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Bone marrow transplantation for paroxysmal nocturnal hemoglobinuria:
eradication of the PNH clone and documentation of complete
lymphohematopoietic engraftment
JH Antin, D Ginsburg, BR Smith, DG Nathan, SH Orkin and JM Rappeport
Paroxysmal nocturnal hemoglobinuria (PNH) involves the proliferation of an
abnormal and possibly premalignant hematopoietic stem cell. Successful
treatment of PNH by marrow grafting requires that the PNH clone be
eradicated by the pretransplant conditioning regimen. Four patients with
PNH-associated marrow aplasia were transplanted with marrow from their
HLA-matched, MLR-nonreactive siblings. Three patients were conditioned with
cyclophosphamide, procarbazine, and antithymocyte serum (CTX/PCZ/ATS), and
one was conditioned with busulfan/CTX/PCZ/ATS. Persistent complete
engraftment of myeloid, lymphoid, and erythroid cell lines was demonstrated
in all four patients by DNA sequence polymorphism analysis or cytogenetics,
and RBC typing. There was no recurrence of the abnormal clone of cells for
up to five years after transplantation despite the use of a conditioning
regimen in three of them, which is not usually associated with permanent
marrow aplasia. Bone marrow transplantation is a curative therapy in
patients whose illness is severe enough to warrant the risk.
Volume 66,
Issue 6,
pp. 1247-1250,
12/01/1985
Copyright © 1985 by The American Society of Hematology
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