PMID- 12890150
OWN - NLM
STAT- MEDLINE
DCOM- 20030917
LR  - 20190910
IS  - 0902-4441 (Print)
IS  - 0902-4441 (Linking)
VI  - 71
IP  - 2
DP  - 2003 Aug
TI  - Allogeneic hematopoietic cell transplantation for paroxysmal nocturnal 
      hemoglobinuria.
PG  - 114-8
AB  - BACKGROUND: Although allogeneic hematopoietic cell transplantation (HCT) has a 
      potential to cure patients with paroxysmal nocturnal hemoglobinuria (PNH), 
      appropriate indication and conditioning regimen for HCT have not been 
      established. PATIENTS AND METHODS: Between July 1999 and December 2001, five 
      patients with PNH underwent allogeneic HCT: three for refractory hemolysis and 
      two for aggravating cytopenia. Four patients with hypercellular marrow received 
      Bu-Fludara-ATG (busulfan 4 mg/kg/d for 2 d, fludarabine 30 mg/m2/d for 6 d, and 
      ATG 20 mg/kg/d for 4 d) for conditioning therapy and one patient with 
      hypocellular marrow was conditioned with Cy-ATG (cyclophosphamide 50 mg/kg/d for 
      4 d and ATG 30 mg/kg/d for 3 d). Three patients received stem cell graft from 
      matched sibling donor and two patients from 1-antigen mismatched unrelated donor. 
      RESULTS: One patient who was conditioned with Bu-Fludara-ATG failed to engraft 
      and died at post-transplant day 62. The other four patients showed three lineage 
      engraftment and normal expression of CD55 and CD59 antigens by flow cytometric 
      analysis. They are alive with stable engraftment and full donor chimerism between 
      post-transplant day 510 and 1116. Acute graft vs. host disease (GVHD) of grade II 
      or more occurred in two patients and extensive chronic GVHD in four. CONCLUSION: 
      HCT using related or unrelated donor could eradicate PNH clones and may cure 
      patients with the disease. Further studies are needed to establish the role of 
      allogeneic HCT, especially with reduced intensity conditioning therapy, in the 
      treatment of PNH.
FAU - Lee, Jae-Lyun
AU  - Lee JL
AD  - Department of Internal Medicine, Asan Medical Center, University of Ulsan College 
      of Medicine, Seoul, Korea.
FAU - Lee, Je-Hwan
AU  - Lee JH
FAU - Lee, Jung-Hee
AU  - Lee JH
FAU - Choi, Seong-Jun
AU  - Choi SJ
FAU - Kim, Shin
AU  - Kim S
FAU - Seol, Miee
AU  - Seol M
FAU - Lee, Young-Shin
AU  - Lee YS
FAU - Chi, Hyun-Sook
AU  - Chi HS
FAU - Park, Chan-Jeoung
AU  - Park CJ
FAU - Kim, Woo-Kun
AU  - Kim WK
FAU - Lee, Jung-Shin
AU  - Lee JS
FAU - Lee, Kyoo-Hyung
AU  - Lee KH
LA  - eng
PT  - Clinical Trial
PT  - Journal Article
PL  - England
TA  - Eur J Haematol
JT  - European journal of haematology
JID - 8703985
RN  - 0 (Antilymphocyte Serum)
RN  - FA2DM6879K (Vidarabine)
RN  - G1LN9045DK (Busulfan)
RN  - P2K93U8740 (fludarabine)
SB  - IM
MH  - Adolescent
MH  - Adult
MH  - Antilymphocyte Serum/administration & dosage
MH  - Busulfan/administration & dosage
MH  - Drug Therapy, Combination
MH  - Female
MH  - Graft Survival
MH  - Graft vs Host Disease
MH  - Hematopoietic Stem Cell Transplantation/*methods
MH  - Hemoglobinuria, Paroxysmal/*therapy
MH  - Histocompatibility
MH  - Humans
MH  - Male
MH  - Transplantation Conditioning/methods
MH  - Transplantation, Homologous
MH  - Vidarabine/administration & dosage/*analogs & derivatives
EDAT- 2003/08/02 05:00
MHDA- 2003/09/18 05:00
CRDT- 2003/08/02 05:00
PHST- 2003/08/02 05:00 [pubmed]
PHST- 2003/09/18 05:00 [medline]
PHST- 2003/08/02 05:00 [entrez]
AID - 097 [pii]
AID - 10.1034/j.1600-0609.2003.00097.x [doi]
PST - ppublish
SO  - Eur J Haematol. 2003 Aug;71(2):114-8. doi: 10.1034/j.1600-0609.2003.00097.x.