Haploidentical bone marrow transplantation with post transplant cyclophosphamide for patients with X-linked adrenoleukodystrophy: a suitable choice in an urgent situation

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dc.contributorSistema FMUSP-HC: Faculdade de Medicina da Universidade de São Paulo (FMUSP) e Hospital das Clínicas da FMUSP
dc.contributor.authorFERNANDES, Juliana Folloni
dc.contributor.authorBONFIM, Carmem
dc.contributor.authorKERBAUY, Fabio Rodrigues
dc.contributor.authorRODRIGUES, Morgani
dc.contributor.authorESTEVES, Iracema
dc.contributor.authorSILVA, Nathalia Halley
dc.contributor.authorAZAMBUJA, Alessandra Prandini
dc.contributor.authorMANTOVANI, Luiz Fernando
dc.contributor.authorKUTNER, Jose Mauro
dc.contributor.authorLOTH, Gisele
dc.contributor.authorKUWAHARA, Cilmara Cristina
dc.contributor.authorBUENO, Clarissa
dc.contributor.authorKONDO, Andrea Tiemi
dc.contributor.authorRIBEIRO, Andreza Alice Feitosa
dc.contributor.authorKOK, Fernando
dc.contributor.authorHAMERSCHLAK, Nelson
dc.date.accessioned2018-07-05T18:03:44Z
dc.date.available2018-07-05T18:03:44Z
dc.date.issued2018
dc.description.abstractAllogeneic hematopoietic stem cell transplantation (HSCT) is the only treatment that enhances survival and stabilizes neurologic symptoms in X-linked adrenoleukodystrophy (X-ALD) with cerebral involvement, a severe demyelinating disease of childhood. Patients with X-ALD who lack a well-matched HLA donor need a rapid alternative. Haploidentical HSCT using post transplant cyclophosphamide (PT/Cy) has been performed in patients with malignant and nonmalignant diseases showing similar outcomes compared to other alternative sources. We describe the outcomes of transplants performed for nine X-ALD patients using haploidentical donors and PT/Cy. Patients received conditioning regimen with fludarabine 150 mg/m(2) , cyclophosphamide 29 mg/kg and 2 Gy total body irradiation (TBI) with or without antithymocyte globulin. Graft-vs.-host disease prophylaxis consisted of cyclophosphamide 50 mg/kg/day on days +3 and +4, tacrolimus or cyclosporine A and mycophenolate mofetil. One patient had a primary graft failure and was not eligible for a second transplant. Three patients had secondary graft failure and were successfully rescued with second haploidentical transplants. Trying to improve engraftment, conditioning regimen was changed, substituting 2 Gy TBI for 4 Gy total lymphoid irradiation. Eight patients are alive and engrafted (17-37 months after transplant). Haploidentical HSCT with PT/Cy is a feasible alternative for X-ALD patients lacking a suitable matched donor. Graft failure has to be addressed in further studies.
dc.description.indexMEDLINE
dc.identifier.citationBONE MARROW TRANSPLANTATION, v.53, n.4, p.392-399, 2018
dc.identifier.doi10.1038/s41409-017-0015-2
dc.identifier.eissn1476-5365
dc.identifier.issn0268-3369
dc.identifier.urihttps://observatorio.fm.usp.br/handle/OPI/26976
dc.language.isoeng
dc.publisherNATURE PUBLISHING GROUP
dc.relation.ispartofBone Marrow Transplantation
dc.rightsrestrictedAccess
dc.rights.holderCopyright NATURE PUBLISHING GROUP
dc.subject.otherhematopoietic-cell transplantation
dc.subject.otherversus-host-disease
dc.subject.otherposttransplantation cyclophosphamide
dc.subject.otheraplastic-anemia
dc.subject.otherirradiation
dc.subject.otherengraftment
dc.subject.otheroutcomes
dc.subject.othertherapy
dc.subject.otherbmt
dc.subject.wosBiophysics
dc.subject.wosOncology
dc.subject.wosHematology
dc.subject.wosImmunology
dc.subject.wosTransplantation
dc.titleHaploidentical bone marrow transplantation with post transplant cyclophosphamide for patients with X-linked adrenoleukodystrophy: a suitable choice in an urgent situation
dc.typearticle
dc.type.categoryoriginal article
dc.type.versionpublishedVersion
dspace.entity.typePublication
hcfmusp.author.externalBONFIM, Carmem:Univ Fed Parana, Hosp Clin, Bone Marrow Transplantat Dept, Curitiba, Parana, Brazil; Hosp Nossa Senhora Gracas, Bone Marrow Transplantat Dept, Curitiba, Parana, Brazil
hcfmusp.author.externalKERBAUY, Fabio Rodrigues:Hosp Israelita Albert Einstein, Hematol & Bone Marrow Transplantat Program, Sao Paulo, Brazil
hcfmusp.author.externalRODRIGUES, Morgani:Hosp Israelita Albert Einstein, Hematol & Bone Marrow Transplantat Program, Sao Paulo, Brazil
hcfmusp.author.externalESTEVES, Iracema:Hosp Israelita Albert Einstein, Hematol & Bone Marrow Transplantat Program, Sao Paulo, Brazil
hcfmusp.author.externalKUTNER, Jose Mauro:Hosp Israelita Albert Einstein, Hematol & Bone Marrow Transplantat Program, Sao Paulo, Brazil
hcfmusp.author.externalLOTH, Gisele:Univ Fed Parana, Hosp Clin, Bone Marrow Transplantat Dept, Curitiba, Parana, Brazil; Hosp Nossa Senhora Gracas, Bone Marrow Transplantat Dept, Curitiba, Parana, Brazil
hcfmusp.author.externalKUWAHARA, Cilmara Cristina:Univ Fed Parana, Hosp Clin, Bone Marrow Transplantat Dept, Curitiba, Parana, Brazil; Hosp Nossa Senhora Gracas, Bone Marrow Transplantat Dept, Curitiba, Parana, Brazil
hcfmusp.author.externalKONDO, Andrea Tiemi:Hosp Israelita Albert Einstein, Hematol & Bone Marrow Transplantat Program, Sao Paulo, Brazil
hcfmusp.author.externalRIBEIRO, Andreza Alice Feitosa:Hosp Israelita Albert Einstein, Hematol & Bone Marrow Transplantat Program, Sao Paulo, Brazil
hcfmusp.author.externalHAMERSCHLAK, Nelson:Hosp Israelita Albert Einstein, Hematol & Bone Marrow Transplantat Program, Sao Paulo, Brazil
hcfmusp.citation.scopus13
hcfmusp.contributor.author-fmusphcJULIANA FOLLONI FERNANDES
hcfmusp.contributor.author-fmusphcNATHALIA DA SILVA HALLEY NEVES
hcfmusp.contributor.author-fmusphcALESSANDRA MILANI PRANDINI DE AZAMBUJA
hcfmusp.contributor.author-fmusphcLUIZ FERNANDO ALVES LIMA MANTOVANI
hcfmusp.contributor.author-fmusphcCLARISSA BUENO
hcfmusp.contributor.author-fmusphcFERNANDO KOK
hcfmusp.description.beginpage392
hcfmusp.description.endpage399
hcfmusp.description.issue4
hcfmusp.description.volume53
hcfmusp.origemWOS
hcfmusp.origem.pubmed29330393
hcfmusp.origem.scopus2-s2.0-85040359860
hcfmusp.origem.wosWOS:000429913700003
hcfmusp.publisher.cityLONDON
hcfmusp.publisher.countryENGLAND
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