeName><settlement type="city">Prague</settlement><region type="région" nuts="2">Bohême centrale</region></placeName></affiliation></author><author><name sortKey="Moore, John" sort="Moore, John" uniqKey="Moore J" first="John" last="Moore">John Moore</name><affiliation wicri:level="3"><nlm:aff id="af9-0950284">Hematology Dept., St Vincents Hospital, Sydney, Australia;</nlm:aff><country xml:lang="fr">Australie</country><wicri:regionArea>Hematology Dept., St Vincents Hospital, Sydney</wicri:regionArea><placeName><settlement type="city">Sydney</settlement><region type="état">Nouvelle-Galles du Sud</region></placeName></affiliation></author><author><name sortKey="Kotter, Ina" sort="Kotter, Ina" uniqKey="Kotter I" first="Ina" last="Kötter">Ina Kötter</name><affiliation wicri:level="3"><nlm:aff id="af10-0950284">Immunology, Rheumatology and Autoimmune Diseases, Univ. of Tübingen, Tübingen, Germany;</nlm:aff><country xml:lang="fr">Allemagne</country><wicri:regionArea>Immunology, Rheumatology and Autoimmune Diseases, Univ. of Tübingen, Tübingen</wicri:regionArea><placeName><region type="land" nuts="1">Bade-Wurtemberg</region><region type="district" nuts="2">District de Tübingen</region><settlement type="city">Tübingen</settlement></placeName></affiliation></author><author><name sortKey="Chesnel, Virginie" sort="Chesnel, Virginie" uniqKey="Chesnel V" first="Virginie" last="Chesnel">Virginie Chesnel</name><affiliation wicri:level="3"><nlm:aff id="af11-0950284">INSERM UMR-S 938, UPMC Univ Paris 06, Paris, France;</nlm:aff><country xml:lang="fr">France</country><wicri:regionArea>INSERM UMR-S 938, UPMC Univ Paris 06, Paris</wicri:regionArea><placeName><region type="region">Île-de-France</region><region type="old region">Île-de-France</region><settlement type="city">Paris</settlement></placeName></affiliation></author><author><name sortKey="Marmont, Alberto" sort="Marmont, Alberto" uniqKey="Marmont A" first="Alberto" last="Marmont">Alberto Marmont</name><affiliation wicri:level="1"><nlm:aff id="af12-0950284">Haematology, Ospedale di San Martino, Genova, Italy;</nlm:aff><country xml:lang="fr">Italie</country><wicri:regionArea>Haematology, Ospedale di San Martino, Genova</wicri:regionArea><wicri:noRegion>Genova</wicri:noRegion></affiliation></author><author><name sortKey="Gratwohl, Alois" sort="Gratwohl, Alois" uniqKey="Gratwohl A" first="Alois" last="Gratwohl">Alois Gratwohl</name><affiliation wicri:level="1"><nlm:aff wicri:cut=" and" id="af13-0950284">Haematology, Kantonspital Basel, Basel, Switzerland</nlm:aff><country xml:lang="fr">Suisse</country><wicri:regionArea>Haematology, Kantonspital Basel, Basel</wicri:regionArea><wicri:noRegion>Basel</wicri:noRegion></affiliation></author><author><name sortKey="Saccardi, Riccardo" sort="Saccardi, Riccardo" uniqKey="Saccardi R" first="Riccardo" last="Saccardi">Riccardo Saccardi</name><affiliation wicri:level="1"><nlm:aff id="af14-0950284">BMT Unit, UO Ematologia, Policlinico Careggi, Florence, Italy</nlm:aff><country xml:lang="fr">Italie</country><wicri:regionArea>BMT Unit, UO Ematologia, Policlinico Careggi, Florence</wicri:regionArea><wicri:noRegion>Florence</wicri:noRegion></affiliation></author></analytic><series><title level="j">Haematologica</title><idno type="ISSN">0390-6078</idno><idno type="eISSN">1592-8721</idno><imprint><date when="2009">2009</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en"><sec><title>Background</title><p>Autologous hematopoietic stem cell transplantation has been used since 1996 for the treatment of severe autoimmune diseases refractory to approved therapies. We evaluated the long-term outcomes of these transplants and aimed to identify potential prognostic factors.</p></sec><sec><title>Design and Methods</title><p>In this observational study we analyzed all first autologous hematopoietic stem cell transplants for autoimmune diseases reported to the European Group for Blood and Marrow Transplantation (EBMT) registry between 1996–2007. The primary end-points for analysis were overall survival, progression-free survival and transplant-related mortality at 100 days.</p></sec><sec><title>Results</title><p>Nine hundred patients with autoimmune diseases (64% female; median age, 35 years) who underwent a first autologous hematopoietic stem cell transplant were included. The main diseases were multiple sclerosis (n=345), systemic sclerosis (n=175), systemic lupus erythematosus (n=85), rheumatoid arthritis (n=89), juvenile arthritis (n=65), and hematologic immune cytopenia (n=37). Among all patients, the 5-year survival was 85% and the progression-free survival 43%, although the rates varied widely according to the type of autoimmune disease. By multivariate analysis, the 100-day transplant-related mortality was associated with the transplant centers’ experience (<italic>P</italic>=0.003) and type of autoimmune disease (<italic>P</italic>=0.03). No significant influence of transplant technique was identified. Age less than 35 years (<italic>P</italic>=0.004), transplantation after 2000 (<italic>P</italic>=0.0015) and diagnosis (<italic>P</italic>=0.0007) were associated with progression-free survival.</p></sec><sec><title>Conclusions</title><p>This largest cohort studied worldwide shows that autologous hematopoietic stem cell transplantation can induce sustained remissions for more than 5 years in patients with severe autoimmune diseases refractory to conventional therapy. The type of autoimmune disease, rather than transplant technique, was the most relevant determinant of outcome. Results improved with time and were associated with the transplant centers’ experience. These data support ongoing and planned phase III trials to evaluate the place of autologous hematopoietic stem cell transplantation in the treatment strategy for severe autoimmune diseases.</p></sec></div></front></TEI><affiliations><list><country><li>Allemagne</li><li>Australie</li><li>France</li><li>Grèce</li><li>Italie</li><li>Royaume-Uni</li><li>République populaire de Chine</li><li>République tchèque</li><li>Suisse</li></country><region><li>Bade-Wurtemberg</li><li>Bohême centrale</li><li>District de Tübingen</li><li>Nouvelle-Galles du Sud</li><li>Île-de-France</li></region><settlement><li>Paris</li><li>Prague</li><li>Sydney</li><li>Tübingen</li></settlement></list><tree><country name="France"><region name="Île-de-France"><name sortKey="Farge, Dominique" sort="Farge, Dominique" uniqKey="Farge D" first="Dominique" last="Farge">Dominique Farge</name></region><name sortKey="Chesnel, Virginie" sort="Chesnel, Virginie" uniqKey="Chesnel V" first="Virginie" last="Chesnel">Virginie Chesnel</name><name sortKey="Labopin, Myriam" sort="Labopin, Myriam" uniqKey="Labopin M" first="Myriam" last="Labopin">Myriam Labopin</name></country><country name="Suisse"><noRegion><name sortKey="Tyndall, Alan" sort="Tyndall, Alan" uniqKey="Tyndall A" first="Alan" last="Tyndall">Alan Tyndall</name></noRegion><name sortKey="Gratwohl, Alois" sort="Gratwohl, Alois" uniqKey="Gratwohl A" first="Alois" last="Gratwohl">Alois Gratwohl</name></country><country name="Grèce"><noRegion><name sortKey="Fassas, Athanasios" sort="Fassas, Athanasios" uniqKey="Fassas A" first="Athanasios" last="Fassas">Athanasios Fassas</name></noRegion></country><country name="Italie"><noRegion><name sortKey="Mancardi, Gian Luigi" sort="Mancardi, Gian Luigi" uniqKey="Mancardi G" first="Gian Luigi" last="Mancardi">Gian Luigi Mancardi</name></noRegion><name sortKey="Marmont, Alberto" sort="Marmont, Alberto" uniqKey="Marmont A" first="Alberto" last="Marmont">Alberto Marmont</name><name sortKey="Saccardi, Riccardo" sort="Saccardi, Riccardo" uniqKey="Saccardi R" first="Riccardo" last="Saccardi">Riccardo Saccardi</name></country><country name="Royaume-Uni"><noRegion><name sortKey="Van Laar, Jaap" sort="Van Laar, Jaap" uniqKey="Van Laar J" first="Jaap" last="Van Laar">Jaap Van Laar</name></noRegion></country><country name="République populaire de Chine"><noRegion><name sortKey="Ouyang, Jian" sort="Ouyang, Jian" uniqKey="Ouyang J" first="Jian" last="Ouyang">Jian Ouyang</name></noRegion></country><country name="République tchèque"><region name="Bohême centrale"><name sortKey="Kozak, Tomas" sort="Kozak, Tomas" uniqKey="Kozak T" first="Tomas" last="Kozak">Tomas Kozak</name></region></country><country name="Australie"><region name="Nouvelle-Galles du Sud"><name sortKey="Moore, John" sort="Moore, John" uniqKey="Moore J" first="John" last="Moore">John Moore</name></region></country><country name="Allemagne"><region name="Bade-Wurtemberg"><name sortKey="Kotter, Ina" sort="Kotter, Ina" uniqKey="Kotter I" first="Ina" last="Kötter">Ina Kötter</name></region></country></tree></affiliations></record>Pour manipuler ce document sous Unix (Dilib)
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