The use of granulocyte colony-stimulating factor (G-CSF) in the treatment of severe aplastic anemia may not have an effect on several long-term outcomes, according to study results reported in Haematologica.

In this multicenter phase 3 trial ( Identifier: NCT01163942), 192 patients with severe aplastic anemia were given immunosuppressive therapy that included horse antithymocyte globulin (ATG) with cyclosporine (CSA). Patients were randomly assigned to receive G-CSF (49.5%) or no G-CSF (50.5%) with treatment. They were evaluated for multiple outcomes.

Related Articles

Median study follow-up duration was 11.7 years (range, 10.9-12.5). The 15-year overall survival rate was 57% for patients receiving G-CSF as part of treatment, and it was 63% for patients not receiving G-CSF (P =.92).

Continue Reading

The event-free survival rates were 24% for patients given G-CSF and 23% for patients not given G-CSF (P =.36).

There were no differences associated with treatment group in the incidences of several outcomes of interest. The 15-year cumulative incidence of myelodysplastic syndrome (MDS), acute myeloid leukemia (AML), or isolated cytogenetic abnormalities was approximately the same for patients in the G-CSF group (8.5%) and in the non-G-CSF group (8.2%; P =.90).

Among the conditions examined in this study, the cumulative incidence of all late events was 50% for patients who received G-CSF and 49% for patients who did not receive G-CSF (P =.65). Late events included in these estimates were MDS/AML, isolated cytogenetic abnormalities, relapse, solid cancer, kidney disease, osteonecrosis, and clinical paroxysmal nocturnal hemoglobinuria.

Age and disease severity upon randomization appeared to be the factors most strongly related to overall survival for patients in this study.

“Taken together, our data suggest that G-CSF added to standard immunosuppressive therapy has no effect on long-term outcomes of patients with acquired aplastic anemia and is not directly related with late effects,” the researchers wrote.


  1. Tichelli A, Peffault de Latour R, Passweg J, et al. Long-term outcome of a randomized controlled study in patients with newly diagnosed severe aplastic anemia treated with antithymocyte globuline, cyclosporine, with or without G-CSF: a Severe Aplastic Anemia Working Party Trial from the European Group of Blood and Marrow Transplantation [published online October 3, 2019]. Haematologica. doi:10.3324/haematol.2019.222562