Successful Allogeneic Hematopoietic Stem Cell Transplantation in XIAP Deficiency Using Reduced-Intensity Conditioning
Hematopoietic stem cell transplantation (HSCT) is currently the only available curative therapy for X‐linked inhibitor of apoptosis (XIAP) deficiency. Myeloablative conditioning regimens are associated with high mortality rates. Reduced‐intensity conditioning (RIC) is recommended in order to decreas...
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Veröffentlicht in: | Pediatric blood & cancer 2016-02, Vol.63 (2), p.355-357 |
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creator | Chellapandian, Deepak Krueger, Joerg Schechter, Tal Gassas, Adam Weitzman, Sheila Naqvi, Ahmed Ali, Muhammad |
description | Hematopoietic stem cell transplantation (HSCT) is currently the only available curative therapy for X‐linked inhibitor of apoptosis (XIAP) deficiency. Myeloablative conditioning regimens are associated with high mortality rates. Reduced‐intensity conditioning (RIC) is recommended in order to decrease treatment‐related toxicities, but RIC regimens increase the risk for mixed donor‐recipient chimerism that may progress to graft loss. We report our experience with a patient with XIAP deficiency who was successfully treated with allogeneic HSCT using a RIC protocol. Post‐transplant chimerism was vigilantly monitored and maintained with donor lymphocyte infusions and a stem cell boost to a level that prevented hemophagocytic lymphohistiocytosis recurrence. Pediatr Blood Cancer © 2015 Wiley Periodicals, Inc. |
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Myeloablative conditioning regimens are associated with high mortality rates. Reduced‐intensity conditioning (RIC) is recommended in order to decrease treatment‐related toxicities, but RIC regimens increase the risk for mixed donor‐recipient chimerism that may progress to graft loss. We report our experience with a patient with XIAP deficiency who was successfully treated with allogeneic HSCT using a RIC protocol. Post‐transplant chimerism was vigilantly monitored and maintained with donor lymphocyte infusions and a stem cell boost to a level that prevented hemophagocytic lymphohistiocytosis recurrence. 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Myeloablative conditioning regimens are associated with high mortality rates. Reduced‐intensity conditioning (RIC) is recommended in order to decrease treatment‐related toxicities, but RIC regimens increase the risk for mixed donor‐recipient chimerism that may progress to graft loss. We report our experience with a patient with XIAP deficiency who was successfully treated with allogeneic HSCT using a RIC protocol. Post‐transplant chimerism was vigilantly monitored and maintained with donor lymphocyte infusions and a stem cell boost to a level that prevented hemophagocytic lymphohistiocytosis recurrence. Pediatr Blood Cancer © 2015 Wiley Periodicals, Inc.</description><subject>Alemtuzumab</subject><subject>Antibodies, Monoclonal, Humanized - administration & dosage</subject><subject>Child, Preschool</subject><subject>donor-recipient chimerism</subject><subject>Genetic Diseases, X-Linked - surgery</subject><subject>Hematology</subject><subject>hematopoietic stem cell transplantation</subject><subject>Hematopoietic Stem Cell Transplantation - methods</subject><subject>Humans</subject><subject>Lymphoproliferative Disorders - surgery</subject><subject>Male</subject><subject>Melphalan - administration & dosage</subject><subject>Myeloablative Agonists - therapeutic use</subject><subject>Oncology</subject><subject>Pediatrics</subject><subject>reduced intensity conditioning</subject><subject>Transplantation Conditioning - methods</subject><subject>Transplantation, Homologous - methods</subject><subject>Vidarabine - administration & dosage</subject><subject>Vidarabine - analogs & derivatives</subject><subject>XIAP</subject><issn>1545-5009</issn><issn>1545-5017</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2016</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNp1kM1u1DAURi1UREthwQsgS6y6SOufOI6XQwrTEQWqTksrNpbHua5cMk6IHcG8PS7Tzq6r6yuf77vSQegdJceUEHYyrOwxE1JUL9ABFaUoBKFyb_cmah-9jvE-oxUR9Su0zyquasnkAZqWk7UQo5s6POu6_g4CeIvPYG1SP_QeUt6WCda4ga7DV6MJcehMSCb5PmAf8O1idoFPwXnrIdgNvo4-3OFLaCcLbbEICUL0aYObPrT-IZS_36CXznQR3j7OQ3T9-dNVc1acf58vmtl5YQWTVcEcb1dSkrJyUCvbUso4IZJwxsqyNmCpXZWOG6askoa3lXNECkVUrWhNS8IP0Ydt7zD2vyeISd_30xjySU1lFlaXNWeZOtpSduxjHMHpYfRrM240JfpBsM6C9X_BmX3_2Dit1tDuyCejGTjZAn98B5vnm_TFx-apstgmfEzwd5cw4y9dSS6Fvvk211-XX9QP2vzUl_wfIuCUAg</recordid><startdate>201602</startdate><enddate>201602</enddate><creator>Chellapandian, Deepak</creator><creator>Krueger, Joerg</creator><creator>Schechter, Tal</creator><creator>Gassas, Adam</creator><creator>Weitzman, Sheila</creator><creator>Naqvi, Ahmed</creator><creator>Ali, Muhammad</creator><general>Blackwell Publishing Ltd</general><general>Wiley Subscription Services, Inc</general><scope>BSCLL</scope><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>7TK</scope><scope>7TO</scope><scope>8FD</scope><scope>FR3</scope><scope>H94</scope><scope>K9.</scope><scope>P64</scope><scope>RC3</scope></search><sort><creationdate>201602</creationdate><title>Successful Allogeneic Hematopoietic Stem Cell Transplantation in XIAP Deficiency Using Reduced-Intensity Conditioning</title><author>Chellapandian, Deepak ; Krueger, Joerg ; Schechter, Tal ; Gassas, Adam ; Weitzman, Sheila ; Naqvi, Ahmed ; Ali, Muhammad</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c5276-2f3db77046fe89cd11230070322448aec1cb4f3a29c97a3d6ff07590989181403</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2016</creationdate><topic>Alemtuzumab</topic><topic>Antibodies, Monoclonal, Humanized - administration & dosage</topic><topic>Child, Preschool</topic><topic>donor-recipient chimerism</topic><topic>Genetic Diseases, X-Linked - surgery</topic><topic>Hematology</topic><topic>hematopoietic stem cell transplantation</topic><topic>Hematopoietic Stem Cell Transplantation - methods</topic><topic>Humans</topic><topic>Lymphoproliferative Disorders - surgery</topic><topic>Male</topic><topic>Melphalan - administration & dosage</topic><topic>Myeloablative Agonists - therapeutic use</topic><topic>Oncology</topic><topic>Pediatrics</topic><topic>reduced intensity conditioning</topic><topic>Transplantation Conditioning - methods</topic><topic>Transplantation, Homologous - methods</topic><topic>Vidarabine - administration & dosage</topic><topic>Vidarabine - analogs & derivatives</topic><topic>XIAP</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Chellapandian, Deepak</creatorcontrib><creatorcontrib>Krueger, Joerg</creatorcontrib><creatorcontrib>Schechter, Tal</creatorcontrib><creatorcontrib>Gassas, Adam</creatorcontrib><creatorcontrib>Weitzman, Sheila</creatorcontrib><creatorcontrib>Naqvi, Ahmed</creatorcontrib><creatorcontrib>Ali, Muhammad</creatorcontrib><collection>Istex</collection><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>Neurosciences Abstracts</collection><collection>Oncogenes and Growth Factors Abstracts</collection><collection>Technology Research Database</collection><collection>Engineering Research Database</collection><collection>AIDS and Cancer Research Abstracts</collection><collection>ProQuest Health & Medical Complete (Alumni)</collection><collection>Biotechnology and BioEngineering Abstracts</collection><collection>Genetics Abstracts</collection><jtitle>Pediatric blood & cancer</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Chellapandian, Deepak</au><au>Krueger, Joerg</au><au>Schechter, Tal</au><au>Gassas, Adam</au><au>Weitzman, Sheila</au><au>Naqvi, Ahmed</au><au>Ali, Muhammad</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Successful Allogeneic Hematopoietic Stem Cell Transplantation in XIAP Deficiency Using Reduced-Intensity Conditioning</atitle><jtitle>Pediatric blood & cancer</jtitle><addtitle>Pediatr Blood Cancer</addtitle><date>2016-02</date><risdate>2016</risdate><volume>63</volume><issue>2</issue><spage>355</spage><epage>357</epage><pages>355-357</pages><issn>1545-5009</issn><eissn>1545-5017</eissn><abstract>Hematopoietic stem cell transplantation (HSCT) is currently the only available curative therapy for X‐linked inhibitor of apoptosis (XIAP) deficiency. Myeloablative conditioning regimens are associated with high mortality rates. Reduced‐intensity conditioning (RIC) is recommended in order to decrease treatment‐related toxicities, but RIC regimens increase the risk for mixed donor‐recipient chimerism that may progress to graft loss. We report our experience with a patient with XIAP deficiency who was successfully treated with allogeneic HSCT using a RIC protocol. Post‐transplant chimerism was vigilantly monitored and maintained with donor lymphocyte infusions and a stem cell boost to a level that prevented hemophagocytic lymphohistiocytosis recurrence. Pediatr Blood Cancer © 2015 Wiley Periodicals, Inc.</abstract><cop>United States</cop><pub>Blackwell Publishing Ltd</pub><pmid>26398727</pmid><doi>10.1002/pbc.25756</doi><tpages>3</tpages></addata></record> |
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subjects | Alemtuzumab Antibodies, Monoclonal, Humanized - administration & dosage Child, Preschool donor-recipient chimerism Genetic Diseases, X-Linked - surgery Hematology hematopoietic stem cell transplantation Hematopoietic Stem Cell Transplantation - methods Humans Lymphoproliferative Disorders - surgery Male Melphalan - administration & dosage Myeloablative Agonists - therapeutic use Oncology Pediatrics reduced intensity conditioning Transplantation Conditioning - methods Transplantation, Homologous - methods Vidarabine - administration & dosage Vidarabine - analogs & derivatives XIAP |
title | Successful Allogeneic Hematopoietic Stem Cell Transplantation in XIAP Deficiency Using Reduced-Intensity Conditioning |
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