Aplastic anemia and paroxysmal nocturnal hemoglobinuria: a follow-up study of the glycosylphosphatidylinositol-anchored proteins defect.
BACKGROUND
Flow cytometry analysis of peripheral blood cells is a simple and reliable method for establishing the diagnosis of paroxysmal nocturnal hemoglobinuria. The behavior of the clone may vary; increasing or diminishing over time but prospective study of such variations have not been reported so far.


METHODS
We report herein the results of a prospective follow-up study of 25 patients. Our aims were twofold: first, to evaluate the behavior of the clone (using flow cytometry) over the time; and second, to evaluate if such variations could predict the occurrence of complications or could be used as a tool for monitoring the residual disease after bone marrow transplantation.


RESULTS
It was found that flow cytometry can be used to specifically follow the residual disease post allogeneic marrow transplantation in four patients, and that even without transplantation the defective clone can significantly decrease or even disappear (three patients).


CONCLUSIONS
We found that most of the patients did have significant change in the amount of affected cells during more than three years, and that an increased size of the clone poorly predicted the occurrence of complications.
Noguera
M E
ME
Laboratoire Central d'Hématologie, Hôpital Saint-Louis, 1 Avenue Claude Vellefaux, 75475 Paris Cedex 10, France.
Leymarie
V
V
Bittencourt
E
E
Gluckman
E
E
Sigaux
F
F
Socié
G
G
D000328:Adult; D000741:Anemia, Aplastic / Q000097:blood / Q000150:complications / Q000188:drug therapy / Q000473:pathology* / Q000628:therapy; D000961:Antilymphocyte Serum / Q000627:therapeutic use; D001772:Blood Cell Count; D016026:Bone Marrow Transplantation; D002999:Clone Cells / Q000473:pathology; D003131:Combined Modality Therapy; D016572:Cyclosporine / Q000627:therapeutic use; D005260:Female; D005434:Flow Cytometry; D005500:Follow-Up Studies; D017261:Glycosylphosphatidylinositols / Q000172:deficiency*; D006457:Hemoglobinuria, Paroxysmal / Q000097:blood / Q000150:complications / Q000188:drug therapy / Q000473:pathology* / Q000628:therapy; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D007167:Immunotherapy; D008297:Male; D008565:Membrane Proteins / Q000172:deficiency / Q000235:genetics; D008875:Middle Aged; D011247:Pregnancy; D011250:Pregnancy Complications, Hematologic / Q000097:blood / Q000473:pathology; D011446:Prospective Studies; D013601:T-Lymphocytes; D014184:Transplantation, Homologous
D000961:Antilymphocyte Serum; D017261:Glycosylphosphatidylinositols; D007166:Immunosuppressive Agents; D008565:Membrane Proteins; C080086:phosphatidylinositol glycan-class A protein; D016572:Cyclosporine