This protocol covers Fanconi anemia presenting with severe bone marrow failure in patients for whom hematopoietic cell transplantation (HCT) carries an exceptional risk or is not a viable option — and in whom a prior course of G-CSF did not achieve the intended improvement in neutrophil count.
Patients in this situation have severe bone marrow failure alongside one or more factors that markedly raise HCT-related mortality: age 35 years or older, severe organ dysfunction, a pre-existing malignancy, or a life-threatening systemic infection. The protocol also applies when cure by HCT is not possible or preferred for other reasons. In these circumstances, maintaining blood counts and quality of life becomes the central focus of management.
The preceding step involved granulocyte colony-stimulating factor (G-CSF), used to address neutropenia associated with recurrent or serious infections, particularly with persistently low neutrophil counts. When G-CSF does not deliver adequate improvement in the neutrophil count, management escalates to this protocol.
At this stage, management centres on sustained transfusion-based blood count support using specially prepared blood products — a strategy directed at maintaining acceptable counts when other treatment pathways are not available. The complete structured regimen, including product specifications and thresholds for intervention, is available via the link below.