Severe Chronic Neutropenia International Registry Protocol
Investigation of Severe Chronic Neutropenia and Its Treatment
Brief description of study.
The objective of the registry is to collect information on the natural course of severe chronic neutropenia (SCN) and its response to treatment with the blood-stimulating factor called granulocyte colony stimulating factor or G-CSF (Neupogen).
Detailed description of study
The objective of the registry is to collect information on the natural course of severe chronic neutropenia (SCN) and its response to treatment with the blood-stimulating factor called granulocyte colony stimulating factor or G-CSF (Neupogen).
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: Severe Chronic Neutropenia,Chronic Neutropenia,Severe Neutropenia,Neutropenia
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Age: 100 years or below
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Gender: All
Inclusion Criteria
A confirmed diagnosis of severe chronic neutropenia based on documented absolute neutrophil counts in the three months prior to enrollment.
Bone marrow aspiration consistent with the diagnosis of congenital, cyclic or idiopathic neutropenia.
Normal cytogenetic evaluation.
History of recurrent infections (i.e. severe mouth ulcers, gingivitis and sinusitis).
Age greater than three months.
Independent of hematological parameters, patients with the following diagnoses may be included: Shwachman-Diamond syndrome (SDS), glycogen storage disease type Ib (GSD1b), Barth syndrome.
Patients with moderately severe chronic neutropenia and recurrent severe infections (i.e., deep tissue infections of subcutaneous areas, lungs, liver, etc.).
Immune neutropenia with positive anti-neutrophil antibodies meeting criteria in 1, 3, 5 and 6.
All SCN patients previously enrolled in Amgen-sponsored SCN studies.
Exclusion Criteria
Neutropenia known to be drug induced.
Primary myelodysplasia
Primary leukemia
Aplastic anemia
Known HIV disease
Systemic autoimmune diseases such as rheumatoid arthritis or systemic lupus, erythematosus
Chemotherapy-induced neutropenia (within the last 5 years)
This study investigates severe chronic neutropenia (SCN), a condition where there are abnormally low levels of neutrophils, a type of white blood cell. This condition can lead to frequent infections. The purpose is to understand the natural progression of SCN and its response to treatment with a blood-stimulating factor called granulocyte colony stimulating factor (G-CSF).
Participants will undergo regular health assessments to monitor their condition and response to the treatment. The study will collect data on various health parameters, including blood counts and infection history, to better understand the effectiveness of the treatment.
- Who can participate: Individuals older than three months with a confirmed diagnosis of severe chronic neutropenia can participate. Key eligibility includes a history of recurrent infections and specific syndromes like Shwachman-Diamond syndrome or glycogen storage disease type Ib.
- Study details: Participants will need to provide health information and undergo regular assessments. The study involves monitoring the response to an investigational treatment, with some participants receiving a placebo, an inactive substance that looks like the investigational medicine but does not contain any medicine.
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