Allogeneic hematopoietic cell transplantation for paroxysmal nocturnal hemoglobinuria.
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.


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.


CONCLUSIONS
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.
Lee
Jae-Lyun
JL
Department of Internal Medicine, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea.
Lee
Je-Hwan
JH
Lee
Jung-Hee
JH
Choi
Seong-Jun
SJ
Kim
Shin
S
Seol
Miee
M
Lee
Young-Shin
YS
Chi
Hyun-Sook
HS
Park
Chan-Jeoung
CJ
Kim
Woo-Kun
WK
Lee
Jung-Shin
JS
Lee
Kyoo-Hyung
KH
D000293:Adolescent; D000328:Adult; D000961:Antilymphocyte Serum / Q000008:administration & dosage; D002066:Busulfan / Q000008:administration & dosage; D004359:Drug Therapy, Combination; D005260:Female; D006085:Graft Survival; D006086:Graft vs Host Disease; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006457:Hemoglobinuria, Paroxysmal / Q000628:therapy*; D006648:Histocompatibility; D006801:Humans; D008297:Male; D019172:Transplantation Conditioning / Q000379:methods; D014184:Transplantation, Homologous; D014740:Vidarabine / Q000008:administration & dosage / Q000031:analogs & derivatives*
D000961:Antilymphocyte Serum; D014740:Vidarabine; D002066:Busulfan; C024352:fludarabine