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Reduced intensity conditioning is effective for hematopoietic SCT in dyskeratosis congenita-related BM failure

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dc.contributor.author Ayas M.
dc.contributor.author Nassar A.
dc.contributor.author Hamidieh A.A.
dc.contributor.author Kharfan-Dabaja M.
dc.contributor.author Othman T.B.
dc.contributor.author Elhaddad A.
dc.contributor.author Seraihy A.
dc.contributor.author Hussain F.
dc.contributor.author Alimoghaddam K.
dc.contributor.author Ladeb S.
dc.contributor.author Fahmy O.
dc.contributor.author Bazarbachi A.
dc.contributor.author Mohamed S.Y.
dc.contributor.author Bakr M.
dc.contributor.author Korthof E.
dc.contributor.author Aljurf M.
dc.contributor.author Ghavamzadeh A.
dc.contributor.editor
dc.date Sep-2013
dc.date.accessioned 2017-10-05T15:40:07Z
dc.date.available 2017-10-05T15:40:07Z
dc.date.issued 2013
dc.identifier 10.1038/bmt.2013.35
dc.identifier.isbn
dc.identifier.issn 02683369
dc.identifier.uri http://hdl.handle.net/10938/17260
dc.description.abstract BM failure (BMF) is a major and frequent complication of dyskeratosis congenita (DKC). Allogeneic hematopoietic SCT (allo-HSCT) represents the only curative treatment for BMF associated with this condition. Transplant-related morbidity-mortality is common especially after myeloablative conditioning regimens. Herein, we report nine cases of patients with DKC who received an allo-SCT at five different member centers within the Eastern Mediterranean Blood and Marrow Transplantation Registry. Between October 1992 and February 2011, nine DKC patients (male, 7 and female, 2), with a median age at transplantation of 19.1 (4.9-31.1) years, underwent an allo-HSCT from HLA-matched, morphologically normal-related donors (100percent). Preparative regimens varied according to different centers, but was reduced intensity conditioning (RIC) in eight patients. Graft source was unstimulated BM in five cases (56percent) and G-CSF-mobilized PBSCs in four (44percent) cases. The median stem cell dose was 6.79 (2.06-12.4) × 106 cells-kg body weight. GVHD prophylaxis consisted of CsA in all nine cases; MTX or mycophenolate mofetil were added in five (56percent) and two (22percent) cases, respectively. Anti-thymocyte globulin was administered at various doses and scheduled in four (44percent) cases. Median time-to-neutrophil engraftment was 21 (17-27) days. In one case, late graft failure was noted at 10.4 months post allo-HSCT. Only one patient developed grade II acute GVHD (11percent). Extensive chronic GVHD was reported in one case, whereas limited chronic GVHD occurred in another four cases. At a median follow-up of 61 (0.8-212) months, seven (78percent) patients were still alive and transfusion independent. One patient died of metastatic gastric adenocarcinoma and graft failure was the cause of death in another patient. This study suggests that RIC preparative regimens are successful in inducing hematopoietic cell engraftment in patients with BMF from DKC. Owing to the limited sample size, the use of registry data and heterogeneity of preparative as well as GVHD prophylaxis regimens reported in this series, we are unable to recommend a particular regimen to be considered as the standard for patients with this disease. © 2013 Macmillan Publishers Limited.
dc.format.extent
dc.format.extent Pages: (1168-1172)
dc.language English
dc.publisher LONDON
dc.relation.ispartof Publication Name: Bone Marrow Transplantation; Publication Year: 2013; Volume: 48; no. 9; Pages: (1168-1172);
dc.relation.ispartofseries
dc.relation.uri
dc.source Scopus
dc.subject.other
dc.title Reduced intensity conditioning is effective for hematopoietic SCT in dyskeratosis congenita-related BM failure
dc.type Article
dc.contributor.affiliation Ayas, M., Adult Hematopoietic Stem Cell Transplantation Program, Oncology Centre, King Faisal Specialist Hospital and Research Center, MBC 64, POB 3354, Riyadh 11211, Saudi Arabia
dc.contributor.affiliation Nassar, A., Adult Hematopoietic Stem Cell Transplantation Program, Oncology Centre, King Faisal Specialist Hospital and Research Center, MBC 64, POB 3354, Riyadh 11211, Saudi Arabia
dc.contributor.affiliation Hamidieh, A.A., HSCT Unit, Tehran University of Medical Sciences, Tehran, Iran
dc.contributor.affiliation Kharfan-Dabaja, M., HSCT Unit, American University Beirut Medical Center, Beirut, Lebanon
dc.contributor.affiliation Othman, T.B., Center National de Greffe de Moelle Osseuse de Tunis, Tunis, Tunisia
dc.contributor.affiliation Elhaddad, A., National Cancer Institute, Cairo University, Cairo, Egypt
dc.contributor.affiliation Seraihy, A., Adult Hematopoietic Stem Cell Transplantation Program, Oncology Centre, King Faisal Specialist Hospital and Research Center, MBC 64, POB 3354, Riyadh 11211, Saudi Arabia
dc.contributor.affiliation Hussain, F., Adult Hematopoietic Stem Cell Transplantation Program, Oncology Centre, King Faisal Specialist Hospital and Research Center, MBC 64, POB 3354, Riyadh 11211, Saudi Arabia
dc.contributor.affiliation Alimoghaddam, K., HSCT Unit, Tehran University of Medical Sciences, Tehran, Iran
dc.contributor.affiliation Ladeb, S., Center National de Greffe de Moelle Osseuse de Tunis, Tunis, Tunisia
dc.contributor.affiliation Fahmy, O., National Cancer Institute, Cairo University, Cairo, Egypt
dc.contributor.affiliation Bazarbachi, A., HSCT Unit, American University Beirut Medical Center, Beirut, Lebanon
dc.contributor.affiliation Mohamed, S.Y., Adult Hematopoietic Stem Cell Transplantation Program, Oncology Centre, King Faisal Specialist Hospital and Research Center, MBC 64, POB 3354, Riyadh 11211, Saudi Arabia
dc.contributor.affiliation Bakr, M., Adult Hematopoietic Stem Cell Transplantation Program, Oncology Centre, King Faisal Specialist Hospital and Research Center, MBC 64, POB 3354, Riyadh 11211, Saudi Arabia
dc.contributor.affiliation Korthof, E., Sanquin-LUMC Jon J van Rood Center for Clinical Transfusion Research, Leiden, Netherlands
dc.contributor.affiliation Aljurf, M., Adult Hematopoietic Stem Cell Transplantation Program, Oncology Centre, King Faisal Specialist Hospital and Research Center, MBC 64, POB 3354, Riyadh 11211, Saudi Arabia
dc.contributor.affiliation Ghavamzadeh, A., HSCT Unit, Tehran University of Medical Sciences, Tehran, Iran
dc.contributor.authorAddress Aljurf, M.; Adult Hematopoietic Stem Cell Transplantation Program, Oncology Centre, King Faisal Specialist Hospital and Research Center, MBC 64, POB 3354, Riyadh 11211, Saudi Arabia; email: maljurf@kfshrc.edu.sa
dc.contributor.authorCorporate University: American University of Beirut Medical Center; Faculty: Faculty of Medicine; Department: Internal Medicine;
dc.contributor.authorDepartment Internal Medicine
dc.contributor.authorDivision
dc.contributor.authorEmail maljurf@kfshrc.edu.sa
dc.contributor.authorFaculty Faculty of Medicine
dc.contributor.authorInitials Ayas, M
dc.contributor.authorInitials Nassar, A
dc.contributor.authorInitials Hamidieh, AA
dc.contributor.authorInitials Kharfan-Dabaja, M
dc.contributor.authorInitials Othman, TB
dc.contributor.authorInitials Elhaddad, A
dc.contributor.authorInitials Seraihy, A
dc.contributor.authorInitials Hussain, F
dc.contributor.authorInitials Alimoghaddam, K
dc.contributor.authorInitials Ladeb, S
dc.contributor.authorInitials Fahmy, O
dc.contributor.authorInitials Bazarbachi, A
dc.contributor.authorInitials Mohamed, SY
dc.contributor.authorInitials Bakr, M
dc.contributor.authorInitials Korthof, E
dc.contributor.authorInitials Aljurf, M
dc.contributor.authorInitials Ghavamzadeh, A
dc.contributor.authorOrcidID
dc.contributor.authorReprintAddress Aljurf, M (reprint author), King Faisal Specialist Hosp and Res Ctr, Ctr Oncol, Adult Hematopoiet Stem Cell Transplantat Program, MBC 64,POB 3354, Riyadh 11211, Saudi Arabia.
dc.contributor.authorResearcherID
dc.contributor.authorUniversity American University of Beirut Medical Center
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dc.description.citedCount 2
dc.description.citedTotWOSCount 4
dc.description.citedWOSCount 4
dc.format.extentCount 5
dc.identifier.articleNo
dc.identifier.coden BMTRE
dc.identifier.pubmedID 23542225
dc.identifier.scopusID 84883742527
dc.identifier.url
dc.publisher.address MACMILLAN BUILDING, 4 CRINAN ST, LONDON N1 9XW, ENGLAND
dc.relation.ispartofConference
dc.relation.ispartofConferenceCode
dc.relation.ispartofConferenceDate
dc.relation.ispartofConferenceHosting
dc.relation.ispartofConferenceLoc
dc.relation.ispartofConferenceSponsor
dc.relation.ispartofConferenceTitle
dc.relation.ispartofFundingAgency
dc.relation.ispartOfISOAbbr Bone Marrow Transplant.
dc.relation.ispartOfIssue 9
dc.relation.ispartOfPart
dc.relation.ispartofPubTitle Bone Marrow Transplantation
dc.relation.ispartofPubTitleAbbr Bone Marrow Transplant.
dc.relation.ispartOfSpecialIssue
dc.relation.ispartOfSuppl
dc.relation.ispartOfVolume 48
dc.source.ID WOS:000324166300004
dc.type.publication Journal
dc.subject.otherAuthKeyword CBT
dc.subject.otherAuthKeyword DKC
dc.subject.otherAuthKeyword EMRO
dc.subject.otherAuthKeyword Hematopoietic SCT
dc.subject.otherAuthKeyword MUD
dc.subject.otherAuthKeyword Trends
dc.subject.otherChemCAS busulfan, 55-98-1
dc.subject.otherChemCAS cyclophosphamide, 50-18-0
dc.subject.otherChemCAS cyclosporin A, 59865-13-3, 63798-73-2
dc.subject.otherChemCAS fludarabine, 21679-14-1
dc.subject.otherChemCAS methotrexate, 15475-56-6, 59-05-2, 7413-34-5
dc.subject.otherChemCAS mycophenolic acid 2 morpholinoethyl ester, 116680-01-4, 128794-94-5
dc.subject.otherIndex busulfan
dc.subject.otherIndex cyclophosphamide
dc.subject.otherIndex cyclosporin A
dc.subject.otherIndex fludarabine
dc.subject.otherIndex granulocyte macrophage colony stimulating factor
dc.subject.otherIndex methotrexate
dc.subject.otherIndex mycophenolic acid 2 morpholinoethyl ester
dc.subject.otherIndex thymocyte antibody
dc.subject.otherIndex adolescent
dc.subject.otherIndex adult
dc.subject.otherIndex allogeneic hematopoietic stem cell transplantation
dc.subject.otherIndex article
dc.subject.otherIndex bone marrow depression
dc.subject.otherIndex cause of death
dc.subject.otherIndex CD34 selection
dc.subject.otherIndex child
dc.subject.otherIndex chronic graft versus host disease
dc.subject.otherIndex clinical article
dc.subject.otherIndex conservative treatment
dc.subject.otherIndex controlled study
dc.subject.otherIndex dyskeratosis congenita
dc.subject.otherIndex engraftment
dc.subject.otherIndex female
dc.subject.otherIndex graft failure
dc.subject.otherIndex graft versus host reaction
dc.subject.otherIndex hematopoietic stem cell transplantation
dc.subject.otherIndex human
dc.subject.otherIndex induction chemotherapy
dc.subject.otherIndex low drug dose
dc.subject.otherIndex male
dc.subject.otherIndex morbidity
dc.subject.otherIndex mortality
dc.subject.otherIndex myeloablative conditioning
dc.subject.otherIndex paraaortic lymph node
dc.subject.otherIndex preschool child
dc.subject.otherIndex priority journal
dc.subject.otherIndex reduced intensity conditioning
dc.subject.otherIndex school child
dc.subject.otherIndex stomach adenocarcinoma
dc.subject.otherIndex thrombocytopenia
dc.subject.otherIndex Adolescent
dc.subject.otherIndex Adult
dc.subject.otherIndex Bone Marrow Diseases
dc.subject.otherIndex Child
dc.subject.otherIndex Child, Preschool
dc.subject.otherIndex Dyskeratosis Congenita
dc.subject.otherIndex Female
dc.subject.otherIndex Hematopoietic Stem Cell Transplantation
dc.subject.otherIndex Humans
dc.subject.otherIndex Male
dc.subject.otherIndex Survival Analysis
dc.subject.otherIndex Transplantation Conditioning
dc.subject.otherIndex Transplantation, Homologous
dc.subject.otherIndex Young Adult
dc.subject.otherKeywordPlus STEM-CELL TRANSPLANTATION
dc.subject.otherKeywordPlus BONE-MARROW-TRANSPLANTATION
dc.subject.otherKeywordPlus INTERSTITIAL PULMONARY-DISEASE
dc.subject.otherKeywordPlus ANTITHYMOCYTE GLOBULIN
dc.subject.otherKeywordPlus APLASTIC-ANEMIA
dc.subject.otherKeywordPlus FLUDARABINE
dc.subject.otherKeywordPlus TELOMERASE
dc.subject.otherKeywordPlus PATIENT
dc.subject.otherKeywordPlus REGIMEN
dc.subject.otherKeywordPlus CYCLOPHOSPHAMIDE
dc.subject.otherWOS Biophysics
dc.subject.otherWOS Oncology
dc.subject.otherWOS Hematology
dc.subject.otherWOS Immunology
dc.subject.otherWOS Transplantation


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