Mobilization of Stem Cells With G-CSF for Collection From Patients With Diamond-Blackfan Anemia
Diamond Blackfan Anemia
About this trial
This is an interventional treatment trial for Diamond Blackfan Anemia focused on measuring G-CSF, Leukapheresis, Stem Cell Cryopreservation, Central Line Placement, G-CSF Administration, Stem Cell Preservation, Diamond Blackfan Anemia, Healthy Volunteer
Eligibility Criteria
INCLUSION CRITERIA: Male or female patients greater than or equal to 4 years of age and weighing at least 12 kg (27 lbs) will be included. Diagnosis of DBA as defined by the onset of anemia before age 2; or, if after age 2, a positive family history of classical DBA or typical physical anomalies Low reticulocyte counts (less than 30,000/ul) Absent or blocked maturation of erythroid precursors in the bone marrow. Red-cell transfusion dependence Adequate birth control while on protocol EXCLUSION CRITERIA: Female patients that are pregnant or lactating Neutrophil count less than 1000/uL or platelet count less than 100,000/uL Positive DEB stimulation test Evidence of transformation to MDS or AML on pre-study marrow aspiration and biopsy, or abnormal marrow cytogenetics. History or evidence of clinically significant autoimmune inflammatory disease or significant cardiopulmonary disease by history or physical exam Uncorrected coagulopathy Renal disease (GFR less than 100mL/min/1/73 M(2)) as estimated for children by Schwartz formula. Adults (age greater than 21) excluded for serum Cr less than 2.5 mg/dL. Hepatic disease (bilirubin greater than 2 mg/dL or transaminases greater than 7 times the upper limit of normal). Seropositivity for hepatitis B (HbsAg), hepatitis C, or HIV will be excluded. Other medical illness or condition which, in the opinion of the Investigators, may contraindicate participation in this study due to risk to patient or comprise of study integrity.
Sites / Locations
- National Heart, Lung and Blood Institute (NHLBI)