Περίληψη:
Purpose: We assessed the value of marrow cultures for defining the
pathophysiology, diagnosis, and therapeutic response to
immunosuppressive therapy in childhood pure red cell aplasia (PRCA).
Patients and Methods: Patients were evaluated either at diagnosis (n =
23) or at the time of treatment failure (n = 2). Twelve patients had
transient erythroblastopenia of childhood (TEC), 4 had Diamont-Blackfan
anemia (DBA), and 9 had acquired sustained PRCA (A-Su-PRCA). Bone marrow
mononuclear cells were cultured with combination of human recombinant
(rhu) erythropoietin (EPO), granulocyte monocyte colony stimulating
factor (GM-CSF), granulocyte colony stimulating factor (G-CSF),
Interleukin 3 (IL-3), either with or without stem cell factor (SCF), and
burst forming unit of erythroid (BFU-E) growth was assessed.
Results: The combination of growth factors without SCF failed to induce
any erythropoiesis (BFU-E <10/10(5) mononuclear cells) in 10 patients (2
with TEC, 2 with DBA, and 6 with A-Su-PRCA), although the growth of
erythroid colonies was substantially lower in the remaining patients
than in controls (45.5 +/- 15.4 Versus 91.7 +/- 12.7, p <0.05). Addition
of SCF restored erythropoiesis in all but 6 patients (5 with A-Su-PRCA
and 1 with DBA). Five of 6 nonresponders did not respond to any
immunomodulating therapy; of the 5, 3 had or developed some evidence of
myelodysplasia.
Conclusion: Our data indicate that in vitro colony studies might prove
to be a useful diagnostic tool, because erythropoiesis’ poor response to
growth factors. including SCF, may suggest the diag nosis of
myelodysplasia. Moreover, it may have predictive value, in cases of
PRCA, regardless of etiology, poor growth of erythropoietic colonies may
predict refractoriness to immunomodulating therapy.
Συγγραφείς:
Gussetis, ES
Peristeri, J
Kitra, V
Liakopoulou, T and
Kattamis, A
Graphakos, S
