PMID- 31209410
OWN - NLM
STAT- MEDLINE
DCOM- 20190807
LR  - 20210626
IS  - 1671-167X (Print)
IS  - 1671-167X (Linking)
VI  - 51
IP  - 3
DP  - 2019 Jun 18
TI  - [Haploidentical allogenetic hematopoietic stem cell transplantation for X-linked 
      adrenoleukodystrophy].
PG  - 409-413
LID - 10.19723/j.issn.1671-167X.2019.03.006 [doi]
AB  - OBJECTIVE: X-linked adrenoleukodystrophy (ALD) is a severe inherited disorder 
      leading to rapid neurological deterioration and premature death. Allogeneic 
      hematopoietic stem cell transplantation (HSCT) is still the only treatment that 
      halts the neurologic symptoms in ALD. However, many patients lack suitable human 
      leukocyte antigen (HLA) matched related donors and must rely on alternative 
      donors for a source of stem cells. The purpose of this study was to explore the 
      outcomes of haploidentical allogeneic stem cell transplantation for ALD patients. 
      METHODS: Between December 2014 and December 2018, eight children with ALD lacking 
      HLA matched related or unrelated donors were treated with haploidentical 
      allogeneic hematopoietic stem cell transplantation. The patients received 
      conditioning regimen with busulfan 9.6 mg/kg, cyclophosphamide 200 mg/kg and 
      fludarabine 90 mg/m(2). Graft-versus-host disease (GVHD) prophylaxis consisted of 
      anti-human thymocyte globulin, cyclosporine A, mycophenolate mofetil and short 
      course of methotrexate. RESULTS: All the 8 children received allogeneic stem cell 
      transplants from their fathers. The median age of the recipients was 8 (range: 
      5-12) years. The median age of the donors was 36 (range: 32-40) years. All the 
      recipients received granulocyte colony-stimulating factor (G-CSF) mobilized bone 
      marrow and peripheral blood-derived stem cells. The median number of total 
      mononuclear cells dose and CD34+ dose was 10.89 (range: 9.40-12.16)x10(8)/kg and 
      7.06 (range: 0.74-7.80)x10(6)/kg, respectively. Neutrophil engraftment occurred a 
      median of 11 days (range:8-13 days) after transplantation. Platelet engraftment 
      occurred a median of 10 days (range:8-12 days) after transplantation. All the 
      patients achieved complete donor chimerism at the time of engraftment. Four 
      patients had grades II-IV acute GVHD and 1 had chronic graft-versus-host disease. 
      No severe chronic GVHD occurred. Among all the children, 2 had cytomegalovirus 
      (CMV) DNAemia and 2 Epstein-Barr virus (EBV) DNAemia. Overall, seven of them 
      survived and had no major complications related to transplantation. One died of 
      cerebral hernia after epilepsy 125 days after transplantation. CONCLUSION: The 
      preliminary observation demonstrates that haploidentical allogeneic stem cell 
      transplantation with this novel regimen could successfully achieve full donor 
      chimerism in ALD patients. According to our experience, haploidentical allogeneic 
      hematopoietic stem cell transplantation is safe and feasible in the treatment of 
      X-linked adrenoleukodystrophy.
FAU - Chen, Y
AU  - Chen Y
AD  - Peking University People's Hospital, Peking University Institute of Hematology, 
      Beijing 100044, China.
AD  - Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation for the 
      Treatment of Hematological Diseases, Beijing 100044, China.
FAU - Zhang, X H
AU  - Zhang XH
AD  - Peking University People's Hospital, Peking University Institute of Hematology, 
      Beijing 100044, China.
AD  - Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation for the 
      Treatment of Hematological Diseases, Beijing 100044, China.
FAU - Xu, L P
AU  - Xu LP
AD  - Peking University People's Hospital, Peking University Institute of Hematology, 
      Beijing 100044, China.
AD  - Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation for the 
      Treatment of Hematological Diseases, Beijing 100044, China.
FAU - Liu, K Y
AU  - Liu KY
AD  - Peking University People's Hospital, Peking University Institute of Hematology, 
      Beijing 100044, China.
AD  - Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation for the 
      Treatment of Hematological Diseases, Beijing 100044, China.
FAU - Qin, J
AU  - Qin J
AD  - Department of Pediatrics, Peking University People's Hospital, Beijing 100044, 
      China.
FAU - Yang, Y L
AU  - Yang YL
AD  - Department of Pediatrics, Peking University First Hospital, Beijing 100034, 
      China.
FAU - Huang, X J
AU  - Huang XJ
AD  - Peking University People's Hospital, Peking University Institute of Hematology, 
      Beijing 100044, China.
AD  - Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation for the 
      Treatment of Hematological Diseases, Beijing 100044, China.
LA  - chi
PT  - Journal Article
PL  - China
TA  - Beijing Da Xue Xue Bao Yi Xue Ban
JT  - Beijing da xue xue bao. Yi xue ban = Journal of Peking University. Health 
      sciences
JID - 101125284
MH  - *Adrenoleukodystrophy/therapy
MH  - Adult
MH  - Bone Marrow Transplantation
MH  - Child
MH  - Child, Preschool
MH  - Chromosomes, Human, X
MH  - *Graft vs Host Disease
MH  - *Hematopoietic Stem Cell Transplantation
MH  - Humans
MH  - Transplantation Conditioning
PMC - PMC7439018
EDAT- 2019/06/19 06:00
MHDA- 2019/08/08 06:00
PMCR- 2019/06/18
CRDT- 2019/06/19 06:00
PHST- 2019/06/19 06:00 [entrez]
PHST- 2019/06/19 06:00 [pubmed]
PHST- 2019/08/08 06:00 [medline]
PHST- 2019/06/18 00:00 [pmc-release]
AID - bjdxxbyxb-51-3-409 [pii]
AID - 10.19723/j.issn.1671-167X.2019.03.006 [doi]
PST - ppublish
SO  - Beijing Da Xue Xue Bao Yi Xue Ban. 2019 Jun 18;51(3):409-413. doi: 
      10.19723/j.issn.1671-167X.2019.03.006.