A validated pediatric disease risk index for allogeneic hematopoietic cell transplantation
A disease risk index (DRI) that was developed for adults with hematologic malignancy who were undergoing hematopoietic cell transplantation is also being used to stratify children and adolescents by disease risk. Therefore, to develop and validate a DRI that can be used to stratify those with AML an...
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| Veröffentlicht in: | Blood 2021-02, Vol.137 (7), p.983-993 |
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| creator | Qayed, Muna Ahn, Kwang Woo Kitko, Carrie L. Johnson, Mariam H. Shah, Nirali N. Dvorak, Christopher Mellgren, Karin Friend, Brian D. Verneris, Michael R. Leung, Wing Toporski, Jacek Levine, John Chewning, Joseph Wayne, Alan Kapoor, Urvi Triplett, Brandon Schultz, Kirk R. Yanik, Gregory A. Eapen, Mary |
| description | A disease risk index (DRI) that was developed for adults with hematologic malignancy who were undergoing hematopoietic cell transplantation is also being used to stratify children and adolescents by disease risk. Therefore, to develop and validate a DRI that can be used to stratify those with AML and ALL by their disease risk, we analyzed 2569 patients aged 8) risk groups was 78%, 53%, 40%, and 25%, respectively (P < .0001). For ALL, 3 risk groups were identified based on age and disease status, including minimal residual disease status at transplantation. The 5-year leukemia-free survival for low (0 points), intermediate (2-4), and high (≥5) risk groups was 68%, 51%, and 33%, respectively (P < .0001). We confirmed that the risk groups could be applied to overall survival, with 5-year survival ranging from 80% to 33% and 73% to 42% for AML and ALL, respectively (P < .0001). This validated pediatric DRI, which includes age and residual disease status, can be used to facilitate prognostication and stratification of children with AML and ALL for allogeneic transplantation.
•The pediatric DRI stratified children with AML and ALL into clinically distinct risk groups based on pretransplantation information.•Risk stratification was based on age at transplant, cytogenetics, and disease status including minimal residual disease at transplant.
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| doi_str_mv | 10.1182/blood.2020009342 |
| format | Article |
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•The pediatric DRI stratified children with AML and ALL into clinically distinct risk groups based on pretransplantation information.•Risk stratification was based on age at transplant, cytogenetics, and disease status including minimal residual disease at transplant.
[Display omitted]</description><identifier>ISSN: 0006-4971</identifier><identifier>EISSN: 1528-0020</identifier><identifier>DOI: 10.1182/blood.2020009342</identifier><identifier>PMID: 33206937</identifier><language>eng</language><publisher>United States: Elsevier Inc</publisher><subject>Adolescent ; Age Factors ; Allografts ; Antineoplastic Combined Chemotherapy Protocols - therapeutic use ; Child ; Child, Preschool ; CME ; Cohort Studies ; Combined Modality Therapy ; Disease-Free Survival ; Female ; Hematopoietic Stem Cell Transplantation ; Humans ; Infant ; Kaplan-Meier Estimate ; Leukemia, Myeloid, Acute - drug therapy ; Leukemia, Myeloid, Acute - mortality ; Leukemia, Myeloid, Acute - pathology ; Leukemia, Myeloid, Acute - therapy ; Male ; Neoplasm, Residual ; Precursor Cell Lymphoblastic Leukemia-Lymphoma - drug therapy ; Precursor Cell Lymphoblastic Leukemia-Lymphoma - mortality ; Precursor Cell Lymphoblastic Leukemia-Lymphoma - pathology ; Precursor Cell Lymphoblastic Leukemia-Lymphoma - therapy ; Prognosis ; Random Allocation ; Risk Assessment ; Risk Factors ; Severity of Illness Index ; Transplantation</subject><ispartof>Blood, 2021-02, Vol.137 (7), p.983-993</ispartof><rights>2021 American Society of Hematology</rights><rights>2021</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c447t-bc03b135d7e283c948cd4ab6ada217d4571fdc6cdd5c36ea8bb83f4761c6622d3</citedby><cites>FETCH-LOGICAL-c447t-bc03b135d7e283c948cd4ab6ada217d4571fdc6cdd5c36ea8bb83f4761c6622d3</cites><orcidid>0000-0001-8220-9980 ; 0000-0003-4567-8037 ; 0000-0001-5554-5890 ; 0000-0001-8652-4400 ; 0000-0001-6012-0651 ; 0000-0002-8474-9080 ; 0000-0002-6146-3952</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/33206937$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Qayed, Muna</creatorcontrib><creatorcontrib>Ahn, Kwang Woo</creatorcontrib><creatorcontrib>Kitko, Carrie L.</creatorcontrib><creatorcontrib>Johnson, Mariam H.</creatorcontrib><creatorcontrib>Shah, Nirali N.</creatorcontrib><creatorcontrib>Dvorak, Christopher</creatorcontrib><creatorcontrib>Mellgren, Karin</creatorcontrib><creatorcontrib>Friend, Brian D.</creatorcontrib><creatorcontrib>Verneris, Michael R.</creatorcontrib><creatorcontrib>Leung, Wing</creatorcontrib><creatorcontrib>Toporski, Jacek</creatorcontrib><creatorcontrib>Levine, John</creatorcontrib><creatorcontrib>Chewning, Joseph</creatorcontrib><creatorcontrib>Wayne, Alan</creatorcontrib><creatorcontrib>Kapoor, Urvi</creatorcontrib><creatorcontrib>Triplett, Brandon</creatorcontrib><creatorcontrib>Schultz, Kirk R.</creatorcontrib><creatorcontrib>Yanik, Gregory A.</creatorcontrib><creatorcontrib>Eapen, Mary</creatorcontrib><title>A validated pediatric disease risk index for allogeneic hematopoietic cell transplantation</title><title>Blood</title><addtitle>Blood</addtitle><description>A disease risk index (DRI) that was developed for adults with hematologic malignancy who were undergoing hematopoietic cell transplantation is also being used to stratify children and adolescents by disease risk. Therefore, to develop and validate a DRI that can be used to stratify those with AML and ALL by their disease risk, we analyzed 2569 patients aged <18 years with acute myeloid (AML; n = 1224) or lymphoblastic (ALL; n = 1345) leukemia who underwent hematopoietic cell transplantation. Training and validation subsets for each disease were generated randomly with 1:1 assignment to the subsets, and separate prognostic models were derived for each disease. For AML, 4 risk groups were identified based on age, cytogenetic risk, and disease status, including minimal residual disease status at transplantation. The 5-year leukemia-free survival for low (0 points), intermediate (2, 3, 5), high (7, 8), and very high (>8) risk groups was 78%, 53%, 40%, and 25%, respectively (P < .0001). For ALL, 3 risk groups were identified based on age and disease status, including minimal residual disease status at transplantation. The 5-year leukemia-free survival for low (0 points), intermediate (2-4), and high (≥5) risk groups was 68%, 51%, and 33%, respectively (P < .0001). We confirmed that the risk groups could be applied to overall survival, with 5-year survival ranging from 80% to 33% and 73% to 42% for AML and ALL, respectively (P < .0001). This validated pediatric DRI, which includes age and residual disease status, can be used to facilitate prognostication and stratification of children with AML and ALL for allogeneic transplantation.
•The pediatric DRI stratified children with AML and ALL into clinically distinct risk groups based on pretransplantation information.•Risk stratification was based on age at transplant, cytogenetics, and disease status including minimal residual disease at transplant.
[Display omitted]</description><subject>Adolescent</subject><subject>Age Factors</subject><subject>Allografts</subject><subject>Antineoplastic Combined Chemotherapy Protocols - therapeutic use</subject><subject>Child</subject><subject>Child, Preschool</subject><subject>CME</subject><subject>Cohort Studies</subject><subject>Combined Modality Therapy</subject><subject>Disease-Free Survival</subject><subject>Female</subject><subject>Hematopoietic Stem Cell Transplantation</subject><subject>Humans</subject><subject>Infant</subject><subject>Kaplan-Meier Estimate</subject><subject>Leukemia, Myeloid, Acute - drug therapy</subject><subject>Leukemia, Myeloid, Acute - mortality</subject><subject>Leukemia, Myeloid, Acute - pathology</subject><subject>Leukemia, Myeloid, Acute - therapy</subject><subject>Male</subject><subject>Neoplasm, Residual</subject><subject>Precursor Cell Lymphoblastic Leukemia-Lymphoma - drug therapy</subject><subject>Precursor Cell Lymphoblastic Leukemia-Lymphoma - mortality</subject><subject>Precursor Cell Lymphoblastic Leukemia-Lymphoma - pathology</subject><subject>Precursor Cell Lymphoblastic Leukemia-Lymphoma - therapy</subject><subject>Prognosis</subject><subject>Random Allocation</subject><subject>Risk Assessment</subject><subject>Risk Factors</subject><subject>Severity of Illness Index</subject><subject>Transplantation</subject><issn>0006-4971</issn><issn>1528-0020</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2021</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNp1kb1vFDEQxS0EIpeDPlW0Jc0m_lrvbgqkKIIkUiQaaGisWXs2cfDZi-07hf8ehwsBCirL4zfPb-ZHyBGjJ4wN_HTyMdoTTjmldBSSvyAr1vGhpbXykqxqVbVy7NkBOcz5nlImBe9ekwMhOFWj6Ffk63mzA-8sFLTNgtZBSc401mWEjE1y-VvjgsWHZo6pAe_jLQasijvcQIlLdFjqzaD3TUkQ8uIhFCguhjfk1Qw-49unc02-fPzw-eKqvfl0eX1xftMaKfvSToaKiYnO9sgHYUY5GCthUmCBs97KrmezNcpY2xmhEIZpGsQse8WMUpxbsSbv977LdtqgNRhqEK-X5DaQfugITv_7Etydvo073Y9sYIOoBu-eDFL8vsVc9Mblx4kgYNxmzaXiktGxxlsTupeaFHNOOD9_w6h-RKJ_IdF_kNSW47_jPTf8ZlAFZ3sB1iXtHCadjcNgKoyEpmgb3f_dfwK8A57Z</recordid><startdate>20210218</startdate><enddate>20210218</enddate><creator>Qayed, Muna</creator><creator>Ahn, Kwang Woo</creator><creator>Kitko, Carrie L.</creator><creator>Johnson, Mariam H.</creator><creator>Shah, Nirali N.</creator><creator>Dvorak, Christopher</creator><creator>Mellgren, Karin</creator><creator>Friend, Brian D.</creator><creator>Verneris, Michael R.</creator><creator>Leung, Wing</creator><creator>Toporski, Jacek</creator><creator>Levine, John</creator><creator>Chewning, Joseph</creator><creator>Wayne, Alan</creator><creator>Kapoor, Urvi</creator><creator>Triplett, Brandon</creator><creator>Schultz, Kirk R.</creator><creator>Yanik, Gregory A.</creator><creator>Eapen, Mary</creator><general>Elsevier Inc</general><general>American Society of Hematology</general><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7X8</scope><scope>5PM</scope><orcidid>https://orcid.org/0000-0001-8220-9980</orcidid><orcidid>https://orcid.org/0000-0003-4567-8037</orcidid><orcidid>https://orcid.org/0000-0001-5554-5890</orcidid><orcidid>https://orcid.org/0000-0001-8652-4400</orcidid><orcidid>https://orcid.org/0000-0001-6012-0651</orcidid><orcidid>https://orcid.org/0000-0002-8474-9080</orcidid><orcidid>https://orcid.org/0000-0002-6146-3952</orcidid></search><sort><creationdate>20210218</creationdate><title>A validated pediatric disease risk index for allogeneic hematopoietic cell transplantation</title><author>Qayed, Muna ; Ahn, Kwang Woo ; Kitko, Carrie L. ; Johnson, Mariam H. ; Shah, Nirali N. ; Dvorak, Christopher ; Mellgren, Karin ; Friend, Brian D. ; Verneris, Michael R. ; Leung, Wing ; Toporski, Jacek ; Levine, John ; Chewning, Joseph ; Wayne, Alan ; Kapoor, Urvi ; Triplett, Brandon ; Schultz, Kirk R. ; Yanik, Gregory A. ; Eapen, Mary</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c447t-bc03b135d7e283c948cd4ab6ada217d4571fdc6cdd5c36ea8bb83f4761c6622d3</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2021</creationdate><topic>Adolescent</topic><topic>Age Factors</topic><topic>Allografts</topic><topic>Antineoplastic Combined Chemotherapy Protocols - therapeutic use</topic><topic>Child</topic><topic>Child, Preschool</topic><topic>CME</topic><topic>Cohort Studies</topic><topic>Combined Modality Therapy</topic><topic>Disease-Free Survival</topic><topic>Female</topic><topic>Hematopoietic Stem Cell Transplantation</topic><topic>Humans</topic><topic>Infant</topic><topic>Kaplan-Meier Estimate</topic><topic>Leukemia, Myeloid, Acute - drug therapy</topic><topic>Leukemia, Myeloid, Acute - mortality</topic><topic>Leukemia, Myeloid, Acute - pathology</topic><topic>Leukemia, Myeloid, Acute - therapy</topic><topic>Male</topic><topic>Neoplasm, Residual</topic><topic>Precursor Cell Lymphoblastic Leukemia-Lymphoma - drug therapy</topic><topic>Precursor Cell Lymphoblastic Leukemia-Lymphoma - mortality</topic><topic>Precursor Cell Lymphoblastic Leukemia-Lymphoma - pathology</topic><topic>Precursor Cell Lymphoblastic Leukemia-Lymphoma - therapy</topic><topic>Prognosis</topic><topic>Random Allocation</topic><topic>Risk Assessment</topic><topic>Risk Factors</topic><topic>Severity of Illness Index</topic><topic>Transplantation</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Qayed, Muna</creatorcontrib><creatorcontrib>Ahn, Kwang Woo</creatorcontrib><creatorcontrib>Kitko, Carrie L.</creatorcontrib><creatorcontrib>Johnson, Mariam H.</creatorcontrib><creatorcontrib>Shah, Nirali N.</creatorcontrib><creatorcontrib>Dvorak, Christopher</creatorcontrib><creatorcontrib>Mellgren, Karin</creatorcontrib><creatorcontrib>Friend, Brian D.</creatorcontrib><creatorcontrib>Verneris, Michael R.</creatorcontrib><creatorcontrib>Leung, Wing</creatorcontrib><creatorcontrib>Toporski, Jacek</creatorcontrib><creatorcontrib>Levine, John</creatorcontrib><creatorcontrib>Chewning, Joseph</creatorcontrib><creatorcontrib>Wayne, Alan</creatorcontrib><creatorcontrib>Kapoor, Urvi</creatorcontrib><creatorcontrib>Triplett, Brandon</creatorcontrib><creatorcontrib>Schultz, Kirk R.</creatorcontrib><creatorcontrib>Yanik, Gregory A.</creatorcontrib><creatorcontrib>Eapen, Mary</creatorcontrib><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>MEDLINE - Academic</collection><collection>PubMed Central (Full Participant titles)</collection><jtitle>Blood</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Qayed, Muna</au><au>Ahn, Kwang Woo</au><au>Kitko, Carrie L.</au><au>Johnson, Mariam H.</au><au>Shah, Nirali N.</au><au>Dvorak, Christopher</au><au>Mellgren, Karin</au><au>Friend, Brian D.</au><au>Verneris, Michael R.</au><au>Leung, Wing</au><au>Toporski, Jacek</au><au>Levine, John</au><au>Chewning, Joseph</au><au>Wayne, Alan</au><au>Kapoor, Urvi</au><au>Triplett, Brandon</au><au>Schultz, Kirk R.</au><au>Yanik, Gregory A.</au><au>Eapen, Mary</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>A validated pediatric disease risk index for allogeneic hematopoietic cell transplantation</atitle><jtitle>Blood</jtitle><addtitle>Blood</addtitle><date>2021-02-18</date><risdate>2021</risdate><volume>137</volume><issue>7</issue><spage>983</spage><epage>993</epage><pages>983-993</pages><issn>0006-4971</issn><eissn>1528-0020</eissn><abstract>A disease risk index (DRI) that was developed for adults with hematologic malignancy who were undergoing hematopoietic cell transplantation is also being used to stratify children and adolescents by disease risk. Therefore, to develop and validate a DRI that can be used to stratify those with AML and ALL by their disease risk, we analyzed 2569 patients aged <18 years with acute myeloid (AML; n = 1224) or lymphoblastic (ALL; n = 1345) leukemia who underwent hematopoietic cell transplantation. Training and validation subsets for each disease were generated randomly with 1:1 assignment to the subsets, and separate prognostic models were derived for each disease. For AML, 4 risk groups were identified based on age, cytogenetic risk, and disease status, including minimal residual disease status at transplantation. The 5-year leukemia-free survival for low (0 points), intermediate (2, 3, 5), high (7, 8), and very high (>8) risk groups was 78%, 53%, 40%, and 25%, respectively (P < .0001). For ALL, 3 risk groups were identified based on age and disease status, including minimal residual disease status at transplantation. The 5-year leukemia-free survival for low (0 points), intermediate (2-4), and high (≥5) risk groups was 68%, 51%, and 33%, respectively (P < .0001). We confirmed that the risk groups could be applied to overall survival, with 5-year survival ranging from 80% to 33% and 73% to 42% for AML and ALL, respectively (P < .0001). This validated pediatric DRI, which includes age and residual disease status, can be used to facilitate prognostication and stratification of children with AML and ALL for allogeneic transplantation.
•The pediatric DRI stratified children with AML and ALL into clinically distinct risk groups based on pretransplantation information.•Risk stratification was based on age at transplant, cytogenetics, and disease status including minimal residual disease at transplant.
[Display omitted]</abstract><cop>United States</cop><pub>Elsevier Inc</pub><pmid>33206937</pmid><doi>10.1182/blood.2020009342</doi><tpages>11</tpages><orcidid>https://orcid.org/0000-0001-8220-9980</orcidid><orcidid>https://orcid.org/0000-0003-4567-8037</orcidid><orcidid>https://orcid.org/0000-0001-5554-5890</orcidid><orcidid>https://orcid.org/0000-0001-8652-4400</orcidid><orcidid>https://orcid.org/0000-0001-6012-0651</orcidid><orcidid>https://orcid.org/0000-0002-8474-9080</orcidid><orcidid>https://orcid.org/0000-0002-6146-3952</orcidid><oa>free_for_read</oa></addata></record> |
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| ispartof | Blood, 2021-02, Vol.137 (7), p.983-993 |
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| source | MEDLINE; EZB-FREE-00999 freely available EZB journals; Alma/SFX Local Collection |
| subjects | Adolescent Age Factors Allografts Antineoplastic Combined Chemotherapy Protocols - therapeutic use Child Child, Preschool CME Cohort Studies Combined Modality Therapy Disease-Free Survival Female Hematopoietic Stem Cell Transplantation Humans Infant Kaplan-Meier Estimate Leukemia, Myeloid, Acute - drug therapy Leukemia, Myeloid, Acute - mortality Leukemia, Myeloid, Acute - pathology Leukemia, Myeloid, Acute - therapy Male Neoplasm, Residual Precursor Cell Lymphoblastic Leukemia-Lymphoma - drug therapy Precursor Cell Lymphoblastic Leukemia-Lymphoma - mortality Precursor Cell Lymphoblastic Leukemia-Lymphoma - pathology Precursor Cell Lymphoblastic Leukemia-Lymphoma - therapy Prognosis Random Allocation Risk Assessment Risk Factors Severity of Illness Index Transplantation |
| title | A validated pediatric disease risk index for allogeneic hematopoietic cell transplantation |
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