Outcome of chronic granulomatous disease ‐ Conventional treatment vs stem cell transplantation
Background Hematopoietic stem cell transplantation (HSCT) can cure chronic granulomatous disease (CGD), but it remains debated whether all conventionally treated CGD patients benefit from HSCT. Methods We retrospectively analyzed 104 conventionally treated CGD patients, of whom 50 patients underwent...
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| Veröffentlicht in: | Pediatric allergy and immunology 2021-04, Vol.32 (3), p.576-585 |
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| creator | Dedieu, Cinzia Albert, Michael H. Mahlaoui, Nizar Hauck, Fabian Hedrich, Christian Baumann, Ulrich Warnatz, Klaus Roesler, Joachim Speckmann, Carsten Schulte, Johannes Fischer, Alain Blanche, Stephane Bernuth, Horst Kühl, Jörn‐Sven Atanaskovic‐Markovic, Marina |
| description | Background
Hematopoietic stem cell transplantation (HSCT) can cure chronic granulomatous disease (CGD), but it remains debated whether all conventionally treated CGD patients benefit from HSCT.
Methods
We retrospectively analyzed 104 conventionally treated CGD patients, of whom 50 patients underwent HSCT.
Results
On conventional treatment, seven patients (13%) died after a median time of 16.2 years (interquartile range [IQR] 7.0‐18.0). Survival without severe complications was 10 ± 3% (mean ± SD) at the age of 20 years; 85% of patients developed at least one infection, 76% one non‐infectious inflammation. After HSCT, 44 patients (88%) were alive at a median follow‐up of 2.3 years (IQR 0.8‐4.9): Six patients (12%) died from infections. Survival after HSCT was significantly better for patients transplanted ≤8 years (96 ± 4%) or for patients without active complications at HSCT (100%). Eight patients suffered from graft failure (16%); six (12%) developed acute graft‐vs‐host disease requiring systemic treatment. Conventionally treated patients developed events that required medical attention at a median frequency of 1.7 (IQR 0.8‐3.2) events per year vs 0 (IQR 0.0‐0.5) in patients beyond the first year post‐HSCT. While most conventionally treated CGD patients failed to thrive, catch‐up growth after HSCT in surviving patients reached the individual percentiles at the age of diagnosis of CGD.
Conclusion
Chronic granulomatous disease patients undergoing HSCT until 8 years of age show excellent survival, but young children need more intense conditioning to avoid graft rejection. Risks and benefits of HSCT for adolescents and adults must still be weighed carefully. |
| doi_str_mv | 10.1111/pai.13402 |
| format | Article |
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Hematopoietic stem cell transplantation (HSCT) can cure chronic granulomatous disease (CGD), but it remains debated whether all conventionally treated CGD patients benefit from HSCT.
Methods
We retrospectively analyzed 104 conventionally treated CGD patients, of whom 50 patients underwent HSCT.
Results
On conventional treatment, seven patients (13%) died after a median time of 16.2 years (interquartile range [IQR] 7.0‐18.0). Survival without severe complications was 10 ± 3% (mean ± SD) at the age of 20 years; 85% of patients developed at least one infection, 76% one non‐infectious inflammation. After HSCT, 44 patients (88%) were alive at a median follow‐up of 2.3 years (IQR 0.8‐4.9): Six patients (12%) died from infections. Survival after HSCT was significantly better for patients transplanted ≤8 years (96 ± 4%) or for patients without active complications at HSCT (100%). Eight patients suffered from graft failure (16%); six (12%) developed acute graft‐vs‐host disease requiring systemic treatment. Conventionally treated patients developed events that required medical attention at a median frequency of 1.7 (IQR 0.8‐3.2) events per year vs 0 (IQR 0.0‐0.5) in patients beyond the first year post‐HSCT. While most conventionally treated CGD patients failed to thrive, catch‐up growth after HSCT in surviving patients reached the individual percentiles at the age of diagnosis of CGD.
Conclusion
Chronic granulomatous disease patients undergoing HSCT until 8 years of age show excellent survival, but young children need more intense conditioning to avoid graft rejection. Risks and benefits of HSCT for adolescents and adults must still be weighed carefully.</description><identifier>ISSN: 0905-6157</identifier><identifier>EISSN: 1399-3038</identifier><identifier>DOI: 10.1111/pai.13402</identifier><identifier>PMID: 33118209</identifier><language>eng</language><publisher>England: Wiley Subscription Services, Inc</publisher><subject>Age ; Chronic granulomatous disease ; chronic granulomatous disease (CGD) ; conventional treatment ; fungal infection ; Graft rejection ; Graft-versus-host reaction ; Grafts ; hematopoietic stem cell transplantation (HSCT) ; Hematopoietic stem cells ; Medical treatment ; outcome ; Patients ; Stem cell transplantation ; Stem cells</subject><ispartof>Pediatric allergy and immunology, 2021-04, Vol.32 (3), p.576-585</ispartof><rights>2020 The Authors. published by European Academy of Allergy and Clinical Immunology and John Wiley & Sons Ltd</rights><rights>2020 The Authors. Pediatric Allergy and Immunology published by European Academy of Allergy and Clinical Immunology and John Wiley & Sons Ltd.</rights><rights>2020. This article is published under http://creativecommons.org/licenses/by-nc/4.0/ (the “License”). Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License.</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c3882-bb77546c74642f2062147c0a538b19f1def3118b9f179c30f07195753fd90ad43</citedby><cites>FETCH-LOGICAL-c3882-bb77546c74642f2062147c0a538b19f1def3118b9f179c30f07195753fd90ad43</cites><orcidid>0000-0002-5812-7675</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%2Fpai.13402$$EPDF$$P50$$Gwiley$$Hfree_for_read</linktopdf><linktohtml>$$Uhttps://onlinelibrary.wiley.com/doi/full/10.1111%2Fpai.13402$$EHTML$$P50$$Gwiley$$Hfree_for_read</linktohtml><link.rule.ids>314,777,781,1412,27905,27906,45555,45556</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/33118209$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><contributor>Atanaskovic‐Markovic, Marina</contributor><creatorcontrib>Dedieu, Cinzia</creatorcontrib><creatorcontrib>Albert, Michael H.</creatorcontrib><creatorcontrib>Mahlaoui, Nizar</creatorcontrib><creatorcontrib>Hauck, Fabian</creatorcontrib><creatorcontrib>Hedrich, Christian</creatorcontrib><creatorcontrib>Baumann, Ulrich</creatorcontrib><creatorcontrib>Warnatz, Klaus</creatorcontrib><creatorcontrib>Roesler, Joachim</creatorcontrib><creatorcontrib>Speckmann, Carsten</creatorcontrib><creatorcontrib>Schulte, Johannes</creatorcontrib><creatorcontrib>Fischer, Alain</creatorcontrib><creatorcontrib>Blanche, Stephane</creatorcontrib><creatorcontrib>Bernuth, Horst</creatorcontrib><creatorcontrib>Kühl, Jörn‐Sven</creatorcontrib><creatorcontrib>Atanaskovic‐Markovic, Marina</creatorcontrib><title>Outcome of chronic granulomatous disease ‐ Conventional treatment vs stem cell transplantation</title><title>Pediatric allergy and immunology</title><addtitle>Pediatr Allergy Immunol</addtitle><description>Background
Hematopoietic stem cell transplantation (HSCT) can cure chronic granulomatous disease (CGD), but it remains debated whether all conventionally treated CGD patients benefit from HSCT.
Methods
We retrospectively analyzed 104 conventionally treated CGD patients, of whom 50 patients underwent HSCT.
Results
On conventional treatment, seven patients (13%) died after a median time of 16.2 years (interquartile range [IQR] 7.0‐18.0). Survival without severe complications was 10 ± 3% (mean ± SD) at the age of 20 years; 85% of patients developed at least one infection, 76% one non‐infectious inflammation. After HSCT, 44 patients (88%) were alive at a median follow‐up of 2.3 years (IQR 0.8‐4.9): Six patients (12%) died from infections. Survival after HSCT was significantly better for patients transplanted ≤8 years (96 ± 4%) or for patients without active complications at HSCT (100%). Eight patients suffered from graft failure (16%); six (12%) developed acute graft‐vs‐host disease requiring systemic treatment. Conventionally treated patients developed events that required medical attention at a median frequency of 1.7 (IQR 0.8‐3.2) events per year vs 0 (IQR 0.0‐0.5) in patients beyond the first year post‐HSCT. While most conventionally treated CGD patients failed to thrive, catch‐up growth after HSCT in surviving patients reached the individual percentiles at the age of diagnosis of CGD.
Conclusion
Chronic granulomatous disease patients undergoing HSCT until 8 years of age show excellent survival, but young children need more intense conditioning to avoid graft rejection. Risks and benefits of HSCT for adolescents and adults must still be weighed carefully.</description><subject>Age</subject><subject>Chronic granulomatous disease</subject><subject>chronic granulomatous disease (CGD)</subject><subject>conventional treatment</subject><subject>fungal infection</subject><subject>Graft rejection</subject><subject>Graft-versus-host reaction</subject><subject>Grafts</subject><subject>hematopoietic stem cell transplantation (HSCT)</subject><subject>Hematopoietic stem cells</subject><subject>Medical treatment</subject><subject>outcome</subject><subject>Patients</subject><subject>Stem cell transplantation</subject><subject>Stem cells</subject><issn>0905-6157</issn><issn>1399-3038</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2021</creationdate><recordtype>article</recordtype><sourceid>24P</sourceid><sourceid>WIN</sourceid><recordid>eNp10LtOwzAYBWALgWgpDLwAssQCQ1pf4jgeq4pLJaQywBwcx4FUSRzspKgbj8Az8iQ4pDAg4cUXfTr6fQA4xWiK_Zo1sphiGiKyB8aYChFQRON9MEYCsSDCjI_AkXNrhDCnET4EI0oxjgkSY_C06lplKg1NDtWLNXWh4LOVdVeaSramczArnJZOw8_3D7gw9UbXbWFqWcLWatlW_go3DrpWV1Dpsn-WtWtKWbeyh8fgIJel0ye7fQIer68eFrfB3epmuZjfBYrGMQnSlHMWRoqHUUhygiKCQ66QZDROschxpvN-5tQfuVAU5YhjwTijeSaQzEI6ARdDbmPNa6ddm1SF6weStfbfSEjIWEyZ4MTT8z90bTrrv-QVQ4KQiFHu1eWglDXOWZ0njS0qabcJRklfe-JrT75r9_Zsl9illc5-5U_PHswG8FaUevt_UnI_Xw6RXzgJjEQ</recordid><startdate>202104</startdate><enddate>202104</enddate><creator>Dedieu, Cinzia</creator><creator>Albert, Michael H.</creator><creator>Mahlaoui, Nizar</creator><creator>Hauck, Fabian</creator><creator>Hedrich, Christian</creator><creator>Baumann, Ulrich</creator><creator>Warnatz, Klaus</creator><creator>Roesler, Joachim</creator><creator>Speckmann, Carsten</creator><creator>Schulte, Johannes</creator><creator>Fischer, Alain</creator><creator>Blanche, Stephane</creator><creator>Bernuth, Horst</creator><creator>Kühl, Jörn‐Sven</creator><creator>Atanaskovic‐Markovic, Marina</creator><general>Wiley Subscription Services, Inc</general><scope>24P</scope><scope>WIN</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7T5</scope><scope>H94</scope><scope>K9.</scope><scope>NAPCQ</scope><scope>7X8</scope><orcidid>https://orcid.org/0000-0002-5812-7675</orcidid></search><sort><creationdate>202104</creationdate><title>Outcome of chronic granulomatous disease ‐ Conventional treatment vs stem cell transplantation</title><author>Dedieu, Cinzia ; Albert, Michael H. ; Mahlaoui, Nizar ; Hauck, Fabian ; Hedrich, Christian ; Baumann, Ulrich ; Warnatz, Klaus ; Roesler, Joachim ; Speckmann, Carsten ; Schulte, Johannes ; Fischer, Alain ; Blanche, Stephane ; Bernuth, Horst ; Kühl, Jörn‐Sven ; Atanaskovic‐Markovic, Marina</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c3882-bb77546c74642f2062147c0a538b19f1def3118b9f179c30f07195753fd90ad43</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2021</creationdate><topic>Age</topic><topic>Chronic granulomatous disease</topic><topic>chronic granulomatous disease (CGD)</topic><topic>conventional treatment</topic><topic>fungal infection</topic><topic>Graft rejection</topic><topic>Graft-versus-host reaction</topic><topic>Grafts</topic><topic>hematopoietic stem cell transplantation (HSCT)</topic><topic>Hematopoietic stem cells</topic><topic>Medical treatment</topic><topic>outcome</topic><topic>Patients</topic><topic>Stem cell transplantation</topic><topic>Stem cells</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Dedieu, Cinzia</creatorcontrib><creatorcontrib>Albert, Michael H.</creatorcontrib><creatorcontrib>Mahlaoui, Nizar</creatorcontrib><creatorcontrib>Hauck, Fabian</creatorcontrib><creatorcontrib>Hedrich, Christian</creatorcontrib><creatorcontrib>Baumann, Ulrich</creatorcontrib><creatorcontrib>Warnatz, Klaus</creatorcontrib><creatorcontrib>Roesler, Joachim</creatorcontrib><creatorcontrib>Speckmann, Carsten</creatorcontrib><creatorcontrib>Schulte, Johannes</creatorcontrib><creatorcontrib>Fischer, Alain</creatorcontrib><creatorcontrib>Blanche, Stephane</creatorcontrib><creatorcontrib>Bernuth, Horst</creatorcontrib><creatorcontrib>Kühl, Jörn‐Sven</creatorcontrib><creatorcontrib>Atanaskovic‐Markovic, Marina</creatorcontrib><collection>Wiley Online Library Open Access</collection><collection>Wiley Free Content</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>Immunology Abstracts</collection><collection>AIDS and Cancer Research Abstracts</collection><collection>ProQuest Health & Medical Complete (Alumni)</collection><collection>Nursing & Allied Health Premium</collection><collection>MEDLINE - Academic</collection><jtitle>Pediatric allergy and immunology</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Dedieu, Cinzia</au><au>Albert, Michael H.</au><au>Mahlaoui, Nizar</au><au>Hauck, Fabian</au><au>Hedrich, Christian</au><au>Baumann, Ulrich</au><au>Warnatz, Klaus</au><au>Roesler, Joachim</au><au>Speckmann, Carsten</au><au>Schulte, Johannes</au><au>Fischer, Alain</au><au>Blanche, Stephane</au><au>Bernuth, Horst</au><au>Kühl, Jörn‐Sven</au><au>Atanaskovic‐Markovic, Marina</au><au>Atanaskovic‐Markovic, Marina</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Outcome of chronic granulomatous disease ‐ Conventional treatment vs stem cell transplantation</atitle><jtitle>Pediatric allergy and immunology</jtitle><addtitle>Pediatr Allergy Immunol</addtitle><date>2021-04</date><risdate>2021</risdate><volume>32</volume><issue>3</issue><spage>576</spage><epage>585</epage><pages>576-585</pages><issn>0905-6157</issn><eissn>1399-3038</eissn><abstract>Background
Hematopoietic stem cell transplantation (HSCT) can cure chronic granulomatous disease (CGD), but it remains debated whether all conventionally treated CGD patients benefit from HSCT.
Methods
We retrospectively analyzed 104 conventionally treated CGD patients, of whom 50 patients underwent HSCT.
Results
On conventional treatment, seven patients (13%) died after a median time of 16.2 years (interquartile range [IQR] 7.0‐18.0). Survival without severe complications was 10 ± 3% (mean ± SD) at the age of 20 years; 85% of patients developed at least one infection, 76% one non‐infectious inflammation. After HSCT, 44 patients (88%) were alive at a median follow‐up of 2.3 years (IQR 0.8‐4.9): Six patients (12%) died from infections. Survival after HSCT was significantly better for patients transplanted ≤8 years (96 ± 4%) or for patients without active complications at HSCT (100%). Eight patients suffered from graft failure (16%); six (12%) developed acute graft‐vs‐host disease requiring systemic treatment. Conventionally treated patients developed events that required medical attention at a median frequency of 1.7 (IQR 0.8‐3.2) events per year vs 0 (IQR 0.0‐0.5) in patients beyond the first year post‐HSCT. While most conventionally treated CGD patients failed to thrive, catch‐up growth after HSCT in surviving patients reached the individual percentiles at the age of diagnosis of CGD.
Conclusion
Chronic granulomatous disease patients undergoing HSCT until 8 years of age show excellent survival, but young children need more intense conditioning to avoid graft rejection. Risks and benefits of HSCT for adolescents and adults must still be weighed carefully.</abstract><cop>England</cop><pub>Wiley Subscription Services, Inc</pub><pmid>33118209</pmid><doi>10.1111/pai.13402</doi><tpages>10</tpages><orcidid>https://orcid.org/0000-0002-5812-7675</orcidid><oa>free_for_read</oa></addata></record> |
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| subjects | Age Chronic granulomatous disease chronic granulomatous disease (CGD) conventional treatment fungal infection Graft rejection Graft-versus-host reaction Grafts hematopoietic stem cell transplantation (HSCT) Hematopoietic stem cells Medical treatment outcome Patients Stem cell transplantation Stem cells |
| title | Outcome of chronic granulomatous disease ‐ Conventional treatment vs stem cell transplantation |
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