{"id":"https://openalex.org/W7225128509","doi":"https://doi.org/10.21203/rs.3.rs-9950362/v1","title":"Allogeneic Hematopoietic Stem Cell Transplantation in Children with Mendelian Susceptibility to Mycobacterial Diseases Caused by Interferon Gamma Receptor 1 Deficiency and Literature Review","display_name":"Allogeneic Hematopoietic Stem Cell Transplantation in Children with Mendelian Susceptibility to Mycobacterial Diseases Caused by Interferon Gamma Receptor 1 Deficiency and Literature Review","publication_year":2026,"publication_date":"2026-11-02","ids":{"openalex":"https://openalex.org/W7225128509","doi":"https://doi.org/10.21203/rs.3.rs-9950362/v1"},"language":"en","primary_location":{"id":"doi:10.21203/rs.3.rs-9950362/v1","doi":"https://doi.org/10.21203/rs.3.rs-9950362/v1","is_oa":true,"landing_page_url":"https://doi.org/10.21203/rs.3.rs-9950362/v1","pdf_url":"https://www.researchsquare.com/article/rs-9950362/latest.pdf","source":{"id":"https://openalex.org/S4306525896","display_name":"Research Square","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":null,"host_organization_name":null,"host_organization_lineage":[],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"acceptedVersion","is_accepted":true,"is_published":false,"raw_source_name":"Research Square","raw_type":"posted-content"},"volume":null,"type":"preprint","indexed_in":["crossref"],"open_access":{"is_oa":true,"oa_status":"green","oa_url":"https://www.researchsquare.com/article/rs-9950362/latest.pdf","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5115594034","display_name":"Lian Liu","orcid":"https://orcid.org/0009-0006-0248-985X","observed_orcids":["https://orcid.org/0009-0006-0248-985X"]},"institutions":[{"id":"https://openalex.org/I4210159329","display_name":"Children's Hospital of Fudan University","ror":"https://ror.org/05n13be63","country_code":"CN","type":"healthcare","lineage":["https://openalex.org/I4210159329"]}],"countries":["CN"],"is_corresponding":false,"raw_author_name":"Lian Liu","raw_affiliation_strings":["Children's Hospital of Fudan University"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Children's Hospital of Fudan University","institution_ids":["https://openalex.org/I4210159329"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5020929077","display_name":"Xiaowen Qian","orcid":"https://orcid.org/0000-0002-0072-6632","observed_orcids":["https://orcid.org/0000-0002-0072-6632"]},"institutions":[{"id":"https://openalex.org/I4210159329","display_name":"Children's Hospital of Fudan University","ror":"https://ror.org/05n13be63","country_code":"CN","type":"healthcare","lineage":["https://openalex.org/I4210159329"]}],"countries":["CN"],"is_corresponding":false,"raw_author_name":"Xiaowen Qian","raw_affiliation_strings":["Children's Hospital of Fudan University"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Children's Hospital of Fudan University","institution_ids":["https://openalex.org/I4210159329"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5160328942","display_name":"Ping Wang","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210159329","display_name":"Children's Hospital of Fudan University","ror":"https://ror.org/05n13be63","country_code":"CN","type":"healthcare","lineage":["https://openalex.org/I4210159329"]}],"countries":["CN"],"is_corresponding":false,"raw_author_name":"Ping Wang","raw_affiliation_strings":["Children's Hospital of Fudan University"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Children's Hospital of Fudan University","institution_ids":["https://openalex.org/I4210159329"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5112699439","display_name":"Wenjin Jiang","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210159329","display_name":"Children's Hospital of Fudan University","ror":"https://ror.org/05n13be63","country_code":"CN","type":"healthcare","lineage":["https://openalex.org/I4210159329"]}],"countries":["CN"],"is_corresponding":false,"raw_author_name":"Wenjin Jiang","raw_affiliation_strings":["Children's Hospital of Fudan University"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Children's Hospital of Fudan University","institution_ids":["https://openalex.org/I4210159329"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5015612045","display_name":"W Wang","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210159329","display_name":"Children's Hospital of Fudan University","ror":"https://ror.org/05n13be63","country_code":"CN","type":"healthcare","lineage":["https://openalex.org/I4210159329"]}],"countries":["CN"],"is_corresponding":false,"raw_author_name":"Wenjie Wang","raw_affiliation_strings":["Children's Hospital of Fudan University"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Children's Hospital of Fudan University","institution_ids":["https://openalex.org/I4210159329"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5143681940","display_name":"Xiaochuan Wang","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210159329","display_name":"Children's Hospital of Fudan University","ror":"https://ror.org/05n13be63","country_code":"CN","type":"healthcare","lineage":["https://openalex.org/I4210159329"]}],"countries":["CN"],"is_corresponding":false,"raw_author_name":"Xiaochuan Wang","raw_affiliation_strings":["Children's Hospital of Fudan University"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Children's Hospital of Fudan University","institution_ids":["https://openalex.org/I4210159329"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5152961093","display_name":"Jinqiao Sun","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210159329","display_name":"Children's Hospital of Fudan University","ror":"https://ror.org/05n13be63","country_code":"CN","type":"healthcare","lineage":["https://openalex.org/I4210159329"]}],"countries":["CN"],"is_corresponding":false,"raw_author_name":"Jinqiao Sun","raw_affiliation_strings":["Children's Hospital of Fudan University"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Children's Hospital of Fudan University","institution_ids":["https://openalex.org/I4210159329"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5133775076","display_name":"Hongsheng Wang","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210159329","display_name":"Children's Hospital of Fudan University","ror":"https://ror.org/05n13be63","country_code":"CN","type":"healthcare","lineage":["https://openalex.org/I4210159329"]}],"countries":["CN"],"is_corresponding":false,"raw_author_name":"Hongsheng Wang","raw_affiliation_strings":["Children's Hospital of Fudan University"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Children's Hospital of Fudan University","institution_ids":["https://openalex.org/I4210159329"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5036392227","display_name":"Xiaowen Zhai","orcid":"https://orcid.org/0000-0002-3505-0302","observed_orcids":["https://orcid.org/0000-0002-3505-0302","https://orcid.org/0000-0002-8127-3232"]},"institutions":[{"id":"https://openalex.org/I4210159329","display_name":"Children's Hospital of Fudan University","ror":"https://ror.org/05n13be63","country_code":"CN","type":"healthcare","lineage":["https://openalex.org/I4210159329"]}],"countries":["CN"],"is_corresponding":false,"raw_author_name":"Xiaowen Zhai","raw_affiliation_strings":["Children's Hospital of Fudan University"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Children's Hospital of Fudan University","institution_ids":["https://openalex.org/I4210159329"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":1,"corresponding_author_ids":[],"corresponding_institution_ids":["https://openalex.org/I4210159329"],"apc_list":null,"apc_paid":null,"fwci":null,"has_fulltext":true,"cited_by_count":0,"citation_normalized_percentile":null,"cited_by_percentile_year":null,"biblio":{"volume":null,"issue":null,"first_page":null,"last_page":null},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T11725","display_name":"Immunodeficiency and Autoimmune Disorders","score":0.9663000106811523,"subfield":{"id":"https://openalex.org/subfields/2403","display_name":"Immunology"},"field":{"id":"https://openalex.org/fields/24","display_name":"Immunology and Microbiology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}},"topics":[{"id":"https://openalex.org/T11725","display_name":"Immunodeficiency and Autoimmune Disorders","score":0.9663000106811523,"subfield":{"id":"https://openalex.org/subfields/2403","display_name":"Immunology"},"field":{"id":"https://openalex.org/fields/24","display_name":"Immunology and Microbiology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}},{"id":"https://openalex.org/T10345","display_name":"Hematopoietic Stem Cell Transplantation","score":0.03180000185966492,"subfield":{"id":"https://openalex.org/subfields/2720","display_name":"Hematology"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}},{"id":"https://openalex.org/T11189","display_name":"Transplantation: Methods and Outcomes","score":0.00039999998989515007,"subfield":{"id":"https://openalex.org/subfields/2746","display_name":"Surgery"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}}],"keywords":[{"id":"https://openalex.org/keywords/ifngr1","display_name":"IFNGR1","score":0.7459999918937683},{"id":"https://openalex.org/keywords/allogeneic-hematopoietic-stem-cell-transplantation","display_name":"allogeneic hematopoietic stem cell transplantation","score":0.9679999947547913},{"id":"https://openalex.org/keywords/disseminated-mycobacterial-infection","display_name":"disseminated mycobacterial infection","score":0.8840000033378601},{"id":"https://openalex.org/keywords/peripheral-blood-stem-cell-transplantation","display_name":"peripheral blood stem cell transplantation","score":0.800000011920929},{"id":"https://openalex.org/keywords/in-vivo-t-cell-depletion","display_name":"in vivo T-cell depletion","score":0.8429999947547913},{"id":"https://openalex.org/keywords/antithymocyte-globulin","display_name":"antithymocyte globulin","score":0.9039999842643738},{"id":"https://openalex.org/keywords/post-transplant-cyclophosphamide","display_name":"post-transplant cyclophosphamide","score":0.9980000257492065},{"id":"https://openalex.org/keywords/haploidentical-donor","display_name":"haploidentical donor","score":0.7820000052452087},{"id":"https://openalex.org/keywords/unrelated-donor","display_name":"unrelated donor","score":0.8809999823570251},{"id":"https://openalex.org/keywords/graft-versus-host-disease","display_name":"graft-versus-host disease","score":0.8980000019073486},{"id":"https://openalex.org/keywords/donor-chimerism","display_name":"donor chimerism","score":0.8679999709129333},{"id":"https://openalex.org/keywords/post-transplant-lymphoproliferative-disorder","display_name":"post-transplant lymphoproliferative disorder","score":0.8619999885559082}],"concepts":[{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.8831999897956848},{"id":"https://openalex.org/C2777408962","wikidata":"https://www.wikidata.org/wiki/Q1128050","display_name":"Hematopoietic stem cell transplantation","level":3,"score":0.603600025177002},{"id":"https://openalex.org/C203014093","wikidata":"https://www.wikidata.org/wiki/Q101929","display_name":"Immunology","level":1,"score":0.5263000130653381},{"id":"https://openalex.org/C2911091166","wikidata":"https://www.wikidata.org/wiki/Q106419912","display_name":"Transplantation","level":2,"score":0.5134000182151794},{"id":"https://openalex.org/C2779019163","wikidata":"https://www.wikidata.org/wiki/Q3043160","display_name":"Primary immunodeficiency","level":3,"score":0.5117999911308289},{"id":"https://openalex.org/C28328180","wikidata":"https://www.wikidata.org/wiki/Q48196","display_name":"Stem cell","level":2,"score":0.4832000136375427},{"id":"https://openalex.org/C26291073","wikidata":"https://www.wikidata.org/wiki/Q310484","display_name":"Ex vivo","level":3,"score":0.43560001254081726},{"id":"https://openalex.org/C2779468541","wikidata":"https://www.wikidata.org/wiki/Q1334408","display_name":"Severe combined immunodeficiency","level":3,"score":0.39660000801086426},{"id":"https://openalex.org/C2776194465","wikidata":"https://www.wikidata.org/wiki/Q31184","display_name":"Sibling","level":2,"score":0.3917999863624573},{"id":"https://openalex.org/C2777607303","wikidata":"https://www.wikidata.org/wiki/Q641307","display_name":"Immunodeficiency","level":3,"score":0.3853999972343445},{"id":"https://openalex.org/C207001950","wikidata":"https://www.wikidata.org/wiki/Q141124","display_name":"In vivo","level":2,"score":0.36489999294281006},{"id":"https://openalex.org/C109159458","wikidata":"https://www.wikidata.org/wiki/Q919283","display_name":"Haematopoiesis","level":3,"score":0.3427000045776367},{"id":"https://openalex.org/C2781161190","wikidata":"https://www.wikidata.org/wiki/Q1055374","display_name":"Adenosine deaminase deficiency","level":4,"score":0.32179999351501465},{"id":"https://openalex.org/C2908554679","wikidata":"https://www.wikidata.org/wiki/Q281721","display_name":"Transplantation Chimera","level":5,"score":0.27090001106262207},{"id":"https://openalex.org/C2780841837","wikidata":"https://www.wikidata.org/wiki/Q24741149","display_name":"Interferon gamma","level":3,"score":0.258899986743927},{"id":"https://openalex.org/C187212893","wikidata":"https://www.wikidata.org/wiki/Q123028","display_name":"Pediatrics","level":1,"score":0.25699999928474426},{"id":"https://openalex.org/C2778384578","wikidata":"https://www.wikidata.org/wiki/Q16930687","display_name":"Therapeutic approach","level":3,"score":0.2522999942302704}],"mesh":[],"locations_count":1,"locations":[{"id":"doi:10.21203/rs.3.rs-9950362/v1","doi":"https://doi.org/10.21203/rs.3.rs-9950362/v1","is_oa":true,"landing_page_url":"https://doi.org/10.21203/rs.3.rs-9950362/v1","pdf_url":"https://www.researchsquare.com/article/rs-9950362/latest.pdf","source":{"id":"https://openalex.org/S4306525896","display_name":"Research Square","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":null,"host_organization_name":null,"host_organization_lineage":[],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"acceptedVersion","is_accepted":true,"is_published":false,"raw_source_name":"Research Square","raw_type":"posted-content"}],"best_oa_location":{"id":"doi:10.21203/rs.3.rs-9950362/v1","doi":"https://doi.org/10.21203/rs.3.rs-9950362/v1","is_oa":true,"landing_page_url":"https://doi.org/10.21203/rs.3.rs-9950362/v1","pdf_url":"https://www.researchsquare.com/article/rs-9950362/latest.pdf","source":{"id":"https://openalex.org/S4306525896","display_name":"Research Square","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":null,"host_organization_name":null,"host_organization_lineage":[],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"acceptedVersion","is_accepted":true,"is_published":false,"raw_source_name":"Research Square","raw_type":"posted-content"},"sustainable_development_goals":[{"id":"https://metadata.un.org/sdg/3","display_name":"Good health and well-being","score":1.0}],"study_designs":[],"awards":[],"funders":[],"has_content":{"pdf":true,"grobid_xml":true},"content_urls":{"pdf":"https://content.openalex.org/works/W7225128509.pdf","grobid_xml":"https://content.openalex.org/works/W7225128509.grobid-xml"},"referenced_works_count":0,"referenced_works":[],"related_works":["https://openalex.org/W2083575604","https://openalex.org/W4411026911","https://openalex.org/W1966020905","https://openalex.org/W2291356462","https://openalex.org/W7171389477","https://openalex.org/W3001555672","https://openalex.org/W2170762856","https://openalex.org/W2080570520","https://openalex.org/W3045432510","https://openalex.org/W7204731604"],"abstract_inverted_index":{"Abstract":[0],"Purpose":[1],"IFNGR1":[2,51,65,179],"deficiency":[3,52,66,180],"is":[4,15,173],"a":[5,75],"rare":[6],"primary":[7],"immunodeficiency":[8],"causing":[9],"severe":[10],"disseminated":[11],"mycobacterial":[12],"infections.":[13],"HSCT":[14,69],"the":[16,36,159],"only":[17],"curative":[18],"option,":[19],"but":[20],"reports":[21],"of":[22,40,162],"success":[23],"are":[24],"limited":[25,129],"and":[26,38,73,87,151,175,187],"optimal":[27],"graft/TCD":[28],"strategies":[29],"remain":[30],"unclear.":[31],"We":[32,57],"aimed":[33],"to":[34],"evaluate":[35],"safety":[37],"efficacy":[39],"peripheral":[41],"blood":[42],"stem":[43],"cell":[44],"transplantation":[45,147,168],"with":[46,63,91,117,169],"in":[47,92,170,193],"vivo":[48,93,171,191],"TCD":[49,94,172,192],"for":[50,177],"using":[53,181],"alternative":[54,100,182],"donors.":[55],"Methods":[56],"retrospectively":[58],"analyzed":[59],"three":[60,81],"pediatric":[61],"patients":[62,82],"complete":[64,178],"who":[67,144],"received":[68,88],"at":[70],"our":[71],"center":[72],"performed":[74],"systematic":[76],"literature":[77],"review.":[78],"Results":[79],"All":[80,110],"lacked":[83],"HLA-matched":[84],"sibling":[85],"donors":[86,101],"PBSC":[89,167],"grafts":[90],"(ATG":[95],"and/or":[96],"post-transplant":[97],"cyclophosphamide)":[98],"from":[99],"(10/10":[102],"unrelated,":[103],"5/10":[104],"haploidentical":[105],"sibling,":[106],"8/10":[107],"mismatched":[108],"unrelated).":[109],"achieved":[111],"durable":[112],"full":[113],"donor":[114,185],"chimerism":[115],"(>99%)":[116],"no":[118],"grade":[119],"III-IV":[120],"acute":[121],"or":[122,139],"extensive":[123],"chronic":[124],"GVHD.":[125],"Manageable":[126],"complications":[127],"(PTLD,":[128],"skin/joint/eye":[130],"GVHD,":[131],"pulmonary":[132],"fungal":[133],"infection)":[134],"occurred":[135],"without":[136],"graft":[137],"failure":[138],"transplant-related":[140],"mortality.":[141],"One":[142],"patient":[143],"failed":[145],"initial":[146],"during":[148],"active":[149],"infection":[150,156,164],"elevated":[152],"IFN\u2011\u03b3":[153],"succeeded":[154],"after":[155],"control,":[157],"underscoring":[158],"critical":[160],"role":[161],"pre-transplant":[163],"management.":[165],"Conclusion":[166],"safe":[174],"effective":[176],"donors,":[183],"expanding":[184],"options":[186],"avoiding":[188],"aggressive":[189],"ex":[190],"this":[194],"vulnerable":[195],"population.":[196]},"counts_by_year":[],"updated_date":"2026-11-04T10:54:05.112534","created_date":"2026-11-03T00:00:00"}