Haematopoietic stem cell transplantation for severe sickle cell disease in childhood: a single centre experience of 50 patients

Summary Despite improvements in medical management, sickle cell disease (SCD) remains associated with severe morbidity and decreased survival. Allogeneic haematopoietic stem cell transplantation (HSCT) remains the only curative approach. We report the outcome of 50 consecutive children with severe S...

Full description

Saved in:

Bibliographic Details
Published in	British journal of haematology Vol. 165; no. 3; pp. 402 - 408
Main Authors	Dedeken, Laurence, Lê, Phu Q., Azzi, Nadira, Brachet, Cécile, Heijmans, Catherine, Huybrechts, Sophie, Devalck, Christine, Rozen, Laurence, Ngalula, Malou, Ferster, Alina
Format	Journal Article
Language	English
Published	Oxford Blackwell 01.05.2014
Subjects	Adolescent Anemia, Sickle Cell - surgery Anemias. Hemoglobinopathies Animals Biological and medical sciences Child Child, Preschool Diseases of red blood cells Female Genotype haematopoietic stem cell transplant Hematologic and hematopoietic diseases Hematopoietic Stem Cell Transplantation - methods Humans hydroxycarbamide Infant Male Medical sciences Rabbits sickle cell disease Survival Analysis Transplantation Conditioning - methods Transplantation, Autologous Tumors Human Antineoplastic agent Hemoglobinopathy Sickle cell anemia Hydroxycarbamide Hematology Stem cell Hematopoietic cell Hemopathy Genetic disease sickle cell disease Hemolytic anemia Cancerology Antimetabolic haematopoietic stem cell transplant Child Hematopoietic stem cell transplantation hydroxycarbamide
Online Access	Get full text

Cover

Loading…

Abstract	Summary Despite improvements in medical management, sickle cell disease (SCD) remains associated with severe morbidity and decreased survival. Allogeneic haematopoietic stem cell transplantation (HSCT) remains the only curative approach. We report the outcome of 50 consecutive children with severe SCD that received HSCT in our unit between November 1988 and April 2013. The stem cell source was bone marrow (n = 39), cord blood (n = 3), bone marrow and cord blood (n = 7) and peripheral blood stem cells (n = 1). All patients had ≥1 severe manifestation: 37 presented with recurrent vaso‐occlusive crises/acute chest syndrome, 27 cerebral vasculopathy and 1 nephropathy. The conditioning regimen consisted of busulfan + cyclophosphamide (BuCy) before November 1991 and BuCy + rabbit antithymocyte globulin after that date. Since 1995, all patients have been treated with hydroxycarbamide (HC) prior to transplantation for a median duration of 2·7 years. Median age at transplantation and median follow‐up was 8·3 and 7·7 years, respectively. Acute graft‐versus‐host disease (GVHD) and chronic GVHD were observed in 11 and 10 patients, respectively. An excellent outcome was achieved, with 8‐year overall survival and event‐free survival (EFS) rates of 94·1% and 85·6%, respectively. Since HC introduction, no graft failure occurred and EFS reached 97·4%. Prior treatment with HC may have contributed to successful engraftment.
AbstractList	Despite improvements in medical management, sickle cell disease (SCD) remains associated with severe morbidity and decreased survival. Allogeneic haematopoietic stem cell transplantation (HSCT) remains the only curative approach. We report the outcome of 50 consecutive children with severe SCD that received HSCT in our unit between November 1988 and April 2013. The stem cell source was bone marrow (n = 39), cord blood (n = 3), bone marrow and cord blood (n = 7) and peripheral blood stem cells (n = 1). All patients had ≥1 severe manifestation: 37 presented with recurrent vaso-occlusive crises/acute chest syndrome, 27 cerebral vasculopathy and 1 nephropathy. The conditioning regimen consisted of busulfan + cyclophosphamide (BuCy) before November 1991 and BuCy + rabbit antithymocyte globulin after that date. Since 1995, all patients have been treated with hydroxycarbamide (HC) prior to transplantation for a median duration of 2·7 years. Median age at transplantation and median follow-up was 8·3 and 7·7 years, respectively. Acute graft-versus-host disease (GVHD) and chronic GVHD were observed in 11 and 10 patients, respectively. An excellent outcome was achieved, with 8-year overall survival and event-free survival (EFS) rates of 94·1% and 85·6%, respectively. Since HC introduction, no graft failure occurred and EFS reached 97·4%. Prior treatment with HC may have contributed to successful engraftment. Despite improvements in medical management, sickle cell disease (SCD) remains associated with severe morbidity and decreased survival. Allogeneic haematopoietic stem cell transplantation (HSCT) remains the only curative approach. We report the outcome of 50 consecutive children with severe SCD that received HSCT in our unit between November 1988 and April 2013. The stem cell source was bone marrow (n = 39), cord blood (n = 3), bone marrow and cord blood (n = 7) and peripheral blood stem cells (n = 1). All patients had greater than or equal to 1 severe manifestation: 37 presented with recurrent vaso-occlusive crises/acute chest syndrome, 27 cerebral vasculopathy and 1 nephropathy. The conditioning regimen consisted of busulfan + cyclophosphamide (BuCy) before November 1991 and BuCy + rabbit antithymocyte globulin after that date. Since 1995, all patients have been treated with hydroxycarbamide (HC) prior to transplantation for a median duration of 2.7 years. Median age at transplantation and median follow-up was 8.3 and 7.7 years, respectively. Acute graft-versus-host disease (GVHD) and chronic GVHD were observed in 11 and 10 patients, respectively. An excellent outcome was achieved, with 8-year overall survival and event-free survival (EFS) rates of 94.1% and 85.6%, respectively. Since HC introduction, no graft failure occurred and EFS reached 97.4%. Prior treatment with HC may have contributed to successful engraftment. Despite improvements in medical management, sickle cell disease (SCD) remains associated with severe morbidity and decreased survival. Allogeneic haematopoietic stem cell transplantation (HSCT) remains the only curative approach. We report the outcome of 50 consecutive children with severe SCD that received HSCT in our unit between November 1988 and April 2013. The stem cell source was bone marrow (n = 39), cord blood (n = 3), bone marrow and cord blood (n = 7) and peripheral blood stem cells (n = 1). All patients had ≥1 severe manifestation: 37 presented with recurrent vaso-occlusive crises/acute chest syndrome, 27 cerebral vasculopathy and 1 nephropathy. The conditioning regimen consisted of busulfan + cyclophosphamide (BuCy) before November 1991 and BuCy + rabbit antithymocyte globulin after that date. Since 1995, all patients have been treated with hydroxycarbamide (HC) prior to transplantation for a median duration of 2·7 years. Median age at transplantation and median follow-up was 8·3 and 7·7 years, respectively. Acute graft-versus-host disease (GVHD) and chronic GVHD were observed in 11 and 10 patients, respectively. An excellent outcome was achieved, with 8-year overall survival and event-free survival (EFS) rates of 94·1% and 85·6%, respectively. Since HC introduction, no graft failure occurred and EFS reached 97·4%. Prior treatment with HC may have contributed to successful engraftment.Despite improvements in medical management, sickle cell disease (SCD) remains associated with severe morbidity and decreased survival. Allogeneic haematopoietic stem cell transplantation (HSCT) remains the only curative approach. We report the outcome of 50 consecutive children with severe SCD that received HSCT in our unit between November 1988 and April 2013. The stem cell source was bone marrow (n = 39), cord blood (n = 3), bone marrow and cord blood (n = 7) and peripheral blood stem cells (n = 1). All patients had ≥1 severe manifestation: 37 presented with recurrent vaso-occlusive crises/acute chest syndrome, 27 cerebral vasculopathy and 1 nephropathy. The conditioning regimen consisted of busulfan + cyclophosphamide (BuCy) before November 1991 and BuCy + rabbit antithymocyte globulin after that date. Since 1995, all patients have been treated with hydroxycarbamide (HC) prior to transplantation for a median duration of 2·7 years. Median age at transplantation and median follow-up was 8·3 and 7·7 years, respectively. Acute graft-versus-host disease (GVHD) and chronic GVHD were observed in 11 and 10 patients, respectively. An excellent outcome was achieved, with 8-year overall survival and event-free survival (EFS) rates of 94·1% and 85·6%, respectively. Since HC introduction, no graft failure occurred and EFS reached 97·4%. Prior treatment with HC may have contributed to successful engraftment. Despite improvements in medical management, sickle cell disease ( SCD ) remains associated with severe morbidity and decreased survival. Allogeneic haematopoietic stem cell transplantation ( HSCT ) remains the only curative approach. We report the outcome of 50 consecutive children with severe SCD that received HSCT in our unit between N ovember 1988 and A pril 2013. The stem cell source was bone marrow ( n = 39), cord blood ( n = 3), bone marrow and cord blood ( n = 7) and peripheral blood stem cells ( n = 1). All patients had ≥1 severe manifestation: 37 presented with recurrent vaso‐occlusive crises/acute chest syndrome, 27 cerebral vasculopathy and 1 nephropathy. The conditioning regimen consisted of busulfan + cyclophosphamide ( B u C y) before November 1991 and B u C y + rabbit antithymocyte globulin after that date. Since 1995, all patients have been treated with hydroxycarbamide ( HC ) prior to transplantation for a median duration of 2·7 years. Median age at transplantation and median follow‐up was 8·3 and 7·7 years, respectively. Acute graft‐versus‐host disease ( GVHD ) and chronic GVHD were observed in 11 and 10 patients, respectively. An excellent outcome was achieved, with 8‐year overall survival and event‐free survival ( EFS ) rates of 94·1% and 85·6%, respectively. Since HC introduction, no graft failure occurred and EFS reached 97·4%. Prior treatment with HC may have contributed to successful engraftment. Summary Despite improvements in medical management, sickle cell disease (SCD) remains associated with severe morbidity and decreased survival. Allogeneic haematopoietic stem cell transplantation (HSCT) remains the only curative approach. We report the outcome of 50 consecutive children with severe SCD that received HSCT in our unit between November 1988 and April 2013. The stem cell source was bone marrow (n = 39), cord blood (n = 3), bone marrow and cord blood (n = 7) and peripheral blood stem cells (n = 1). All patients had ≥1 severe manifestation: 37 presented with recurrent vaso‐occlusive crises/acute chest syndrome, 27 cerebral vasculopathy and 1 nephropathy. The conditioning regimen consisted of busulfan + cyclophosphamide (BuCy) before November 1991 and BuCy + rabbit antithymocyte globulin after that date. Since 1995, all patients have been treated with hydroxycarbamide (HC) prior to transplantation for a median duration of 2·7 years. Median age at transplantation and median follow‐up was 8·3 and 7·7 years, respectively. Acute graft‐versus‐host disease (GVHD) and chronic GVHD were observed in 11 and 10 patients, respectively. An excellent outcome was achieved, with 8‐year overall survival and event‐free survival (EFS) rates of 94·1% and 85·6%, respectively. Since HC introduction, no graft failure occurred and EFS reached 97·4%. Prior treatment with HC may have contributed to successful engraftment.
Author	Dedeken, Laurence Ferster, Alina Heijmans, Catherine Azzi, Nadira Devalck, Christine Rozen, Laurence Lê, Phu Q. Ngalula, Malou Brachet, Cécile Huybrechts, Sophie
Author_xml	– sequence: 1 givenname: Laurence surname: Dedeken fullname: Dedeken, Laurence organization: Université Libre de Bruxelles – sequence: 2 givenname: Phu Q. surname: Lê fullname: Lê, Phu Q. organization: Université Libre de Bruxelles – sequence: 3 givenname: Nadira surname: Azzi fullname: Azzi, Nadira organization: Université Libre de Bruxelles – sequence: 4 givenname: Cécile surname: Brachet fullname: Brachet, Cécile organization: Université Libre de Bruxelles – sequence: 5 givenname: Catherine surname: Heijmans fullname: Heijmans, Catherine organization: Université Libre de Bruxelles – sequence: 6 givenname: Sophie surname: Huybrechts fullname: Huybrechts, Sophie organization: Université Libre de Bruxelles – sequence: 7 givenname: Christine surname: Devalck fullname: Devalck, Christine organization: Université Libre de Bruxelles – sequence: 8 givenname: Laurence surname: Rozen fullname: Rozen, Laurence organization: Université Libre de Bruxelles – sequence: 9 givenname: Malou surname: Ngalula fullname: Ngalula, Malou organization: Université Libre de Bruxelles – sequence: 10 givenname: Alina surname: Ferster fullname: Ferster, Alina organization: Université Libre de Bruxelles
BackLink	http://pascal-francis.inist.fr/vibad/index.php?action=getRecordDetail&idt=28427852$$DView record in Pascal Francis https://www.ncbi.nlm.nih.gov/pubmed/24433465$$D View this record in MEDLINE/PubMed
BookMark	eNqF0T1vFDEQBmALBZFLSMEfQG6QoNjE3_bRQQQcUSQaUq-83lnOwWsv9h6Qir-Okz1AQgq4ceFnxpp5j9BBTBEQekLJKa3nrLvenlKmuX6AVpQr2TAq6AFaEUJ0Q4kwh-iolGtCKCeSPkKHTAjOhZIr9GNjYbRzmpKH2TtcZhixgxDwnG0sU7BxtrNPEQ8p4wJfIQMu3n0OsLDeF7AFsI_YbX3otyn1L7GtJn66M3GuFfB9guwhOsBpwJLgqTatT-UxejjYUOBkfx-jq7dvPp5vmssP796fv7psnGBcN3owxjGmhDasp8R1stNScMuAK847YXoK1vZmrQYhlei7NSgFXAvHQQq95sfo-dJ3yunLDsrcjr7cDmAjpF1pqZZcSG2U-D-VtH5hDJWVPt3TXTdC307ZjzbftL_2W8GzPbDF2TDUnTpf_jgjmDaSVfdicS6nUjIMvwkl7W3Gbc24vcu42rO_rPNLRjUyH_5V8c0HuLm_dfv6YrNU_AQ87Lbq
CODEN	BJHEAL
CitedBy_id	crossref_primary_10_1016_j_bcmd_2015_05_004 crossref_primary_10_1111_ejh_13828 crossref_primary_10_1016_j_anpedi_2016_03_014 crossref_primary_10_1111_bjh_13326 crossref_primary_10_1002_pbc_25608 crossref_primary_10_1259_bjr_20130699 crossref_primary_10_1016_j_anpede_2016_03_012 crossref_primary_10_1517_21678707_2015_1076724 crossref_primary_10_1080_21548331_2018_1464363 crossref_primary_10_1016_j_jtct_2020_10_007 crossref_primary_10_1182_blood_2016_10_745711 crossref_primary_10_1080_17474086_2023_2268271 crossref_primary_10_1016_j_jns_2021_117510 crossref_primary_10_1177_09636897241246351 crossref_primary_10_1016_j_hoc_2014_08_014 crossref_primary_10_1080_14656566_2024_2317336 crossref_primary_10_1080_17474086_2018_1486703 crossref_primary_10_1016_j_bbmt_2020_01_016 crossref_primary_10_1182_bloodadvances_2017007708 crossref_primary_10_1111_bjh_14167 crossref_primary_10_1016_j_hemonc_2017_03_003 crossref_primary_10_1111_ejh_12447 crossref_primary_10_1016_j_jcyt_2021_09_003 crossref_primary_10_1111_bjh_13352 crossref_primary_10_1002_hem3_70089 crossref_primary_10_1182_blood_2015_03_636803 crossref_primary_10_1053_j_seminhematol_2016_01_001 crossref_primary_10_1177_0969141317701166 crossref_primary_10_3390_jcm8111796 crossref_primary_10_1016_j_bbmt_2017_08_034 crossref_primary_10_1097_MOH_0000000000000282 crossref_primary_10_3389_fendo_2022_985525 crossref_primary_10_5409_wjcp_v5_i1_25 crossref_primary_10_1002_pbc_25904 crossref_primary_10_3390_jcm11092318 crossref_primary_10_1182_blood_2014_05_575209 crossref_primary_10_1016_j_jtct_2023_08_016 crossref_primary_10_1016_j_blre_2021_100868 crossref_primary_10_1016_j_jsps_2024_102049 crossref_primary_10_3174_ajnr_A5830 crossref_primary_10_1097_MOH_0000000000000136 crossref_primary_10_1016_j_bbmt_2015_10_017 crossref_primary_10_1080_16078454_2022_2163357 crossref_primary_10_1016_j_bbmt_2015_08_040 crossref_primary_10_1038_s41409_021_01415_6 crossref_primary_10_1002_ajh_24116 crossref_primary_10_1007_s00441_016_2369_y crossref_primary_10_1038_s41409_018_0111_y crossref_primary_10_1182_bloodadvances_2017005462 crossref_primary_10_1186_s12955_022_02021_w crossref_primary_10_3390_jcm11133856 crossref_primary_10_1182_asheducation_2014_1_468 crossref_primary_10_1002_pbc_27601 crossref_primary_10_1053_j_seminhematol_2018_04_011 crossref_primary_10_1016_j_bbmt_2017_01_009
Cites_doi	10.1097/00005792-198801000-00005 10.1002/pbc.22269 10.1056/NEJM199202273260905 10.1111/ejh.12082 10.1002/9781118032985 10.1182/blood-2012-03-419879 10.1182/blood.V85.4.879.bloodjournal854879 10.1056/NEJMoa0904971 10.1002/pbc.24177 10.1016/S0140-6736(11)60355-3 10.1002/pbc.24557 10.1056/NEJM199608083350601 10.1111/j.1365-2141.2007.06592.x 10.1182/blood-2007-03-079665 10.1038/sj.bmt.1705943 10.1038/sj.bmt.1704443 10.1038/sj.bmt.1701291 10.1182/blood-2013-03-489112 10.1182/blood-2009-07-233700
ContentType	Journal Article
Copyright	2014 John Wiley & Sons Ltd 2015 INIST-CNRS 2014 John Wiley & Sons Ltd.
Copyright_xml	– notice: 2014 John Wiley & Sons Ltd – notice: 2015 INIST-CNRS – notice: 2014 John Wiley & Sons Ltd.
DBID	AAYXX CITATION IQODW CGR CUY CVF ECM EIF NPM 7X8 7T5 H94
DOI	10.1111/bjh.12737
DatabaseName	CrossRef Pascal-Francis Medline MEDLINE MEDLINE (Ovid) MEDLINE MEDLINE PubMed MEDLINE - Academic Immunology Abstracts AIDS and Cancer Research Abstracts
DatabaseTitle	CrossRef MEDLINE Medline Complete MEDLINE with Full Text PubMed MEDLINE (Ovid) MEDLINE - Academic AIDS and Cancer Research Abstracts Immunology Abstracts
DatabaseTitleList	MEDLINE AIDS and Cancer Research Abstracts MEDLINE - Academic CrossRef
Database_xml	– sequence: 1 dbid: NPM name: PubMed url: https://proxy.k.utb.cz/login?url=http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?db=PubMed sourceTypes: Index Database – sequence: 2 dbid: EIF name: MEDLINE url: https://proxy.k.utb.cz/login?url=https://www.webofscience.com/wos/medline/basic-search sourceTypes: Index Database
DeliveryMethod	fulltext_linktorsrc
Discipline	Medicine
EISSN	1365-2141
EndPage	408
ExternalDocumentID	24433465 28427852 10_1111_bjh_12737 BJH12737
Genre	article Journal Article
GroupedDBID	--- .3N .55 .GA .GJ .Y3 05W 0R~ 10A 1KJ 1OB 1OC 23N 24P 31~ 33P 36B 3O- 3SF 4.4 50Y 50Z 51W 51X 52M 52N 52O 52P 52R 52S 52T 52U 52V 52W 52X 53G 5GY 5HH 5LA 5RE 5VS 66C 6J9 6P2 702 7PT 8-0 8-1 8-3 8-4 8-5 8F7 8UM 930 A01 A03 AAESR AAEVG AAHHS AAHQN AAIPD AAKAS AAMNL AANHP AANLZ AAONW AASGY AAXRX AAYCA AAYEP AAZKR ABCQN ABCUV ABEML ABJNI ABLJU ABOCM ABPVW ABQWH ABXGK ACAHQ ACBWZ ACCFJ ACCZN ACFBH ACGFO ACGFS ACGOF ACMXC ACPOU ACPRK ACRPL ACSCC ACXBN ACXQS ACYXJ ADBBV ADBTR ADEOM ADIZJ ADKYN ADMGS ADNMO ADOZA ADXAS ADZCM ADZMN ADZOD AEEZP AEGXH AEIGN AEIMD AENEX AEQDE AEUQT AEUYR AFBPY AFEBI AFFNX AFFPM AFGKR AFPWT AFRAH AFWVQ AFZJQ AHBTC AHEFC AI. AIACR AIAGR AITYG AIURR AIWBW AJBDE ALAGY ALMA_UNASSIGNED_HOLDINGS ALUQN ALVPJ AMBMR AMYDB ASPBG ATUGU AVWKF AZBYB AZFZN AZVAB BAFTC BDRZF BFHJK BHBCM BMXJE BROTX BRXPI BY8 C45 CAG COF CS3 D-6 D-7 D-E D-F DCZOG DPXWK DR2 DRFUL DRMAN DRSTM DU5 EBS EGARE EJD EMOBN ESX EX3 F00 F01 F04 F5P FEDTE FUBAC FZ0 G-S G.N GODZA H.X HF~ HGLYW HVGLF HZI HZ~ IH2 IHE IX1 J0M J5H K48 KBYEO L7B LATKE LC2 LC3 LEEKS LH4 LITHE LOXES LP6 LP7 LUTES LW6 LYRES MEWTI MK4 MRFUL MRMAN MRSTM MSFUL MSMAN MSSTM MXFUL MXMAN MXSTM N04 N05 N4W N9A NF~ O66 O9- OIG OK1 OVD P2P P2W P2X P2Z P4B P4D PALCI Q.N Q11 QB0 R.K RIWAO RJQFR ROL RX1 SAMSI SUPJJ TEORI UB1 V8K V9Y VH1 W8V W99 WBKPD WHWMO WIH WIJ WIK WIN WOHZO WOW WQJ WRC WUP WVDHM WXI WXSBR X7M XG1 YFH YOC YUY ZGI ZXP ZZTAW ~IA ~WT AAYXX AEYWJ AGHNM AGQPQ AGYGG CITATION IQODW AAMMB AEFGJ AGXDD AIDQK AIDYY CGR CUY CVF ECM EIF NPM 7X8 7T5 H94
ID	FETCH-LOGICAL-c4237-7f88c2264782d10cb5b7543a2e3633b48d1eaad896f4564db9e66e374c3e54793
IEDL.DBID	DR2
ISSN	0007-1048 1365-2141
IngestDate	Fri Jul 11 02:47:13 EDT 2025 Fri Jul 11 07:29:24 EDT 2025 Mon Jul 21 05:29:26 EDT 2025 Wed Apr 02 07:14:59 EDT 2025 Thu Apr 24 23:09:36 EDT 2025 Tue Jul 01 02:05:20 EDT 2025 Wed Jan 22 16:35:08 EST 2025
IsDoiOpenAccess	false
IsOpenAccess	true
IsPeerReviewed	true
IsScholarly	true
Issue	3
Keywords	Human Antineoplastic agent Hemoglobinopathy Sickle cell anemia Hydroxycarbamide Hematology Stem cell Hematopoietic cell Hemopathy Genetic disease sickle cell disease Hemolytic anemia Cancerology Antimetabolic haematopoietic stem cell transplant Child Hematopoietic stem cell transplantation hydroxycarbamide
Language	English
License	CC BY 4.0 2014 John Wiley & Sons Ltd.
LinkModel	DirectLink
MergedId	FETCHMERGED-LOGICAL-c4237-7f88c2264782d10cb5b7543a2e3633b48d1eaad896f4564db9e66e374c3e54793
Notes	ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 content type line 23
OpenAccessLink	https://onlinelibrary.wiley.com/doi/pdfdirect/10.1111/bjh.12737
PMID	24433465
PQID	1515648815
PQPubID	23479
PageCount	7
ParticipantIDs	proquest_miscellaneous_1753457864 proquest_miscellaneous_1515648815 pubmed_primary_24433465 pascalfrancis_primary_28427852 crossref_primary_10_1111_bjh_12737 crossref_citationtrail_10_1111_bjh_12737 wiley_primary_10_1111_bjh_12737_BJH12737
ProviderPackageCode	CITATION AAYXX
PublicationCentury	2000
PublicationDate	May 2014
PublicationDateYYYYMMDD	2014-05-01
PublicationDate_xml	– month: 05 year: 2014 text: May 2014
PublicationDecade	2010
PublicationPlace	Oxford
PublicationPlace_xml	– name: Oxford – name: England
PublicationTitle	British journal of haematology
PublicationTitleAlternate	Br J Haematol
PublicationYear	2014
Publisher	Blackwell
Publisher_xml	– name: Blackwell
References	2004; 33 2007; 137 1995; 85 2010; 54 2012; 120 2011; 377 2007; 110 2010; 115 1992; 326 2013; 122 2013; 90 2000; 95 2013; 60 1988; 67 2008; 41 2009; 361 2002 2012; 59 1996; 335 1998; 22 e_1_2_6_10_1 e_1_2_6_20_1 Lederman H.M. (e_1_2_6_9_1) 2012; 120 e_1_2_6_8_1 e_1_2_6_19_1 e_1_2_6_5_1 e_1_2_6_4_1 e_1_2_6_7_1 e_1_2_6_6_1 e_1_2_6_13_1 e_1_2_6_14_1 e_1_2_6_3_1 e_1_2_6_11_1 Walters M.C. (e_1_2_6_21_1) 2000; 95 e_1_2_6_2_1 e_1_2_6_12_1 e_1_2_6_22_1 e_1_2_6_17_1 e_1_2_6_18_1 e_1_2_6_15_1 e_1_2_6_16_1
References_xml	– volume: 33 start-page: 799 year: 2004 end-page: 803 article-title: Hydroxyurea treatment for sickle cell disease: impact on haematopoietic stem cell transplantation's outcome publication-title: Bone Marrow Transplantation – volume: 59 start-page: 372 year: 2012 end-page: 376 article-title: Allogeneic hematopoietic stem cell transplantation in children with sickle cell disease publication-title: Pediatric Blood & Cancer – volume: 60 start-page: 1482 year: 2013 end-page: 1486 article-title: Sickle cell disease related mortality in the United States (1999‐2009) publication-title: Pediatric Blood & Cancer – volume: 120 start-page: 4304 year: 2012 end-page: 4310 article-title: Impact of hydroxyurea on clinical events in the BABY HUG trial publication-title: Blood – volume: 95 start-page: 1918 year: 2000 end-page: 1924 article-title: Impact of bone marrow transplantation for symptomatic sickle cell disease: an interim report. multicenter investigation of bone marrow transplantation for sickle cell disease publication-title: Blood – volume: 122 start-page: 1072 year: 2013 end-page: 1078 article-title: Outcome of patients with hemoglobinopathies given either cord blood or bone marrow transplantation from an HLA‐identical sibling publication-title: Blood – volume: 54 start-page: 250 year: 2010 end-page: 255 article-title: The pediatric hydroxyurea phase III clinical trial (BABY HUG): challenges of study design publication-title: Pediatric Blood & Cancer – volume: 85 start-page: 879 year: 1995 end-page: 884 article-title: Neurologic complications after allogeneic marrow transplantation for sickle cell anemia publication-title: Blood – volume: 377 start-page: 1663 year: 2011 end-page: 1672 article-title: Hydroxycarbamide in very young children with sickle‐cell anaemia: a multicentre, randomised, controlled trial (BABY HUG) publication-title: Lancet – year: 2002 – volume: 335 start-page: 369 year: 1996 end-page: 376 article-title: Bone marrow transplantation for sickle cell disease publication-title: The New England Journal of Medicine – volume: 67 start-page: 66 year: 1988 end-page: 76 article-title: Sickle cell chronic lung disease: prior morbidity and the risk of pulmonary failure publication-title: Medicine (Baltimore) – volume: 361 start-page: 2309 year: 2009 end-page: 2317 article-title: Allogeneic hematopoietic stem‐cell transplantation for sickle cell disease publication-title: New England Journal of Medicine – volume: 137 start-page: 479 year: 2007 end-page: 485 article-title: Matched‐related donor transplantation for sickle cell disease: report from the center for international blood and transplant research publication-title: British Journal of Haematology – volume: 115 start-page: 3447 year: 2010 end-page: 3452 article-title: Improved survival of children and adolescents with sickle cell disease publication-title: Blood – volume: 120 start-page: 243 year: 2012 article-title: Effects of hydroxyurea (HU) On lymphocyte subsets and the immune response to pneumococcal, measles, mumps and rubella vaccination in the pediatric hydroxyurea phase III clinical trial ‐ BABY HUG ‐ (ClinicalTrials.gov Identifier: NCT00006400) publication-title: ASH Annual Meeting Abstracts – volume: 22 start-page: 1 year: 1998 end-page: 6 article-title: Haematopoietic stem cell transplantation for sickle cell anaemia: the first 50 patients transplanted in Belgium publication-title: Bone Marrow Transplantation – volume: 326 start-page: 605 year: 1992 end-page: 610 article-title: The use of transcranial ultrasonography to predict stroke in sickle cell disease publication-title: New England journal of Medicine – volume: 90 start-page: 308 year: 2013 end-page: 312 article-title: Stem cell transplantation after reduced‐intensity conditioning for sickle cell disease publication-title: European Journal of Haematology – volume: 110 start-page: 2749 year: 2007 end-page: 2756 article-title: Long‐term results of related myeloablative stem‐cell transplantation to cure sickle cell disease publication-title: Blood – volume: 41 start-page: 109 year: 2008 end-page: 117 article-title: Hematopoietic cell transplantation for thalassemia and sickle cell disease: past, present and future publication-title: Bone Marrow Transplantation – ident: e_1_2_6_14_1 doi: 10.1097/00005792-198801000-00005 – ident: e_1_2_6_16_1 doi: 10.1002/pbc.22269 – ident: e_1_2_6_2_1 doi: 10.1056/NEJM199202273260905 – ident: e_1_2_6_12_1 doi: 10.1111/ejh.12082 – ident: e_1_2_6_8_1 doi: 10.1002/9781118032985 – ident: e_1_2_6_17_1 doi: 10.1182/blood-2012-03-419879 – ident: e_1_2_6_19_1 doi: 10.1182/blood.V85.4.879.bloodjournal854879 – ident: e_1_2_6_7_1 doi: 10.1056/NEJMoa0904971 – ident: e_1_2_6_10_1 doi: 10.1002/pbc.24177 – ident: e_1_2_6_22_1 doi: 10.1016/S0140-6736(11)60355-3 – ident: e_1_2_6_6_1 doi: 10.1002/pbc.24557 – ident: e_1_2_6_20_1 doi: 10.1056/NEJM199608083350601 – ident: e_1_2_6_13_1 doi: 10.1111/j.1365-2141.2007.06592.x – ident: e_1_2_6_3_1 doi: 10.1182/blood-2007-03-079665 – ident: e_1_2_6_4_1 doi: 10.1038/sj.bmt.1705943 – ident: e_1_2_6_5_1 doi: 10.1038/sj.bmt.1704443 – volume: 120 start-page: 243 year: 2012 ident: e_1_2_6_9_1 article-title: Effects of hydroxyurea (HU) On lymphocyte subsets and the immune response to pneumococcal, measles, mumps and rubella vaccination in the pediatric hydroxyurea phase III clinical trial ‐ BABY HUG ‐ (ClinicalTrials.gov Identifier: NCT00006400) publication-title: ASH Annual Meeting Abstracts – ident: e_1_2_6_18_1 doi: 10.1038/sj.bmt.1701291 – ident: e_1_2_6_11_1 doi: 10.1182/blood-2013-03-489112 – ident: e_1_2_6_15_1 doi: 10.1182/blood-2009-07-233700 – volume: 95 start-page: 1918 year: 2000 ident: e_1_2_6_21_1 article-title: Impact of bone marrow transplantation for symptomatic sickle cell disease: an interim report. multicenter investigation of bone marrow transplantation for sickle cell disease publication-title: Blood
SSID	ssj0013051
Score	2.366877
Snippet	Summary Despite improvements in medical management, sickle cell disease (SCD) remains associated with severe morbidity and decreased survival. Allogeneic... Despite improvements in medical management, sickle cell disease ( SCD ) remains associated with severe morbidity and decreased survival. Allogeneic... Despite improvements in medical management, sickle cell disease (SCD) remains associated with severe morbidity and decreased survival. Allogeneic...
SourceID	proquest pubmed pascalfrancis crossref wiley
SourceType	Aggregation Database Index Database Enrichment Source Publisher
StartPage	402
SubjectTerms	Adolescent Anemia, Sickle Cell - surgery Anemias. Hemoglobinopathies Animals Biological and medical sciences Child Child, Preschool Diseases of red blood cells Female Genotype haematopoietic stem cell transplant Hematologic and hematopoietic diseases Hematopoietic Stem Cell Transplantation - methods Humans hydroxycarbamide Infant Male Medical sciences Rabbits sickle cell disease Survival Analysis Transplantation Conditioning - methods Transplantation, Autologous Tumors
Title	Haematopoietic stem cell transplantation for severe sickle cell disease in childhood: a single centre experience of 50 patients
URI	https://onlinelibrary.wiley.com/doi/abs/10.1111%2Fbjh.12737 https://www.ncbi.nlm.nih.gov/pubmed/24433465 https://www.proquest.com/docview/1515648815 https://www.proquest.com/docview/1753457864
Volume	165
hasFullText	1
inHoldings	1
isFullTextHit
isPrint
link	http://utb.summon.serialssolutions.com/2.0.0/link/0/eLvHCXMwnV1Li9RAEC6WPYggvh_jY2nFg5cMmfQjGT2puAwL60Fc2IMQ-lHBdYdk2Jm5ePGvW9WdZB1ZRbzlUCHdnXp83V31FcBL5U2RS6sy6eY-UyXazAVVZlg0lbTBYYjs_McfzeJEHZ3q0z14M9TCJH6I8cCNLSP6azZw69a_GLn79nU6o-DLleScq8WA6FNxeYOQ675bXkmuRlU9qxBn8Yxv7sSiGyu7pmVpUj-LqwDnLn6NAejwFnwZhp7yTs6n242b-u-_sTr-59xuw80emIq3SZPuwB62d-HacX_1fg9-LCzTu3ar7ozrHgUTQAs-9hebyI--tKmIqRUEgwXFW7xAQSpwvsQk1l8FibNW-IFP-bWwgg8rogwzbAocqZdF1widi574dX0fTg4_fH6_yPruDZnnVJusbKrKc5kuYZAwy73TrtRK2gKlkdKpKszQ2lDNTcOUNsHN0RiUpfISNZ_3PYD9tmvxEYhgAqFU25SO2ZLyuats7sj7ILfPsqgn8Gr4j7Xvqc25w8ayHrY4tKB1XNAJvBhFV4nP4yqhgx1lGCUplhdlpYsJPB-0oyZz5FW0LXbbdc340JBTnOm_yNAWUZGnNGoCD5NqXX5BKSmV4SlFBfnzIOt3R4v48PjfRZ_AdQJ8KiVsPoX9zcUWnxGo2riDaD0_AYdlHVk
linkProvider	Wiley-Blackwell
linkToHtml	http://utb.summon.serialssolutions.com/2.0.0/link/0/eLvHCXMwnV1Lb9QwEB6VIgFSxbOP5VEM4sAlq2zsOFnEhVcVSrcH1Eq9oMiPiVq6Slbd3Usv_HVm8iqLCkLccpgotjOPz-PxNwCvlNNRKI0KpB27QCVoAutVEmBUpNJ4i75m558c6uxY7Z_EJ2vwtrsL0_BD9Ak3tozaX7OBc0L6Fyu330-HI4q-yQ24yR29mTn_49fo6gwhjNt-eQk5G5W2vEJcx9O_uhKNNmZmTgtTNB0troOcqwi2DkF79-BbN_im8uR8uFzYobv8jdfxf2d3H-622FS8a5TpAaxh-RBuTdrT90fwIzPM8FrNqjO--iiYA1pw5l8saor0qWnuMZWCkLCgkIsXKEgLzqfYiLWnQeKsFK6jVH4jjOB8RS3DJJsCe_ZlURUiDkXL_TrfhOO9T0cfsqBt4BA4rrYJkiJNHd_UJRjiR6GzsU1iJU2EUktpVepHaIxPx7pgVhtvx6g1ykQ5iTGn_LZgvaxK3AHhtSegaorEMmFSOLapCS05IOQOWgbjAbzufmTuWnZzbrIxzbtdDi1oXi_oAF72orOG0uM6od0VbeglKZxHSRpHA3jRqUdOFsmraEqslvOcIaImvziK_yJDu0RFzlKrAWw3unX1BaWkJIWmKdUa8udB5u_3s_rh8b-LPofb2dHkID_4fPjlCdwh_Kea-s2nsL64WOIzwlgLu1ub0k9G9CF1
linkToPdf	http://utb.summon.serialssolutions.com/2.0.0/link/0/eLvHCXMwnV1Lb9QwEB6VIlVIqDxLt4ViEAcuWWXjV1JOQFkthVYIUakHpMiviNJVsuruXnrhr-Oxk5RFBSFuOUwUezKPz2P7G4AXzIgspYolVBcmYdKpRFsmE5dVOVVWOxvY-Y-OxeSEHZ7y0zV41d2FifwQfcENPSPEa3Twma1-cXL9_dtw5JOvvAE3mUgL7Ntw8Dm72kJIedsuT_pYw_KWVgiP8fSvriSj2zM193qpYkOL6xDnKoANGWh8B752Y48HT86Hy4UemsvfaB3_c3J3YbNFpuR1NKV7sObq-7Bx1O69P4AfE4X8rs2sOcOLjwQZoAnW_ckiEKRPVbzFVBOPg4lPuO7CEW8D51MXxdq9IHJWE9MRKu8TRbBaEWSQYpO4nnuZNBXhKWmZX-cP4WT87svbSdK2b0gMnrVJZJXnBu_pehBiR6nRXEvOqMocFZRqltuRU8rmhaiQ08bqwgnhqGSGOo4Fvy1Yr5vabQOxwnqYqiqpkS4pLXSuUu3Dj8P-WcrxAbzs_mNpWm5zbLExLbs1jldoGRQ6gOe96CwSelwntLdiDL2kT-aZzHk2gGeddZTeH1GLqnbNcl4iQBQ-Ko74X2T8GpH5UCnYAB5F07r6AmOUMoFTCgby50GWbw4n4WHn30Wfwsang3H58f3xh1245cEfi4c3H8P64mLpnniAtdB7wZF-Asz-ICQ
openUrl	ctx_ver=Z39.88-2004&ctx_enc=info%3Aofi%2Fenc%3AUTF-8&rfr_id=info%3Asid%2Fsummon.serialssolutions.com&rft_val_fmt=info%3Aofi%2Ffmt%3Akev%3Amtx%3Ajournal&rft.genre=article&rft.atitle=Haematopoietic+stem+cell+transplantation+for+severe+sickle+cell+disease+in+childhood%3A+a+single+centre+experience+of+50+patients&rft.jtitle=British+journal+of+haematology&rft.au=Dedeken%2C+Laurence&rft.au=L%C3%AA%2C+Phu+Q&rft.au=Azzi%2C+Nadira&rft.au=Brachet%2C+C%C3%A9cile&rft.date=2014-05-01&rft.eissn=1365-2141&rft.volume=165&rft.issue=3&rft.spage=402&rft_id=info:doi/10.1111%2Fbjh.12737&rft_id=info%3Apmid%2F24433465&rft.externalDocID=24433465
thumbnail_l	http://covers-cdn.summon.serialssolutions.com/index.aspx?isbn=/lc.gif&issn=0007-1048&client=summon
thumbnail_m	http://covers-cdn.summon.serialssolutions.com/index.aspx?isbn=/mc.gif&issn=0007-1048&client=summon
thumbnail_s	http://covers-cdn.summon.serialssolutions.com/index.aspx?isbn=/sc.gif&issn=0007-1048&client=summon