PMID- 33606297
OWN - NLM
STAT- MEDLINE
DCOM- 20210513
LR  - 20210513
IS  - 1096-8652 (Electronic)
IS  - 0361-8609 (Linking)
VI  - 96
IP  - 5
DP  - 2021 May 1
TI  - Haplo-identical or mismatched unrelated donor hematopoietic cell transplantation 
      for Fanconi anemia: Results from the Severe Aplastic Anemia Working Party of the 
      EBMT.
PG  - 571-579
LID - 10.1002/ajh.26135 [doi]
AB  - Allogeneic hematopoietic cell transplantation (HCT) is the only curative option 
      for bone marrow failure or hematopoietic malignant diseases for Fanconi anemia 
      (FA) patients. Although results have improved over the last decades, reaching 
      more than 90% survival when a human leukocyte antigen (HLA)-identical donor is 
      available, alternative HCT donors are still less reported. We compared HCT 
      outcomes using HLA-mismatched unrelated donors (MMUD; n = 123) or haplo-identical 
      donors (HDs), either using only in vivo T cell depletion (n = 33) or T cells 
      depleted in vivo with some type of graft manipulation ex vivo (n = 59) performed 
      for FA between 2000 and 2018. Overall survival (OS) by 24 months was 62% (53-71%) 
      for MMUD, versus 80% (66-95%) for HDs with only in vivo T cell depletion and 60% 
      (47-73%) for HDs with in vivo and ex vivo T cell depletion (p = .22). Event-free 
      survival (EFS) was better for HD-transplanted FA patients with only in vivo T 
      cell depletion 86% (73-99%) than for those transplanted from a MMUD 58% (48-68%) 
      or those with graft manipulation 56% (42-69%) (p = .046). Grade II-IV acute 
      graft-versus-host disease (GVHD) was 41% (MMUD) versus 40% (HDs with no graft 
      manipulation) versus 17% (HDs with T cell depleted graft), (p = .005). No 
      differences were found for the other transplant related outcomes. These data 
      suggest that HDs might be considered as an alternative option for FA patients 
      with better EFS using unmanipulated grafts.
CI  - (c) 2021 Wiley Periodicals LLC.
FAU - Zubicaray, Josune
AU  - Zubicaray J
AD  - Nino Jesus Children's Hospital, Fundacion para la investigacion del HIUNJ, 
      Madrid, Spain.
FAU - Pagliara, Daria
AU  - Pagliara D
AD  - IRRCS Ospedale Pediatrico Bambino Gesu, Rome, Italy.
FAU - Sevilla, Julian
AU  - Sevilla J
AUID- ORCID: 0000-0002-6852-1860
AD  - Nino Jesus Children's Hospital, Fundacion para la investigacion del HIUNJ, 
      Madrid, Spain.
FAU - Eikema, Dirk-Jan
AU  - Eikema DJ
AD  - EBMT Statistical Unit Leiden, Leiden, The Netherlands.
FAU - Bosman, Paul
AU  - Bosman P
AUID- ORCID: 0000-0002-1473-9442
AD  - EBMT Data Office Leiden, Leiden, The Netherlands.
FAU - Ayas, Mouhab
AU  - Ayas M
AD  - King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia.
FAU - Zecca, Marco
AU  - Zecca M
AD  - Pediatric Hematology/Oncology, Fondazione IRCCS Policlinico San Matteo, Pavia, 
      Italy.
FAU - Yesilipek, Akif
AU  - Yesilipek A
AD  - Medical Park Antalya Hospital, Antalya, Turkey.
FAU - Kansoy, Savas
AU  - Kansoy S
AD  - Ege University Pediatric BMT Centre, Izmir, Turkey.
FAU - Renard, Cecile
AU  - Renard C
AD  - Institut d'Hematologie et d'Oncologie Pediatrique, Hospices Civils de Lyon, Lyon, 
      France.
FAU - Dalle, Jean H
AU  - Dalle JH
AD  - Hemato-Immunology Department, Robert-Debre Hospital, GHU Nord-Universite de 
      Paris, Paris, France.
FAU - Campos, Antonio
AU  - Campos A
AD  - Inst. Portugues de Oncologia do Porto, Porto, Portugal.
FAU - Faraci, Maura
AU  - Faraci M
AD  - HSCT Unit, Instituto G. Gaslini, Genoa, Italy.
FAU - Kupesiz, Alphan
AU  - Kupesiz A
AD  - Akdeniz University Medical School, Antalya, Turkey.
FAU - Smiers, Frans J W
AU  - Smiers FJW
AD  - Department of Pediatrics, Leiden University Medical Center, Leiden, The 
      Netherlands.
FAU - Velardi, Andrea
AU  - Velardi A
AD  - Sezione di Ematologia, Perugia, Italy.
FAU - Abecasis, Manuel
AU  - Abecasis M
AD  - Inst. Portugues Oncologia, Lisbon, Portugal.
FAU - Corti, Paola
AU  - Corti P
AD  - Clinica Pediatrica, Fondazione MBBM, Universita degli Studi di Milano Bicocca, 
      Monza, Italy.
FAU - Fagioli, Franca
AU  - Fagioli F
AD  - Pediatric Onco-Hematology and Stem Cell Transplantation Division, Regina 
      Margherita Children's Hospital, University of Turin, Turin, Italy.
FAU - Gonzalez Muniz, Soledad
AU  - Gonzalez Muniz S
AD  - Hospital Universitario Central de Asturias, Oviedo, Spain.
FAU - Krivan, Gergely
AU  - Krivan G
AD  - Department for Pediatric Hematology and Hemopoietic Stem Cell Transplantation, 
      Central Hospital of Southern Pest - National Institute of Hematology and 
      Infectious Diseases, Budapest, Hungary.
FAU - Dufour, Carlo
AU  - Dufour C
AD  - Istituto Giannina Gaslini, IRCSS, Genova, Italy.
FAU - Risitano, Antonio
AU  - Risitano A
AUID- ORCID: 0000-0002-4744-5109
AD  - AORN San Giuseppe Moscati, Hematology and Hematopoietic Stem Cell Transplantation 
      Unit, Avellino, Italy.
AD  - Federico II University of Naples, Naples, Italy.
FAU - Corbacioglu, Selim
AU  - Corbacioglu S
AD  - Department of Pediatric Hematology, Oncology and Stem Cell Transplantation, 
      University of Regensburg, Regensburg, Germany.
FAU - Peffault de Latour, Regis
AU  - Peffault de Latour R
AUID- ORCID: 0000-0001-6222-4753
AD  - French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal 
      Hemoglobinuria, Saint Louis Hospital and University Paris Diderot, Paris, France.
LA  - eng
PT  - Journal Article
PT  - Multicenter Study
DEP - 20210304
PL  - United States
TA  - Am J Hematol
JT  - American journal of hematology
JID - 7610369
RN  - 0 (HLA Antigens)
SB  - IM
MH  - Adolescent
MH  - Allografts
MH  - *Bone Marrow Transplantation/statistics & numerical data
MH  - Child
MH  - Fanconi Anemia/genetics/mortality/*therapy
MH  - Female
MH  - Graft Survival
MH  - Graft vs Host Disease/epidemiology/etiology/prevention & control
MH  - HLA Antigens/genetics/*immunology
MH  - Haplotypes
MH  - *Histocompatibility/genetics/immunology
MH  - Histocompatibility Testing
MH  - Humans
MH  - Kaplan-Meier Estimate
MH  - Living Donors
MH  - Lymphocyte Depletion
MH  - Male
MH  - *Peripheral Blood Stem Cell Transplantation/statistics & numerical data
MH  - Primary Graft Dysfunction/epidemiology
MH  - Progression-Free Survival
MH  - Proportional Hazards Models
MH  - Prospective Studies
MH  - Siblings
MH  - T-Lymphocyte Subsets/immunology
MH  - Treatment Outcome
EDAT- 2021/02/20 06:00
MHDA- 2021/05/14 06:00
CRDT- 2021/02/19 12:15
PHST- 2021/02/16 00:00 [revised]
PHST- 2020/11/10 00:00 [received]
PHST- 2021/02/18 00:00 [accepted]
PHST- 2021/02/20 06:00 [pubmed]
PHST- 2021/05/14 06:00 [medline]
PHST- 2021/02/19 12:15 [entrez]
AID - 10.1002/ajh.26135 [doi]
PST - ppublish
SO  - Am J Hematol. 2021 May 1;96(5):571-579. doi: 10.1002/ajh.26135. Epub 2021 Mar 4.