Korean J Hematol.  2002 May;37(2):114-119.

Hematopoietic Stem Cell Transplantation for Paroxysmal Nocturnal Hemoglobinuria

• Shin HJ
• Kim HJ
• Park CY
• Park YH
• Kim YJ
• Lee S
• Min CK
• Kim DW
• Lee JW
• Kim CC
• Min WS

Abstract

BACKGROUND: Paroxysmal nocturnal hemogolbinuria (PNH) is an acquired clonal hematological disorder characterized by intermittent episodes of hemolysis, a predisposition to venous thrombosis, defective hemopoiesis, and occasionally, transition to leukemia. Because PNH is a stem cell disorder, treatment with androgen or corticosteroids may only be a palliative measure, and allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative treatment. We report here our experience of eight PNH patients who underwent HSCT.
METHODS
Between January 1997 and July 2001, 8 patients, aged 16 to 47 (median 27.5), underwent HSCT for PNH at the Catholic Hemopoietic Stem Cell Transplantation Center. Median time from diagnosis to HSCT was 60.5 months (range 5-165 months). Two different conditioning regimens included busulfan (16mg/ kg) and cyclophosphamide (120mg/kg) or TBI 1,200cGy and cyclophosphamide (120mg/kg). Graft-versus-host disease (GVHD) prevention was cyclosporine with methotrexate (MTX)(N= 6), FK506 with MTX (N=1) or cyclosporine with T-cell depletion of donor marrow (N=1).
RESULTS
Four deaths occurred in the first post-transplant year. Deaths were from graft failure (N=1), pneumonia (N=1) and veno- cclusive disease/thrombotic thrombocytopenic purpura (N=2). Four patients (50%) remain alive at a median of 26 months (range 1-60 months) and 5-year probability of survival was 66.7% after HLA-matched sibling HSCT. Grade I or II acute GVHDs occurred in 3 patients and chronic GVHD did not develop in 5 patients other than 3 patients who died within 100 days post-transplant.
CONCLUSION
This study suggests that HSCT is an effective therapeutic option for PNH. Further studies are needed to decide the appropriate conditioning regimens to overcome treatment-related mortality after transplantation.

Keyword

Paroxysmal nocturnal hemoglobinuria; Graft versus host disease; Hematopoietic stem cell transplantation

MeSH Terms

Adrenal Cortex Hormones
Bone Marrow
Busulfan
Cyclophosphamide
Cyclosporine
Diagnosis
Graft vs Host Disease
Hematopoietic Stem Cell Transplantation*
Hematopoietic Stem Cells*
Hemoglobinuria*
Hemoglobinuria, Paroxysmal
Hemolysis
Humans
Leukemia
Methotrexate
Mortality
Pneumonia
Purpura, Thrombocytopenic
Siblings
Stem Cell Transplantation
Stem Cells
T-Lymphocytes
Tacrolimus
Tissue Donors
Transplants
Venous Thrombosis
Adrenal Cortex Hormones
Busulfan
Cyclophosphamide
Cyclosporine
Methotrexate
Tacrolimus