Real-life experience with hydroxyurea in sickle cell disease: A multicenter study in a cohort of patients with heterogeneous descent
We conducted the first nation-wide cohort study of sickle cell disease (SCD) in Italy, a Southern European country exposed to intense recent flux migration from endemic areas for SCD. We evaluate the impact of hydroxyurea on a total of 652 pediatric and adult patients from 33 Reference Centers for S...
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| Veröffentlicht in: | Blood cells, molecules, & diseases molecules, & diseases, 2018-03, Vol.69, p.82-89 |
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| creator | Rigano, Paolo De Franceschi, Lucia Sainati, Laura Piga, Antonio Piel, Frédéric B. Cappellini, Maria Domenica Fidone, Carmelo Masera, Nicoletta Palazzi, Giovanni Gianesin, Barbara Forni, Gian Luca |
| description | We conducted the first nation-wide cohort study of sickle cell disease (SCD) in Italy, a Southern European country exposed to intense recent flux migration from endemic areas for SCD. We evaluate the impact of hydroxyurea on a total of 652 pediatric and adult patients from 33 Reference Centers for SCD (mean age 24.5±15years, 51.4% males). Hydroxyurea median treatment duration was 7years (range: |
| doi_str_mv | 10.1016/j.bcmd.2017.08.017 |
| format | Article |
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We evaluate the impact of hydroxyurea on a total of 652 pediatric and adult patients from 33 Reference Centers for SCD (mean age 24.5±15years, 51.4% males). Hydroxyurea median treatment duration was 7years (range: <1year to 29years) at a mean therapeutic dose of 18±4.7mg/kg/day. Hydroxyurea was associated with a significant increase in mean total and fetal hemoglobin and a significant decrease in mean hemoglobin S, white blood and platelet counts, and lactate dehydrogenase levels. Hydroxyurea was associated with a significant reduction in the incidence of acute chest syndrome (−29.3%, p<0.001), vaso-occlusive crisis (−34.1%, p<0.001), hospitalization (−53.2%, p<0.001), and bone necrosis (−6.9%, p<0.001). New silent cerebral infarction (SCI) occurred during treatment (+42.4%, p<0.001) but not stroke (+0.5%, p=0.572). These observations were generally consistent upon stratification for age, descent (Caucasian or African), genotype (βS/βS, βS/β0 or βS/β+) and duration of treatment (< or ≥10years). There were no new safety concerns observed compared to those commonly reported in the literature. Our study, conducted on a large population of patients with different descent and compound state supports the benefits of hydroxyurea therapy as a treatment option. Registered at clinical trials.gov (NCT02709681).]]></description><identifier>ISSN: 1079-9796</identifier><identifier>EISSN: 1096-0961</identifier><identifier>DOI: 10.1016/j.bcmd.2017.08.017</identifier><identifier>PMID: 29107441</identifier><language>eng</language><publisher>United States: Elsevier Inc</publisher><subject>Adolescent ; Adult ; Aged ; Anemia, Sickle Cell - diagnosis ; Anemia, Sickle Cell - drug therapy ; Anemia, Sickle Cell - genetics ; Anemia, Sickle Cell - mortality ; Antisickling Agents - administration & dosage ; Antisickling Agents - adverse effects ; Antisickling Agents - therapeutic use ; Biomarkers ; Cause of Death ; Child ; Child, Preschool ; Complications ; Disease Management ; Erythrocyte Indices ; Female ; Genotype ; Humans ; Hydroxycarbamide ; Hydroxyurea ; Hydroxyurea - administration & dosage ; Hydroxyurea - adverse effects ; Hydroxyurea - therapeutic use ; Infant ; Kaplan-Meier Estimate ; Male ; Management ; Middle Aged ; Real-world ; Retrospective Studies ; Sickle cell disease ; Treatment Outcome ; Young Adult</subject><ispartof>Blood cells, molecules, & diseases, 2018-03, Vol.69, p.82-89</ispartof><rights>2017 Elsevier Inc.</rights><rights>Copyright © 2017 Elsevier Inc. All rights reserved.</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c400t-2e88032848951932c0d090c5282f58a93efdc72c042e34d5717184b5a562479d3</citedby><cites>FETCH-LOGICAL-c400t-2e88032848951932c0d090c5282f58a93efdc72c042e34d5717184b5a562479d3</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktohtml>$$Uhttps://dx.doi.org/10.1016/j.bcmd.2017.08.017$$EHTML$$P50$$Gelsevier$$H</linktohtml><link.rule.ids>314,780,784,3548,27922,27923,45993</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/29107441$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Rigano, Paolo</creatorcontrib><creatorcontrib>De Franceschi, Lucia</creatorcontrib><creatorcontrib>Sainati, Laura</creatorcontrib><creatorcontrib>Piga, Antonio</creatorcontrib><creatorcontrib>Piel, Frédéric B.</creatorcontrib><creatorcontrib>Cappellini, Maria Domenica</creatorcontrib><creatorcontrib>Fidone, Carmelo</creatorcontrib><creatorcontrib>Masera, Nicoletta</creatorcontrib><creatorcontrib>Palazzi, Giovanni</creatorcontrib><creatorcontrib>Gianesin, Barbara</creatorcontrib><creatorcontrib>Forni, Gian Luca</creatorcontrib><creatorcontrib>on behalf of The Italian Multicenter Study of Hydroxyurea in Sickle Cell Anemia Investigators</creatorcontrib><creatorcontrib>Italian Multicenter Study of Hydroxyurea in Sickle Cell Anemia Investigators</creatorcontrib><title>Real-life experience with hydroxyurea in sickle cell disease: A multicenter study in a cohort of patients with heterogeneous descent</title><title>Blood cells, molecules, & diseases</title><addtitle>Blood Cells Mol Dis</addtitle><description><![CDATA[We conducted the first nation-wide cohort study of sickle cell disease (SCD) in Italy, a Southern European country exposed to intense recent flux migration from endemic areas for SCD. We evaluate the impact of hydroxyurea on a total of 652 pediatric and adult patients from 33 Reference Centers for SCD (mean age 24.5±15years, 51.4% males). Hydroxyurea median treatment duration was 7years (range: <1year to 29years) at a mean therapeutic dose of 18±4.7mg/kg/day. Hydroxyurea was associated with a significant increase in mean total and fetal hemoglobin and a significant decrease in mean hemoglobin S, white blood and platelet counts, and lactate dehydrogenase levels. Hydroxyurea was associated with a significant reduction in the incidence of acute chest syndrome (−29.3%, p<0.001), vaso-occlusive crisis (−34.1%, p<0.001), hospitalization (−53.2%, p<0.001), and bone necrosis (−6.9%, p<0.001). New silent cerebral infarction (SCI) occurred during treatment (+42.4%, p<0.001) but not stroke (+0.5%, p=0.572). These observations were generally consistent upon stratification for age, descent (Caucasian or African), genotype (βS/βS, βS/β0 or βS/β+) and duration of treatment (< or ≥10years). There were no new safety concerns observed compared to those commonly reported in the literature. Our study, conducted on a large population of patients with different descent and compound state supports the benefits of hydroxyurea therapy as a treatment option. Registered at clinical trials.gov (NCT02709681).]]></description><subject>Adolescent</subject><subject>Adult</subject><subject>Aged</subject><subject>Anemia, Sickle Cell - diagnosis</subject><subject>Anemia, Sickle Cell - drug therapy</subject><subject>Anemia, Sickle Cell - genetics</subject><subject>Anemia, Sickle Cell - mortality</subject><subject>Antisickling Agents - administration & dosage</subject><subject>Antisickling Agents - adverse effects</subject><subject>Antisickling Agents - therapeutic use</subject><subject>Biomarkers</subject><subject>Cause of Death</subject><subject>Child</subject><subject>Child, Preschool</subject><subject>Complications</subject><subject>Disease Management</subject><subject>Erythrocyte Indices</subject><subject>Female</subject><subject>Genotype</subject><subject>Humans</subject><subject>Hydroxycarbamide</subject><subject>Hydroxyurea</subject><subject>Hydroxyurea - administration & dosage</subject><subject>Hydroxyurea - adverse effects</subject><subject>Hydroxyurea - therapeutic use</subject><subject>Infant</subject><subject>Kaplan-Meier Estimate</subject><subject>Male</subject><subject>Management</subject><subject>Middle Aged</subject><subject>Real-world</subject><subject>Retrospective Studies</subject><subject>Sickle cell disease</subject><subject>Treatment Outcome</subject><subject>Young Adult</subject><issn>1079-9796</issn><issn>1096-0961</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2018</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNp9kDtPBCEUhYnR-P4DFobSZkZgXmBsjPGVmJgYrQkLd1zWmWUFRt3eHy7jrpYW5BA45-TeD6EjSnJKaH06yye6NzkjtMkJz5NsoF1KRJ2lQzfHeyMy0Yh6B-2FMCOEUCr4NtphIn2VJd1FX4-guqyzLWD4XIC3MNeAP2yc4unSePe5HDwobOc4WP3aAdbQddjYACrAGb7A_dBFq2EeweMQB7McvQprN3U-YtfihYqpNIZ1KSSje4E5uCFgA2GMHqCtVnUBDte6j56vr54ub7P7h5u7y4v7TJeExIwB56RgvOSioqJgmhgiiK4YZ23FlSigNbpJzyWDojRVQxvKy0mlqpqVjTDFPjpZ9S68exsgRNnbMC6kfsaRNGGri6bgLFnZyqq9C8FDKxfe9sovJSVypC9ncqQvR_qScJkkhY7X_cOkB_MX-cWdDOcrA6Qt3y14GfQPcWM96CiNs__1fwMjcJba</recordid><startdate>201803</startdate><enddate>201803</enddate><creator>Rigano, Paolo</creator><creator>De Franceschi, Lucia</creator><creator>Sainati, Laura</creator><creator>Piga, Antonio</creator><creator>Piel, Frédéric B.</creator><creator>Cappellini, Maria Domenica</creator><creator>Fidone, Carmelo</creator><creator>Masera, Nicoletta</creator><creator>Palazzi, Giovanni</creator><creator>Gianesin, Barbara</creator><creator>Forni, Gian Luca</creator><general>Elsevier Inc</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></search><sort><creationdate>201803</creationdate><title>Real-life experience with hydroxyurea in sickle cell disease: A multicenter study in a cohort of patients with heterogeneous descent</title><author>Rigano, Paolo ; De Franceschi, Lucia ; Sainati, Laura ; Piga, Antonio ; Piel, Frédéric B. ; Cappellini, Maria Domenica ; Fidone, Carmelo ; Masera, Nicoletta ; Palazzi, Giovanni ; Gianesin, Barbara ; Forni, Gian Luca</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c400t-2e88032848951932c0d090c5282f58a93efdc72c042e34d5717184b5a562479d3</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2018</creationdate><topic>Adolescent</topic><topic>Adult</topic><topic>Aged</topic><topic>Anemia, Sickle Cell - diagnosis</topic><topic>Anemia, Sickle Cell - drug therapy</topic><topic>Anemia, Sickle Cell - genetics</topic><topic>Anemia, Sickle Cell - mortality</topic><topic>Antisickling Agents - administration & dosage</topic><topic>Antisickling Agents - adverse effects</topic><topic>Antisickling Agents - therapeutic use</topic><topic>Biomarkers</topic><topic>Cause of Death</topic><topic>Child</topic><topic>Child, Preschool</topic><topic>Complications</topic><topic>Disease Management</topic><topic>Erythrocyte Indices</topic><topic>Female</topic><topic>Genotype</topic><topic>Humans</topic><topic>Hydroxycarbamide</topic><topic>Hydroxyurea</topic><topic>Hydroxyurea - administration & dosage</topic><topic>Hydroxyurea - adverse effects</topic><topic>Hydroxyurea - therapeutic use</topic><topic>Infant</topic><topic>Kaplan-Meier Estimate</topic><topic>Male</topic><topic>Management</topic><topic>Middle Aged</topic><topic>Real-world</topic><topic>Retrospective Studies</topic><topic>Sickle cell disease</topic><topic>Treatment Outcome</topic><topic>Young Adult</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Rigano, Paolo</creatorcontrib><creatorcontrib>De Franceschi, Lucia</creatorcontrib><creatorcontrib>Sainati, Laura</creatorcontrib><creatorcontrib>Piga, Antonio</creatorcontrib><creatorcontrib>Piel, Frédéric B.</creatorcontrib><creatorcontrib>Cappellini, Maria Domenica</creatorcontrib><creatorcontrib>Fidone, Carmelo</creatorcontrib><creatorcontrib>Masera, Nicoletta</creatorcontrib><creatorcontrib>Palazzi, Giovanni</creatorcontrib><creatorcontrib>Gianesin, Barbara</creatorcontrib><creatorcontrib>Forni, Gian Luca</creatorcontrib><creatorcontrib>on behalf of The Italian Multicenter Study of Hydroxyurea in Sickle Cell Anemia Investigators</creatorcontrib><creatorcontrib>Italian Multicenter Study of Hydroxyurea in Sickle Cell Anemia Investigators</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><jtitle>Blood cells, molecules, & diseases</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Rigano, Paolo</au><au>De Franceschi, Lucia</au><au>Sainati, Laura</au><au>Piga, Antonio</au><au>Piel, Frédéric B.</au><au>Cappellini, Maria Domenica</au><au>Fidone, Carmelo</au><au>Masera, Nicoletta</au><au>Palazzi, Giovanni</au><au>Gianesin, Barbara</au><au>Forni, Gian Luca</au><aucorp>on behalf of The Italian Multicenter Study of Hydroxyurea in Sickle Cell Anemia Investigators</aucorp><aucorp>Italian Multicenter Study of Hydroxyurea in Sickle Cell Anemia Investigators</aucorp><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Real-life experience with hydroxyurea in sickle cell disease: A multicenter study in a cohort of patients with heterogeneous descent</atitle><jtitle>Blood cells, molecules, & diseases</jtitle><addtitle>Blood Cells Mol Dis</addtitle><date>2018-03</date><risdate>2018</risdate><volume>69</volume><spage>82</spage><epage>89</epage><pages>82-89</pages><issn>1079-9796</issn><eissn>1096-0961</eissn><abstract><![CDATA[We conducted the first nation-wide cohort study of sickle cell disease (SCD) in Italy, a Southern European country exposed to intense recent flux migration from endemic areas for SCD. We evaluate the impact of hydroxyurea on a total of 652 pediatric and adult patients from 33 Reference Centers for SCD (mean age 24.5±15years, 51.4% males). Hydroxyurea median treatment duration was 7years (range: <1year to 29years) at a mean therapeutic dose of 18±4.7mg/kg/day. Hydroxyurea was associated with a significant increase in mean total and fetal hemoglobin and a significant decrease in mean hemoglobin S, white blood and platelet counts, and lactate dehydrogenase levels. Hydroxyurea was associated with a significant reduction in the incidence of acute chest syndrome (−29.3%, p<0.001), vaso-occlusive crisis (−34.1%, p<0.001), hospitalization (−53.2%, p<0.001), and bone necrosis (−6.9%, p<0.001). New silent cerebral infarction (SCI) occurred during treatment (+42.4%, p<0.001) but not stroke (+0.5%, p=0.572). These observations were generally consistent upon stratification for age, descent (Caucasian or African), genotype (βS/βS, βS/β0 or βS/β+) and duration of treatment (< or ≥10years). There were no new safety concerns observed compared to those commonly reported in the literature. Our study, conducted on a large population of patients with different descent and compound state supports the benefits of hydroxyurea therapy as a treatment option. Registered at clinical trials.gov (NCT02709681).]]></abstract><cop>United States</cop><pub>Elsevier Inc</pub><pmid>29107441</pmid><doi>10.1016/j.bcmd.2017.08.017</doi><tpages>8</tpages><oa>free_for_read</oa></addata></record> |
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| ispartof | Blood cells, molecules, & diseases, 2018-03, Vol.69, p.82-89 |
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| language | eng |
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| source | MEDLINE; ScienceDirect Journals (5 years ago - present) |
| subjects | Adolescent Adult Aged Anemia, Sickle Cell - diagnosis Anemia, Sickle Cell - drug therapy Anemia, Sickle Cell - genetics Anemia, Sickle Cell - mortality Antisickling Agents - administration & dosage Antisickling Agents - adverse effects Antisickling Agents - therapeutic use Biomarkers Cause of Death Child Child, Preschool Complications Disease Management Erythrocyte Indices Female Genotype Humans Hydroxycarbamide Hydroxyurea Hydroxyurea - administration & dosage Hydroxyurea - adverse effects Hydroxyurea - therapeutic use Infant Kaplan-Meier Estimate Male Management Middle Aged Real-world Retrospective Studies Sickle cell disease Treatment Outcome Young Adult |
| title | Real-life experience with hydroxyurea in sickle cell disease: A multicenter study in a cohort of patients with heterogeneous descent |
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