Stable to improved cardiac and pulmonary function in children with high-risk sickle cell disease following haploidentical stem cell transplantation
Children with sickle cell disease (SCD) are at high-risk of progressive, chronic pulmonary and cardiac dysfunction. In this prospective multicenter Phase II trial of myeloimmunoablative conditioning followed by haploidentical stem cell transplantation in children with high-risk SCD, 19 patients, 2.0...
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| Veröffentlicht in: | Bone marrow transplantation (Basingstoke) 2021-09, Vol.56 (9), p.2221-2230 |
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| creator | Friedman, Deborah Dozor, Allen J. Milner, Jordan D’Souza, Marise Talano, Julie-An Moore, Theodore B. Shenoy, Shalini Shi, Qiuhu Walters, Mark C. Vichinsky, Elliott Parsons, Susan K. Braniecki, Suzanne Moorthy, Chitti R. Ayello, Janet Flower, Allyson Morris, Erin Mahanti, Harshini Fabricatore, Sandra Klejmont, Liana van de Ven, Carmella Baxter-Lowe, Lee Ann Cairo, Mitchell S. |
| description | Children with sickle cell disease (SCD) are at high-risk of progressive, chronic pulmonary and cardiac dysfunction. In this prospective multicenter Phase II trial of myeloimmunoablative conditioning followed by haploidentical stem cell transplantation in children with high-risk SCD, 19 patients, 2.0–21.0 years of age, were enrolled with one or more of the following: history of (1) overt stroke; (2) silent stroke; (3) elevated transcranial Doppler velocity; (4) multiple vaso-occlusive crises; and/or (5) two or more acute chest syndromes and received haploidentical transplants from 18 parental donors. Cardiac and pulmonary centralized cores were established. Pulmonary function results were expressed as percent of the median of healthy reference cohorts, matched for age, sex, height and race. At 2 years, pulmonary functions including forced expiratory volume (FEV), FEV
1
/ forced vital capacity (FVC), total lung capacity (TLC), diffusing capacity of lung for carbon monoxide (DLCO) were stable to improved compared to baseline values. Importantly, specific airway conductance was significantly improved at 2 years (
p
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| doi_str_mv | 10.1038/s41409-021-01298-7 |
| format | Article |
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1
/ forced vital capacity (FVC), total lung capacity (TLC), diffusing capacity of lung for carbon monoxide (DLCO) were stable to improved compared to baseline values. Importantly, specific airway conductance was significantly improved at 2 years (
p
< 0.004). Left ventricular systolic function (fractional shortening) and tricuspid regurgitant velocity were stable at 2 years. These results demonstrate that haploidentical stem cell transplantation can stabilize or improve cardiopulmonary function in patients with SCD.</description><identifier>ISSN: 0268-3369</identifier><identifier>EISSN: 1476-5365</identifier><identifier>DOI: 10.1038/s41409-021-01298-7</identifier><identifier>PMID: 33958740</identifier><language>eng</language><publisher>London: Nature Publishing Group UK</publisher><subject>692/699/1541 ; 692/700/1720 ; Anemia in children ; Anemia, Sickle Cell - therapy ; Carbon monoxide ; Cardiac conditioning ; Care and treatment ; Cell Biology ; Child ; Children ; Complications and side effects ; Health risks ; Heart diseases ; Hematology ; Hematopoietic Stem Cell Transplantation ; Humans ; Immunotherapy ; Internal Medicine ; Lung ; Lung diseases ; Lungs ; Medicine ; Medicine & Public Health ; Patient outcomes ; Pediatric research ; Prospective Studies ; Public Health ; Pulmonary functions ; Respiratory function ; Risk ; Risk factors ; Sickle cell anemia ; Sickle cell disease ; Stem cell transplantation ; Stem Cells ; Transplantation ; Transplants ; Ultrasound ; Velocity ; Ventricle ; Vital Capacity</subject><ispartof>Bone marrow transplantation (Basingstoke), 2021-09, Vol.56 (9), p.2221-2230</ispartof><rights>The Author(s), under exclusive licence to Springer Nature Limited 2021</rights><rights>2021. The Author(s), under exclusive licence to Springer Nature Limited.</rights><rights>COPYRIGHT 2021 Nature Publishing Group</rights><rights>The Author(s), under exclusive licence to Springer Nature Limited 2021.</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c572t-85bd0e0f431ee28e3b7ace1aeb4bc3b80c852bd44a229c1551eaf8711de908983</citedby><cites>FETCH-LOGICAL-c572t-85bd0e0f431ee28e3b7ace1aeb4bc3b80c852bd44a229c1551eaf8711de908983</cites><orcidid>0000-0002-2075-434X</orcidid></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><link.rule.ids>230,314,780,784,885,27924,27925</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/33958740$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Friedman, Deborah</creatorcontrib><creatorcontrib>Dozor, Allen J.</creatorcontrib><creatorcontrib>Milner, Jordan</creatorcontrib><creatorcontrib>D’Souza, Marise</creatorcontrib><creatorcontrib>Talano, Julie-An</creatorcontrib><creatorcontrib>Moore, Theodore B.</creatorcontrib><creatorcontrib>Shenoy, Shalini</creatorcontrib><creatorcontrib>Shi, Qiuhu</creatorcontrib><creatorcontrib>Walters, Mark C.</creatorcontrib><creatorcontrib>Vichinsky, Elliott</creatorcontrib><creatorcontrib>Parsons, Susan K.</creatorcontrib><creatorcontrib>Braniecki, Suzanne</creatorcontrib><creatorcontrib>Moorthy, Chitti R.</creatorcontrib><creatorcontrib>Ayello, Janet</creatorcontrib><creatorcontrib>Flower, Allyson</creatorcontrib><creatorcontrib>Morris, Erin</creatorcontrib><creatorcontrib>Mahanti, Harshini</creatorcontrib><creatorcontrib>Fabricatore, Sandra</creatorcontrib><creatorcontrib>Klejmont, Liana</creatorcontrib><creatorcontrib>van de Ven, Carmella</creatorcontrib><creatorcontrib>Baxter-Lowe, Lee Ann</creatorcontrib><creatorcontrib>Cairo, Mitchell S.</creatorcontrib><title>Stable to improved cardiac and pulmonary function in children with high-risk sickle cell disease following haploidentical stem cell transplantation</title><title>Bone marrow transplantation (Basingstoke)</title><addtitle>Bone Marrow Transplant</addtitle><addtitle>Bone Marrow Transplant</addtitle><description>Children with sickle cell disease (SCD) are at high-risk of progressive, chronic pulmonary and cardiac dysfunction. In this prospective multicenter Phase II trial of myeloimmunoablative conditioning followed by haploidentical stem cell transplantation in children with high-risk SCD, 19 patients, 2.0–21.0 years of age, were enrolled with one or more of the following: history of (1) overt stroke; (2) silent stroke; (3) elevated transcranial Doppler velocity; (4) multiple vaso-occlusive crises; and/or (5) two or more acute chest syndromes and received haploidentical transplants from 18 parental donors. Cardiac and pulmonary centralized cores were established. Pulmonary function results were expressed as percent of the median of healthy reference cohorts, matched for age, sex, height and race. At 2 years, pulmonary functions including forced expiratory volume (FEV), FEV
1
/ forced vital capacity (FVC), total lung capacity (TLC), diffusing capacity of lung for carbon monoxide (DLCO) were stable to improved compared to baseline values. Importantly, specific airway conductance was significantly improved at 2 years (
p
< 0.004). Left ventricular systolic function (fractional shortening) and tricuspid regurgitant velocity were stable at 2 years. These results demonstrate that haploidentical stem cell transplantation can stabilize or improve cardiopulmonary function in patients with SCD.</description><subject>692/699/1541</subject><subject>692/700/1720</subject><subject>Anemia in children</subject><subject>Anemia, Sickle Cell - therapy</subject><subject>Carbon monoxide</subject><subject>Cardiac conditioning</subject><subject>Care and treatment</subject><subject>Cell Biology</subject><subject>Child</subject><subject>Children</subject><subject>Complications and side effects</subject><subject>Health risks</subject><subject>Heart diseases</subject><subject>Hematology</subject><subject>Hematopoietic Stem Cell Transplantation</subject><subject>Humans</subject><subject>Immunotherapy</subject><subject>Internal Medicine</subject><subject>Lung</subject><subject>Lung diseases</subject><subject>Lungs</subject><subject>Medicine</subject><subject>Medicine & Public Health</subject><subject>Patient outcomes</subject><subject>Pediatric research</subject><subject>Prospective Studies</subject><subject>Public Health</subject><subject>Pulmonary functions</subject><subject>Respiratory function</subject><subject>Risk</subject><subject>Risk factors</subject><subject>Sickle cell anemia</subject><subject>Sickle cell disease</subject><subject>Stem cell transplantation</subject><subject>Stem Cells</subject><subject>Transplantation</subject><subject>Transplants</subject><subject>Ultrasound</subject><subject>Velocity</subject><subject>Ventricle</subject><subject>Vital Capacity</subject><issn>0268-3369</issn><issn>1476-5365</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2021</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><sourceid>ABUWG</sourceid><sourceid>AFKRA</sourceid><sourceid>AZQEC</sourceid><sourceid>BENPR</sourceid><sourceid>CCPQU</sourceid><sourceid>DWQXO</sourceid><sourceid>GNUQQ</sourceid><recordid>eNp9kt1qFTEUhQdR7LH6Al5IQBBvpuZ_MjdCKf5BwQv1OmQye86kzSRjMtPic_jC5nhq2yMiuQgk31o7e2dV1XOCTwhm6k3mhOO2xpTUmNBW1c2DakN4I2vBpHhYbTCVqmZMtkfVk5wvMCacY_G4OmKsFarheFP9_LKYzgNaInLTnOIV9Mia1DtjkQk9mlc_xWDSDzSswS4uBuQCsqPzfYKArt0yotFtxzq5fImys5fFzIL3qHcZTAY0RO_jtQtbNJrZR9dDWJw1HuUFpj26JBPy7E1YzK7C0-rRYHyGZzf7cfXt_buvZx_r888fPp2dntdWNHSpleh6DHjgjABQBaxrjAVioOOdZZ3CVgna9ZwbSltLhCBgBtUQ0kOLVavYcfV27zuv3QS9LQ9Lxus5uak0rKNx-vAmuFFv45VWnMiGy2Lw-sYgxe8r5EVPLu86MgHimjUVlDOhWiIK-vIv9CKuKZT2CiVV-U-G6R21NR60C0Msde3OVJ_KhrWSSMkKdfIPqqweJmdjgMGV8wPBq3uCEYxfxhz9uht2PgTpHrQp5pxguB0GwXqXOb3PnC6Z078zp5sienF_jLeSPyErANsDuVyFLaS73v9j-wt6Z-Rc</recordid><startdate>20210901</startdate><enddate>20210901</enddate><creator>Friedman, 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to improved cardiac and pulmonary function in children with high-risk sickle cell disease following haploidentical stem cell transplantation</title><author>Friedman, Deborah ; Dozor, Allen J. ; Milner, Jordan ; D’Souza, Marise ; Talano, Julie-An ; Moore, Theodore B. ; Shenoy, Shalini ; Shi, Qiuhu ; Walters, Mark C. ; Vichinsky, Elliott ; Parsons, Susan K. ; Braniecki, Suzanne ; Moorthy, Chitti R. ; Ayello, Janet ; Flower, Allyson ; Morris, Erin ; Mahanti, Harshini ; Fabricatore, Sandra ; Klejmont, Liana ; van de Ven, Carmella ; Baxter-Lowe, Lee Ann ; Cairo, Mitchell S.</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c572t-85bd0e0f431ee28e3b7ace1aeb4bc3b80c852bd44a229c1551eaf8711de908983</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2021</creationdate><topic>692/699/1541</topic><topic>692/700/1720</topic><topic>Anemia in children</topic><topic>Anemia, Sickle Cell - therapy</topic><topic>Carbon monoxide</topic><topic>Cardiac conditioning</topic><topic>Care and treatment</topic><topic>Cell Biology</topic><topic>Child</topic><topic>Children</topic><topic>Complications and side effects</topic><topic>Health risks</topic><topic>Heart diseases</topic><topic>Hematology</topic><topic>Hematopoietic Stem Cell Transplantation</topic><topic>Humans</topic><topic>Immunotherapy</topic><topic>Internal Medicine</topic><topic>Lung</topic><topic>Lung diseases</topic><topic>Lungs</topic><topic>Medicine</topic><topic>Medicine & Public Health</topic><topic>Patient outcomes</topic><topic>Pediatric research</topic><topic>Prospective Studies</topic><topic>Public Health</topic><topic>Pulmonary functions</topic><topic>Respiratory function</topic><topic>Risk</topic><topic>Risk factors</topic><topic>Sickle cell anemia</topic><topic>Sickle cell disease</topic><topic>Stem cell transplantation</topic><topic>Stem 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transplantation (Basingstoke)</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Friedman, Deborah</au><au>Dozor, Allen J.</au><au>Milner, Jordan</au><au>D’Souza, Marise</au><au>Talano, Julie-An</au><au>Moore, Theodore B.</au><au>Shenoy, Shalini</au><au>Shi, Qiuhu</au><au>Walters, Mark C.</au><au>Vichinsky, Elliott</au><au>Parsons, Susan K.</au><au>Braniecki, Suzanne</au><au>Moorthy, Chitti R.</au><au>Ayello, Janet</au><au>Flower, Allyson</au><au>Morris, Erin</au><au>Mahanti, Harshini</au><au>Fabricatore, Sandra</au><au>Klejmont, Liana</au><au>van de Ven, Carmella</au><au>Baxter-Lowe, Lee Ann</au><au>Cairo, Mitchell S.</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Stable to improved cardiac and pulmonary function in children with high-risk sickle cell disease following haploidentical stem cell transplantation</atitle><jtitle>Bone marrow transplantation (Basingstoke)</jtitle><stitle>Bone Marrow Transplant</stitle><addtitle>Bone Marrow Transplant</addtitle><date>2021-09-01</date><risdate>2021</risdate><volume>56</volume><issue>9</issue><spage>2221</spage><epage>2230</epage><pages>2221-2230</pages><issn>0268-3369</issn><eissn>1476-5365</eissn><abstract>Children with sickle cell disease (SCD) are at high-risk of progressive, chronic pulmonary and cardiac dysfunction. In this prospective multicenter Phase II trial of myeloimmunoablative conditioning followed by haploidentical stem cell transplantation in children with high-risk SCD, 19 patients, 2.0–21.0 years of age, were enrolled with one or more of the following: history of (1) overt stroke; (2) silent stroke; (3) elevated transcranial Doppler velocity; (4) multiple vaso-occlusive crises; and/or (5) two or more acute chest syndromes and received haploidentical transplants from 18 parental donors. Cardiac and pulmonary centralized cores were established. Pulmonary function results were expressed as percent of the median of healthy reference cohorts, matched for age, sex, height and race. At 2 years, pulmonary functions including forced expiratory volume (FEV), FEV
1
/ forced vital capacity (FVC), total lung capacity (TLC), diffusing capacity of lung for carbon monoxide (DLCO) were stable to improved compared to baseline values. Importantly, specific airway conductance was significantly improved at 2 years (
p
< 0.004). Left ventricular systolic function (fractional shortening) and tricuspid regurgitant velocity were stable at 2 years. These results demonstrate that haploidentical stem cell transplantation can stabilize or improve cardiopulmonary function in patients with SCD.</abstract><cop>London</cop><pub>Nature Publishing Group UK</pub><pmid>33958740</pmid><doi>10.1038/s41409-021-01298-7</doi><tpages>10</tpages><orcidid>https://orcid.org/0000-0002-2075-434X</orcidid><oa>free_for_read</oa></addata></record> |
| fulltext | fulltext |
| identifier | ISSN: 0268-3369 |
| ispartof | Bone marrow transplantation (Basingstoke), 2021-09, Vol.56 (9), p.2221-2230 |
| issn | 0268-3369 1476-5365 |
| language | eng |
| recordid | cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_8416746 |
| source | MEDLINE; EZB-FREE-00999 freely available EZB journals; Alma/SFX Local Collection |
| subjects | 692/699/1541 692/700/1720 Anemia in children Anemia, Sickle Cell - therapy Carbon monoxide Cardiac conditioning Care and treatment Cell Biology Child Children Complications and side effects Health risks Heart diseases Hematology Hematopoietic Stem Cell Transplantation Humans Immunotherapy Internal Medicine Lung Lung diseases Lungs Medicine Medicine & Public Health Patient outcomes Pediatric research Prospective Studies Public Health Pulmonary functions Respiratory function Risk Risk factors Sickle cell anemia Sickle cell disease Stem cell transplantation Stem Cells Transplantation Transplants Ultrasound Velocity Ventricle Vital Capacity |
| title | Stable to improved cardiac and pulmonary function in children with high-risk sickle cell disease following haploidentical stem cell transplantation |
| url | https://sfx.bib-bvb.de/sfx_tum?ctx_ver=Z39.88-2004&ctx_enc=info:ofi/enc:UTF-8&ctx_tim=2024-12-27T05%3A47%3A52IST&url_ver=Z39.88-2004&url_ctx_fmt=infofi/fmt:kev:mtx:ctx&rfr_id=info:sid/primo.exlibrisgroup.com:primo3-Article-gale_pubme&rft_val_fmt=info:ofi/fmt:kev:mtx:journal&rft.genre=article&rft.atitle=Stable%20to%20improved%20cardiac%20and%20pulmonary%20function%20in%20children%20with%20high-risk%20sickle%20cell%20disease%20following%20haploidentical%20stem%20cell%20transplantation&rft.jtitle=Bone%20marrow%20transplantation%20(Basingstoke)&rft.au=Friedman,%20Deborah&rft.date=2021-09-01&rft.volume=56&rft.issue=9&rft.spage=2221&rft.epage=2230&rft.pages=2221-2230&rft.issn=0268-3369&rft.eissn=1476-5365&rft_id=info:doi/10.1038/s41409-021-01298-7&rft_dat=%3Cgale_pubme%3EA673961663%3C/gale_pubme%3E%3Curl%3E%3C/url%3E&disable_directlink=true&sfx.directlink=off&sfx.report_link=0&rft_id=info:oai/&rft_pqid=2568103302&rft_id=info:pmid/33958740&rft_galeid=A673961663&rfr_iscdi=true |