Haploidentical transplantation and posttransplant cyclophosphamide for treating aplastic anemia patients: a report from the EBMT Severe Aplastic Anemia Working Party
In the absence of an HLA-matched donor, the best treatment for acquired aplastic anemia patients refractory to immunosuppression is unclear. We collected and analyzed data from all acquired aplastic anemia patients who underwent a haploidentical transplantation with posttransplant cyclophosphamide i...
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| Veröffentlicht in: | Bone marrow transplantation (Basingstoke) 2020-06, Vol.55 (6), p.1050-1058 |
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| creator | Prata, Pedro H. Eikema, Dirk-Jan Afansyev, Boris Bosman, Paul Smiers, Frans Diez-Martin, José L. Arrais-Rodrigues, Celso Koc, Yener Poiré, Xavier Sirvent, Anne Kröger, Nicolaus Porta, Fulvio Holter, Wolfgang Bloor, Adrian Jubert, Charlotte Ganser, Arnold Tanase, Alina Ménard, Anne-Lise Pioltelli, Pietro Pérez-Simón, José A. Ho, Aloysius Aljurf, Mahmoud Russell, Nigel Labussiere-Wallet, Helene Kerre, Tessa Rocha, Vanderson Socié, Gérard Risitano, Antonio Dufour, Carlo Peffault de Latour, Régis |
| description | In the absence of an HLA-matched donor, the best treatment for acquired aplastic anemia patients refractory to immunosuppression is unclear. We collected and analyzed data from all acquired aplastic anemia patients who underwent a haploidentical transplantation with posttransplant cyclophosphamide in Europe from 2011 to 2017 (
n
= 33). The cumulative incidence of neutrophil engraftment was 67% (CI
95%
: 51–83%) at D +28 and was unaffected by age group, stem cell source, ATG use, or Baltimore conditioning regimen. The cumulative incidence of grades II–III acute GvHD was 23% at D +100, and limited chronic GvHD was 10% (0–20) at 2 years, without cases of grade IV acute or extensive chronic GvHD. Two-year overall survival was 78% (64–93), and 2-year graft-versus-host disease-free survival was 63% (46–81). In univariate analysis, the 2-year OS was higher among patients who received the Baltimore conditioning regimen (93% (81–100) versus 64% (41–87),
p
= 0.03), whereas age group, stem cell source, and ATG use had no effect. Our results using unmanipulated haploidentical transplantation and posttransplant cyclophosphamide for treating refractory AA patients are encouraging, but warrant confirmation in a prospective study with a larger number of patients and longer follow-up. |
| doi_str_mv | 10.1038/s41409-019-0773-0 |
| format | Article |
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n
= 33). The cumulative incidence of neutrophil engraftment was 67% (CI
95%
: 51–83%) at D +28 and was unaffected by age group, stem cell source, ATG use, or Baltimore conditioning regimen. The cumulative incidence of grades II–III acute GvHD was 23% at D +100, and limited chronic GvHD was 10% (0–20) at 2 years, without cases of grade IV acute or extensive chronic GvHD. Two-year overall survival was 78% (64–93), and 2-year graft-versus-host disease-free survival was 63% (46–81). In univariate analysis, the 2-year OS was higher among patients who received the Baltimore conditioning regimen (93% (81–100) versus 64% (41–87),
p
= 0.03), whereas age group, stem cell source, and ATG use had no effect. Our results using unmanipulated haploidentical transplantation and posttransplant cyclophosphamide for treating refractory AA patients are encouraging, but warrant confirmation in a prospective study with a larger number of patients and longer follow-up.</description><identifier>ISSN: 0268-3369</identifier><identifier>EISSN: 1476-5365</identifier><identifier>DOI: 10.1038/s41409-019-0773-0</identifier><identifier>PMID: 31844137</identifier><language>eng</language><publisher>London: Nature Publishing Group UK</publisher><subject>692/699/1541/13 ; 692/700/565/2319 ; Analysis ; Anemia ; Aplastic anemia ; Bone marrow ; Care and treatment ; Cell Biology ; Conditioning ; Cyclophosphamide ; Data acquisition ; Eltrombopag ; Graft-versus-host reaction ; Hematology ; Histocompatibility antigen HLA ; Immunosuppression ; Internal Medicine ; Medical research ; Medicine ; Medicine & Public Health ; Medicine, Experimental ; Public Health ; Stem cell transplantation ; Stem Cells ; Survival ; Transplantation</subject><ispartof>Bone marrow transplantation (Basingstoke), 2020-06, Vol.55 (6), p.1050-1058</ispartof><rights>The Author(s), under exclusive licence to Springer Nature Limited 2019</rights><rights>COPYRIGHT 2020 Nature Publishing Group</rights><rights>The Author(s), under exclusive licence to Springer Nature Limited 2019.</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c564t-25a961511ca28055a6c2096b65acbc99ec1ccc5c5988b32406bb31ab0c09f13a3</citedby><cites>FETCH-LOGICAL-c564t-25a961511ca28055a6c2096b65acbc99ec1ccc5c5988b32406bb31ab0c09f13a3</cites><orcidid>0000-0003-0235-7890 ; 0000-0002-2114-7533 ; 0000-0003-1897-0227 ; 0000-0002-4744-5109</orcidid></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><link.rule.ids>314,780,784,27924,27925</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/31844137$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Prata, Pedro H.</creatorcontrib><creatorcontrib>Eikema, Dirk-Jan</creatorcontrib><creatorcontrib>Afansyev, Boris</creatorcontrib><creatorcontrib>Bosman, Paul</creatorcontrib><creatorcontrib>Smiers, Frans</creatorcontrib><creatorcontrib>Diez-Martin, José L.</creatorcontrib><creatorcontrib>Arrais-Rodrigues, Celso</creatorcontrib><creatorcontrib>Koc, Yener</creatorcontrib><creatorcontrib>Poiré, Xavier</creatorcontrib><creatorcontrib>Sirvent, Anne</creatorcontrib><creatorcontrib>Kröger, Nicolaus</creatorcontrib><creatorcontrib>Porta, Fulvio</creatorcontrib><creatorcontrib>Holter, Wolfgang</creatorcontrib><creatorcontrib>Bloor, Adrian</creatorcontrib><creatorcontrib>Jubert, Charlotte</creatorcontrib><creatorcontrib>Ganser, Arnold</creatorcontrib><creatorcontrib>Tanase, Alina</creatorcontrib><creatorcontrib>Ménard, Anne-Lise</creatorcontrib><creatorcontrib>Pioltelli, Pietro</creatorcontrib><creatorcontrib>Pérez-Simón, José A.</creatorcontrib><creatorcontrib>Ho, Aloysius</creatorcontrib><creatorcontrib>Aljurf, Mahmoud</creatorcontrib><creatorcontrib>Russell, Nigel</creatorcontrib><creatorcontrib>Labussiere-Wallet, Helene</creatorcontrib><creatorcontrib>Kerre, Tessa</creatorcontrib><creatorcontrib>Rocha, Vanderson</creatorcontrib><creatorcontrib>Socié, Gérard</creatorcontrib><creatorcontrib>Risitano, Antonio</creatorcontrib><creatorcontrib>Dufour, Carlo</creatorcontrib><creatorcontrib>Peffault de Latour, Régis</creatorcontrib><creatorcontrib>SAA WP of the EBMT</creatorcontrib><creatorcontrib>on behalf of the SAA WP of the EBMT</creatorcontrib><title>Haploidentical transplantation and posttransplant cyclophosphamide for treating aplastic anemia patients: a report from the EBMT Severe Aplastic Anemia Working Party</title><title>Bone marrow transplantation (Basingstoke)</title><addtitle>Bone Marrow Transplant</addtitle><addtitle>Bone Marrow Transplant</addtitle><description>In the absence of an HLA-matched donor, the best treatment for acquired aplastic anemia patients refractory to immunosuppression is unclear. We collected and analyzed data from all acquired aplastic anemia patients who underwent a haploidentical transplantation with posttransplant cyclophosphamide in Europe from 2011 to 2017 (
n
= 33). The cumulative incidence of neutrophil engraftment was 67% (CI
95%
: 51–83%) at D +28 and was unaffected by age group, stem cell source, ATG use, or Baltimore conditioning regimen. The cumulative incidence of grades II–III acute GvHD was 23% at D +100, and limited chronic GvHD was 10% (0–20) at 2 years, without cases of grade IV acute or extensive chronic GvHD. Two-year overall survival was 78% (64–93), and 2-year graft-versus-host disease-free survival was 63% (46–81). In univariate analysis, the 2-year OS was higher among patients who received the Baltimore conditioning regimen (93% (81–100) versus 64% (41–87),
p
= 0.03), whereas age group, stem cell source, and ATG use had no effect. Our results using unmanipulated haploidentical transplantation and posttransplant cyclophosphamide for treating refractory AA patients are encouraging, but warrant confirmation in a prospective study with a larger number of patients and longer follow-up.</description><subject>692/699/1541/13</subject><subject>692/700/565/2319</subject><subject>Analysis</subject><subject>Anemia</subject><subject>Aplastic anemia</subject><subject>Bone marrow</subject><subject>Care and treatment</subject><subject>Cell Biology</subject><subject>Conditioning</subject><subject>Cyclophosphamide</subject><subject>Data acquisition</subject><subject>Eltrombopag</subject><subject>Graft-versus-host reaction</subject><subject>Hematology</subject><subject>Histocompatibility antigen HLA</subject><subject>Immunosuppression</subject><subject>Internal Medicine</subject><subject>Medical research</subject><subject>Medicine</subject><subject>Medicine & Public Health</subject><subject>Medicine, Experimental</subject><subject>Public Health</subject><subject>Stem cell transplantation</subject><subject>Stem Cells</subject><subject>Survival</subject><subject>Transplantation</subject><issn>0268-3369</issn><issn>1476-5365</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2020</creationdate><recordtype>article</recordtype><sourceid>ABUWG</sourceid><sourceid>AFKRA</sourceid><sourceid>AZQEC</sourceid><sourceid>BENPR</sourceid><sourceid>CCPQU</sourceid><sourceid>DWQXO</sourceid><sourceid>GNUQQ</sourceid><recordid>eNp9kl9r1TAYxoso7jj9AN5IQBBvOvO_jXfHMZ0wUXDiZXibk552tklNcoTzgfY9l9LNbaISQuDN73mSN3mK4jnBRwSz-k3khGNVYpJnVbESPyhWhFeyFEyKh8UKU1mXjEl1UDyJ8QJjwjkWj4sDRmrOCatWxeUpTIPvN9al3sCAUgAXpwFcgtR7h8Bt0ORjuq0jszeDnzofpw7GrEStD1lns8BtUbaDmL2y0o49oCmXs3l8iwAFO_mQUBv8iFJn0cm7T-foq_1lg0XrG9160X334cfs9wVC2j8tHrUwRPvsej0svr0_OT8-Lc8-f_h4vD4rjZA8lVSAkkQQYoDWWAiQhmIlGynANEYpa4gxRhih6rphlGPZNIxAgw1WLWHADovXi-8U_M-djUmPfTR2yH1bv4uaMlopJjGtM_ryD_TC74LLt9OUV1wpUnH8fwrPjGR3vLYwWN271ufnNvPRei2pqBiXFc_U0V-oPDb5wYx3tu1z_Z7g1R1BZ2FIXfTDbv7YeB8kC2iCjzHYVk-hHyHsNcF6TppekqZz0vScND139uK6s10z2s1vxU20MkAXIOYtt7XhtvV_u14B_urd9A</recordid><startdate>20200601</startdate><enddate>20200601</enddate><creator>Prata, Pedro H.</creator><creator>Eikema, Dirk-Jan</creator><creator>Afansyev, Boris</creator><creator>Bosman, Paul</creator><creator>Smiers, Frans</creator><creator>Diez-Martin, José L.</creator><creator>Arrais-Rodrigues, Celso</creator><creator>Koc, Yener</creator><creator>Poiré, Xavier</creator><creator>Sirvent, Anne</creator><creator>Kröger, Nicolaus</creator><creator>Porta, Fulvio</creator><creator>Holter, Wolfgang</creator><creator>Bloor, Adrian</creator><creator>Jubert, Charlotte</creator><creator>Ganser, Arnold</creator><creator>Tanase, Alina</creator><creator>Ménard, Anne-Lise</creator><creator>Pioltelli, Pietro</creator><creator>Pérez-Simón, José A.</creator><creator>Ho, Aloysius</creator><creator>Aljurf, Mahmoud</creator><creator>Russell, Nigel</creator><creator>Labussiere-Wallet, Helene</creator><creator>Kerre, Tessa</creator><creator>Rocha, Vanderson</creator><creator>Socié, Gérard</creator><creator>Risitano, Antonio</creator><creator>Dufour, Carlo</creator><creator>Peffault de 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transplantation and posttransplant cyclophosphamide for treating aplastic anemia patients: a report from the EBMT Severe Aplastic Anemia Working Party</title><author>Prata, Pedro H. ; Eikema, Dirk-Jan ; Afansyev, Boris ; Bosman, Paul ; Smiers, Frans ; Diez-Martin, José L. ; Arrais-Rodrigues, Celso ; Koc, Yener ; Poiré, Xavier ; Sirvent, Anne ; Kröger, Nicolaus ; Porta, Fulvio ; Holter, Wolfgang ; Bloor, Adrian ; Jubert, Charlotte ; Ganser, Arnold ; Tanase, Alina ; Ménard, Anne-Lise ; Pioltelli, Pietro ; Pérez-Simón, José A. ; Ho, Aloysius ; Aljurf, Mahmoud ; Russell, Nigel ; Labussiere-Wallet, Helene ; Kerre, Tessa ; Rocha, Vanderson ; Socié, Gérard ; Risitano, Antonio ; Dufour, Carlo ; Peffault de Latour, 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Tessa</au><au>Rocha, Vanderson</au><au>Socié, Gérard</au><au>Risitano, Antonio</au><au>Dufour, Carlo</au><au>Peffault de Latour, Régis</au><aucorp>SAA WP of the EBMT</aucorp><aucorp>on behalf of the SAA WP of the EBMT</aucorp><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Haploidentical transplantation and posttransplant cyclophosphamide for treating aplastic anemia patients: a report from the EBMT Severe Aplastic Anemia Working Party</atitle><jtitle>Bone marrow transplantation (Basingstoke)</jtitle><stitle>Bone Marrow Transplant</stitle><addtitle>Bone Marrow Transplant</addtitle><date>2020-06-01</date><risdate>2020</risdate><volume>55</volume><issue>6</issue><spage>1050</spage><epage>1058</epage><pages>1050-1058</pages><issn>0268-3369</issn><eissn>1476-5365</eissn><abstract>In the absence of an HLA-matched donor, the best treatment for acquired aplastic anemia patients refractory to immunosuppression is unclear. We collected and analyzed data from all acquired aplastic anemia patients who underwent a haploidentical transplantation with posttransplant cyclophosphamide in Europe from 2011 to 2017 (
n
= 33). The cumulative incidence of neutrophil engraftment was 67% (CI
95%
: 51–83%) at D +28 and was unaffected by age group, stem cell source, ATG use, or Baltimore conditioning regimen. The cumulative incidence of grades II–III acute GvHD was 23% at D +100, and limited chronic GvHD was 10% (0–20) at 2 years, without cases of grade IV acute or extensive chronic GvHD. Two-year overall survival was 78% (64–93), and 2-year graft-versus-host disease-free survival was 63% (46–81). In univariate analysis, the 2-year OS was higher among patients who received the Baltimore conditioning regimen (93% (81–100) versus 64% (41–87),
p
= 0.03), whereas age group, stem cell source, and ATG use had no effect. Our results using unmanipulated haploidentical transplantation and posttransplant cyclophosphamide for treating refractory AA patients are encouraging, but warrant confirmation in a prospective study with a larger number of patients and longer follow-up.</abstract><cop>London</cop><pub>Nature Publishing Group UK</pub><pmid>31844137</pmid><doi>10.1038/s41409-019-0773-0</doi><tpages>9</tpages><orcidid>https://orcid.org/0000-0003-0235-7890</orcidid><orcidid>https://orcid.org/0000-0002-2114-7533</orcidid><orcidid>https://orcid.org/0000-0003-1897-0227</orcidid><orcidid>https://orcid.org/0000-0002-4744-5109</orcidid></addata></record> |
| fulltext | fulltext |
| identifier | ISSN: 0268-3369 |
| ispartof | Bone marrow transplantation (Basingstoke), 2020-06, Vol.55 (6), p.1050-1058 |
| issn | 0268-3369 1476-5365 |
| language | eng |
| recordid | cdi_proquest_miscellaneous_2327936028 |
| source | Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals; Alma/SFX Local Collection |
| subjects | 692/699/1541/13 692/700/565/2319 Analysis Anemia Aplastic anemia Bone marrow Care and treatment Cell Biology Conditioning Cyclophosphamide Data acquisition Eltrombopag Graft-versus-host reaction Hematology Histocompatibility antigen HLA Immunosuppression Internal Medicine Medical research Medicine Medicine & Public Health Medicine, Experimental Public Health Stem cell transplantation Stem Cells Survival Transplantation |
| title | Haploidentical transplantation and posttransplant cyclophosphamide for treating aplastic anemia patients: a report from the EBMT Severe Aplastic Anemia Working Party |
| url | https://sfx.bib-bvb.de/sfx_tum?ctx_ver=Z39.88-2004&ctx_enc=info:ofi/enc:UTF-8&ctx_tim=2024-12-19T07%3A55%3A30IST&url_ver=Z39.88-2004&url_ctx_fmt=infofi/fmt:kev:mtx:ctx&rfr_id=info:sid/primo.exlibrisgroup.com:primo3-Article-gale_proqu&rft_val_fmt=info:ofi/fmt:kev:mtx:journal&rft.genre=article&rft.atitle=Haploidentical%20transplantation%20and%20posttransplant%20cyclophosphamide%20for%20treating%20aplastic%20anemia%20patients:%20a%20report%20from%20the%20EBMT%20Severe%20Aplastic%20Anemia%20Working%20Party&rft.jtitle=Bone%20marrow%20transplantation%20(Basingstoke)&rft.au=Prata,%20Pedro%20H.&rft.aucorp=SAA%20WP%20of%20the%20EBMT&rft.date=2020-06-01&rft.volume=55&rft.issue=6&rft.spage=1050&rft.epage=1058&rft.pages=1050-1058&rft.issn=0268-3369&rft.eissn=1476-5365&rft_id=info:doi/10.1038/s41409-019-0773-0&rft_dat=%3Cgale_proqu%3EA625734674%3C/gale_proqu%3E%3Curl%3E%3C/url%3E&disable_directlink=true&sfx.directlink=off&sfx.report_link=0&rft_id=info:oai/&rft_pqid=2409174638&rft_id=info:pmid/31844137&rft_galeid=A625734674&rfr_iscdi=true |