Sound Advice From The Experts

Dr. Melvin Freedman, "Neutropenia Support Assoc. Inc. Newsletter", Volume 9, 1997

For patients with severe chronic Neutropenia, their families, and treating Physicians:

The Severe Chronic Neutropenia International Registry (SCNIR) meets regularly to monitor disease and treatment outcomes of patients with various forms of severe chronic Neutropenia (SCN). The SCNIR has a large database with long term information on more than 500 patients. Using this valuable material, the SCNIR and its Sub-committee on Safety have been able to make recommendations to patients, their families and their treating physicians about the administration of G-CSF (Neupogen) and about the monitoring of bone marrow function. The following two recommendations are in effect for 1997 and 1998.

Annual Bone Marrow Evaluation

Approximately 10% of SCN patients with the congenital or Kostmann’s form of Neutropenia have developed myelodysplastic syndrome (MDS) and/or acute myeloblastic leukemia (AML). SCNIR research studies have shown that marrow cells of patients who have developed MDS/AML have also showed chromosome abnormalities and other cellular changes indicative of the malignant transformation. Using this knowledge, the SCNIR recommends that all patients with congenital or Kostmann’s Neutropenia receiving G-CSF have an annual bone marrow evaluation for microscopic inspection of the specimen, and for chromosome and possibly other studies that may identify those patients who are at risk of MDS/AML. This recommendation also applies to patients with Shwachman-Diamond syndrome because of their predisposition to MDS/AML. Early detection will allow the initiation of a treatment plan before overt disease, which is more difficult to manage, becomes manifested.

Since MDS/AML has not yet been seen in SCN patients with glycogen storage disease type 1b, with cyclic Neutropenia, or with idiopathic forms of Neutropenia, annual bone marrow testing for these patients can be waived at the discretion of physician and patient, but careful, serial monitoring of blood counts and physical status should continue.

The Need to Continue G-CSF Treatment

G-CSF treatment of SCN patients is highly effective in more than 90% of cases and induces the production of neutrophils to levels that prevent infection and related complications. For congenital and Kostmann’s Neutropenia and for cyclic Neutropenia, G-CSF doses and scheduling of administration may vary somewhat from patient to patient. But G-CSF cannot be stopped. Discontinuing G-CSF therapy in these patients inevitably leads to recurrence of severe Neutropenia. Until an equally effective treatment becomes available, the SCNIR currently believes that G-CSF therapy in these patients is for the long-term.

Similarly, most patients with idiopathic Neutropenia require long-term G-CSF therapy. However, there may be small numbers of patients in this subgroup who spontaneously have an improvement in neutrophil numbers and will not require continuous G-CSF therapy. This will only happen in a minority of cases and must be determined by the treating physician on a case-by-case basis.