Long‐term follow‐up of children with risk organ‐negative Langerhans cell histiocytosis after 2‐chlorodeoxyadenosine treatment
Summary The nucleoside analogue, 2‐chlorodeoxyadenosine (2CDA), was reported to be an active treatment for childhood Langerhans cell histiocytosis (LCH) without risk organ (RO−) involvement. However, we lack data on long‐term effects of 2CDA treatment, including the disease reactivation rate, perman...
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| Veröffentlicht in: | British journal of haematology 2020-12, Vol.191 (5), p.825-834 |
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| creator | Barkaoui, Mohamed‐Aziz Queheille, Emma Aladjidi, Nathalie Plat, Geneviève Jeziorski, Eric Moshous, Despina Lambilliotte, Anne Kebaili, Kamila Pacquement, Hélène Leverger, Guy Mansuy, Ludovic Entz‐Werlé, Natacha Bodet, Damien Schneider, Pascale Pagnier, Anne Lutun, Anne Gillibert‐Yvert, Marion Millot, Fréderic Toutain, Fabienne Reguerre, Yves Thomas, Caroline Tazi, Abdelatif Emile, Jean‐François Donadieu, Jean Héritier, Sébastien |
| description | Summary
The nucleoside analogue, 2‐chlorodeoxyadenosine (2CDA), was reported to be an active treatment for childhood Langerhans cell histiocytosis (LCH) without risk organ (RO−) involvement. However, we lack data on long‐term effects of 2CDA treatment, including the disease reactivation rate, permanent sequelae and long‐term tolerance. This study included 44 children from the French LCH registry, treated for a RO− LCH with 2CDA monotherapy (median number of six courses). The median age at the beginning of 2CDA was 3·6 years (range, 0·3–19·7 years) and the median follow‐up after was 5·4 years (range, 0·6–15·1 years). Objective response to 2CDA was observed in 25 patients (56·8%), while six patients (13·6%) had stable disease and 13 patients (29·5%) exhibited progressive disease. Among patients without progression, only two experienced disease reactivation after 2CDA discontinuation. The five‐year cumulative incidence of disease progression or reactivation after 2CDA therapy initiation was 34·3%. The lymphopenia reported in all cases [72% below absolute lymphocyte count (ALC) of 0·5 G/l], was addressed with appropriate prophylactic measures. Other toxicities above grade 2 were uncommon, and no second malignant neoplasm or neuropathy was reported. The five‐year overall survival was 97·7%. In conclusion, we could confirm that 2CDA monotherapy was a beneficial long‐term therapy for treating patients with RO− LCH. Appropriate management of induced immune deficiency is mandatory. |
| doi_str_mv | 10.1111/bjh.16944 |
| format | Article |
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The nucleoside analogue, 2‐chlorodeoxyadenosine (2CDA), was reported to be an active treatment for childhood Langerhans cell histiocytosis (LCH) without risk organ (RO−) involvement. However, we lack data on long‐term effects of 2CDA treatment, including the disease reactivation rate, permanent sequelae and long‐term tolerance. This study included 44 children from the French LCH registry, treated for a RO− LCH with 2CDA monotherapy (median number of six courses). The median age at the beginning of 2CDA was 3·6 years (range, 0·3–19·7 years) and the median follow‐up after was 5·4 years (range, 0·6–15·1 years). Objective response to 2CDA was observed in 25 patients (56·8%), while six patients (13·6%) had stable disease and 13 patients (29·5%) exhibited progressive disease. Among patients without progression, only two experienced disease reactivation after 2CDA discontinuation. The five‐year cumulative incidence of disease progression or reactivation after 2CDA therapy initiation was 34·3%. The lymphopenia reported in all cases [72% below absolute lymphocyte count (ALC) of 0·5 G/l], was addressed with appropriate prophylactic measures. Other toxicities above grade 2 were uncommon, and no second malignant neoplasm or neuropathy was reported. The five‐year overall survival was 97·7%. In conclusion, we could confirm that 2CDA monotherapy was a beneficial long‐term therapy for treating patients with RO− LCH. Appropriate management of induced immune deficiency is mandatory.</description><identifier>ISSN: 0007-1048</identifier><identifier>EISSN: 1365-2141</identifier><identifier>DOI: 10.1111/bjh.16944</identifier><identifier>PMID: 32700439</identifier><language>eng</language><publisher>England: Blackwell Publishing Ltd</publisher><subject>2‐chlorodeoxyadenosine ; Adolescent ; Cell number ; Child ; Child, Preschool ; Children ; cladribine ; Cladribine - administration & dosage ; Cladribine - adverse effects ; Complications ; Disease-Free Survival ; Female ; Follow-Up Studies ; France ; Hematology ; Histiocytosis ; Histiocytosis, Langerhans-Cell - blood ; Histiocytosis, Langerhans-Cell - drug therapy ; Histiocytosis, Langerhans-Cell - mortality ; Humans ; Immunological tolerance ; Infant ; Langerhans cell histiocytosis ; Life Sciences ; long‐term follow‐up ; Lymphocyte Count ; Lymphocytes ; Lymphopenia ; Male ; Neuropathy ; Nucleoside analogs ; Registries ; Survival Rate</subject><ispartof>British journal of haematology, 2020-12, Vol.191 (5), p.825-834</ispartof><rights>2020 British Society for Haematology and John Wiley & Sons Ltd</rights><rights>2020 British Society for Haematology and John Wiley & Sons Ltd.</rights><rights>Copyright © 2020 John Wiley & Sons Ltd</rights><rights>Distributed under a Creative Commons Attribution 4.0 International License</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c4224-bef80a34e636325e5cbc83dd779e67e23be574325fa35c2b3c68536f7f7fc99d3</citedby><cites>FETCH-LOGICAL-c4224-bef80a34e636325e5cbc83dd779e67e23be574325fa35c2b3c68536f7f7fc99d3</cites><orcidid>0000-0003-0384-6370 ; 0000-0003-0231-4460 ; 0000-0002-4485-146X ; 0000-0001-6719-3693</orcidid></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://onlinelibrary.wiley.com/doi/pdf/10.1111%2Fbjh.16944$$EPDF$$P50$$Gwiley$$H</linktopdf><linktohtml>$$Uhttps://onlinelibrary.wiley.com/doi/full/10.1111%2Fbjh.16944$$EHTML$$P50$$Gwiley$$H</linktohtml><link.rule.ids>230,314,780,784,885,1417,1433,27924,27925,45574,45575,46409,46833</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/32700439$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink><backlink>$$Uhttps://hal.science/hal-03109204$$DView record in HAL$$Hfree_for_read</backlink></links><search><creatorcontrib>Barkaoui, Mohamed‐Aziz</creatorcontrib><creatorcontrib>Queheille, Emma</creatorcontrib><creatorcontrib>Aladjidi, Nathalie</creatorcontrib><creatorcontrib>Plat, Geneviève</creatorcontrib><creatorcontrib>Jeziorski, Eric</creatorcontrib><creatorcontrib>Moshous, Despina</creatorcontrib><creatorcontrib>Lambilliotte, Anne</creatorcontrib><creatorcontrib>Kebaili, Kamila</creatorcontrib><creatorcontrib>Pacquement, Hélène</creatorcontrib><creatorcontrib>Leverger, Guy</creatorcontrib><creatorcontrib>Mansuy, Ludovic</creatorcontrib><creatorcontrib>Entz‐Werlé, Natacha</creatorcontrib><creatorcontrib>Bodet, Damien</creatorcontrib><creatorcontrib>Schneider, Pascale</creatorcontrib><creatorcontrib>Pagnier, Anne</creatorcontrib><creatorcontrib>Lutun, Anne</creatorcontrib><creatorcontrib>Gillibert‐Yvert, Marion</creatorcontrib><creatorcontrib>Millot, Fréderic</creatorcontrib><creatorcontrib>Toutain, Fabienne</creatorcontrib><creatorcontrib>Reguerre, Yves</creatorcontrib><creatorcontrib>Thomas, Caroline</creatorcontrib><creatorcontrib>Tazi, Abdelatif</creatorcontrib><creatorcontrib>Emile, Jean‐François</creatorcontrib><creatorcontrib>Donadieu, Jean</creatorcontrib><creatorcontrib>Héritier, Sébastien</creatorcontrib><title>Long‐term follow‐up of children with risk organ‐negative Langerhans cell histiocytosis after 2‐chlorodeoxyadenosine treatment</title><title>British journal of haematology</title><addtitle>Br J Haematol</addtitle><description>Summary
The nucleoside analogue, 2‐chlorodeoxyadenosine (2CDA), was reported to be an active treatment for childhood Langerhans cell histiocytosis (LCH) without risk organ (RO−) involvement. However, we lack data on long‐term effects of 2CDA treatment, including the disease reactivation rate, permanent sequelae and long‐term tolerance. This study included 44 children from the French LCH registry, treated for a RO− LCH with 2CDA monotherapy (median number of six courses). The median age at the beginning of 2CDA was 3·6 years (range, 0·3–19·7 years) and the median follow‐up after was 5·4 years (range, 0·6–15·1 years). Objective response to 2CDA was observed in 25 patients (56·8%), while six patients (13·6%) had stable disease and 13 patients (29·5%) exhibited progressive disease. Among patients without progression, only two experienced disease reactivation after 2CDA discontinuation. The five‐year cumulative incidence of disease progression or reactivation after 2CDA therapy initiation was 34·3%. The lymphopenia reported in all cases [72% below absolute lymphocyte count (ALC) of 0·5 G/l], was addressed with appropriate prophylactic measures. Other toxicities above grade 2 were uncommon, and no second malignant neoplasm or neuropathy was reported. The five‐year overall survival was 97·7%. In conclusion, we could confirm that 2CDA monotherapy was a beneficial long‐term therapy for treating patients with RO− LCH. Appropriate management of induced immune deficiency is mandatory.</description><subject>2‐chlorodeoxyadenosine</subject><subject>Adolescent</subject><subject>Cell number</subject><subject>Child</subject><subject>Child, Preschool</subject><subject>Children</subject><subject>cladribine</subject><subject>Cladribine - administration & dosage</subject><subject>Cladribine - adverse effects</subject><subject>Complications</subject><subject>Disease-Free Survival</subject><subject>Female</subject><subject>Follow-Up Studies</subject><subject>France</subject><subject>Hematology</subject><subject>Histiocytosis</subject><subject>Histiocytosis, Langerhans-Cell - blood</subject><subject>Histiocytosis, Langerhans-Cell - drug therapy</subject><subject>Histiocytosis, Langerhans-Cell - mortality</subject><subject>Humans</subject><subject>Immunological tolerance</subject><subject>Infant</subject><subject>Langerhans cell histiocytosis</subject><subject>Life Sciences</subject><subject>long‐term follow‐up</subject><subject>Lymphocyte Count</subject><subject>Lymphocytes</subject><subject>Lymphopenia</subject><subject>Male</subject><subject>Neuropathy</subject><subject>Nucleoside analogs</subject><subject>Registries</subject><subject>Survival Rate</subject><issn>0007-1048</issn><issn>1365-2141</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2020</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNp1kb9uFDEQhy1ERI5AwQsgS1QUm_jfenfLEAFHtBIN1JbXO3vrw2cfti_HdTT0PCNPgpNLQoWnsDzzzSdLP4ReUXJOy7kY1vM5lZ0QT9CCcllXjAr6FC0IIU1FiWhP0fOU1oRQTmr6DJ1y1hAieLdAv_rgV39-_s4QN3gKzoV9ee22OEzYzNaNETze2zzjaNM3HOJK-wJ4WOlsbwD32q8gztonbMA5PNuUbTCHHJJNWE_Fi1lZMLMLMYwQfhz0CL5MPeAcQecN-PwCnUzaJXh5f5-hrx_ef7laVv3nj5-uLvvKCMZENcDUEs0FSC45q6E2g2n5ODZNB7IBxgeoG1Emk-a1YQM3sq25nJpSputGfobeHr2zdmob7UbHgwraquVlr257hFPSMSJuaGHfHNltDN93kLJah1305XuKCSllywlv_xlNDClFmB61lKjbcFQJR92FU9jX98bdsIHxkXxIowAXR2BvHRz-b1LvrpdH5V-g955w</recordid><startdate>202012</startdate><enddate>202012</enddate><creator>Barkaoui, Mohamed‐Aziz</creator><creator>Queheille, Emma</creator><creator>Aladjidi, Nathalie</creator><creator>Plat, Geneviève</creator><creator>Jeziorski, Eric</creator><creator>Moshous, Despina</creator><creator>Lambilliotte, Anne</creator><creator>Kebaili, Kamila</creator><creator>Pacquement, Hélène</creator><creator>Leverger, Guy</creator><creator>Mansuy, Ludovic</creator><creator>Entz‐Werlé, Natacha</creator><creator>Bodet, Damien</creator><creator>Schneider, Pascale</creator><creator>Pagnier, Anne</creator><creator>Lutun, Anne</creator><creator>Gillibert‐Yvert, Marion</creator><creator>Millot, Fréderic</creator><creator>Toutain, Fabienne</creator><creator>Reguerre, Yves</creator><creator>Thomas, Caroline</creator><creator>Tazi, Abdelatif</creator><creator>Emile, Jean‐François</creator><creator>Donadieu, Jean</creator><creator>Héritier, Sébastien</creator><general>Blackwell Publishing Ltd</general><general>Wiley</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>7T5</scope><scope>H94</scope><scope>1XC</scope><orcidid>https://orcid.org/0000-0003-0384-6370</orcidid><orcidid>https://orcid.org/0000-0003-0231-4460</orcidid><orcidid>https://orcid.org/0000-0002-4485-146X</orcidid><orcidid>https://orcid.org/0000-0001-6719-3693</orcidid></search><sort><creationdate>202012</creationdate><title>Long‐term follow‐up of children with risk organ‐negative Langerhans cell histiocytosis after 2‐chlorodeoxyadenosine treatment</title><author>Barkaoui, Mohamed‐Aziz ; Queheille, Emma ; Aladjidi, Nathalie ; Plat, Geneviève ; Jeziorski, Eric ; Moshous, Despina ; Lambilliotte, Anne ; Kebaili, Kamila ; Pacquement, Hélène ; Leverger, Guy ; Mansuy, Ludovic ; Entz‐Werlé, Natacha ; Bodet, Damien ; Schneider, Pascale ; Pagnier, Anne ; Lutun, Anne ; Gillibert‐Yvert, Marion ; Millot, Fréderic ; Toutain, Fabienne ; Reguerre, Yves ; Thomas, Caroline ; Tazi, Abdelatif ; Emile, Jean‐François ; Donadieu, Jean ; Héritier, Sébastien</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c4224-bef80a34e636325e5cbc83dd779e67e23be574325fa35c2b3c68536f7f7fc99d3</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2020</creationdate><topic>2‐chlorodeoxyadenosine</topic><topic>Adolescent</topic><topic>Cell number</topic><topic>Child</topic><topic>Child, Preschool</topic><topic>Children</topic><topic>cladribine</topic><topic>Cladribine - administration & dosage</topic><topic>Cladribine - adverse effects</topic><topic>Complications</topic><topic>Disease-Free Survival</topic><topic>Female</topic><topic>Follow-Up Studies</topic><topic>France</topic><topic>Hematology</topic><topic>Histiocytosis</topic><topic>Histiocytosis, Langerhans-Cell - blood</topic><topic>Histiocytosis, Langerhans-Cell - drug therapy</topic><topic>Histiocytosis, Langerhans-Cell - mortality</topic><topic>Humans</topic><topic>Immunological tolerance</topic><topic>Infant</topic><topic>Langerhans cell histiocytosis</topic><topic>Life Sciences</topic><topic>long‐term follow‐up</topic><topic>Lymphocyte Count</topic><topic>Lymphocytes</topic><topic>Lymphopenia</topic><topic>Male</topic><topic>Neuropathy</topic><topic>Nucleoside analogs</topic><topic>Registries</topic><topic>Survival Rate</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Barkaoui, Mohamed‐Aziz</creatorcontrib><creatorcontrib>Queheille, Emma</creatorcontrib><creatorcontrib>Aladjidi, Nathalie</creatorcontrib><creatorcontrib>Plat, Geneviève</creatorcontrib><creatorcontrib>Jeziorski, Eric</creatorcontrib><creatorcontrib>Moshous, Despina</creatorcontrib><creatorcontrib>Lambilliotte, Anne</creatorcontrib><creatorcontrib>Kebaili, Kamila</creatorcontrib><creatorcontrib>Pacquement, Hélène</creatorcontrib><creatorcontrib>Leverger, Guy</creatorcontrib><creatorcontrib>Mansuy, Ludovic</creatorcontrib><creatorcontrib>Entz‐Werlé, Natacha</creatorcontrib><creatorcontrib>Bodet, Damien</creatorcontrib><creatorcontrib>Schneider, Pascale</creatorcontrib><creatorcontrib>Pagnier, Anne</creatorcontrib><creatorcontrib>Lutun, Anne</creatorcontrib><creatorcontrib>Gillibert‐Yvert, Marion</creatorcontrib><creatorcontrib>Millot, Fréderic</creatorcontrib><creatorcontrib>Toutain, Fabienne</creatorcontrib><creatorcontrib>Reguerre, Yves</creatorcontrib><creatorcontrib>Thomas, Caroline</creatorcontrib><creatorcontrib>Tazi, Abdelatif</creatorcontrib><creatorcontrib>Emile, Jean‐François</creatorcontrib><creatorcontrib>Donadieu, Jean</creatorcontrib><creatorcontrib>Héritier, Sébastien</creatorcontrib><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>Immunology Abstracts</collection><collection>AIDS and Cancer Research Abstracts</collection><collection>Hyper Article en Ligne (HAL)</collection><jtitle>British journal of haematology</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Barkaoui, Mohamed‐Aziz</au><au>Queheille, Emma</au><au>Aladjidi, Nathalie</au><au>Plat, Geneviève</au><au>Jeziorski, Eric</au><au>Moshous, Despina</au><au>Lambilliotte, Anne</au><au>Kebaili, Kamila</au><au>Pacquement, Hélène</au><au>Leverger, Guy</au><au>Mansuy, Ludovic</au><au>Entz‐Werlé, Natacha</au><au>Bodet, Damien</au><au>Schneider, Pascale</au><au>Pagnier, Anne</au><au>Lutun, Anne</au><au>Gillibert‐Yvert, Marion</au><au>Millot, Fréderic</au><au>Toutain, Fabienne</au><au>Reguerre, Yves</au><au>Thomas, Caroline</au><au>Tazi, Abdelatif</au><au>Emile, Jean‐François</au><au>Donadieu, Jean</au><au>Héritier, Sébastien</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Long‐term follow‐up of children with risk organ‐negative Langerhans cell histiocytosis after 2‐chlorodeoxyadenosine treatment</atitle><jtitle>British journal of haematology</jtitle><addtitle>Br J Haematol</addtitle><date>2020-12</date><risdate>2020</risdate><volume>191</volume><issue>5</issue><spage>825</spage><epage>834</epage><pages>825-834</pages><issn>0007-1048</issn><eissn>1365-2141</eissn><abstract>Summary
The nucleoside analogue, 2‐chlorodeoxyadenosine (2CDA), was reported to be an active treatment for childhood Langerhans cell histiocytosis (LCH) without risk organ (RO−) involvement. However, we lack data on long‐term effects of 2CDA treatment, including the disease reactivation rate, permanent sequelae and long‐term tolerance. This study included 44 children from the French LCH registry, treated for a RO− LCH with 2CDA monotherapy (median number of six courses). The median age at the beginning of 2CDA was 3·6 years (range, 0·3–19·7 years) and the median follow‐up after was 5·4 years (range, 0·6–15·1 years). Objective response to 2CDA was observed in 25 patients (56·8%), while six patients (13·6%) had stable disease and 13 patients (29·5%) exhibited progressive disease. Among patients without progression, only two experienced disease reactivation after 2CDA discontinuation. The five‐year cumulative incidence of disease progression or reactivation after 2CDA therapy initiation was 34·3%. The lymphopenia reported in all cases [72% below absolute lymphocyte count (ALC) of 0·5 G/l], was addressed with appropriate prophylactic measures. Other toxicities above grade 2 were uncommon, and no second malignant neoplasm or neuropathy was reported. The five‐year overall survival was 97·7%. In conclusion, we could confirm that 2CDA monotherapy was a beneficial long‐term therapy for treating patients with RO− LCH. Appropriate management of induced immune deficiency is mandatory.</abstract><cop>England</cop><pub>Blackwell Publishing Ltd</pub><pmid>32700439</pmid><doi>10.1111/bjh.16944</doi><tpages>10</tpages><orcidid>https://orcid.org/0000-0003-0384-6370</orcidid><orcidid>https://orcid.org/0000-0003-0231-4460</orcidid><orcidid>https://orcid.org/0000-0002-4485-146X</orcidid><orcidid>https://orcid.org/0000-0001-6719-3693</orcidid><oa>free_for_read</oa></addata></record> |
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| ispartof | British journal of haematology, 2020-12, Vol.191 (5), p.825-834 |
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| subjects | 2‐chlorodeoxyadenosine Adolescent Cell number Child Child, Preschool Children cladribine Cladribine - administration & dosage Cladribine - adverse effects Complications Disease-Free Survival Female Follow-Up Studies France Hematology Histiocytosis Histiocytosis, Langerhans-Cell - blood Histiocytosis, Langerhans-Cell - drug therapy Histiocytosis, Langerhans-Cell - mortality Humans Immunological tolerance Infant Langerhans cell histiocytosis Life Sciences long‐term follow‐up Lymphocyte Count Lymphocytes Lymphopenia Male Neuropathy Nucleoside analogs Registries Survival Rate |
| title | Long‐term follow‐up of children with risk organ‐negative Langerhans cell histiocytosis after 2‐chlorodeoxyadenosine treatment |
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