Pediatric acute GVHD: clinical phenotype and response to upfront steroids
To better understand the clinical phenotype of acute graft-versus-host disease (GVHD) in children, we examined the GVHD clinical stage, grade, and response to prednisone 60 mg/m 2 /day PO in a diverse group of 370 pediatric patients with acute GVHD treated from 1990 to 2016 at a single institution....
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| Veröffentlicht in: | Bone marrow transplantation (Basingstoke) 2020-01, Vol.55 (1), p.165-171 |
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| creator | MacMillan, Margaret L. Holtan, Shernan G. Rashidi, Armin DeFor, Todd E. Blazar, Bruce R. Weisdorf, Daniel J. |
| description | To better understand the clinical phenotype of acute graft-versus-host disease (GVHD) in children, we examined the GVHD clinical stage, grade, and response to prednisone 60 mg/m
2
/day PO in a diverse group of 370 pediatric patients with acute GVHD treated from 1990 to 2016 at a single institution. Overall response [complete response (CR) + partial response (PR)] at day 28 occurred in 65%, (CR 52%; PR 13%). Initial GVHD grade did not predict day 28 response. However, the Minnesota GVHD Risk Score predicted response with 68% standard risk (SR)-GVHD patients achieving CR/PR at day 28 versus 48% high risk (HR)-GVHD patients (
p
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| doi_str_mv | 10.1038/s41409-019-0651-9 |
| format | Article |
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2
/day PO in a diverse group of 370 pediatric patients with acute GVHD treated from 1990 to 2016 at a single institution. Overall response [complete response (CR) + partial response (PR)] at day 28 occurred in 65%, (CR 52%; PR 13%). Initial GVHD grade did not predict day 28 response. However, the Minnesota GVHD Risk Score predicted response with 68% standard risk (SR)-GVHD patients achieving CR/PR at day 28 versus 48% high risk (HR)-GVHD patients (
p
< 0.01). Multivariable analysis confirmed that response rates were lower in patients with HR-GVHD [odds ratio (OR), 0.4,
p
< 0.01] and in recipients of HLA mismatched URD (OR 0.4,
p
= 0.03). Transplant-related mortality (TRM) at 2 years was greater in HR-GVHD patients, recipients of HLA-partially matched or mismatched unrelated donor (URD) grafts, but not umbilical cord blood (UCB). These data highlight the importance of including children in novel acute GVHD treatment trials. Compared with initial GVHD grade, the Minnesota GVHD Risk Score better demarcates risk of steroid failure and TRM in children and could be used for risk stratification in pediatric acute GVHD studies.</description><identifier>ISSN: 0268-3369</identifier><identifier>EISSN: 1476-5365</identifier><identifier>DOI: 10.1038/s41409-019-0651-9</identifier><identifier>PMID: 31477785</identifier><language>eng</language><publisher>London: Nature Publishing Group UK</publisher><subject>692/308/2171 ; 692/699/249/1529 ; 692/700/1720 ; 692/700/1750 ; Cell Biology ; Child ; Children ; Clinical trials ; Cord blood ; Corticosteroids ; Genetic aspects ; Graft versus host disease ; Graft vs Host Disease - drug therapy ; Graft-versus-host reaction ; Grafts ; Health aspects ; Hematology ; Hematopoietic Stem Cell Transplantation ; Histocompatibility antigen HLA ; Humans ; Internal Medicine ; Medicine ; Medicine & Public Health ; Patients ; Pediatrics ; Phenotype ; Phenotypes ; Prednisone ; Prednisone - therapeutic use ; Public Health ; Risk ; Stem cell transplantation ; Stem Cells ; Steroid hormones ; Steroids ; Umbilical cord ; Unrelated Donors</subject><ispartof>Bone marrow transplantation (Basingstoke), 2020-01, Vol.55 (1), p.165-171</ispartof><rights>The Author(s), under exclusive licence to Springer Nature Limited 2019</rights><rights>COPYRIGHT 2020 Nature Publishing Group</rights><rights>Copyright Nature Publishing Group Jan 2020</rights><rights>The Author(s), under exclusive licence to Springer Nature Limited 2019.</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c596t-d0d005c5662732c830e7dbb14b516cca754ba1d615b288bb901055b87dabe5b43</citedby><cites>FETCH-LOGICAL-c596t-d0d005c5662732c830e7dbb14b516cca754ba1d615b288bb901055b87dabe5b43</cites><orcidid>0000-0002-0755-0852</orcidid></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://link.springer.com/content/pdf/10.1038/s41409-019-0651-9$$EPDF$$P50$$Gspringer$$H</linktopdf><linktohtml>$$Uhttps://link.springer.com/10.1038/s41409-019-0651-9$$EHTML$$P50$$Gspringer$$H</linktohtml><link.rule.ids>230,314,776,780,881,27901,27902,41464,42533,51294</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/31477785$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>MacMillan, Margaret L.</creatorcontrib><creatorcontrib>Holtan, Shernan G.</creatorcontrib><creatorcontrib>Rashidi, Armin</creatorcontrib><creatorcontrib>DeFor, Todd E.</creatorcontrib><creatorcontrib>Blazar, Bruce R.</creatorcontrib><creatorcontrib>Weisdorf, Daniel J.</creatorcontrib><title>Pediatric acute GVHD: clinical phenotype and response to upfront steroids</title><title>Bone marrow transplantation (Basingstoke)</title><addtitle>Bone Marrow Transplant</addtitle><addtitle>Bone Marrow Transplant</addtitle><description>To better understand the clinical phenotype of acute graft-versus-host disease (GVHD) in children, we examined the GVHD clinical stage, grade, and response to prednisone 60 mg/m
2
/day PO in a diverse group of 370 pediatric patients with acute GVHD treated from 1990 to 2016 at a single institution. Overall response [complete response (CR) + partial response (PR)] at day 28 occurred in 65%, (CR 52%; PR 13%). Initial GVHD grade did not predict day 28 response. However, the Minnesota GVHD Risk Score predicted response with 68% standard risk (SR)-GVHD patients achieving CR/PR at day 28 versus 48% high risk (HR)-GVHD patients (
p
< 0.01). Multivariable analysis confirmed that response rates were lower in patients with HR-GVHD [odds ratio (OR), 0.4,
p
< 0.01] and in recipients of HLA mismatched URD (OR 0.4,
p
= 0.03). Transplant-related mortality (TRM) at 2 years was greater in HR-GVHD patients, recipients of HLA-partially matched or mismatched unrelated donor (URD) grafts, but not umbilical cord blood (UCB). These data highlight the importance of including children in novel acute GVHD treatment trials. Compared with initial GVHD grade, the Minnesota GVHD Risk Score better demarcates risk of steroid failure and TRM in children and could be used for risk stratification in pediatric acute GVHD studies.</description><subject>692/308/2171</subject><subject>692/699/249/1529</subject><subject>692/700/1720</subject><subject>692/700/1750</subject><subject>Cell Biology</subject><subject>Child</subject><subject>Children</subject><subject>Clinical trials</subject><subject>Cord blood</subject><subject>Corticosteroids</subject><subject>Genetic aspects</subject><subject>Graft versus host disease</subject><subject>Graft vs Host Disease - drug therapy</subject><subject>Graft-versus-host reaction</subject><subject>Grafts</subject><subject>Health aspects</subject><subject>Hematology</subject><subject>Hematopoietic Stem Cell Transplantation</subject><subject>Histocompatibility antigen HLA</subject><subject>Humans</subject><subject>Internal Medicine</subject><subject>Medicine</subject><subject>Medicine & Public Health</subject><subject>Patients</subject><subject>Pediatrics</subject><subject>Phenotype</subject><subject>Phenotypes</subject><subject>Prednisone</subject><subject>Prednisone - 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Academic</collection><collection>PubMed Central (Full Participant titles)</collection><jtitle>Bone marrow transplantation (Basingstoke)</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>MacMillan, Margaret L.</au><au>Holtan, Shernan G.</au><au>Rashidi, Armin</au><au>DeFor, Todd E.</au><au>Blazar, Bruce R.</au><au>Weisdorf, Daniel J.</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Pediatric acute GVHD: clinical phenotype and response to upfront steroids</atitle><jtitle>Bone marrow transplantation (Basingstoke)</jtitle><stitle>Bone Marrow Transplant</stitle><addtitle>Bone Marrow Transplant</addtitle><date>2020-01-01</date><risdate>2020</risdate><volume>55</volume><issue>1</issue><spage>165</spage><epage>171</epage><pages>165-171</pages><issn>0268-3369</issn><eissn>1476-5365</eissn><abstract>To better understand the clinical phenotype of acute graft-versus-host disease (GVHD) in children, we examined the GVHD clinical stage, grade, and response to prednisone 60 mg/m
2
/day PO in a diverse group of 370 pediatric patients with acute GVHD treated from 1990 to 2016 at a single institution. Overall response [complete response (CR) + partial response (PR)] at day 28 occurred in 65%, (CR 52%; PR 13%). Initial GVHD grade did not predict day 28 response. However, the Minnesota GVHD Risk Score predicted response with 68% standard risk (SR)-GVHD patients achieving CR/PR at day 28 versus 48% high risk (HR)-GVHD patients (
p
< 0.01). Multivariable analysis confirmed that response rates were lower in patients with HR-GVHD [odds ratio (OR), 0.4,
p
< 0.01] and in recipients of HLA mismatched URD (OR 0.4,
p
= 0.03). Transplant-related mortality (TRM) at 2 years was greater in HR-GVHD patients, recipients of HLA-partially matched or mismatched unrelated donor (URD) grafts, but not umbilical cord blood (UCB). These data highlight the importance of including children in novel acute GVHD treatment trials. Compared with initial GVHD grade, the Minnesota GVHD Risk Score better demarcates risk of steroid failure and TRM in children and could be used for risk stratification in pediatric acute GVHD studies.</abstract><cop>London</cop><pub>Nature Publishing Group UK</pub><pmid>31477785</pmid><doi>10.1038/s41409-019-0651-9</doi><tpages>7</tpages><orcidid>https://orcid.org/0000-0002-0755-0852</orcidid><oa>free_for_read</oa></addata></record> |
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| issn | 0268-3369 1476-5365 |
| language | eng |
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| source | MEDLINE; Springer Nature - Complete Springer Journals; Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals |
| subjects | 692/308/2171 692/699/249/1529 692/700/1720 692/700/1750 Cell Biology Child Children Clinical trials Cord blood Corticosteroids Genetic aspects Graft versus host disease Graft vs Host Disease - drug therapy Graft-versus-host reaction Grafts Health aspects Hematology Hematopoietic Stem Cell Transplantation Histocompatibility antigen HLA Humans Internal Medicine Medicine Medicine & Public Health Patients Pediatrics Phenotype Phenotypes Prednisone Prednisone - therapeutic use Public Health Risk Stem cell transplantation Stem Cells Steroid hormones Steroids Umbilical cord Unrelated Donors |
| title | Pediatric acute GVHD: clinical phenotype and response to upfront steroids |
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