Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes

• Status: Completed
• Trial end date: 1969-12-31
• Target enrollment: 0
• Participant gender: All

Summary

OBJECTIVES: I. Assess the efficacy of recombinant human granulocyte colony-stimulating factor (G-CSF) in raising the absolute neutrophil count, platelet count, and hemoglobin level in patients with inherited bone marrow failure syndromes.

II. Assess the efficacy of a reduced maintenance dose in patients who respond to daily G-CSF.

III. Assess the toxic effects of G-CSF in these patients. IV. Measure bone marrow progenitor colonies before and after G-CSF. V. Measure CD34-positive cells in marrow and blood before and after G-CSF using flow cytometry and immunohistochemistry.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: National Center for Research Resources (NCRR)
• Collaborator: James Whitcomb Riley Hospital for Children
• Treatments: Lenograstim

Criteria

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics--

Inherited bone marrow failure syndrome, including:

• Fanconi's anemia

• Dyskeratosis congenita

• Shwachman syndrome

• Amegakaryocytic thrombocytopenia

• Decreased megakaryocytes in infancy

• No thrombocytopenia with absent radius syndrome (TAR)

• No trisomy 13 or 18

• No clonal bone marrow karyotype

--Prior/Concurrent Therapy--

• At least 4 weeks since growth factors

• Concurrent therapy allowed if not altered for 30 days prior to entry through week 8

• No concurrent investigational drugs

--Patient Characteristics--

• Hematopoietic: ANC <1000

• No leukemia

• Other: No medical or psychiatric contraindication to protocol participation

• No pregnant or nursing women