دورية
Diamond-Blackfan Anemia: In Vitro Response of Erythroid Progenitors to the Ligand for c-kit
| العنوان: | Diamond-Blackfan Anemia: In Vitro Response of Erythroid Progenitors to the Ligand for c-kit |
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| المؤلفون: | Abkowitz, Janis L., Sabo, Kathleen M., Nakamoto, Betty, Blau, C. Anthony, Martin, Frank H., Zsebo, Krisztina M., Papayannopoulou, Thalia |
| المصدر: | Blood; November 1991, Vol. 78 Issue: 9 p2198-2202, 5p |
| مستخلص: | To provide insights into the pathogenesis of Diamond-Blackfan anemia, we examined the in vitro response of erythroid progenitors to the recently isolated ligand for c-kit (stem cell factor, SCF). For these studies, marrow or blood mononuclear cells from 10 Diamond-Blackfan patients were cultured with erythropoietin (Ep), Ep and interleukin-3, Ep and granulocyte-macrophage colony-stimulating factor, or Ep and lymphocyte conditioned media (LCM). These combinations were tested in the presence or absence of SCF. The mean number of cells per erythroid burst increased 5 to 50-fold in cultures containing SCF. Furthermore, many additional erythroid bursts were seen (mean increment 3.2 x baseline values). Although burst-forming unit-erythroid (BFU-E) from all patients responded, there were differences among individuals in the sensitivity of their BFU-E to SCF. In six patients and all control studies, plateau frequencies of erythroid bursts were achieved with < 10 ng/mL SCF, whereas in studies from the other four patients, over 50 ng/mL SCF was required. These data invite speculation that the c-kit recep-tor/ligand axis is involved in the pathogenesis of Diamond-Blackfan anemia. More importantly and regardless of whether the observed patterns of response reflect the primary defect or an epiphenomenon, our data strongly support a therapeutic trial of SCF in patients with Diamond-Blackfan anemia. |
| قاعدة البيانات: | Supplemental Index |
| تدمد: | 00064971 15280020 |
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| DOI: | 10.1182/blood.V78.9.2198.2198 |