Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes
Shwachman Syndrome, Fanconi's Anemia, Dyskeratosis Congenita
About this trial
This is an interventional treatment trial for Shwachman Syndrome focused on measuring Fanconi's anemia, Shwachman syndrome, aplastic anemia, dermatologic disorders, dyskeratosis congenita, hematologic disorders, rare disease, thrombocytopenia
Eligibility 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