G-CSF Treatment for Inherited Bone Marrow Failure Syndromes
filgrastim
Congenital Abnormalities+32
+ Bone Marrow Failure Disorders
+ Congenital Bone Marrow Failure Syndromes
Treatment Study
Summary
Study start date: December 1, 1994
Actual date on which the first participant was enrolled.PROTOCOL OUTLINE: Patients receive granulocyte colony-stimulating factor (G-CSF) subcutaneously every day for 8 weeks; nonresponders receive an increased dose for an additional 8 weeks. Patients who respond at week 8 or 16 are then tapered to a lower maintenance dose of G-CSF administered every other day through week 40. The dose is adjusted to maintain an absolute neutrophil count above 1500. Patients are removed from study for failure to achieve a complete response by week 16, unacceptable nonhematologic toxicity, the identification of a clonal karyotype in marrow, or the onset of leukemia.
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.20 patients to be enrolled
Total number of participants that the clinical trial aims to recruit.Treatment Study
Eligibility
Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.Any sex
Biological sex of participants that are eligible to enroll.Healthy volunteers not allowed
If individuals who are healthy and do not have the condition being studied can participate.Conditions
Pathology
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