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Haematopoietic SCT in severe autoimmune diseases: updated guidelines of the European Group for Blood and Marrow Transplantation

Identifieur interne : 000018 ( Pmc/Curation ); précédent : 000017; suivant : 000019

Haematopoietic SCT in severe autoimmune diseases: updated guidelines of the European Group for Blood and Marrow Transplantation

Auteurs : J A Snowden [Royaume-Uni] ; R. Saccardi [Italie] ; M. Allez [France] ; S. Ardizzone [Italie] ; R. Arnold [Allemagne] ; R. Cervera [Espagne] ; C. Denton [Royaume-Uni] ; C. Hawkey [Royaume-Uni] ; M. Labopin [France] ; G. Mancardi [Italie] ; R. Martin [Allemagne] ; J J Moore [Australie] ; J. Passweg [Suisse] ; C. Peters [Autriche] ; M. Rabusin [Italie] ; M. Rovira [Espagne] ; J M Van Laar [Royaume-Uni] ; D. Farge [France]

Source :

RBID : PMC:3371413

Abstract

In 1997, the first consensus guidelines for haematopoietic SCT (HSCT) in autoimmune diseases (ADs) were published, while an international coordinated clinical programme was launched. These guidelines provided broad principles for the field over the following decade and were accompanied by comprehensive data collection in the European Group for Blood and Marrow Transplantation (EBMT) AD Registry. Subsequently, retrospective analyses and prospective phase I/II studies generated evidence to support the feasibility, safety and efficacy of HSCT in several types of severe, treatment-resistant ADs, which became the basis for larger-scale phase II and III studies. In parallel, there has also been an era of immense progress in biological therapy in ADs. The aim of this document is to provide revised and updated guidelines for both the current application and future development of HSCT in ADs in relation to the benefits, risks and health economic considerations of other modern treatments. Patient safety considerations are central to guidance on patient selection and HSCT procedural aspects within appropriately experienced and Joint Accreditation Committee of International Society for Cellular Therapy and EBMT accredited centres. A need for prospective interventional and non-interventional studies, where feasible, along with systematic data reporting, in accordance with EBMT policies and procedures, is emphasized.


Url:
DOI: 10.1038/bmt.2011.185
PubMed: 22002489
PubMed Central: 3371413

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PMC:3371413

Le document en format XML

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<institution>Department of Haematology, Sheffield Teaching Hospitals NHS Trust</institution>
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<institution>Department of Autoimmune Diseases, Hospital Clinic</institution>
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<country xml:lang="fr">Espagne</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
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<country xml:lang="fr">Allemagne</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
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<institution>St Vincent's Hospital</institution>
, Sydney, NSW,
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<country xml:lang="fr">Australie</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
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<institution>Universitaetsspital Basel</institution>
, Basel,
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<country xml:lang="fr">Suisse</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
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, Sheffield,
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<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
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</nlm:aff>
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<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
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<name sortKey="Cervera, R" sort="Cervera, R" uniqKey="Cervera R" first="R" last="Cervera">R. Cervera</name>
<affiliation wicri:level="1">
<nlm:aff id="aff7">
<institution>Department of Autoimmune Diseases, Hospital Clinic</institution>
, Barcelona,
<country>Spain</country>
</nlm:aff>
<country xml:lang="fr">Espagne</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
</affiliation>
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<name sortKey="Denton, C" sort="Denton, C" uniqKey="Denton C" first="C" last="Denton">C. Denton</name>
<affiliation wicri:level="1">
<nlm:aff id="aff8">
<institution>Centre for Rheumatology, Royal Free and University College Medical School</institution>
, Hampstead, London,
<country>UK</country>
</nlm:aff>
<country xml:lang="fr">Royaume-Uni</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
</affiliation>
</author>
<author>
<name sortKey="Hawkey, C" sort="Hawkey, C" uniqKey="Hawkey C" first="C" last="Hawkey">C. Hawkey</name>
<affiliation wicri:level="1">
<nlm:aff id="aff9">
<institution>Nottingham Digestive Diseases Centre, University of Nottingham</institution>
, Nottingham,
<country>UK</country>
</nlm:aff>
<country xml:lang="fr">Royaume-Uni</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
</affiliation>
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<name sortKey="Labopin, M" sort="Labopin, M" uniqKey="Labopin M" first="M" last="Labopin">M. Labopin</name>
<affiliation wicri:level="1">
<nlm:aff id="aff10">
<institution>Hôpital Saint Antoine, Service d'Hématologie et Thérapie Cellulaire, AP-HP, UPMC Univ Paris 06</institution>
, Paris,
<country>France</country>
</nlm:aff>
<country xml:lang="fr">France</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
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<name sortKey="Mancardi, G" sort="Mancardi, G" uniqKey="Mancardi G" first="G" last="Mancardi">G. Mancardi</name>
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<nlm:aff id="aff11">
<institution>Department of Neuroscience, Ophthalmology and Genetics, University of Genova</institution>
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</nlm:aff>
<country xml:lang="fr">Italie</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
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<name sortKey="Martin, R" sort="Martin, R" uniqKey="Martin R" first="R" last="Martin">R. Martin</name>
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<nlm:aff id="aff12">
<institution>Institute for Neuroimmunology and Clinical MS Research</institution>
, Hamburg,
<country>Germany</country>
</nlm:aff>
<country xml:lang="fr">Allemagne</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
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<author>
<name sortKey="Moore, J J" sort="Moore, J J" uniqKey="Moore J" first="J J" last="Moore">J J Moore</name>
<affiliation wicri:level="1">
<nlm:aff id="aff13">
<institution>St Vincent's Hospital</institution>
, Sydney, NSW,
<country>Australia</country>
</nlm:aff>
<country xml:lang="fr">Australie</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
</affiliation>
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<author>
<name sortKey="Passweg, J" sort="Passweg, J" uniqKey="Passweg J" first="J" last="Passweg">J. Passweg</name>
<affiliation wicri:level="1">
<nlm:aff id="aff14">
<institution>Universitaetsspital Basel</institution>
, Basel,
<country>Switzerland</country>
</nlm:aff>
<country xml:lang="fr">Suisse</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
</affiliation>
</author>
<author>
<name sortKey="Peters, C" sort="Peters, C" uniqKey="Peters C" first="C" last="Peters">C. Peters</name>
<affiliation wicri:level="1">
<nlm:aff id="aff15">
<institution>BMT Unit, St Anna Children's Hospital</institution>
, Vienna,
<country>Austria</country>
</nlm:aff>
<country xml:lang="fr">Autriche</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
</affiliation>
</author>
<author>
<name sortKey="Rabusin, M" sort="Rabusin, M" uniqKey="Rabusin M" first="M" last="Rabusin">M. Rabusin</name>
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<institution>BMT Unit, Department of Pediatrics, Institute of Maternal and Child Health Burlo Garofolo</institution>
, Trieste,
<country>Italy</country>
</nlm:aff>
<country xml:lang="fr">Italie</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
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<name sortKey="Rovira, M" sort="Rovira, M" uniqKey="Rovira M" first="M" last="Rovira">M. Rovira</name>
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<institution>SCT Unit, Hematology Department, Hospital Clinic</institution>
, Barcelona,
<country>Spain</country>
</nlm:aff>
<country xml:lang="fr">Espagne</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
</affiliation>
</author>
<author>
<name sortKey="Van Laar, J M" sort="Van Laar, J M" uniqKey="Van Laar J" first="J M" last="Van Laar">J M Van Laar</name>
<affiliation wicri:level="1">
<nlm:aff id="aff18">
<institution>Newcastle University</institution>
, Newcastle-Upon-Tyne,
<country>UK</country>
</nlm:aff>
<country xml:lang="fr">Royaume-Uni</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
</affiliation>
</author>
<author>
<name sortKey="Farge, D" sort="Farge, D" uniqKey="Farge D" first="D" last="Farge">D. Farge</name>
<affiliation wicri:level="1">
<nlm:aff id="aff19">
<institution>Department of Internal Medicine, INSERM U 796, Hôpital St Louis</institution>
, Paris,
<country>France</country>
</nlm:aff>
<country xml:lang="fr">France</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
</affiliation>
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</analytic>
<series>
<title level="j">Bone Marrow Transplantation</title>
<idno type="ISSN">0268-3369</idno>
<idno type="eISSN">1476-5365</idno>
<imprint>
<date when="2011">2011</date>
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<div type="abstract" xml:lang="en">
<p>In 1997, the first consensus guidelines for haematopoietic SCT (HSCT) in autoimmune diseases (ADs) were published, while an international coordinated clinical programme was launched. These guidelines provided broad principles for the field over the following decade and were accompanied by comprehensive data collection in the European Group for Blood and Marrow Transplantation (EBMT) AD Registry. Subsequently, retrospective analyses and prospective phase I/II studies generated evidence to support the feasibility, safety and efficacy of HSCT in several types of severe, treatment-resistant ADs, which became the basis for larger-scale phase II and III studies. In parallel, there has also been an era of immense progress in biological therapy in ADs. The aim of this document is to provide revised and updated guidelines for both the current application and future development of HSCT in ADs in relation to the benefits, risks and health economic considerations of other modern treatments. Patient safety considerations are central to guidance on patient selection and HSCT procedural aspects within appropriately experienced and Joint Accreditation Committee of International Society for Cellular Therapy and EBMT accredited centres. A need for prospective interventional and non-interventional studies, where feasible, along with systematic data reporting, in accordance with EBMT policies and procedures, is emphasized.</p>
</div>
</front>
<back>
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<name sortKey="Ikehara, S" uniqKey="Ikehara S">S Ikehara</name>
</author>
<author>
<name sortKey="Good, Ra" uniqKey="Good R">RA Good</name>
</author>
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<name sortKey="Inoue, S" uniqKey="Inoue S">S Inoue</name>
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<name sortKey="Oo, Mm" uniqKey="Oo M">MM Oo</name>
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</TEI>
<pmc article-type="other">
<pmc-dir>properties open_access</pmc-dir>
<front>
<journal-meta>
<journal-id journal-id-type="nlm-ta">Bone Marrow Transplant</journal-id>
<journal-id journal-id-type="iso-abbrev">Bone Marrow Transplant</journal-id>
<journal-title-group>
<journal-title>Bone Marrow Transplantation</journal-title>
</journal-title-group>
<issn pub-type="ppub">0268-3369</issn>
<issn pub-type="epub">1476-5365</issn>
<publisher>
<publisher-name>Nature Publishing Group</publisher-name>
</publisher>
</journal-meta>
<article-meta>
<article-id pub-id-type="pmid">22002489</article-id>
<article-id pub-id-type="pmc">3371413</article-id>
<article-id pub-id-type="pii">bmt2011185</article-id>
<article-id pub-id-type="doi">10.1038/bmt.2011.185</article-id>
<article-categories>
<subj-group subj-group-type="heading">
<subject>Special Report</subject>
</subj-group>
</article-categories>
<title-group>
<article-title>Haematopoietic SCT in severe autoimmune diseases: updated guidelines of the European Group for Blood and Marrow Transplantation</article-title>
<alt-title alt-title-type="running">Updated guidelines for HSCT in autoimmune diseases</alt-title>
</title-group>
<contrib-group>
<contrib contrib-type="author">
<name>
<surname>Snowden</surname>
<given-names>J A</given-names>
</name>
<xref ref-type="aff" rid="aff1">1</xref>
<xref ref-type="aff" rid="aff2">2</xref>
<xref ref-type="corresp" rid="caf1">*</xref>
</contrib>
<contrib contrib-type="author">
<name>
<surname>Saccardi</surname>
<given-names>R</given-names>
</name>
<xref ref-type="aff" rid="aff3">3</xref>
</contrib>
<contrib contrib-type="author">
<name>
<surname>Allez</surname>
<given-names>M</given-names>
</name>
<xref ref-type="aff" rid="aff4">4</xref>
</contrib>
<contrib contrib-type="author">
<name>
<surname>Ardizzone</surname>
<given-names>S</given-names>
</name>
<xref ref-type="aff" rid="aff5">5</xref>
</contrib>
<contrib contrib-type="author">
<name>
<surname>Arnold</surname>
<given-names>R</given-names>
</name>
<xref ref-type="aff" rid="aff6">6</xref>
</contrib>
<contrib contrib-type="author">
<name>
<surname>Cervera</surname>
<given-names>R</given-names>
</name>
<xref ref-type="aff" rid="aff7">7</xref>
</contrib>
<contrib contrib-type="author">
<name>
<surname>Denton</surname>
<given-names>C</given-names>
</name>
<xref ref-type="aff" rid="aff8">8</xref>
</contrib>
<contrib contrib-type="author">
<name>
<surname>Hawkey</surname>
<given-names>C</given-names>
</name>
<xref ref-type="aff" rid="aff9">9</xref>
</contrib>
<contrib contrib-type="author">
<name>
<surname>Labopin</surname>
<given-names>M</given-names>
</name>
<xref ref-type="aff" rid="aff10">10</xref>
</contrib>
<contrib contrib-type="author">
<name>
<surname>Mancardi</surname>
<given-names>G</given-names>
</name>
<xref ref-type="aff" rid="aff11">11</xref>
</contrib>
<contrib contrib-type="author">
<name>
<surname>Martin</surname>
<given-names>R</given-names>
</name>
<xref ref-type="aff" rid="aff12">12</xref>
</contrib>
<contrib contrib-type="author">
<name>
<surname>Moore</surname>
<given-names>J J</given-names>
</name>
<xref ref-type="aff" rid="aff13">13</xref>
<xref ref-type="author-notes" rid="note1">
<sup>20</sup>
</xref>
</contrib>
<contrib contrib-type="author">
<name>
<surname>Passweg</surname>
<given-names>J</given-names>
</name>
<xref ref-type="aff" rid="aff14">14</xref>
</contrib>
<contrib contrib-type="author">
<name>
<surname>Peters</surname>
<given-names>C</given-names>
</name>
<xref ref-type="aff" rid="aff15">15</xref>
<xref ref-type="author-notes" rid="note2">
<sup>21</sup>
</xref>
</contrib>
<contrib contrib-type="author">
<name>
<surname>Rabusin</surname>
<given-names>M</given-names>
</name>
<xref ref-type="aff" rid="aff16">16</xref>
<xref ref-type="author-notes" rid="note2">
<sup>21</sup>
</xref>
</contrib>
<contrib contrib-type="author">
<name>
<surname>Rovira</surname>
<given-names>M</given-names>
</name>
<xref ref-type="aff" rid="aff17">17</xref>
</contrib>
<contrib contrib-type="author">
<name>
<surname>van Laar</surname>
<given-names>J M</given-names>
</name>
<xref ref-type="aff" rid="aff18">18</xref>
</contrib>
<contrib contrib-type="author">
<name>
<surname>Farge</surname>
<given-names>D</given-names>
</name>
<xref ref-type="aff" rid="aff19">19</xref>
<xref ref-type="corresp" rid="caf2">*</xref>
</contrib>
<on-behalf-of>on behalf of the EBMT Autoimmune Disease Working Party (ADWP) and Paediatric Diseases Working Party (PDWP)</on-behalf-of>
<aff id="aff1">
<label>1</label>
<institution>Department of Haematology, Sheffield Teaching Hospitals NHS Trust</institution>
, Sheffield,
<country>UK</country>
</aff>
<aff id="aff2">
<label>2</label>
<institution>Department of Oncology, University of Sheffield</institution>
, Sheffield
<country>, UK</country>
</aff>
<aff id="aff3">
<label>3</label>
<institution>Department of Haematology, Careggi University Hospital</institution>
, Firenze,
<country>Italy</country>
</aff>
<aff id="aff4">
<label>4</label>
<institution>Service de Gastroentérologie, INSERM U 662, Hôpital St Louis</institution>
, Paris,
<country>France</country>
</aff>
<aff id="aff5">
<label>5</label>
<institution>Department of Gastroenterology, Sacco University Hospital</institution>
, Milan,
<country>Italy</country>
</aff>
<aff id="aff6">
<label>6</label>
<institution>Charite Hospital Berlin</institution>
, Berlin,
<country>Germany</country>
</aff>
<aff id="aff7">
<label>7</label>
<institution>Department of Autoimmune Diseases, Hospital Clinic</institution>
, Barcelona,
<country>Spain</country>
</aff>
<aff id="aff8">
<label>8</label>
<institution>Centre for Rheumatology, Royal Free and University College Medical School</institution>
, Hampstead, London,
<country>UK</country>
</aff>
<aff id="aff9">
<label>9</label>
<institution>Nottingham Digestive Diseases Centre, University of Nottingham</institution>
, Nottingham,
<country>UK</country>
</aff>
<aff id="aff10">
<label>10</label>
<institution>Hôpital Saint Antoine, Service d'Hématologie et Thérapie Cellulaire, AP-HP, UPMC Univ Paris 06</institution>
, Paris,
<country>France</country>
</aff>
<aff id="aff11">
<label>11</label>
<institution>Department of Neuroscience, Ophthalmology and Genetics, University of Genova</institution>
, Genova,
<country>Italy</country>
</aff>
<aff id="aff12">
<label>12</label>
<institution>Institute for Neuroimmunology and Clinical MS Research</institution>
, Hamburg,
<country>Germany</country>
</aff>
<aff id="aff13">
<label>13</label>
<institution>St Vincent's Hospital</institution>
, Sydney, NSW,
<country>Australia</country>
</aff>
<aff id="aff14">
<label>14</label>
<institution>Universitaetsspital Basel</institution>
, Basel,
<country>Switzerland</country>
</aff>
<aff id="aff15">
<label>15</label>
<institution>BMT Unit, St Anna Children's Hospital</institution>
, Vienna,
<country>Austria</country>
</aff>
<aff id="aff16">
<label>16</label>
<institution>BMT Unit, Department of Pediatrics, Institute of Maternal and Child Health Burlo Garofolo</institution>
, Trieste,
<country>Italy</country>
</aff>
<aff id="aff17">
<label>17</label>
<institution>SCT Unit, Hematology Department, Hospital Clinic</institution>
, Barcelona,
<country>Spain</country>
</aff>
<aff id="aff18">
<label>18</label>
<institution>Newcastle University</institution>
, Newcastle-Upon-Tyne,
<country>UK</country>
</aff>
<aff id="aff19">
<label>19</label>
<institution>Department of Internal Medicine, INSERM U 796, Hôpital St Louis</institution>
, Paris,
<country>France</country>
</aff>
</contrib-group>
<author-notes>
<corresp id="caf1">
<label>*</label>
<institution>Department of Haematology, Sheffield Teaching Hospitals NHS Trust, University of Sheffield</institution>
, Glossop Road, Sheffield S10 2JF,
<country>UK</country>
. E-mail:
<email>john.snowden@sth.nhs.uk</email>
</corresp>
<corresp id="caf2">
<label>*</label>
<institution>EBMT Autoimmune Diseases Working Party (ADWP), EBMT Paris Office</institution>
, Paris,
<country>France</country>
. E-mail:
<email>dominique.farge-bancel@sls.aphp.fr</email>
</corresp>
<fn fn-type="present-address" id="note1">
<label>20</label>
<p>Representative of the Bone Marrow Transplantation Society of Australia and New Zealand (BMTSANZ).</p>
</fn>
<fn fn-type="present-address" id="note2">
<label>21</label>
<p>Representative of the EBMT Paediatric Diseases Working Party (PDWP).</p>
</fn>
</author-notes>
<pub-date pub-type="ppub">
<month>06</month>
<year>2012</year>
</pub-date>
<pub-date pub-type="epub">
<day>17</day>
<month>10</month>
<year>2011</year>
</pub-date>
<volume>47</volume>
<issue>6</issue>
<fpage>770</fpage>
<lpage>790</lpage>
<history>
<date date-type="received">
<day>03</day>
<month>05</month>
<year>2011</year>
</date>
<date date-type="rev-recd">
<day>04</day>
<month>07</month>
<year>2011</year>
</date>
<date date-type="accepted">
<day>04</day>
<month>07</month>
<year>2011</year>
</date>
</history>
<permissions>
<copyright-statement>Copyright © 2012 Macmillan Publishers Limited</copyright-statement>
<copyright-year>2012</copyright-year>
<copyright-holder>Macmillan Publishers Limited</copyright-holder>
<license license-type="open-access" xlink:href="http://creativecommons.org/licenses/by-nc-nd/3.0/">
<pmc-comment>author-paid</pmc-comment>
<license-p>This work is licensed under the Creative Commons Attribution-NonCommercial-No Derivative Works 3.0 Unported License. To view a copy of this license, visit http://creativecommons.org/licenses/by-nc-nd/3.0/</license-p>
</license>
</permissions>
<abstract>
<p>In 1997, the first consensus guidelines for haematopoietic SCT (HSCT) in autoimmune diseases (ADs) were published, while an international coordinated clinical programme was launched. These guidelines provided broad principles for the field over the following decade and were accompanied by comprehensive data collection in the European Group for Blood and Marrow Transplantation (EBMT) AD Registry. Subsequently, retrospective analyses and prospective phase I/II studies generated evidence to support the feasibility, safety and efficacy of HSCT in several types of severe, treatment-resistant ADs, which became the basis for larger-scale phase II and III studies. In parallel, there has also been an era of immense progress in biological therapy in ADs. The aim of this document is to provide revised and updated guidelines for both the current application and future development of HSCT in ADs in relation to the benefits, risks and health economic considerations of other modern treatments. Patient safety considerations are central to guidance on patient selection and HSCT procedural aspects within appropriately experienced and Joint Accreditation Committee of International Society for Cellular Therapy and EBMT accredited centres. A need for prospective interventional and non-interventional studies, where feasible, along with systematic data reporting, in accordance with EBMT policies and procedures, is emphasized.</p>
</abstract>
<kwd-group>
<kwd>haematopoietic SCT</kwd>
<kwd>autoimmune diseases</kwd>
<kwd>autologous</kwd>
<kwd>allogeneic</kwd>
<kwd>guidelines</kwd>
<kwd>recommendations</kwd>
</kwd-group>
</article-meta>
</front>
<floats-group>
<table-wrap id="tbl1">
<label>Table 1</label>
<caption>
<title>EBMT database of autoimmune diseases, June 2011</title>
</caption>
<table frame="hsides" rules="groups" border="1">
<colgroup>
<col align="left"></col>
<col align="char" char="."></col>
<col align="char" char="."></col>
<col align="char" char="."></col>
</colgroup>
<thead valign="bottom">
<tr>
<th align="left" valign="top" charoff="50">
<italic>Diseases</italic>
</th>
<th colspan="3" align="center" valign="top" char="." charoff="50">
<italic>No. of patients</italic>
<hr></hr>
</th>
</tr>
<tr>
<th align="left" valign="top" charoff="50"> </th>
<th align="center" valign="top" char="." charoff="50">
<italic>Paediatrics</italic>
<xref ref-type="fn" rid="t1-fn2">a</xref>
</th>
<th align="center" valign="top" char="." charoff="50">
<italic>Adults</italic>
</th>
<th align="center" valign="top" char="." charoff="50">
<italic>Total</italic>
</th>
</tr>
<tr>
<th align="left" valign="top" charoff="50"> </th>
<th align="center" valign="top" char="." charoff="50">
<italic>Auto/Allo</italic>
</th>
<th align="center" valign="top" char="." charoff="50">
<italic>Auto/Allo</italic>
</th>
<th align="center" valign="top" char="." charoff="50">
<italic>Auto/Allo</italic>
</th>
</tr>
</thead>
<tbody valign="top">
<tr>
<td align="left" valign="top" charoff="50">
<italic>Neurological diseases</italic>
</td>
<td align="char" valign="top" char="." charoff="50">9/0</td>
<td align="char" valign="top" char="." charoff="50">493/4</td>
<td align="char" valign="top" char="." charoff="50">502/4</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50"> Multiple sclerosis</td>
<td align="char" valign="top" char="." charoff="50">7/0</td>
<td align="char" valign="top" char="." charoff="50">462/3</td>
<td align="char" valign="top" char="." charoff="50">469/3</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50"> Other neurological diseases, for example, myasthenia gravis</td>
<td align="char" valign="top" char="." charoff="50">2/0</td>
<td align="char" valign="top" char="." charoff="50">31/1</td>
<td align="char" valign="top" char="." charoff="50">33/1</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50"> </td>
<td align="char" valign="top" char="." charoff="50"> </td>
<td align="char" valign="top" char="." charoff="50"> </td>
<td align="char" valign="top" char="." charoff="50"> </td>
</tr>
<tr>
<td align="left" valign="top" charoff="50">
<italic>Connective tissue diseases</italic>
</td>
<td align="char" valign="top" char="." charoff="50">29/1</td>
<td align="char" valign="top" char="." charoff="50">367/1</td>
<td align="char" valign="top" char="." charoff="50">396/2</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50"> Systemic sclerosis</td>
<td align="char" valign="top" char="." charoff="50">9/0</td>
<td align="char" valign="top" char="." charoff="50">257/0</td>
<td align="char" valign="top" char="." charoff="50">266/0</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50"> Systemic lupus erythematosus</td>
<td align="char" valign="top" char="." charoff="50">17/1</td>
<td align="char" valign="top" char="." charoff="50">78/1</td>
<td align="char" valign="top" char="." charoff="50">95/2</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50"> Polymyositis–dermatomyositis</td>
<td align="char" valign="top" char="." charoff="50">0</td>
<td align="char" valign="top" char="." charoff="50">16/0</td>
<td align="char" valign="top" char="." charoff="50">16/0</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50"> Other connective tissue disease, including Sjogren's disease</td>
<td align="char" valign="top" char="." charoff="50">2/0</td>
<td align="char" valign="top" char="." charoff="50">16/0</td>
<td align="char" valign="top" char="." charoff="50">18/0</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50"> </td>
<td align="char" valign="top" char="." charoff="50"> </td>
<td align="char" valign="top" char="." charoff="50"> </td>
<td align="char" valign="top" char="." charoff="50"> </td>
</tr>
<tr>
<td align="left" valign="top" charoff="50">
<italic>Arthritis</italic>
</td>
<td align="char" valign="top" char="." charoff="50">62/4</td>
<td align="char" valign="top" char="." charoff="50">90/2</td>
<td align="char" valign="top" char="." charoff="50">152/6</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50"> Rheumatoid arthritis</td>
<td align="char" valign="top" char="." charoff="50">1/0</td>
<td align="char" valign="top" char="." charoff="50">75/2</td>
<td align="char" valign="top" char="." charoff="50">76/2</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50"> Juvenile chronic arthritis</td>
<td align="char" valign="top" char="." charoff="50">61/3</td>
<td align="char" valign="top" char="." charoff="50">10/0</td>
<td align="char" valign="top" char="." charoff="50">71/3</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50"> Other arthritis, including psoriatic</td>
<td align="char" valign="top" char="." charoff="50">0/1</td>
<td align="char" valign="top" char="." charoff="50">5/0</td>
<td align="char" valign="top" char="." charoff="50">5/1</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50"> </td>
<td align="char" valign="top" char="." charoff="50"> </td>
<td align="char" valign="top" char="." charoff="50"> </td>
<td align="char" valign="top" char="." charoff="50"> </td>
</tr>
<tr>
<td align="left" valign="top" charoff="50">Vasculitis</td>
<td align="char" valign="top" char="." charoff="50">3/3</td>
<td align="char" valign="top" char="." charoff="50">26/1</td>
<td align="char" valign="top" char="." charoff="50">29/4</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50"> </td>
<td align="char" valign="top" char="." charoff="50"> </td>
<td align="char" valign="top" char="." charoff="50"> </td>
<td align="char" valign="top" char="." charoff="50"> </td>
</tr>
<tr>
<td align="left" valign="top" charoff="50">
<italic>Haematological</italic>
</td>
<td align="char" valign="top" char="." charoff="50">7/21</td>
<td align="char" valign="top" char="." charoff="50">31/13</td>
<td align="char" valign="top" char="." charoff="50">38/34</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50"> Immune thrombocytopenia</td>
<td align="char" valign="top" char="." charoff="50">3/1</td>
<td align="char" valign="top" char="." charoff="50">18/3</td>
<td align="char" valign="top" char="." charoff="50">21/4</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50"> Autoimmune haemolytic anaemia</td>
<td align="char" valign="top" char="." charoff="50">1/3</td>
<td align="char" valign="top" char="." charoff="50">8/2</td>
<td align="char" valign="top" char="." charoff="50">9/5</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50"> Evans' syndrome</td>
<td align="char" valign="top" char="." charoff="50">2/10</td>
<td align="char" valign="top" char="." charoff="50">3/2</td>
<td align="char" valign="top" char="." charoff="50">5/12</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50"> Other haematological, including pure red/white cell aplasia</td>
<td align="char" valign="top" char="." charoff="50">1/7</td>
<td align="char" valign="top" char="." charoff="50">2/6</td>
<td align="char" valign="top" char="." charoff="50">3/13</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50"> </td>
<td align="char" valign="top" char="." charoff="50"> </td>
<td align="char" valign="top" char="." charoff="50"> </td>
<td align="char" valign="top" char="." charoff="50"> </td>
</tr>
<tr>
<td align="left" valign="top" charoff="50">
<italic>Inflammatory bowel disease</italic>
</td>
<td align="char" valign="top" char="." charoff="50">8/5</td>
<td align="char" valign="top" char="." charoff="50">51/1</td>
<td align="char" valign="top" char="." charoff="50">59/6</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50"> Crohn's disease</td>
<td align="char" valign="top" char="." charoff="50">7/1</td>
<td align="char" valign="top" char="." charoff="50">49/0</td>
<td align="char" valign="top" char="." charoff="50">56/1</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50"> Ulcerative colitis</td>
<td align="char" valign="top" char="." charoff="50">1/2</td>
<td align="char" valign="top" char="." charoff="50">1/0</td>
<td align="char" valign="top" char="." charoff="50">1/3</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50"> Other</td>
<td align="char" valign="top" char="." charoff="50">0/2</td>
<td align="char" valign="top" char="." charoff="50">1/1</td>
<td align="char" valign="top" char="." charoff="50">1/3</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50"> </td>
<td align="char" valign="top" char="." charoff="50"> </td>
<td align="char" valign="top" char="." charoff="50"> </td>
<td align="char" valign="top" char="." charoff="50"> </td>
</tr>
<tr>
<td align="left" valign="top" charoff="50">Type 1 diabetes</td>
<td align="char" valign="top" char="." charoff="50"> </td>
<td align="char" valign="top" char="." charoff="50">10/0</td>
<td align="char" valign="top" char="." charoff="50">10/0</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50">Others</td>
<td align="char" valign="top" char="." charoff="50">1/6</td>
<td align="char" valign="top" char="." charoff="50">22/2</td>
<td align="char" valign="top" char="." charoff="50">23/8</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50">Total</td>
<td align="char" valign="top" char="." charoff="50">119/40</td>
<td align="char" valign="top" char="." charoff="50">1090/24</td>
<td align="char" valign="top" char="." charoff="50">1209/64</td>
</tr>
</tbody>
</table>
<table-wrap-foot>
<fn id="t1-fn1">
<p>Abbreviation: EBMT=European Group for Blood and Marrow Transplantation.</p>
</fn>
<fn id="t1-fn2">
<label>a</label>
<p>Paediatric=less than 18 years old at HSCT.</p>
</fn>
</table-wrap-foot>
</table-wrap>
<table-wrap id="tbl2">
<label>Table 2</label>
<caption>
<title>EBMT disease indications—(a) adults and (b) paediatrics</title>
</caption>
<table frame="hsides" rules="groups" border="1">
<colgroup>
<col align="left"></col>
<col align="center"></col>
<col align="center"></col>
<col align="center"></col>
</colgroup>
<thead valign="bottom">
<tr>
<th align="left" valign="top" charoff="50">
<italic>Disease</italic>
</th>
<th align="center" valign="top" charoff="50">
<italic>Sibling donor</italic>
</th>
<th align="center" valign="top" charoff="50">
<italic>Well-matched unrelated</italic>
<xref ref-type="fn" rid="t2-fn2">a</xref>
</th>
<th align="center" valign="top" charoff="50">
<italic>Autologous</italic>
</th>
</tr>
</thead>
<tbody valign="top">
<tr>
<td colspan="4" align="left" valign="top" charoff="50">
<italic>(a)</italic>
</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50">MS</td>
<td align="center" valign="top" charoff="50">D/III</td>
<td align="center" valign="top" charoff="50">GNR/III</td>
<td align="center" valign="top" charoff="50">CO/II</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50">SSc</td>
<td align="center" valign="top" charoff="50">D/III</td>
<td align="center" valign="top" charoff="50">GNR/III</td>
<td align="center" valign="top" charoff="50">CO/II</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50">SLE</td>
<td align="center" valign="top" charoff="50">D/III</td>
<td align="center" valign="top" charoff="50">GNR/III</td>
<td align="center" valign="top" charoff="50">CO/II</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50">Crohn's</td>
<td align="center" valign="top" charoff="50">GNR/III</td>
<td align="center" valign="top" charoff="50">GNR/III</td>
<td align="center" valign="top" charoff="50">CO/II</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50">RA</td>
<td align="center" valign="top" charoff="50">GNR/III</td>
<td align="center" valign="top" charoff="50">GNR/III</td>
<td align="center" valign="top" charoff="50">CO/II</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50">Vasculitis</td>
<td align="center" valign="top" charoff="50">GNR/III</td>
<td align="center" valign="top" charoff="50">GNR/III</td>
<td align="center" valign="top" charoff="50">CO/II</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50">Polymyositis–dermatomyositis</td>
<td align="center" valign="top" charoff="50">GNR/III</td>
<td align="center" valign="top" charoff="50">GNR/III</td>
<td align="center" valign="top" charoff="50">CO/II</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50">CIPD</td>
<td align="center" valign="top" charoff="50">GNR/III</td>
<td align="center" valign="top" charoff="50">GNR/III</td>
<td align="center" valign="top" charoff="50">CO/II</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50">NMO</td>
<td align="center" valign="top" charoff="50">GNR/III</td>
<td align="center" valign="top" charoff="50">GNR/III</td>
<td align="center" valign="top" charoff="50">CO/II</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50">Cytopenia</td>
<td align="center" valign="top" charoff="50">CO/II</td>
<td align="center" valign="top" charoff="50">D/III</td>
<td align="center" valign="top" charoff="50">CO/II</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50">T1D</td>
<td align="center" valign="top" charoff="50">GNR/III</td>
<td align="center" valign="top" charoff="50">GNR/III</td>
<td align="center" valign="top" charoff="50">D/III</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50">RCD II</td>
<td align="center" valign="top" charoff="50">GNR/III</td>
<td align="center" valign="top" charoff="50">GNR/III</td>
<td align="center" valign="top" charoff="50">D/III</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50"> </td>
<td align="center" valign="top" charoff="50"> </td>
<td align="center" valign="top" charoff="50"> </td>
<td align="center" valign="top" charoff="50"> </td>
</tr>
<tr>
<td colspan="4" align="left" valign="top" charoff="50">
<italic>(b)</italic>
</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50">JIA</td>
<td align="center" valign="top" charoff="50">D/III</td>
<td align="center" valign="top" charoff="50">GNR/III</td>
<td align="center" valign="top" charoff="50">CO/II</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50">JSSc</td>
<td align="center" valign="top" charoff="50">D/III</td>
<td align="center" valign="top" charoff="50">GNR/III</td>
<td align="center" valign="top" charoff="50">CO/II</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50">JSLE</td>
<td align="center" valign="top" charoff="50">D/III</td>
<td align="center" valign="top" charoff="50">GNR/III</td>
<td align="center" valign="top" charoff="50">CO/II</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50">Crohn's</td>
<td align="center" valign="top" charoff="50">GNR/III</td>
<td align="center" valign="top" charoff="50">GNR/III</td>
<td align="center" valign="top" charoff="50">CO/II</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50">Vasculitis</td>
<td align="center" valign="top" charoff="50">GNR/III</td>
<td align="center" valign="top" charoff="50">GNR/III</td>
<td align="center" valign="top" charoff="50">CO/II</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50">Polymyositis–dermatomyositis</td>
<td align="center" valign="top" charoff="50">GNR/III</td>
<td align="center" valign="top" charoff="50">GNR/III</td>
<td align="center" valign="top" charoff="50">CO/II</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50">Cytopenia</td>
<td align="center" valign="top" charoff="50">CO/II</td>
<td align="center" valign="top" charoff="50">CO/II</td>
<td align="center" valign="top" charoff="50">CO/II</td>
</tr>
<tr>
<td align="left" valign="top" charoff="50">T1D</td>
<td align="center" valign="top" charoff="50">GNR/III</td>
<td align="center" valign="top" charoff="50">GNR/III</td>
<td align="center" valign="top" charoff="50">D/III</td>
</tr>
</tbody>
</table>
<table-wrap-foot>
<fn id="t2-fn1">
<p>Abbreviations: CIPD=chronic inflammatory demyelinating polyradiculoneuropathy; EBMT=European Group for Blood and Marrow Transplantation; JIA=juvenile chronic arthritis; JSLE=juvenile systemic lupus erythematosus; JSSc=juvenile systemic sclerosis; MS=multiple sclerosis; NMO=neuromyelitis optica; T1D=type 1 diabetes; RA=rheumatoid arthritis; RCD II=refractory type II coeliac disease; SSc=systemic sclerosis; SLE=systemic lupus erythematosus.</p>
</fn>
<fn id="t2-fn2">
<label>a</label>
<p>A well-matched unrelated donor is defined as a 9/10 or 10/10 identical donor based on HLA high-resolution typing for class I (HLA-A, -B, -C) and II (HLA-DRB1, DQB1).</p>
</fn>
<fn id="t2-fn3">
<p>EBMT grades of evidence:
<sup>
<xref ref-type="bibr" rid="bib78">78</xref>
</sup>
I=evidence from at least one well-executed randomized trial; II=evidence from at least one well-designed clinical trial without randomization: cohort or case-controlled analytical studies (preferably from more than one centre), multiple time-series studies: or dramatic results from uncontrolled experiments; III=evidence from opinions of respected authorities based on clinical experience, descriptive studies or reports from expert committee.</p>
</fn>
<fn id="t2-fn4">
<p>EBMT grades of recommendation:
<sup>
<xref ref-type="bibr" rid="bib78">78</xref>
</sup>
CO=Clinical Option: can be carried out after careful assessment of risks and benefits; D=developmental; GNR=generally not recommended; S=standard: generally indicated in suitable patients.</p>
</fn>
</table-wrap-foot>
</table-wrap>
</floats-group>
</pmc>
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