Mobilization of Stem Cells With G-CSF for Collection From Patients With Diamond-Blackfan Anemia

Plain-language summary

This study examined the use of the drug G-CSF to mobilize stem cells, together with leukapheresis, in order to collect stem cells from patients with Diamond-Blackfan anemia. Patients aged 4 and over, weighing at least 12 kg, with a diagnosis of Diamond-Blackfan anemia could take part. The study was carried out in Phase 2 and has been completed.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Phase 2

Status

Completed

Sponsor

National Heart, Lung, and Blood Institute (NHLBI)

Intervention

G-CSF, Leukapheresis

Start date

2/1/2001

Estimated completion

4/1/2006

Countries

United States

Related genes

Locations

• Bethesda, United States

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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.